NCT00531232 is a Phase 2 clinical trial of Clofarabine in Myelodysplastic Syndromes, sponsored by Genzyme, a Sanofi Company.

Who is sponsoring NCT00531232?

NCT00531232 is sponsored by Genzyme, a Sanofi Company.

What drugs are being tested in NCT00531232?

Interventions in NCT00531232: Clofarabine.

What condition does NCT00531232 study?

NCT00531232 studies Myelodysplastic Syndromes, Secondary Acute Myeloid Leukemia (AML).

Is NCT00531232 still recruiting?

NCT00531232 is currently completed. Last updated 24 March 2022.

Are results published for NCT00531232?

Yes — primary outcome results have been posted to ClinicalTrials.gov. See the outcomes section above for details.

Last reviewed 2022-03-24 · How we verify

NCT00531232

Name: Drug Landscape Pharmaceutical Database
Creator: Drug Landscape
Published: 2026-01-01
License: https://creativecommons.org/licenses/by/4.0/

A Dose Confirmation Study of Oral Clofarabine for Adult Patients Previously Treated for Myelodysplastic Syndromes (MDS)

Completed Phase 2 Results posted Last updated 24 March 2022

What this trial tests

Phase 2 trial testing Clofarabine in Myelodysplastic Syndromes in 38 participants. Completed in 12 May 2011.

Timeline

7 May 2007

Primary endpoint
12 May 2011

12 May 2011

Quick facts

Lead sponsor	Genzyme, a Sanofi Company
Phase	Phase 2
Status	Completed
Study type	INTERVENTIONAL
Allocation	non randomized
Design	parallel
Masking	none
Primary purpose	treatment
Enrollment	38
Start date	7 May 2007
Primary completion	12 May 2011
Estimated completion	12 May 2011
Sites	6 locations across United States

Drugs / interventions tested

Clofarabine (CLOFARABINE) — full drug profile →

Conditions studied

Myelodysplastic Syndromes — all drugs for Myelodysplastic Syndromes →
Secondary Acute Myeloid Leukemia (AML) — all drugs for Secondary Acute Myeloid Leukemia (AML) →

Sponsor

Genzyme, a Sanofi Company — full company profile →

Who can join

18 and older, any sex, with Myelodysplastic Syndromes or Secondary Acute Myeloid Leukemia (AML). Patients with the condition only — healthy volunteers not accepted.

Results — posted to ClinicalTrials.gov

Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.

Percentage of Participants With Overall Response Primary · From date of randomization until disease recurrence or death, whichever occurred first (maximum study duration: up to 4 years)

Overall Response: complete remission(CR) or marrow CR or partial remission (PR), or hematologic improvement (HI) in participants with myelodysplastic syndromes (MDS);CR or CR with incomplete count recovery(CRi),or PR in participants with secondary acute myeloid leukemia (sAML). Per International Working Group (IWG) criteria, CR:\<= 5% myeloblasts in bone marrow; persistent dysplasia had to be noted; peripheral blood showing hemoglobin (Hgb)\>=11g/dL, platelets \>=100\*10\^9/L,neutrophils \>=1\*10\^9/L, blasts 0%. Marrow CR:\<=5%myeloblasts in bone marrow and decreased by \>=50% over pretreatme

Group	Value	95% CI
Clofarabine 35 mg/Day	13	0.00 – 26.75
Clofarabine 25 mg/Day	25	10.58 – 37.77

Duration of Response (DoR) Secondary · From first documentation of response to date of documentation of disease relapse, progression or death due to any cause, whichever occurs first (maximum study duration: up to 4 years)

DoR: time (in months) from 1st documentation of response to date of 1st documentation of disease relapse, progression or death due to any cause, whichever occured first. Response defined as CR, marrow CR, or PR (MDS participants) and CR/CRi (sAML participants). Per IWG criteria, relapse defined as: return to pretreatment bone marrow blast %; decrease of \>=50% from maximum remission levels; reduction in Hgb by \>=1.5g/dL.CR:\<= 5%myeloblasts in bone marrow; persistent dysplasia; peripheral blood showing Hgb\>=11g/dL. Marrow CR:\<=5%myeloblasts (bone marrow) and decreased by \>=50% over pretrea

Group	Value	95% CI
Clofarabine 35 mg/Day	12.5
Clofarabine 25 mg/Day	3.2	1.6 – 6.2

Number of Participants Who Achieved Hematologic Improvement (HI) Secondary · From date of randomization until disease recurrence or death, whichever occurred first (maximum study duration: up to 4 years)

Per IWG criteria, HI was defined as meeting any of the erythroid, platelet,and/or neutrophil independence categories for at least 8 consecutive weeks. Erythroid response (pre-treatment,\<11 grams per deciliter \[g/dL\]) was defined as Hgb increased by \>=1.5 g/dL;relevant reduction of units of red blood cell (RBC) transfusion by an absolute number of at least 4 RBC transfusion/8 weeks compared with the pretreatment transfusion number in the previous 8 weeks; only RBC transfusions given for Hgb of \<=9.0 g/dL pre-treatment counted in the RBC transfusion response evaluation. Platelet response(pr

Group	Value	95% CI
Clofarabine 35 mg/Day	0
Clofarabine 25 mg/Day	3

Percentage of Participants Achieving Overall Remission (OR) Secondary · From date of randomization until disease recurrence or death, whichever occurred first (maximum study duration: up to 4 years)

OR was defined as a best response of CR, marrow CR, or PR in participants with MDS or a best response of CR or CRi in participants with sAML. As per IWG criteria, CR was defined as the following: bone marrow \<=5% myeloblasts with normal maturation of all cell lines; persistent dysplasia had to be noted; peripheral blood showing hemoglobin \>=11 g/dL, platelets \>=100\*10\^9/L, neutrophils \>=1\*10\^9/L, blasts 0%. Marrow CR was defined as: bone marrow \<=5% myeloblasts and decreased by \>=50% over pretreatment; any HI response in peripheral blood had to be noted. CRi was defined as meeting al

Group	Value	95% CI
Clofarabine 35 mg/Day	13	0.00 – 26.75
Clofarabine 25 mg/Day	25	10.58 – 37.77

Time to Acute Myeloid Leukemia (AML) Transformation Secondary · From date of randomization until disease recurrence or death, whichever occurred first (maximum study duration: up to 4 years)

Time to AML transformation was defined as time (in months) from date of the first dose of oral Clofarabine to the date of AML transformation, (i.e., the earliest date when participants experienced bone marrow or peripheral blasts \>30%). The analysis was performed by Kaplan-Meier method.

Group	Value	95% CI
Clofarabine 35 mg/Day	4.2	3.1 – NA
Clofarabine 25 mg/Day	NA	5.2 – NA

Overall Survival (OS) Secondary · From date of first dose of study drug until date of death due to any cause (maximum study duration: up to 4 years)

OS defined as date of the first dose of oral Clofarabine until date of death due to any cause. If death was not observed, the participant was censored at the earliest of the last date the participant was known to be alive or the study cut-off date. The analysis was performed by Kaplan-Meier method.

Group	Value	95% CI
Clofarabine 35 mg/Day	4.8	0.6 – 7.4
Clofarabine 25 mg/Day	7.3	2.8 – NA

Maximum Tolerated Dose (MTD) of Oral Clofarabine Secondary · Cycle 1 (28 days)

The MTD was the highest dose level of Clofarabine that caused less than (\<) 2 participants to experience unacceptable drug-related toxicities after receiving 1 or more of the initial 5 daily doses of Clofarabine. Unacceptable drug-related toxicities included: drug-related prolonged myelosuppression, which was defined as hypocellular bone marrow with \<5 percent (%) cellularity at 6 weeks after starting treatment, which required a 1-dose level reduction for subsequent cycles; Common Terminology Criteria for Adverse Events (CTCAE) Grade 4 drug-related non-hematologic toxicity; CTCAE Grade 3 dru

Group	Value	95% CI
Clofarabine 55 mg/Day	25
Clofarabine 35 mg/Day	25
Clofarabine 25 mg/Day	25

Number of Participants With Febrile Neutropenia Secondary · From Baseline up to 45 days post last dose of study drug (maximum study duration: up to 4 years)

Febrile neutropenia was defined as fever (e.g., greater than or equal to (\>=) 38.5 Celsius (°C) on a single occasion, or greater than (\>) 38°C on 2 occasions within 12 hours) in the setting of neutropenia (defined as Absolute Neutrophil Count \<1.0\*10\^9/liter \[L\]).

Group	Value	95% CI
Clofarabine 55 mg/Day	1
Clofarabine 35 mg/Day	1
Clofarabine 25 mg/Day	13

Number of Participants With Adverse Events (AEs) Secondary · From Baseline up to 45 days post last dose of study drug (maximum duration: up to 4 years)

Adverse Event (AE): any undesirable physical, psychological/behavioral effect experienced by participant during their participation in clinical study, with study drug usage, whether or not product related.Treatment Emergent AEs (TEAEs): AEs that developed, worsened, or became serious during treatment period (from signature of informed consent form up to 45 days post last dose). Serious AE (SAE):any untoward medical occurrence that at any dose resulted in death, was life-threatening, required inpatient hospitalization/prolongation of existing hospitalization, resulted in persistent or significa

Any TEAE

Group	Value	95% CI
Clofarabine 55 mg/Day	4
Clofarabine 35 mg/Day	8
Clofarabine 25 mg/Day	24

Any treatment emergent SAE

Group	Value	95% CI
Clofarabine 55 mg/Day	4
Clofarabine 35 mg/Day	6
Clofarabine 25 mg/Day	19

Any TEAE leading to discontinuation

Group	Value	95% CI
Clofarabine 55 mg/Day	1
Clofarabine 35 mg/Day	2
Clofarabine 25 mg/Day	2

Any TEAE leading to death

Group	Value	95% CI
Clofarabine 55 mg/Day	4
Clofarabine 35 mg/Day	7
Clofarabine 25 mg/Day	14

Deaths within 45 days of last dose

Group	Value	95% CI
Clofarabine 55 mg/Day	3
Clofarabine 35 mg/Day	3
Clofarabine 25 mg/Day	3

Grade 4 Toxicities

Group	Value	95% CI
Clofarabine 55 mg/Day	1
Clofarabine 35 mg/Day	4
Clofarabine 25 mg/Day	18

Grade 5 Toxicities

Group	Value	95% CI
Clofarabine 55 mg/Day	3
Clofarabine 35 mg/Day	4
Clofarabine 25 mg/Day	4

Number of Participants Who Reported Death Within 30 Days of First Dose Secondary · Within 30 days of first dose administered on Day 1 of Cycle 1

Group	Value	95% CI
Clofarabine 55 mg/Day	2
Clofarabine 35 mg/Day	1
Clofarabine 25 mg/Day	1

Number of Participants With Unacceptable Drug-related Toxicities During Cycle 1 Secondary · Cycle 1 (28 days)

Unacceptable drug-related toxicities included: drug-related prolonged myelosuppression, which was defined as hypocellular bone marrow with \<5% cellularity at 6 weeks after starting treatment, which required a 1-dose level reduction for subsequent cycles; CTCAE Grade 4 drug-related non-hematologic toxicity; CTCAE Grade 3 drug-related non-hematologic toxicity that either recovered to Baseline grade but was recurrent to Grade 3 upon re-treatment during the first cycle or did not recover to Grade 1 or Baseline within 3 weeks when the drug was held or dose reduced; drug-related non-hematologic tox

Any unacceptable drug-related toxicities

Group	Value	95% CI
Clofarabine 55 mg/Day	0
Clofarabine 35 mg/Day	1
Clofarabine 25 mg/Day	0

Drug-related prolonged myelosuppression

Group	Value	95% CI
Clofarabine 55 mg/Day	0
Clofarabine 35 mg/Day	0
Clofarabine 25 mg/Day	0

Grade 4 drug-related non-haematologic toxicity

Group	Value	95% CI
Clofarabine 55 mg/Day	0
Clofarabine 35 mg/Day	1
Clofarabine 25 mg/Day	0

Grade 3 drug-related recurrent or persistent non-haematologic toxicity

Group	Value	95% CI
Clofarabine 55 mg/Day	0
Clofarabine 35 mg/Day	0
Clofarabine 25 mg/Day	0

Drug-related toxicity precluding completion of at least 4 daily doses

Group	Value	95% CI
Clofarabine 55 mg/Day	0
Clofarabine 35 mg/Day	0
Clofarabine 25 mg/Day	0

Pharmacokinetics (PK) Parameter: Maximum Observed Plasma Concentration (Cmax) of Clofarabine Secondary · Pre-dose and at 1, 3, and 5 hours Post-dose on Day 1 of Cycle 1

Cmax was defined as maximum observed plasma concentration of study drug.

Group	Value	95% CI
Clofarabine 55 mg/Day	158.25	± 55.446
Clofarabine 35 mg/Day	79.613	± 39.597
Clofarabine 25 mg/Day	47.813	± 44.745

Adverse events — posted to ClinicalTrials.gov

Time frame: AE data were collected from signature of the informed consent form up to 45 days post last dose of study drug (maximum study duration: up to 4 years).. Reporting threshold: 5%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

Clofarabine 25 mg/Day

Serious: 19/24 (79%)

Deaths: 14/24

Clofarabine 35 mg/Day

Serious: 6/8 (75%)

Deaths: 7/8

Clofarabine 55 mg/Day

Serious: 4/4 (100%)

Deaths: 4/4

Serious adverse events (56 terms)

Reaction	System	Clofarabine 25 mg/Day	Clofarabine 35 mg/Day	Clofarabine 55 mg/Day
Febrile Neutropenia	Blood and lymphatic system disorders	—	—	—
Pneumonia	Infections and infestations	—	—	—
Pyrexia	General disorders	—	—	—
Neutropenia	Blood and lymphatic system disorders	—	—	—
Clostridium Difficile Colitis	Infections and infestations	—	—	—
Sepsis	Infections and infestations	—	—	—
Septic Shock	Infections and infestations	—	—	—
Staphylococcal Bacteraemia	Infections and infestations	—	—	—
Haematuria	Renal and urinary disorders	—	—	—
Renal Failure	Renal and urinary disorders	—	—	—
Renal Failure Acute	Renal and urinary disorders	—	—	—
Respiratory Failure	Respiratory, thoracic and mediastinal disorders	—	—	—
Splenic Infarction	Blood and lymphatic system disorders	—	—	—
Thrombocytopenia	Blood and lymphatic system disorders	—	—	—
Arrhythmia	Cardiac disorders	—	—	—
Atrial Fibrillation	Cardiac disorders	—	—	—
Atrial Flutter	Cardiac disorders	—	—	—
Cardiac Failure Congestive	Cardiac disorders	—	—	—
Constipation	Gastrointestinal disorders	—	—	—
Gastrointestinal Haemorrhage	Gastrointestinal disorders	—	—	—
Nausea	Gastrointestinal disorders	—	—	—
Vomiting	Gastrointestinal disorders	—	—	—
Chest Pain	General disorders	—	—	—
Mucosal Inflammation	General disorders	—	—	—
Bacteraemia	Infections and infestations	—	—	—

Other adverse events (223 terms — click to expand)

Reaction	System	Clofarabine 25 mg/Day	Clofarabine 35 mg/Day	Clofarabine 55 mg/Day
Leukopenia	Blood and lymphatic system disorders	—	—	—
Neutropenia	Blood and lymphatic system disorders	—	—	—
Nausea	Gastrointestinal disorders	—	—	—
Hypokalaemia	Metabolism and nutrition disorders	—	—	—
Anaemia	Blood and lymphatic system disorders	—	—	—
Hypophosphataemia	Metabolism and nutrition disorders	—	—	—
Thrombocytopenia	Blood and lymphatic system disorders	—	—	—
Vomiting	Gastrointestinal disorders	—	—	—
Hypomagnesaemia	Metabolism and nutrition disorders	—	—	—
Chills	General disorders	—	—	—
Diarrhoea	Gastrointestinal disorders	—	—	—
Hypertension	Vascular disorders	—	—	—
Asthenia	General disorders	—	—	—
Fatigue	General disorders	—	—	—
Alanine Aminotransferase Increased	Investigations	—	—	—
Aspartate Aminotransferase Increased	Investigations	—	—	—
Decreased Appetite	Metabolism and nutrition disorders	—	—	—
Headache	Nervous system disorders	—	—	—
Proteinuria	Renal and urinary disorders	—	—	—
Petechiae	Skin and subcutaneous tissue disorders	—	—	—
Tachycardia	Cardiac disorders	—	—	—
Constipation	Gastrointestinal disorders	—	—	—
Haematuria	Renal and urinary disorders	—	—	—
Ecchymosis	Skin and subcutaneous tissue disorders	—	—	—
Abdominal Pain	Gastrointestinal disorders	—	—	—
Blood Bilirubin Increased	Investigations	—	—	—
Weight Decreased	Investigations	—	—	—
Mucosal Inflammation	General disorders	—	—	—
Oedema Peripheral	General disorders	—	—	—
Hypocalcaemia	Metabolism and nutrition disorders	—	—	—
Hyponatraemia	Metabolism and nutrition disorders	—	—	—
Dysuria	Renal and urinary disorders	—	—	—
Cough	Respiratory, thoracic and mediastinal disorders	—	—	—
Epistaxis	Respiratory, thoracic and mediastinal disorders	—	—	—
Rash	Skin and subcutaneous tissue disorders	—	—	—
Hypotension	Vascular disorders	—	—	—
Conjunctival Haemorrhage	Eye disorders	—	—	—
Pyrexia	General disorders	—	—	—
Hyperbilirubinaemia	Hepatobiliary disorders	—	—	—
Oral Candidiasis	Infections and infestations	—	—	—

Most-reported serious reactions: Febrile Neutropenia, Pneumonia, Pyrexia, Neutropenia, Clostridium Difficile Colitis, Sepsis, Septic Shock, Staphylococcal Bacteraemia.

Data from ClinicalTrials.gov NCT00531232 adverse events section.

Sponsor's own description

There was no well accepted standard of care for participants who failed or were intolerant to any of the currently approved therapies for myelodysplastic syndromes (MDS). In this study, participants were initially assigned to receive 55 or 35 milligrams (mg) of oral clofarabine daily for 5 days. After safety review of the first participants enrolled, the dose was reduced to 25 milligrams per day (mg/day) for up to 8 cycles as long as the participants continued to benefit and in the absence of progressive disease.

Publications & conference data

No peer-reviewed publications indexed yet for this trial. Completed trials usually publish results within 12-18 months.

Verify or expand the search:

Other trials of Clofarabine

Trials testing the same drug.

NCT07566377 — Cord Blood Transplantation in Children and Young Adults With Blood Cancer · Phase 2 · recruiting
NCT06252870 — Study Testing Two Conditioning Regimen With a Single Prophylaxis of GVHD by Cyclophosphamide and Methotrexate Post-trans · Phase 2 · recruiting
NCT05917405 — Study Comparing the Efficacy of 2 RIC Regimens (Clofarabine vs Fludarabine) in Adults With AML Eligible to Allo-SCT · Phase 2 · recruiting
NCT04994808 — Treosulfan-Based Versus Clofarabine-Based Conditioning Before Donor Hematopoietic Stem Cell Transplant for the Treatment · Phase 2 · active not recruiting
NCT05569512 — Uproleselan With Pre-Transplant Conditioning in Hematopoietic Stem Cell Transplantation for AML · Phase 1 · terminated

Other recruiting trials for Myelodysplastic Syndromes

Currently open trials in the same condition.

NCT07566377 — Cord Blood Transplantation in Children and Young Adults With Blood Cancer · Phase 2 · recruiting
NCT06303193 — Pacritinib, a Kinase Inhibitor of CSF1R, IRAK1, JAK2, and FLT3, in Adults and Pediatric Participants 12 Years of Age or · Phase 1, PHASE2 · recruiting
NCT07071155 — Momelotinib in Combination With Hypomethylating Agent for Chronic Phase Myelodysplastic Syndromes/Myeloproliferative Ove · EARLY_PHASE1 · recruiting
NCT07283900 — Ascorbate in Myelodysplastic Syndrome · Phase 2 · recruiting
NCT06487247 — HEME Home Transfusion Program · NA · recruiting

Other Genzyme, a Sanofi Company trials

Trials by the same sponsor.

NCT06666413 — China Post-approval Commitment (PAC) Study of Avalglucosidase Alfa in Participants With IOPD · Phase 4 · recruiting
NCT05164055 — Avalglucosidase Alfa French Post-trial Access for Participants With Pompe Disease (PTA Avalglucosidase) · Phase 4 · active not recruiting
NCT05134571 — China Post-marketing Surveillance (PMS) Study of Aldurazyme® · Phase 4 · completed
NCT05054387 — China Post-marketing Surveillance (PMS) Study of Fabrazyme® · Phase 4 · completed
NCT04676373 — Study to Evaluate Efficacy and Safety in Chinese Patients With Late Onset Pompe Disease With Alglucosidase Alfa Treatmen · Phase 4 · completed

Verify against primary sources

ClinicalTrials.gov — authoritative US registry record
WHO ICTRP — international registry index
EU Clinical Trials Register
Sponsor press releases (Google)

Data sources for this page

Trial protocol + status: ClinicalTrials.gov NCT00531232 (US National Library of Medicine, public domain)
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by Genzyme, a Sanofi Company
Last refreshed: 24 March 2022

Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT00531232.

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