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Stem Cell Transplantation in Patients With High-Risk and Recurrent Pediatric Sarcomas
Phase 2 trial testing F-18 Fluorodeoxyglucose in Sarcoma in 60 participants. Completed in 14 December 2011.
| Lead sponsor | National Cancer Institute (NCI) |
|---|---|
| Phase | Phase 2 |
| Status | Completed |
| Study type | INTERVENTIONAL |
| Allocation | non randomized |
| Design | parallel |
| Masking | none |
| Primary purpose | treatment |
| Enrollment | 60 |
| Start date | 19 September 2002 |
| Primary completion | 1 May 2009 |
| Estimated completion | 14 December 2011 |
| Sites | 1 location across United States |
National Cancer Institute (NCI)
Adults 5 to 35, any sex, with Sarcoma. Patients with the condition only — healthy volunteers not accepted.
Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.
Engraftment is defined as rapid conversion to complete donor chimerism and is assessed by blood counts and chimerism, \>95% donor engraftment at day 100 in \>75% of patients.
| Group | Value | 95% CI |
|---|---|---|
| Arm 2-Recipients | 23 |
Here is the number of participants with adverse events. For a detailed list of adverse events see the adverse event module.
| Group | Value | 95% CI |
|---|---|---|
| Arm 2-Recipients | 30 |
Acute GVHD as by Modified Glucksberg Criteria occurring before day 100. Chronic GVHD as per Seattle criteria occurring after day 100.
| Group | Value | 95% CI |
|---|---|---|
| Recipients -Cyclosporine GVHD Prophylaxis | 12 | |
| Recipients -Tacrolimus/Sirolimus GVHD Prophylaxis | 5 |
| Group | Value | 95% CI |
|---|---|---|
| Recipients -Cyclosporine GVHD Prophylaxis | 12 | |
| Recipients -Tacrolimus/Sirolimus GVHD Prophylaxis | 5 |
Days for participants to achieve a neutrophil count of 500/mm(3).
| Group | Value | 95% CI |
|---|---|---|
| Arm 2-Recipients | 9 | 8 – 12 |
Days for participants to achieve a platelet count of 50,000/mm(3).
| Group | Value | 95% CI |
|---|---|---|
| Arm 2-Recipients | 15 | 10 – 43 |
Participants who experienced recurrence or progression of disease following transplant.
| Group | Value | 95% CI |
|---|---|---|
| Arm 2-Recipients | 100 | 28 – 300 |
Progression free survival was based on the time from on-study date until progression or last follow-up.
| Group | Value | 95% CI |
|---|---|---|
| Arm 2-Recipients | 15.9 | 2.2 – 77.0 |
Participants who are alive at two years following Allo-Hematopoietic Stem Cell Transplant.
| Group | Value | 95% CI |
|---|---|---|
| Arm 2-Recipients | 39.1 |
| Group | Value | 95% CI |
|---|---|---|
| Arm 2-Recipients | 34.8 |
Participants who tolerated the transplantation regimen and accepted \>95% of the donors blood, marrow, and/or tissue.
| Group | Value | 95% CI |
|---|---|---|
| Arm 2-Recipients | 23 |
| Group | Value | 95% CI |
|---|---|---|
| Arm 2-Recipients | 23 |
The median CD4 count with a range of 85-1565 (absolute count) was used to determine recovery and were considered recovered if in this range. The CD4 count was established by flow cytometry testing.
| Group | Value | 95% CI |
|---|---|---|
| Arm 2-Recipients | 284 | 85 – 1042 |
Response is defined by the Response Evaluation Criteria in Solid Tumors (RECIST). RECIST criteria offer a simplified, conservative, extraction of imaging data for wide application in clinical trials. They presume that linear measures are an adequate substitute for 2-D (dimensional) methods and registers four response categories: Complete response (CR) is disappearance of all target lesions. Partial response (PR) is 30% increase in the sum of the longest diameter of target lesions. Progressive disease (PD) is 20% increase in the sum of the longest diameter of target lesions. Stable disease (SD)
| Group | Value | 95% CI |
|---|---|---|
| Arm 2-Recipients | 2 |
| Group | Value | 95% CI |
|---|---|---|
| Arm 2-Recipients | 4 |
| Group | Value | 95% CI |
|---|---|---|
| Arm 2-Recipients | 4 |
| Group | Value | 95% CI |
|---|---|---|
| Arm 2-Recipients | 2 |
Median survival from date of progression is based on the time from on-study date until progression or last follow-up.
| Group | Value | 95% CI |
|---|---|---|
| Arm 2-Recipients | 3.3 | 2.2 – 11.2 |
| Group | Value | 95% CI |
|---|---|---|
| Arm 2-Recipients | 19.1 | 5.6 – 77.0 |
Time frame: 16.5 months. Reporting threshold: 0%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.
| Reaction | System | Arm 2-Recipients |
|---|---|---|
| Death: Death not associated with CTCAE term: Disease progression NOS | General disorders | — |
| Gastrointestinal: Diarrhea | Gastrointestinal disorders | — |
| Neurology: Seizure | Nervous system disorders | — |
| Pulmonary/Upper Respiratory: Dyspnea (shortness of breath) | Respiratory, thoracic and mediastinal disorders | — |
| Pulmonary/Upper Respiratory: Hypoxia | Respiratory, thoracic and mediastinal disorders | — |
| Thrombosis | Cardiac disorders | — |
| Cardiac General: Hypotension | Cardiac disorders | — |
| Constitutional Symptoms: Fever | General disorders | — |
| Creatinine | Renal and urinary disorders | — |
| Gastrointestinal:Mucositis/stomatitis (clinical exam)::oral cavity | Gastrointestinal disorders | — |
| GI, other | Gastrointestinal disorders | — |
| Infection with neutropenia | Infections and infestations | — |
| Infection without neutropenia | Infections and infestations | — |
| Infection: Febrile neutropenia | Infections and infestations | — |
| Intussception | Gastrointestinal disorders | — |
| Metabolic/Laboratory: Bilirubin, serum-high (hyperbilirubinemia) | Metabolism and nutrition disorders | — |
| Metabolic/Laboratory: ALT, SGPT (serum glutamic pyruvic transaminase) | Metabolism and nutrition disorders | — |
| Metabolic/Laboratory: AST, SGOT (serum glutamic oxaloacetic transaminase) | Metabolism and nutrition disorders | — |
| Mood alteration; suicide gesture | Nervous system disorders | — |
| Neurology: Mood alteration: anxiety | Nervous system disorders | — |
| Pain - right chest wall | General disorders | — |
| Pericardial effusion | Cardiac disorders | — |
| Pulmonary/Upper Respiratory: Pneumonitis/pulmonary infiltrates | Respiratory, thoracic and mediastinal disorders | — |
| Pulmonary - Other (pulmonary edema w/normal 02 sat) | Respiratory, thoracic and mediastinal disorders | — |
| Radiation Recall Reaction (anterior chest) | Skin and subcutaneous tissue disorders | — |
| Reaction | System | Arm 2-Recipients |
|---|---|---|
| Blood/Bone Marrow:Hemoglobin | Blood and lymphatic system disorders | — |
| Coagulation: PTT (partial thromboplastin time) | Blood and lymphatic system disorders | — |
| Blood/Bone Marrow:Leukocytes (total WBC) | Blood and lymphatic system disorders | — |
| Metabolic/Laboratory: Glucose, serum-high (hyperglycemia) | Metabolism and nutrition disorders | — |
| Blood/Bone Marrow:Platelets | Blood and lymphatic system disorders | — |
| Blood/Bone Marrow:Neutrophils/granulocytes (ANC/AGC) | Blood and lymphatic system disorders | — |
| Metabolic/Laboratory: Sodium, serum-low (hyponatremia) | Metabolism and nutrition disorders | — |
| Metabolic/Laboratory: AST, SGOT (serum glutamic oxaloacetic) | Metabolism and nutrition disorders | — |
| Metabolic/Laboratory: Calcium, serum-low (hypocalcemia) | Metabolism and nutrition disorders | — |
| Gastrointestinal:Diarrhea | Gastrointestinal disorders | — |
| Metabolic/Laboratory: Magnesium, serum-low (hypomagnesemia) | Metabolism and nutrition disorders | — |
| Gastrointestinal:Nausea | Gastrointestinal disorders | — |
| Metabolic/Laboratory: Bilirubin, serum-high (hyperbilirubinemia) | Metabolism and nutrition disorders | — |
| Constitutional Symptoms: Fatigue (asthenia, lethargy, malaise) | General disorders | — |
| Constitutional Symptoms: Fever | General disorders | — |
| Metabolic/Laboratory: Metabolic/Laboratory: Other, (Specify, LDH) | Metabolism and nutrition disorders | — |
| Allergy/Immunology: Allergic rhinitis (including sneezing, nasal stuffiness, postnasal drip) | Immune system disorders | — |
| Pulmonary/Upper Respiratory: Cough | Respiratory, thoracic and mediastinal disorders | — |
| Dermatology/Skin: Rash/desquamation | Skin and subcutaneous tissue disorders | — |
| Metabolic/Laboratory: Magnesium, serum-high (hypermagnesemia) | Metabolism and nutrition disorders | — |
| Gastrointestinal:Constipation | Gastrointestinal disorders | — |
| Metabolic/Laboratory: Albumin, serum-low (hypoalbuminemia) | Metabolism and nutrition disorders | — |
| Neurology: mood alteration: depression | Nervous system disorders | — |
| Dermatology/Skin: Hair loss/alopecia (scalp or body) | Skin and subcutaneous tissue disorders | — |
| Metabolic/Laboratory: ALT, SGPT (serum glutamic pyruvic transaminase) | Metabolism and nutrition disorders | — |
| Metabolic/Laboratory:Potassium, serum-low (hypokalemia) | Metabolism and nutrition disorders | — |
| Gastrointestinal: Anorexia | Gastrointestinal disorders | — |
| Metabolic/Laboratory: Potassium, serum-low (hypokalemia) | Metabolism and nutrition disorders | — |
| Pulmonary/Upper Respiratory:Dyspnea (shortness of breath) | Respiratory, thoracic and mediastinal disorders | — |
| Cardiac General:Hypotension | Cardiac disorders | — |
| Dermatology/Skin: Dry skin | Skin and subcutaneous tissue disorders | — |
| Endocrine: Cushingoid appearance | Endocrine disorders | — |
| Gastrointestinal:Mucositis/stomatitis (clinical exam)::oral cavity | Gastrointestinal disorders | — |
| Metabolic/Laboratory: Potassium, serum-high (hyperkalemia) | Metabolism and nutrition disorders | — |
| Neurology: mood alteration: anxiety | Nervous system disorders | — |
| Neurology: tremor | Nervous system disorders | — |
| Pain/Musculoskeletal:Extremity-limb | Musculoskeletal and connective tissue disorders | — |
| Vomiting | Gastrointestinal disorders | — |
| Pain/Gastrointestinal::Abdomen NOS | Gastrointestinal disorders | — |
| Metabolic/Laboratory: Other (Urea nitrogen, low???) | Metabolism and nutrition disorders | — |
Most-reported serious reactions: Death: Death not associated with CTCAE term: Disease progression NOS, Gastrointestinal: Diarrhea, Neurology: Seizure, Pulmonary/Upper Respiratory: Dyspnea (shortness of breath), Pulmonary/Upper Respiratory: Hypoxia, Thrombosis, Cardiac General: Hypotension, Constitutional Symptoms: Fever.
Data from ClinicalTrials.gov NCT00043979 adverse events section.
This study will examine the safety and effectiveness of stem cell transplantation for treating patients with sarcomas (tumors of the bone, nerves, or soft tissue). Stem cells are immature cells in the bone marrow and blood stream that develop into blood cells. Stem cells transplanted from a healthy donor travel to the patient's bone marrow and begin producing normal cells. In patients with certain cancers, such as leukemia and lymphoma, the donor's immune cells attack the patient's cancer cells in what is called a "graft-versus-tumor" effect, contributing to cure of the disease. This study will determine whether this treatment can be used successfully to treat patients with sarcomas. Patients between 4 and 35 years of age with a sarcoma that has spread from the primary site or cannot be removed surgically, and for whom effective treatment is not available, may be eligible for this study. Candidates must have been diagnosed by the age of 30 at the time of enrollment. They must have a matched donor (usually a sibling). Participants undergo the following procedures: Donors: Stem cells are collected from the donor. To do this, the hormone granulocyte colony stimulating factor (G-CSF) is injected under the skin for several days to move stem cells out of the bone marrow into the bloodstream. Then, the cells are collected by apheresis. In this procedure the blood is drawn through a needle placed in one arm and pumped into a machine where the stem cells are separated out and removed. The rest of the blood is returned to the donor through a needle in the other arm. Patients: For patients who do not already have a central venous catheter (plastic tube), one is placed into a major vein. This tube can stay in the body the entire treatment period for giving medications, transfusing blood, , withdrawing blood samples, and delivering the donated stem cells. Before the transplant procedure, patients receive from one to three cycles of "induction" chemotherapy, with each cycle consisting of 5 days of fludarabine, cyclophosphamide, etoposide, doxorubicin, vincristine, and prednisone followed by at least a 17-day rest period. All the drugs are infused through the catheter except prednisone, which is taken by mouth. After the induction therapy, the patient is admitted to the hospital for 5 days of chemotherapy with high doses of cyclophosphamide, melphalan, and fludarabine. Two days later, the stem cells are infused. The anticipated hospital stay is about 3 weeks, but may be longer if complications arise. Patients are discharged when their white cell count is near normal, they have no fever or infection, they can take sufficient food and fluids by mouth, and they have no signs of serious graft-versus-host disease (GVHD)-a condition in which the donor's cells "see" the patient's cells as foreign and mount an immune response against them. After hospital discharge, patients are followed in the clinic at least once or twice weekly for a medical history, physical exam, and blood tests for 100 days. They receive medications to prevent infection and GVHD and, if needed, blood transfusions. If GVHD has not developed by about 120 days post transplant, patients receive additional white cells to boost the immune response. After 100 days, follow-up visits may be less frequent. Follow-up continues for at least 5 years. During the course of the study, patients undergo repeated medical evaluations, including blood tests and radiology studies, to check on the cancer and on any treatment side effects. On four occasions, white blood cells may be collected through apheresis to see if immune responses can be generated against the sarcomas treated in this study. Positron emission tomography (PET) scans may be done on five occasions. This test uses a radioactive material to produce images useful in detecting primary tumors and cancer that has spread.
2 peer-reviewed publications reference this trial (live from Europe PMC):
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