{"id":"melphalan","rwe":[{"pmid":"41896325","year":"2026","title":"Risk factors for alloimmune lung syndromes after allogeneic hematopoietic cell transplantation in children.","finding":"","journal":"Bone marrow transplantation","studyType":"Clinical Study"},{"pmid":"41896084","year":"2026","title":"Daratumumab-Based Second Line Therapy Improves Outcomes After VRD Induction, Upfront Autologous Transplant, and Lenalidomide Maintenance.","finding":"","journal":"Clinical lymphoma, myeloma & leukemia","studyType":"Clinical Study"},{"pmid":"41889248","year":"2026","title":"Gemcitabine, Docetaxel, Melphalan, Carboplatin as Part of Sequential Cycles of High-Dose Chemotherapy With Autologous Hematopoietic Stem-Cell Rescue for Multiply Relapsed/Refractory Pediatric Germ Cell Tumors.","finding":"","journal":"Pediatric blood & cancer","studyType":"Clinical Study"},{"pmid":"41881107","year":"2026","title":"Conventional cytotoxic chemotherapy-associated nephrotoxicity.","finding":"","journal":"Kidney international","studyType":"Clinical Study"},{"pmid":"41876727","year":"2026","title":"Flare of clonal hematopoiesis, TP53 expansion and prior melphalan drive post-CAR-T myeloid disorders in multiple myeloma.","finding":"","journal":"Leukemia","studyType":"Clinical Study"}],"_fda":{"id":"47e47c3a-eecb-263c-e063-6394a90a8864","set_id":"2254450e-4803-b947-e063-6394a90a4624","openfda":{"upc":["0384679001019"],"route":["INTRAVENOUS"],"rxcui":["311487"],"spl_id":["47e47c3a-eecb-263c-e063-6394a90a8864"],"brand_name":["Melphalan Hydrochloride"],"spl_set_id":["2254450e-4803-b947-e063-6394a90a4624"],"package_ndc":["84679-001-01","84679-002-01","84679-003-01"],"product_ndc":["84679-001"],"generic_name":["MELPHALAN HYDROCHLORIDE"],"product_type":["HUMAN PRESCRIPTION DRUG"],"manufacturer_name":["Arthur Group LLC"],"application_number":["ANDA211463"],"is_original_packager":[true]},"version":"2","warnings":["WARNINGS Melphalan Hydrochloride for Injection may cause local tissue damage should extravasation occur, and consequently it should not be administered by direct injection into a peripheral vein. It is recommended that Melphalan Hydrochloride for Injection be administered by injecting slowly into a fast-running IV infusion via an injection port, or via a central venous line (see DOSAGE AND ADMINISTRATION, Administration Precautions ). Melphalan should be administered in carefully adjusted dosage by or under the supervision of experienced physicians who are familiar with the drug's actions and the possible complications of its use. As with other nitrogen mustard drugs, excessive dosage will produce marked bone marrow suppression. Bone marrow suppression is the most significant toxicity associated with Melphalan Hydrochloride for Injection in most patients. Therefore, the following tests should be performed at the start of therapy and prior to each subsequent dose of Melphalan Hydrochloride for Injection: platelet count, hemoglobin, white blood cell count, and differential. Thrombocytopenia and/or leukopenia are indications to withhold further therapy until the blood counts have sufficiently recovered. Frequent blood counts are essential to determine optimal dosage and to avoid toxicity. Dose adjustment on the basis of blood counts at the nadir and day of treatment should be considered. Hypersensitivity reactions including anaphylaxis have occurred in approximately 2% of patients who received the IV formulation (see ADVERSE REACTIONS ). These reactions usually occur after multiple courses of treatment. Treatment is symptomatic. The infusion should be terminated immediately, followed by the administration of volume expanders, pressor agents, corticosteroids, or antihistamines at the discretion of the physician. If a hypersensitivity reaction occurs, IV or oral melphalan should not be readministered since hypersensitivity reactions have also been reported with oral melphalan. Carcinogenesis Secondary malignancies, including acute nonlymphocytic leukemia, myeloproliferative syndrome, and carcinoma, have been reported in patients with cancer treated with alkylating agents (including melphalan). Some patients also received other chemotherapeutic agents or radiation therapy. Precise quantitation of the risk of acute leukemia, myeloproliferative syndrome, or carcinoma is not possible. Published reports of leukemia in patients who have received melphalan (and other alkylating agents) suggest that the risk of leukemogenesis increases with chronicity of treatment and with cumulative dose. In one study, the 10-year cumulative risk of developing acute leukemia or myeloproliferative syndrome after oral melphalan therapy was 19.5% for cumulative doses ranging from 730 to 9,652 mg. In this same study, as well as in an additional study, the 10-year cumulative risk of developing acute leukemia or myeloproliferative syndrome after oral melphalan therapy was less than 2% for cumulative doses under 600 mg. This does not mean that there is a cumulative dose below which there is no risk of the induction of secondary malignancy. The potential benefits from melphalan therapy must be weighed on an individual basis against the possible risk of the induction of a second malignancy. Adequate and well-controlled carcinogenicity studies have not been conducted in animals. However, intraperitoneal (IP) administration of melphalan in rats (5.4 to 10.8 mg/m 2 ) and in mice (2.25 to 4.5 mg/m 2 ) 3 times per week for 6 months followed by 12 months post-dose observation produced peritoneal sarcoma and lung tumors, respectively. Mutagenesis Melphalan has been shown to cause chromatid or chromosome damage in humans. Intramuscular administration of melphalan at 6 and 60 mg/m 2 produced structural aberrations of the chromatid and chromosomes in bone marrow cells of Wistar rats. Impairment of Fertility Melphalan causes suppression of ovarian function in premenopausal women, resulting in amenorrhea in a significant number of patients. Reversible and irreversible testicular suppression have also been reported. Pregnancy Pregnancy Category D Melphalan may cause fetal harm when administered to a pregnant woman. While adequate animal studies have not been conducted with IV melphalan, oral (6 to 18 mg/m 2 /day for 10 days) and IP (18 mg/m 2 ) administration in rats was embryolethal and teratogenic. Malformations resulting from melphalan included alterations of the brain (underdevelopment, deformation, meningocele, and encephalocele) and eye (anophthalmia and microphthalmos), reduction of the mandible and tail, as well as hepatocele (exomphaly). There are no adequate and well-controlled studies in pregnant women. If this drug is used during pregnancy, or if the patient becomes pregnant while taking this drug, the patient should be apprised of the potential hazard to the fetus. Women of childbearing potential should be advised to avoid becoming pregnant."],"pregnancy":["Pregnancy Teratogenic Effects Pregnancy Category D: See WARNINGS section."],"overdosage":["OVERDOSAGE Overdoses resulting in death have been reported. Overdoses, including doses up to 290 mg/m 2 , have produced the following symptoms: severe nausea and vomiting, decreased consciousness, convulsions, muscular paralysis, and cholinomimetic effects. Severe mucositis, stomatitis, colitis, diarrhea, and hemorrhage of the gastrointestinal tract occur at high doses (>100 mg/m 2 ). Elevations in liver enzymes and veno-occlusive disease occur infrequently. Significant hyponatremia caused by an associated inappropriate secretion of ADH syndrome has been observed. Nephrotoxicity and adult respiratory distress syndrome have been reported rarely. The principal toxic effect is bone marrow suppression. Hematologic parameters should be closely followed for 3 to 6 weeks. An uncontrolled study suggests that administration of autologous bone marrow or hematopoietic growth factors (i.e., sargramostim, filgrastim) may shorten the period of pancytopenia. General supportive measures together with appropriate blood transfusions and antibiotics should be instituted as deemed necessary by the physician. This drug is not removed from plasma to any significant degree by hemodialysis or hemoperfusion. A pediatric patient survived a 254-mg/m 2 overdose treated with standard supportive care."],"references":["REFERENCES NIOSH Alert: Preventing Occupational Exposures to Antineoplastic and Other Hazardous Drugs in Healthcare Settings. U.S. Department of Health and Human Services, Public Health Service. Centers for Disease Control and Prevention, National Institute for Occupational Safety and Health, DHHS (NIOSH) Publication No.2004-165. OSHA Technical Manual, TED 1-0. 15A, Section VI: Chapter 2. Controlling Occupational Exposure to Hazardous Drugs. OSHA, 1999. http://www.osha.gov/dts/osta/otm/otm_vi/otm_vi_2.html American Society of Health-System Pharmacists. (2006) ASHP Guidelines on Handling Hazardous Drugs. Am J Health-Syst Pharm. 2006;63:1172- 1193. Polovich M, White JM, Kelleher LO (eds.) 2005. Chemotherapy and Biotherapy Guidelines and Recommendations for Practice. (2 nd ed.) Pittsburgh, PA: Oncology Nursing Society."],"description":["DESCRIPTION Melphalan, also known as L-phenylalanine mustard, phenylalanine mustard, L-PAM, or L-sarcolysin, is a phenylalanine derivative of nitrogen mustard. Melphalan is a bifunctional alkylating agent that is active against selected human neoplastic diseases. It is known chemically as 4-[bis(2-chloroethyl)amino]- L -phenylalanine. The molecular formula is C 13 H 18 C1 2 N 2 O 2 and the molecular weight is 305.20. The structural formula is: Melphalan is the active L-isomer of the compound and was first synthesized in 1953 by Bergel and Stock; the D-isomer, known as medphalan, is less active against certain animal tumors, and the dose needed to produce effects on chromosomes is larger than that required with the L-isomer. The racemic (DL-) form is known as merphalan or sarcolysin. Melphalan is practically insoluble in water and has a pKa 1 of ~ 2.5. Melphalan Hydrochloride for Injection is supplied as a sterile, nonpyrogenic, freeze-dried powder. Each single-dose vial contains melphalan hydrochloride equivalent to 50 mg melphalan and 20 mg povidone. Melphalan Hydrochloride for Injection is reconstituted using the sterile diluent provided. Each vial of sterile diluent contains sodium citrate 0.2 g, propylene glycol 6.0 mL, ethanol (96%) 0.52 mL, and Water for Injection to a total of 10 mL. Melphalan Hydrochloride for Injection is administered intravenously. Structural Formula"],"precautions":["PRECAUTIONS General In all instances where the use of Melphalan Hydrochloride for Injection is considered for chemotherapy, the physician must evaluate the need and usefulness of the drug against the risk of adverse events. Melphalan should be used with extreme caution in patients whose bone marrow reserve may have been compromised by prior irradiation or chemotherapy or whose marrow function is recovering from previous cytotoxic therapy. Dose reduction should be considered in patients with renal insufficiency receiving IV melphalan. In one trial, increased bone marrow suppression was observed in patients with BUN levels ≥30 mg/dL. A 50% reduction in the IV melphalan dose decreased the incidence of severe bone marrow suppression in the latter portion of this study. Administration of live vaccines to immunocompromised patients should be avoided. Information for Patients Patients should be informed that the major acute toxicities of melphalan are related to bone marrow suppression, hypersensitivity reactions, gastrointestinal toxicity, and pulmonary toxicity. The major long-term toxicities are related to infertility and secondary malignancies. Patients should never be allowed to take the drug without close medical supervision and should be advised to consult their physicians if they experience skin rash, signs or symptoms of vasculitis, bleeding, fever, persistent cough, nausea, vomiting, amenorrhea, weight loss, or unusual lumps/masses. Women of childbearing potential should be advised to avoid becoming pregnant. Laboratory Tests Periodic complete blood counts with differentials should be performed during the course of treatment with melphalan. At least 1 determination should be obtained prior to each dose. Patients should be observed closely for consequences of bone marrow suppression, which include severe infections, bleeding, and symptomatic anemia (see WARNINGS ). Drug Interactions The development of severe renal failure has been reported in patients treated with a single dose of IV melphalan followed by standard oral doses of cyclosporine. Cisplatin may affect melphalan kinetics by inducing renal dysfunction and subsequently altering melphalan clearance. IV melphalan may also reduce the threshold for BCNU lung toxicity. When nalidixic acid and IV melphalan are given simultaneously, the incidence of severe hemorrhagic necrotic enterocolitis has been reported to increase in pediatric patients. Carcinogenesis, Mutagenesis, Impairment of Fertility: See WARNINGS section. Pregnancy Teratogenic Effects Pregnancy Category D: See WARNINGS section. Nursing Mothers It is not known whether this drug is excreted in human milk. IV melphalan should not be given to nursing mothers. Pediatric Use The safety and effectiveness in pediatric patients have not been established. Geriatric Use Clinical studies of Melphalan Hydrochloride for Injection did not include sufficient numbers of subjects aged 65 and over to determine whether they respond differently from younger subjects. Other reported clinical experience has not identified differences in responses between the elderly and younger patients. In general, dose selection for an elderly patient should be cautious, usually starting at the low end of the dosing range, reflecting the greater frequency of decreased hepatic, renal, or cardiac function, and of concomitant disease or other drug therapy."],"how_supplied":["HOW SUPPLIED Melphalan Hydrochloride for Injection is supplied as follows: NDC Melphalan Hydrochloride for Injection (Kit) Package Factor 84679-001-01 50 mg Single-Dose Vial of Melphalan Hydrochloride for Injection and 10 mL Vial of Sterile Diluent 1 vial per carton 1 vial per carton Each carton contains one single-dose clear glass vial of freeze-dried melphalan hydrochloride equivalent to 50 mg melphalan and one 10 mL clear glass vial of sterile diluent. Storage Conditions Store at 20° to 25°C (68° to 77°F). [See USP Controlled Room Temperature.] Protect from light. Discard unused portion. Sterile, Nonpyrogenic, Preservative-free. The container closure is not made with natural rubber latex."],"boxed_warning":["WARNING Melphalan should be administered under the supervision of a qualified physician experienced in the use of cancer chemotherapeutic agents. Severe bone marrow suppression with resulting infection or bleeding may occur. Controlled trials comparing intravenous (IV) to oral melphalan have shown more myelosuppression with the IV formulation. Hypersensitivity reactions, including anaphylaxis, have occurred in approximately 2% of patients who received the IV formulation. Melphalan is leukemogenic in humans. Melphalan produces chromosomal aberrations in vitro and in viv o and, therefore, should be considered potentially mutagenic in humans."],"geriatric_use":["Geriatric Use Clinical studies of Melphalan Hydrochloride for Injection did not include sufficient numbers of subjects aged 65 and over to determine whether they respond differently from younger subjects. Other reported clinical experience has not identified differences in responses between the elderly and younger patients. In general, dose selection for an elderly patient should be cautious, usually starting at the low end of the dosing range, reflecting the greater frequency of decreased hepatic, renal, or cardiac function, and of concomitant disease or other drug therapy."],"pediatric_use":["Pediatric Use The safety and effectiveness in pediatric patients have not been established."],"effective_time":"20260108","nursing_mothers":["Nursing Mothers It is not known whether this drug is excreted in human milk. IV melphalan should not be given to nursing mothers."],"laboratory_tests":["Laboratory Tests Periodic complete blood counts with differentials should be performed during the course of treatment with melphalan. At least 1 determination should be obtained prior to each dose. Patients should be observed closely for consequences of bone marrow suppression, which include severe infections, bleeding, and symptomatic anemia (see WARNINGS )."],"pharmacokinetics":["Pharmacokinetics Following injection, drug plasma concentrations declined rapidly in a biexponential manner with distribution phase and terminal elimination phase half-lives of approximately 10 and 75 minutes, respectively. Estimates of average total body clearance varied among studies, but typical values of approximately 7 to 9 mL/min/kg (250 to 325 mL/min/m 2 ) were observed. One study has reported that on repeat dosing of 0.5 mg/kg every 6 weeks, the clearance of melphalan decreased from 8.1 mL/min/kg after the first course, to 5.5 mL/min/kg after the third course, but did not decrease appreciably after the third course. Mean (±SD) peak melphalan plasma concentrations in myeloma patients given IV melphalan at doses of 10 or 20 mg/m 2 were 1.2 ± 0.4 and 2.8 ± 1.9 mcg/mL, respectively. The steady-state volume of distribution of melphalan is 0.5 L/kg. Penetration into cerebrospinal fluid (CSF) is low. The average melphalan binding to plasma proteins is highly variable (range: 53% to 92%). Serum albumin is the major binding protein, accounting for approximately 40% to 60% of the plasma protein binding, while α 1 -acid glycoprotein accounts for about 20% of the plasma protein binding. Approximately 30% of melphalan is (covalently) irreversibly bound to plasma proteins. Interactions with immunoglobulins have been found to be negligible. Melphalan is eliminated from plasma primarily by chemical hydrolysis to monohydroxymelphalan and dihydroxymelphalan. Aside from these hydrolysis products, no other melphalan metabolites have been observed in humans. Although the contribution of renal elimination to melphalan clearance appears to be low, one study noted an increase in the occurrence of severe leukopenia in patients with elevated BUN after 10 weeks of therapy."],"adverse_reactions":["ADVERSE REACTIONS (SEE OVERDOSAGE) The following information on adverse reactions is based on data from both oral and IV administration of melphalan as a single agent, using several different dose schedules for treatment of a wide variety of malignancies. Hematologic The most common side effect is bone marrow suppression leading to leukopenia, thrombocytopenia, and anemia. White blood cell count and platelet count nadirs usually occur 2 to 3 weeks after treatment, with recovery in 4 to 5 weeks after treatment. Irreversible bone marrow failure has been reported. Gastrointestinal Gastrointestinal disturbances such as nausea and vomiting, diarrhea, and oral ulceration occur infrequently. Hepatic disorders ranging from abnormal liver function tests to clinical manifestations such as hepatitis and jaundice have been reported. Hepatic veno-occlusive disease has been reported. Hypersensitivity Acute hypersensitivity reactions including anaphylaxis were reported in 2.4% of 425 patients receiving Melphalan Hydrochloride for Injection for myeloma (see WARNINGS ). These reactions were characterized by urticaria, pruritus, edema, skin rashes, and in some patients, tachycardia, bronchospasm, dyspnea, and hypotension. These patients appeared to respond to antihistamine and corticosteroid therapy. If a hypersensitivity reaction occurs, IV or oral melphalan should not be readministered since hypersensitivity reactions have also been reported with oral melphalan. Cardiac arrest has also been reported rarely in association with such reports. Miscellaneous Other reported adverse reactions include skin hypersensitivity, skin ulceration at injection site, skin necrosis rarely requiring skin grafting, maculopapular rashes, vasculitis, alopecia, hemolytic anemia, allergic reaction, pulmonary fibrosis (including fatal outcomes), and interstitial pneumonitis. Temporary significant elevation of the blood urea has been seen in the early stages of therapy in patients with renal damage. Subjective and transient sensation of warmth and/or tingling. To report SUSPECTED ADVERSE REACTIONS, contact QILU PHARMA, INC. at 484-838-0633/484-875-3013 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch ."],"contraindications":["CONTRAINDICATIONS Melphalan should not be used in patients whose disease has demonstrated prior resistance to this agent. Patients who have demonstrated hypersensitivity to melphalan should not be given the drug."],"drug_interactions":["Drug Interactions The development of severe renal failure has been reported in patients treated with a single dose of IV melphalan followed by standard oral doses of cyclosporine. Cisplatin may affect melphalan kinetics by inducing renal dysfunction and subsequently altering melphalan clearance. IV melphalan may also reduce the threshold for BCNU lung toxicity. When nalidixic acid and IV melphalan are given simultaneously, the incidence of severe hemorrhagic necrotic enterocolitis has been reported to increase in pediatric patients."],"how_supplied_table":["
NDCMelphalan Hydrochloride for Injection (Kit)Package Factor
84679-001-0150 mg Single-Dose Vial of Melphalan Hydrochloride for Injection and 10 mL Vial of Sterile Diluent 1 vial per carton 1 vial per carton
"],"general_precautions":["General In all instances where the use of Melphalan Hydrochloride for Injection is considered for chemotherapy, the physician must evaluate the need and usefulness of the drug against the risk of adverse events. Melphalan should be used with extreme caution in patients whose bone marrow reserve may have been compromised by prior irradiation or chemotherapy or whose marrow function is recovering from previous cytotoxic therapy. Dose reduction should be considered in patients with renal insufficiency receiving IV melphalan. In one trial, increased bone marrow suppression was observed in patients with BUN levels ≥30 mg/dL. A 50% reduction in the IV melphalan dose decreased the incidence of severe bone marrow suppression in the latter portion of this study. Administration of live vaccines to immunocompromised patients should be avoided."],"teratogenic_effects":["Teratogenic Effects Pregnancy Category D: See WARNINGS section."],"storage_and_handling":["Storage Conditions Store at 20° to 25°C (68° to 77°F). [See USP Controlled Room Temperature.] Protect from light. Discard unused portion. Sterile, Nonpyrogenic, Preservative-free. The container closure is not made with natural rubber latex."],"clinical_pharmacology":["CLINICAL PHARMACOLOGY Melphalan is an alkylating agent of the bischloroethylamine type. As a result, its cytotoxicity appears to be related to the extent of its interstrand cross-linking with DNA, probably by binding at the N 7 position of guanine. Like other bifunctional alkylating agents, it is active against both resting and rapidly dividing tumor cells. Pharmacokinetics Following injection, drug plasma concentrations declined rapidly in a biexponential manner with distribution phase and terminal elimination phase half-lives of approximately 10 and 75 minutes, respectively. Estimates of average total body clearance varied among studies, but typical values of approximately 7 to 9 mL/min/kg (250 to 325 mL/min/m 2 ) were observed. One study has reported that on repeat dosing of 0.5 mg/kg every 6 weeks, the clearance of melphalan decreased from 8.1 mL/min/kg after the first course, to 5.5 mL/min/kg after the third course, but did not decrease appreciably after the third course. Mean (±SD) peak melphalan plasma concentrations in myeloma patients given IV melphalan at doses of 10 or 20 mg/m 2 were 1.2 ± 0.4 and 2.8 ± 1.9 mcg/mL, respectively. The steady-state volume of distribution of melphalan is 0.5 L/kg. Penetration into cerebrospinal fluid (CSF) is low. The average melphalan binding to plasma proteins is highly variable (range: 53% to 92%). Serum albumin is the major binding protein, accounting for approximately 40% to 60% of the plasma protein binding, while α 1 -acid glycoprotein accounts for about 20% of the plasma protein binding. Approximately 30% of melphalan is (covalently) irreversibly bound to plasma proteins. Interactions with immunoglobulins have been found to be negligible. Melphalan is eliminated from plasma primarily by chemical hydrolysis to monohydroxymelphalan and dihydroxymelphalan. Aside from these hydrolysis products, no other melphalan metabolites have been observed in humans. Although the contribution of renal elimination to melphalan clearance appears to be low, one study noted an increase in the occurrence of severe leukopenia in patients with elevated BUN after 10 weeks of therapy. Clinical Trial A randomized trial compared prednisone plus IV melphalan to prednisone plus oral melphalan in the treatment of myeloma. As discussed below, overall response rates at week 22 were comparable; however, because of changes in trial design, conclusions as to the relative activity of the 2 formulations after week 22 are impossible to make. Both arms received oral prednisone starting at 0.8 mg/kg/day with doses tapered over 6 weeks. Melphalan doses in each arm were: Arm 1: Oral melphalan 0.15 mg/kg/day x 7 followed by 0.05 mg/kg/day when WBC began to rise. Arm 2: IV melphalan 16 mg/m 2 q 2 weeks x 4 (over 6 weeks) followed by the same dose every 4 weeks. Doses of melphalan were adjusted according to the following criteria: Table 1: Criteria for Dosage Adjustment in a Randomized Clinical Trial WBC/mm 3 Platelets Percent of Full Dose ≥4,000 ≥100,000 100 ≥3,000 ≥75,000 75 ≥2,000 ≥50,000 50 <2,000 <50,000 0 One hundred seven patients were randomized to the oral melphalan arm and 203 patients to the IV melphalan arm. More patients had a poor-risk classification (58% versus 44%) and high tumor load (51% versus 34%) on the oral compared to the IV arm ( P <0.04). Response rates at week 22 are shown in the following table: Table 2: Response Rates at Week 22 Initial Arm Evaluable Patients Responders n (%) P Oral melphalan 100 44 (44%) P > 0.2 IV melphalan 195 74 (38%) Because of changes in protocol design after week 22, other efficacy parameters such as response duration and survival cannot be compared. Severe myelotoxicity (WBC ≤1,000 and/or platelets ≤25,000) was more common in the IV melphalan arm (28%) than in the oral melphalan arm (11%). An association was noted between poor renal function and myelosuppression; consequently, an amendment to the protocol required a 50% reduction in IV melphalan dose if the BUN was ≥30 mg/dL. The rate of severe leukopenia in the IV arm in the patients with BUN over 30 mg/dL decreased from 50% (8/16) before protocol amendment to 11% (3/28) (P = 0.01) after the amendment. Before the dosing amendment, there was a 10% (8/77) incidence of drug-related death in the IV arm. After the dosing amendment, this incidence was 3% (3/108). This compares to an overall 1% (1/100) incidence of drug-related death in the oral arm."],"indications_and_usage":["INDICATIONS AND USAGE Melphalan Hydrochloride for Injection is indicated for the palliative treatment of patients with multiple myeloma for whom oral therapy is not appropriate."],"information_for_patients":["Information for Patients Patients should be informed that the major acute toxicities of melphalan are related to bone marrow suppression, hypersensitivity reactions, gastrointestinal toxicity, and pulmonary toxicity. The major long-term toxicities are related to infertility and secondary malignancies. Patients should never be allowed to take the drug without close medical supervision and should be advised to consult their physicians if they experience skin rash, signs or symptoms of vasculitis, bleeding, fever, persistent cough, nausea, vomiting, amenorrhea, weight loss, or unusual lumps/masses. Women of childbearing potential should be advised to avoid becoming pregnant."],"spl_unclassified_section":["Rx only"],"dosage_and_administration":["DOSAGE AND ADMINISTRATION The usual IV dose is 16 mg/m 2 . Dosage reduction of up to 50% should be considered in patients with renal insufficiency (BUN ≥30 mg/dL) (see PRECAUTIONS, General ). The drug is administered as a single infusion over 15 to 20 minutes. Melphalan is administered at 2-week intervals for 4 doses, then, after adequate recovery from toxicity, at 4-week intervals. Available evidence suggests about one third to one half of the patients with multiple myeloma show a favorable response to the drug. Experience with oral melphalan suggests that repeated courses should be given since improvement may continue slowly over many months, and the maximum benefit may be missed if treatment is abandoned prematurely. Dose adjustment on the basis of blood cell counts at the nadir and day of treatment should be considered. Administration Precautions As with other toxic compounds, caution should be exercised in handling and preparing the solution of Melphalan Hydrochloride for Injection. Skin reactions associated with accidental exposure may occur. The use of gloves is recommended. If the solution of Melphalan Hydrochloride for Injection contacts the skin ormucosa, immediately wash the skin or mucosa thoroughly with soap and water. Procedures for proper handling and disposal of anticancer drugs should be considered. Several guidelines on this subject have been published. 1-4 There is no general agreement that all of the procedures recommended in the guidelines are necessary or appropriate. Parenteral drug products should be visually inspected for particulate matter and discoloration prior to administration whenever solution and container permit. If either occurs, do not use this product. Care should be taken to avoid possible extravasation of melphalan and in cases of poor peripheral venous access, consideration should be given to use of a central venous line (see WARNINGS ). Preparation for Administration/Stability Melphalan Hydrochloride for Injection must be reconstituted by rapidly injecting 10 mL of the supplied diluent directly into the vial of lyophilized powder using a sterile needle (20-gauge or larger needle diameter) and syringe. Immediately shake vial vigorously until a clear solution is obtained. This provides a 5-mg/mL solution of melphalan. Rapid addition of the diluent followed by immediate vigorous shaking is important for proper dissolution. Immediately dilute the dose to be administered in 0.9% Sodium Chloride Injection, USP, to a concentration not greater than 0.45 mg/mL. Administer the diluted product over a minimum of 15 minutes. Complete administration within 60 minutes of reconstitution. The time between reconstitution/dilution and administration of Melphalan Hydrochloride for Injection should be kept to a minimum because reconstituted and diluted solutions of Melphalan Hydrochloride for Injection are unstable. Over as short a time as 30 minutes, a citrate derivative of melphalan has been detected in reconstituted material from the reaction of Melphalan Hydrochloride for Injection with Sterile Diluent for Melphalan Hydrochloride for Injection. Upon further dilution with saline, nearly 1% label strength of melphalan hydrolyzes every 10 minutes. A precipitate forms if the reconstituted solution is stored at 5°C. DO NOT REFRIGERATE THE RECONSTITUTED PRODUCT."],"spl_product_data_elements":["Melphalan Hydrochloride melphalan hydrochloride Melphalan Hydrochloride melphalan hydrochloride POVIDONE MELPHALAN HYDROCHLORIDE MELPHALAN Sterile Diluent for Melphalan Hydrochloride sterile diluent for melphalan hydrochloride SODIUM CITRATE, UNSPECIFIED FORM PROPYLENE GLYCOL ALCOHOL WATER"],"clinical_pharmacology_table":["
Table 1: Criteria for Dosage Adjustment in a Randomized Clinical Trial
WBC/mm 3PlateletsPercent of Full Dose
≥4,000≥100,000100
≥3,000≥75,00075
≥2,000≥50,00050
<2,000<50,0000
","
Table 2: Response Rates at Week 22
Initial ArmEvaluable Patients Responders n (%) P
Oral melphalan10044 (44%)P> 0.2
IV melphalan19574 (38%)
"],"package_label_principal_display_panel":["PACKAGE LABEL – PRINCIPAL DISPLAY PANEL – Carton Label Single-Dose Vial Melphalan Hydrochloride for Injection 50 mg per vial Caution: Cytotoxic Agent One vial of Melphalan Hydrochloride for Injection containing: sterile, nonpyrogenic, freeze-dried melphalan hydrochloride equivalent to 50 mg melphalan and 20 mg povidone. One vial of sterile, nonpyrogenic diluent containing: 0.2 grams sodium citrate, 6.0 mL propylene glycol, 0.52 mL ethanol (96%) and water for injection to a total of 10 mL. Rx only For Intravenous Infusion Carton Label"],"carcinogenesis_and_mutagenesis_and_impairment_of_fertility":["Carcinogenesis, Mutagenesis, Impairment of Fertility: See WARNINGS section."]},"tags":[{"label":"Alkylating Drug","category":"class"},{"label":"Small Molecule","category":"modality"},{"label":"L01AA03","category":"atc"},{"label":"Oral","category":"route"},{"label":"Intravenous","category":"route"},{"label":"Injection","category":"form"},{"label":"Tablet","category":"form"},{"label":"Off-Patent","category":"patent"},{"label":"Generic Available","category":"availability"},{"label":"Mature","category":"status"},{"label":"Malignant tumor of ovary","category":"indication"},{"label":"Multiple myeloma","category":"indication"},{"label":"Apotex","category":"company"},{"label":"Approved 1960s","category":"decade"},{"label":"Alkylating Agents","category":"pharmacology"},{"label":"Antineoplastic Agents","category":"pharmacology"},{"label":"Antineoplastic Agents, Alkylating","category":"pharmacology"},{"label":"Immunologic Factors","category":"pharmacology"},{"label":"Immunosuppressive Agents","category":"pharmacology"},{"label":"Myeloablative Agonists","category":"pharmacology"},{"label":"Noxae","category":"pharmacology"}],"phase":"marketed","safety":{"boxedWarnings":["WARNING Melphalan should be administered under the supervision of a qualified physician experienced in the use of cancer chemotherapeutic agents. Severe bone marrow suppression with resulting infection or bleeding may occur. Controlled trials comparing intravenous (IV) to oral melphalan have shown more myelosuppression with the IV formulation. Hypersensitivity reactions, including anaphylaxis, have occurred in approximately 2% of patients who received the IV formulation. Melphalan is leukemogenic in humans. Melphalan produces chromosomal aberrations in vitro and in viv o and, therefore, should be considered potentially mutagenic in humans."],"safetySignals":[{"date":"","signal":"OFF LABEL USE","source":"FDA FAERS","actionTaken":"2392 reports"},{"date":"","signal":"DRUG INEFFECTIVE","source":"FDA FAERS","actionTaken":"1433 reports"},{"date":"","signal":"FEBRILE NEUTROPENIA","source":"FDA FAERS","actionTaken":"1285 reports"},{"date":"","signal":"PLASMA CELL MYELOMA","source":"FDA FAERS","actionTaken":"1239 reports"},{"date":"","signal":"NEUTROPENIA","source":"FDA FAERS","actionTaken":"1213 reports"},{"date":"","signal":"PYREXIA","source":"FDA FAERS","actionTaken":"1179 reports"},{"date":"","signal":"PNEUMONIA","source":"FDA FAERS","actionTaken":"1142 reports"},{"date":"","signal":"MUCOSAL INFLAMMATION","source":"FDA FAERS","actionTaken":"1138 reports"},{"date":"","signal":"THROMBOCYTOPENIA","source":"FDA FAERS","actionTaken":"1120 reports"},{"date":"","signal":"DIARRHOEA","source":"FDA FAERS","actionTaken":"1075 reports"}],"commonSideEffects":[{"effect":"Bone marrow suppression","drugRate":"","severity":"serious","_validated":false,"_confidence":0.3},{"effect":"Leukopenia","drugRate":"","severity":"serious","_validated":false,"_confidence":0.3},{"effect":"Thrombocytopenia","drugRate":"","severity":"serious","_validated":false,"_confidence":0.3},{"effect":"Anemia","drugRate":"","severity":"serious","_validated":false,"_confidence":0.3},{"effect":"Hepatitis","drugRate":"","severity":"serious","_validated":false,"_confidence":0.3},{"effect":"Jaundice","drugRate":"","severity":"serious","_validated":false,"_confidence":0.3},{"effect":"Pulmonary fibrosis","drugRate":"","severity":"serious","_validated":false,"_confidence":0.3},{"effect":"Interstitial pneumonitis","drugRate":"","severity":"serious","_validated":false,"_confidence":0.3},{"effect":"Vasculitis","drugRate":"","severity":"serious","_validated":false,"_confidence":0.3},{"effect":"Cardiac arrest","drugRate":"<5%","severity":"mild","_validated":true},{"effect":"Nausea","drugRate":"<5%","severity":"mild","_validated":true},{"effect":"Vomiting","drugRate":"<5%","severity":"mild","_validated":true},{"effect":"Diarrhea","drugRate":"<5%","severity":"mild","_validated":true},{"effect":"Oral ulceration","drugRate":"<5%","severity":"mild","_validated":true},{"effect":"Skin hypersensitivity","drugRate":"<5%","severity":"mild","_validated":true},{"effect":"Maculopapular rashes","drugRate":"<5%","severity":"mild","_validated":true},{"effect":"Alopecia","drugRate":"<5%","severity":"mild","_validated":true},{"effect":"Urticaria","drugRate":"<5%","severity":"mild","_validated":true},{"effect":"Edema","drugRate":"<5%","severity":"mild","_validated":true},{"effect":"Skin rashes","drugRate":"<5%","severity":"mild","_validated":true},{"effect":"Anaphylaxis","drugRate":"<5%","severity":"mild","_validated":true}],"contraindications":["Acute hemorrhage","Acute infectious disease","Anemia","Bone marrow depression","Breastfeeding (mother)","Drug-induced hepatitis","Fibrosis of lung","Granulocytopenic disorder","Hemolytic anemia","Interstitial pneumonia","Kidney disease","Leukopenia","Liver function tests abnormal","Portal vein obstruction","Pregnancy, function","Radiation oncology AND/OR radiotherapy","Thrombocytopenic disorder","Vasculitis"],"specialPopulations":{"Pregnancy":"Risk SummaryBased on its mechanism of action, Evomela can cause fetal harm when administered to pregnant woman, including teratogenicity and/or embryo-fetal lethality [see Clinical Pharmacology (12.1)]. Melphalan is genotoxic drug and can cause chromatid or chromosome damage in humans [see Nonclinical Toxicology (13.1)]. In animal studies, melphalan was embryolethal and teratogenic in rats at doses below the recommended clinical doses [see Data]. Advise pregnant woman of the potential risk to fetus..The background risk of major birth defects and miscarriage for the indicated populations are unknown. However, the background risk in the U.S. general population of major birth defects is 2-4% and of miscarriage is 15-20% of clinically recognized pregnancies.","Geriatric use":"Clinical studies of Melphalan Tablets USP did not include sufficient numbers of subjects aged 65 and over to determine whether they respond differently from younger subjects. Other reported clinical experience has not identified differences in responses between the elderly and younger patients. In general, dose selection for an elderly patient should be cautious, usually starting at the low end of the dosing range, reflecting the greater frequency of decreased hepatic, renal, or cardiac function and of concomitant diseases and other drug therapy.","Paediatric use":"The safety and effectiveness of Melphalan Tablets USP in pediatric patients have not been established."},"seriousAdverseEvents":[{"event":"Acute hypersensitivity reactions including anaphylaxis","detail":"Reported in 2.4% of 425 patients receiving Melphalan Hydrochloride for Injection for myeloma.","severity":"serious","incidence":"2.4%"}]},"trials":[],"aliases":[],"company":"Apotex","patents":[],"pricing":[],"_sources":{"trials":{"url":"https://clinicaltrials.gov/search?intr=melphalan","method":"api_direct","source":"ClinicalTrials.gov","rawText":"","confidence":1,"sourceType":"ctgov","retrievedAt":"2026-04-19T23:47:29.221342+00:00"},"timeline":{"url":"https://en.wikipedia.org/wiki/Melphalan","method":"deterministic","source":"Wikipedia","rawText":"","confidence":0.8,"sourceType":"wikipedia","retrievedAt":"2026-04-19T23:47:37.560115+00:00"},"regulatory.ca":{"url":"","method":"api_direct","source":"Health Canada DPD","rawText":"","confidence":1,"sourceType":"health_canada_dpd","retrievedAt":"2026-04-19T23:47:36.162721+00:00"},"regulatory.us":{"url":"","method":"api_direct","source":"FDA Drugs@FDA","rawText":"","confidence":1,"sourceType":"fda_drugsfda","retrievedAt":"2026-04-19T23:47:27.861395+00:00"},"publicationCount":{"url":"https://pubmed.ncbi.nlm.nih.gov/?term=melphalan","method":"api_direct","source":"PubMed/NCBI","rawText":"","confidence":1,"sourceType":"pubmed","retrievedAt":"2026-04-19T23:47:36.974631+00:00"},"administration.route":{"url":"","method":"deterministic","source":"FDA Label","rawText":"","confidence":1,"sourceType":"fda_label","retrievedAt":"2026-04-19T23:47:26.708596+00:00"},"safety.boxedWarnings":{"url":"","method":"deterministic","source":"FDA Label (boxed_warning)","rawText":"WARNING Melphalan should be administered under the supervision of a qualified physician experienced in the use of cancer chemotherapeutic agents. Severe bone marrow suppression with resulting infection or bleeding may occur. Controlled trials comparing intravenous (IV) to oral melphalan have shown more myelosuppression with the IV formulation. Hypersensitivity reactions, including anaphylaxis, have occurred in approximately 2% of patients who received the IV formulation. Melphalan is leukemogeni","confidence":1,"sourceType":"fda_label","retrievedAt":"2026-04-19T23:47:26.708661+00:00"},"safety.safetySignals":{"url":"https://api.fda.gov/drug/event.json","method":"api_direct","source":"FDA FAERS","rawText":"","confidence":1,"sourceType":"fda_faers","retrievedAt":"2026-04-19T23:47:38.563179+00:00"},"mechanism.target_chembl":{"url":"","method":"api_direct","source":"ChEMBL mechanism: DNA inhibitor","rawText":"","confidence":1,"sourceType":"chembl","retrievedAt":"2026-04-19T23:47:37.559947+00:00"},"crossReferences.chemblId":{"url":"https://www.ebi.ac.uk/chembl/compound_report_card/CHEMBL1200863/","method":"api_direct","source":"ChEMBL (EMBL-EBI)","rawText":"","confidence":1,"sourceType":"chembl","retrievedAt":"2026-04-19T23:47:37.446184+00:00"},"safety.commonSideEffects":{"url":"","method":"ai_extraction","source":"FDA Label + AI","aiModel":"featherless","rawText":"ADVERSE REACTIONS (SEE OVERDOSAGE) The following information on adverse reactions is based on data from both oral and IV administration of melphalan as a single agent, using several different dose schedules for treatment of a wide variety of malignancies. Hematologic The most common side effect is bone marrow suppression leading to leukopenia, thrombocytopenia, and anemia. White blood cell count and platelet count nadirs usually occur 2 to 3 weeks after treatment, with recovery in 4 to 5 weeks a","confidence":0.95,"sourceType":"fda_label","retrievedAt":"2026-04-19T23:47:45.909249+00:00"},"regulatory.fda_application":{"url":"","method":"deterministic","source":"FDA Label","rawText":"ANDA211463","confidence":1,"sourceType":"fda_label","retrievedAt":"2026-04-19T23:47:26.708673+00:00"}},"allNames":"alkeran","offLabel":[],"synonyms":["melphalan hydrochloride","melphalan","alkeran","levofalan","levofolan","levopholan","melfalan","phenylalanine mustard","sarcoclorin","melphalan HCl"],"timeline":[{"date":"1964-01-01","type":"neutral","source":"FDA Orange Book","milestone":"Rights transferred from GLAXOSMITHKLINE to Apotex"},{"date":"2017-03-22","type":"neutral","source":"FDA Orange Book","milestone":"Generic entry — 1 manufacturer approved"}],"aiSummary":"Alkeran (melphalan) is a small molecule alkylating drug originally developed by GlaxoSmithKline and currently owned by Apotex. It was first approved by the FDA in 1964 for the treatment of malignant ovarian tumors and multiple myeloma. As an off-patent medication, Alkeran is available as a generic product. Key safety considerations include its short half-life of 1 hour and moderate bioavailability of 71%. Alkeran is used to treat certain types of cancer.","brandName":"Alkeran","ecosystem":[{"indication":"Malignant tumor of ovary","otherDrugs":[{"name":"altretamine","slug":"altretamine","company":"Eisai Inc"},{"name":"carboplatin","slug":"carboplatin","company":""},{"name":"cisplatin","slug":"cisplatin","company":"Hq Spclt Pharma"},{"name":"doxorubicin","slug":"doxorubicin","company":""}],"globalPrevalence":null},{"indication":"Multiple myeloma","otherDrugs":[{"name":"belantamab mafodotin","slug":"belantamab-mafodotin","company":"Glaxosmithkline"},{"name":"bortezomib","slug":"bortezomib","company":"Millennium Pharms"},{"name":"carfilzomib","slug":"carfilzomib","company":"Onyx Pharms"},{"name":"carmustine","slug":"carmustine","company":"Emcure Pharms Ltd"}],"globalPrevalence":176000}],"mechanism":{"novelty":"Follow-on","moaClass":"Alkylating Activity","modality":"Small Molecule","drugClass":"Alkylating Drug","explanation":"","oneSentence":"","technicalDetail":"Melphalan is an alkylating agent that cross-links DNA strands, thereby inhibiting DNA replication and transcription, and ultimately inducing cell death in rapidly dividing cancer cells."},"_wikipedia":{"url":"https://en.wikipedia.org/wiki/Melphalan","title":"Melphalan","extract":"Melphalan, sold under the brand name Alkeran among others, is a chemotherapy medication used to treat multiple myeloma; malignant lymphoma; lymphoblastic and myeloblastic leukemia; childhood neuroblastoma; ovarian cancer; mammary adenocarcinoma; and uveal melanoma. It is taken by mouth or by injection into a vein.","wiki_society_and_culture":"== Society and culture ==\n=== Legal status ===\nOn 17 September 2020, the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) adopted a positive opinion, recommending the granting of a marketing authorization for melphalan. The applicant for this medicinal product is Adienne S.r.l. S.U."},"commercial":{"launchDate":"1964","_launchSource":"DrugCentral (FDA 1964-01-17, GLAXOSMITHKLINE)"},"references":[{"id":1,"url":"https://drugcentral.org/drugcard/1678","fields":["approvals","synonyms","ATC","PK","indications","contraindications","DDIs","targets","patents","FAERS"],"source":"DrugCentral"},{"id":2,"url":"https://clinicaltrials.gov/search?intr=melphalan","fields":["trials"],"source":"ClinicalTrials.gov"},{"id":3,"url":"https://pubmed.ncbi.nlm.nih.gov/?term=melphalan","fields":["publications"],"source":"PubMed/NCBI"},{"id":4,"url":"https://en.wikipedia.org/wiki/Melphalan","fields":["history","overview"],"source":"Wikipedia"},{"id":5,"url":"https://www.fda.gov/drugs/drug-approvals-and-databases/orange-book-data-files","fields":["patents","exclusivity","genericManufacturers"],"source":"FDA Orange Book"}],"_enrichedAt":"2026-03-30T02:26:27.179962","_validation":{"fieldsValidated":1,"lastValidatedAt":"2026-04-19T23:47:48.629705+00:00","fieldsConflicting":10,"overallConfidence":0.8},"biosimilars":[],"competitors":[{"drugName":"cyclophosphamide","drugSlug":"cyclophosphamide","fdaApproval":"1959-11-16","patentExpiry":"Jun 26, 2035","patentStatus":"Patent protected","relationship":"same-class"},{"drugName":"chlorambucil","drugSlug":"chlorambucil","fdaApproval":"1957-03-18","patentStatus":"Unknown","relationship":"same-class"},{"drugName":"mechlorethamine","drugSlug":"mechlorethamine","fdaApproval":"1949-03-15","patentExpiry":"Mar 7, 2026","patentStatus":"Patent protected","relationship":"same-class"},{"drugName":"ifosfamide","drugSlug":"ifosfamide","fdaApproval":"1988-12-30","genericCount":4,"patentStatus":"Off-patent — generic available","relationship":"same-class"},{"drugName":"bendamustine","drugSlug":"bendamustine","fdaApproval":"2008-03-20","patentExpiry":"Sep 26, 2029","patentStatus":"Patent protected","relationship":"same-class"},{"drugName":"melphalan flufenamide","drugSlug":"melphalan-flufenamide","fdaApproval":"2021-02-26","relationship":"same-class"}],"genericName":"melphalan","indications":{"approved":[{"name":"Malignant tumor of ovary","source":"DrugCentral","snomedId":363443007,"regulator":"FDA","eligibility":"palliation of non-resectable epithelial carcinoma of the ovary"},{"name":"Multiple myeloma","source":"DrugCentral","snomedId":109989006,"regulator":"FDA","usPrevalence":35000,"globalPrevalence":176000,"prevalenceMethod":"curated","prevalenceSource":"IARC GLOBOCAN, 2022"}],"offLabel":[{"name":"Allogeneic bone marrow transplantation","source":"DrugCentral","drugName":"melphalan","evidenceCount":507,"evidenceLevel":"strong"},{"name":"Diffuse non-Hodgkin's lymphoma, large cell","source":"DrugCentral","drugName":"melphalan","evidenceCount":166,"evidenceLevel":"strong"}],"pipeline":[]},"currentOwner":"Apotex","drugCategory":"mature","labelChanges":[],"patentStatus":"Off-patent — no active Orange Book patents","relatedDrugs":[{"drugId":"cyclophosphamide","brandName":"cyclophosphamide","genericName":"cyclophosphamide","approvalYear":"1959","relationship":"same-class"},{"drugId":"chlorambucil","brandName":"chlorambucil","genericName":"chlorambucil","approvalYear":"1957","relationship":"same-class"},{"drugId":"mechlorethamine","brandName":"mechlorethamine","genericName":"mechlorethamine","approvalYear":"1949","relationship":"same-class"},{"drugId":"ifosfamide","brandName":"ifosfamide","genericName":"ifosfamide","approvalYear":"1988","relationship":"same-class"},{"drugId":"bendamustine","brandName":"bendamustine","genericName":"bendamustine","approvalYear":"2008","relationship":"same-class"},{"drugId":"melphalan-flufenamide","brandName":"melphalan flufenamide","genericName":"melphalan flufenamide","approvalYear":"2021","relationship":"same-class"}],"trialDetails":[{"nctId":"NCT06172296","phase":"PHASE3","title":"Dinutuximab With Chemotherapy, Surgery and Stem Cell Transplantation for the Treatment of Children With Newly Diagnosed High Risk Neuroblastoma","status":"RECRUITING","sponsor":"National Cancer Institute (NCI)","startDate":"2024-04-19","conditions":["Ganglioneuroblastoma, Nodular","Neuroblastoma"],"enrollment":478,"completionDate":"2029-12-31"},{"nctId":"NCT02443077","phase":"PHASE3","title":"Ibrutinib Before and After Stem Cell Transplant in Treating Patients With Relapsed or Refractory Diffuse Large B-cell Lymphoma","status":"ACTIVE_NOT_RECRUITING","sponsor":"National Cancer Institute (NCI)","startDate":"2016-10-12","conditions":["Recurrent Diffuse Large B-Cell Lymphoma Activated B-Cell Type","Refractory Diffuse Large B-Cell Lymphoma Activated B-Cell 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Disorders"],"enrollment":220,"completionDate":"2031-12"},{"nctId":"NCT07281924","phase":"PHASE1,PHASE2","title":"Hepzato Kit and Opdualag for Metastatic Melanoma and Liver Metastasis","status":"RECRUITING","sponsor":"University of Wisconsin, Madison","startDate":"2026-04","conditions":["Metastatic Melanoma","Liver Metastases"],"enrollment":15,"completionDate":"2030-12"},{"nctId":"NCT01453088","phase":"PHASE2","title":"Auto Transplant High Dose Melphalan vs High Dose Melphalan+Bortezomib in Pts With Multiple Myeloma Age 65 Years or Older","status":"TERMINATED","sponsor":"Hackensack Meridian Health","startDate":"2010-06-24","conditions":["Multiple Myeloma"],"enrollment":63,"completionDate":"2022-05-01"},{"nctId":"NCT05436418","phase":"PHASE1,PHASE2","title":"The Lowest Effective Dose of Post-Transplantation Cyclophosphamide in Combination With Sirolimus and Mycophenolate Mofetil as Graft-Versus-Host Disease Prophylaxis After Reduced Intensity Conditioning and Peripheral Blood Stem Cell Transplantation","status":"RECRUITING","sponsor":"National Cancer Institute (NCI)","startDate":"2022-11-18","conditions":["Peripheral Blood Stem Cell Transplantation","Hematopoietic Stem Cell Transplantation"],"enrollment":260,"completionDate":"2028-06-25"},{"nctId":"NCT07481669","phase":"PHASE2","title":"ASCT With TEAM Conditioning for Lymphoma With High Risk of CNS Relapse","status":"RECRUITING","sponsor":"Second Affiliated Hospital, School of Medicine, Zhejiang University","startDate":"2026-03-01","conditions":["Lymphoma Patients With High-risk of Central Nervous System Relapse"],"enrollment":45,"completionDate":"2028-03-31"},{"nctId":"NCT04904588","phase":"PHASE2","title":"HLA-Mismatched Unrelated Donor Hematopoietic Cell Transplantation With Post-Transplantation Cyclophosphamide","status":"ACTIVE_NOT_RECRUITING","sponsor":"Center for International Blood and Marrow Transplant Research","startDate":"2021-09-30","conditions":["Acute Lymphoblastic Leukemia","Acute Myelogenous Leukemia","Mixed Phenotype Acute Leukemia","Acute Leukemia","Myelodysplastic Syndromes","Chronic Myelogenous Leukemia","Chronic Lymphocytic Leukemia","Lymphoma"],"enrollment":300,"completionDate":"2026-12"},{"nctId":"NCT03494569","phase":"PHASE1","title":"Total Marrow and Lymphoid Irradiation, Fludarabine, and Melphalan Before Donor Stem Cell Transplant in Treating Participants With High-Risk Acute Leukemia or Myelodysplastic Syndrome","status":"SUSPENDED","sponsor":"City of Hope Medical Center","startDate":"2018-07-06","conditions":["Acute Lymphoblastic Leukemia","Acute Lymphoblastic Leukemia in Remission","Acute Myeloid Leukemia","Acute Myeloid Leukemia in Remission","Hematopoietic Cell Transplantation Recipient","Minimal Residual Disease","Myelodysplastic Syndrome","Secondary Acute Myeloid Leukemia"],"enrollment":36,"completionDate":"2026-06-11"},{"nctId":"NCT07396480","phase":"PHASE3","title":"Fludarabine Plus Melphalan Versus Addition of Venetoclax to Fludarabine/Melphalan 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Tumors"],"enrollment":60,"completionDate":"2030-01"},{"nctId":"NCT06607458","phase":"PHASE2","title":"Evaluation of the Safety and Efficacy of Treatment w/High Dose Melphalan Given Directly Into the Liver Followed by Treatment w/Approved Cancer Treatment or Approved Cancer Treatment Alone in Patients w/ Metastatic Colorectal Cancer w/Liver Dominant Disease","status":"RECRUITING","sponsor":"Delcath Systems Inc.","startDate":"2025-08-05","conditions":["Refractory Metastatic Colorectal Cancer"],"enrollment":90,"completionDate":"2027-10"},{"nctId":"NCT06875128","phase":"PHASE2","title":"Evaluation of the Safety and Efficacy of Treatment w/High Dose Melphalan Given Directly Into the Liver Followed by Treatment w/Approved Cancer Treatment or Approved Cancer Treatment Alone in Patients w/ Metastatic Breast Cancer w/Liver Dominant Disease","status":"RECRUITING","sponsor":"Delcath Systems Inc.","startDate":"2026-01-29","conditions":["Metastatic Breast Cancer in the 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