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Oral Clofarabine Study in Patients With Myelodysplastic Syndrome

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ClinicalTrials.gov Identifier: NCT00299156
Recruitment Status : Completed
First Posted : March 6, 2006
Results First Posted : September 18, 2015
Last Update Posted : November 3, 2015
Sponsor:
Collaborator:
Genzyme, a Sanofi Company
Information provided by (Responsible Party):
M.D. Anderson Cancer Center

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Conditions Myelodysplastic Syndrome
Chronic Myelomonocytic Leukemia
Intervention Drug: Clofarabine
Enrollment 65

Recruitment Details Recruitment Period: March 2006 to July 2012. All patients registered at The University of Texas M.D. Anderson Cancer Center.
Pre-assignment Details Sixty-five participants were registered, one participant did not receive study treatment. The study consisted of two parts, Phase 1 and a randomized Phase 2 portion.
Arm/Group Title Oral Clofarabine Randomized, Oral Clofarabine 10mg Randomized, Oral Clofarabine 20mg
Hide Arm/Group Description Original dose of 40 mg oral reduced to 30mg and than lower dose of either 10 mg (Group 1) or 20 mg (Group 2) tablets once a day for 5 days in a row and repeated every 4-8 week cycle. Arm A: 10 mg tablets once a day for 5 days in a row and repeated every 4-8 week cycle. Arm B: 20 mg tablets once a day for 5 days in a row and repeated every 4-8 week cycle.
Period Title: Period 1 (Non-Randomized)
Started 32 0 0
Completed 32 0 0
Not Completed 0 0 0
Period Title: Period 2 (Randomized)
Started 0 17 16
Completed 0 16 16
Not Completed 0 1 0
Reason Not Completed
Screen Failure             0             1             0
Arm/Group Title Oral Clofarabine Randomized, Oral Clofarabine 10mg Randomized, Oral Clofarabine 20mg Total
Hide Arm/Group Description Original dose of 40 mg oral reduced to 30mg and than lower dose of either 10 mg (Group 1) or 20 mg (Group 2) tablets once a day for 5 days in a row and repeated every 4-8 week cycle. Arm A: 10 mg tablets once a day for 5 days in a row and repeated every 4-8 week cycle. Arm B: 20 mg tablets once a day for 5 days in a row and repeated every 4-8 week cycle. Total of all reporting groups
Overall Number of Baseline Participants 32 17 16 65
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Median (Full Range)
Unit of measure:  Years
Number Analyzed 32 participants 17 participants 16 participants 65 participants
70.0
(53 to 86)
67.5
(43 to 87)
72.5
(54 to 84)
70.0
(43 to 87)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 32 participants 17 participants 16 participants 65 participants
Female
9
  28.1%
7
  41.2%
7
  43.8%
23
  35.4%
Male
23
  71.9%
10
  58.8%
9
  56.3%
42
  64.6%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 32 participants 17 participants 16 participants 65 participants
32 17 16 65
1.Primary Outcome
Title Participants With a Complete Remission (CR)
Hide Description

Complete Remission (CR): Normalization of the peripheral blood and bone marrow with <5% bone marrow blasts, a peripheral blood granulocyte count > (1.0 x 109/ L, and a platelet count > 100 x 109/L).

Partial Remission: as above except for the presence of 6-15% marrow blasts, or 50% reduction if <15% at start of treatment.

Hematologic Improvement: meets all criteria for CR except for platelet recovery to >100 x 109/L.

Clinical Benefit: Platelets increase by 50% and to above 30 x 109/L untransfused (if lower than that pretherapy); or granulocytes increase by 100% and to above 109/L (if lower than that pretherapy); or hemoglobin increase by 2 g/dl; or transfusion independent; or splenomegaly reduction by > 50%; or monocytosis reduction by > 50% if pretreatment > 5 x 109/L.

Time Frame After 3 courses of treatment, up to 24 weeks.
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Oral Clofarabine Randomized, Oral Clofarabine 10mg Randomized, Oral Clofarabine 20mg
Hide Arm/Group Description:
Original dose of 40 mg oral reduced to 30mg and than lower dose of either 10 mg (Group 1) or 20 mg (Group 2) tablets once a day for 5 days in a row and repeated every 4-8 week cycle.
Arm A: 10 mg tablets once a day for 5 days in a row and repeated every 4-8 week cycle.
Arm B: 20 mg tablets once a day for 5 days in a row and repeated every 4-8 week cycle.
Overall Number of Participants Analyzed 32 16 16
Measure Type: Number
Unit of Measure: Participants
Complete Remission 8 3 0
Partial Remission 0 0 0
Hematologic Improvement 3 1 3
Clinical Benefit 3 0 0
Time Frame Adverse event collection with each 4-8 weeks course, up to a total of 12 courses of therapy. Overall study period, three years and four months.
Adverse Event Reporting Description The first participant of the phase II portion of this study started treatment in August 2009.
 
Arm/Group Title Oral Clofarabine Randomized, Oral Clofarabine 10mg Randomized, Oral Clofarabine 20mg
Hide Arm/Group Description Original dose of 40 mg oral reduced to 30mg and than lower dose of either 10 mg (Group 1) or 20 mg (Group 2) tablets once a day for 5 days in a row and repeated every 4-8 week cycle. Arm A: 10 mg tablets once a day for 5 days in a row and repeated every 4-8 week cycle. Arm B: 20 mg tablets once a day for 5 days in a row and repeated every 4-8 week cycle.
All-Cause Mortality
Oral Clofarabine Randomized, Oral Clofarabine 10mg Randomized, Oral Clofarabine 20mg
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   --/--      --/--      --/--    
Show Serious Adverse Events Hide Serious Adverse Events
Oral Clofarabine Randomized, Oral Clofarabine 10mg Randomized, Oral Clofarabine 20mg
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   16/32 (50.00%)      8/16 (50.00%)      12/16 (75.00%)    
Blood and lymphatic system disorders       
Gastrointestinal Hemorrhage  1  2/32 (6.25%)  3 0/16 (0.00%)  0 1/16 (6.25%)  1
Vaginal Hemorrhage  1  1/32 (3.13%)  1 0/16 (0.00%)  0 0/16 (0.00%)  0
Cardiac disorders       
Cardiac Arrythmia  1  1/32 (3.13%)  1 0/16 (0.00%)  0 0/16 (0.00%)  0
Supraventricular Tachycardia  1  1/32 (3.13%)  1 0/16 (0.00%)  0 0/16 (0.00%)  0
Pericardial Effusion  1  0/32 (0.00%)  0 0/16 (0.00%)  0 1/16 (6.25%)  1
Gastrointestinal disorders       
Dehydration  1  1/32 (3.13%)  1 0/16 (0.00%)  0 0/16 (0.00%)  0
Diarrhea  1  1/32 (3.13%)  1 0/16 (0.00%)  0 0/16 (0.00%)  0
Nausea  1  1/32 (3.13%)  1 0/16 (0.00%)  0 0/16 (0.00%)  0
Constipation  1  0/32 (0.00%)  0 1/16 (6.25%)  1 0/16 (0.00%)  0
General disorders       
Death  1  4/32 (12.50%)  4 1/16 (6.25%)  1 0/16 (0.00%)  0
Tumor Lysis Syndrome  1 [1]  1/32 (3.13%)  1 0/16 (0.00%)  0 0/16 (0.00%)  0
Abdominal Pain  1  1/32 (3.13%)  1 0/16 (0.00%)  0 0/16 (0.00%)  0
Fever  1  1/32 (3.13%)  1 1/16 (6.25%)  1 2/16 (12.50%)  2
Bone Pain  1  0/32 (0.00%)  0 1/16 (6.25%)  1 0/16 (0.00%)  0
Chest Pain  1  0/32 (0.00%)  0 0/16 (0.00%)  0 2/16 (12.50%)  2
Fatigue  1  0/32 (0.00%)  0 0/16 (0.00%)  0 1/16 (6.25%)  1
Pain  1  0/32 (0.00%)  0 0/16 (0.00%)  0 1/16 (6.25%)  1
Infections and infestations       
Infection  1  7/32 (21.88%)  8 1/16 (6.25%)  1 3/16 (18.75%)  4
Fungal Pneumonia  1  1/32 (3.13%)  1 0/16 (0.00%)  0 0/16 (0.00%)  0
Pneumonia  1  1/32 (3.13%)  1 0/16 (0.00%)  0 2/16 (12.50%)  3
Cellulitis  1  0/32 (0.00%)  0 1/16 (6.25%)  1 1/16 (6.25%)  1
E. Coli in Urine and Blood  1  0/32 (0.00%)  0 0/16 (0.00%)  0 1/16 (6.25%)  1
Hemolytic Strep  1  0/32 (0.00%)  0 0/16 (0.00%)  0 1/16 (6.25%)  1
Herpetic Mouth Sores  1  0/32 (0.00%)  0 1/16 (6.25%)  1 0/16 (0.00%)  0
Neutropenic Fever  1  0/32 (0.00%)  0 4/16 (25.00%)  6 7/16 (43.75%)  8
Pseudomonas  1  0/32 (0.00%)  0 1/16 (6.25%)  2 0/16 (0.00%)  0
Nervous system disorders       
Cerebrovascular Ischemia  1  1/32 (3.13%)  1 0/16 (0.00%)  0 0/16 (0.00%)  0
Ataxia  1  1/32 (3.13%)  1 0/16 (0.00%)  0 0/16 (0.00%)  0
Syncope  1  1/32 (3.13%)  1 0/16 (0.00%)  0 0/16 (0.00%)  0
Altered Mental Status  1  0/32 (0.00%)  0 0/16 (0.00%)  0 1/16 (6.25%)  1
Renal and urinary disorders       
Renal Failure  1  2/32 (6.25%)  2 0/16 (0.00%)  0 0/16 (0.00%)  0
Elevated Creatinine  1  1/32 (3.13%)  1 0/16 (0.00%)  0 0/16 (0.00%)  0
Respiratory, thoracic and mediastinal disorders       
Respiratory Failure  1 [1]  1/32 (3.13%)  1 0/16 (0.00%)  0 0/16 (0.00%)  0
Indicates events were collected by systematic assessment
1
Term from vocabulary, CTCAE (3.0)
[1]
Death
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Oral Clofarabine Randomized, Oral Clofarabine 10mg Randomized, Oral Clofarabine 20mg
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   32/32 (100.00%)      16/16 (100.00%)      16/16 (100.00%)    
Blood and lymphatic system disorders       
Edema  1  4/32 (12.50%)  4 0/16 (0.00%)  0 0/16 (0.00%)  0
Gastrointestinal Hemorrhage  1  3/32 (9.38%)  3 0/16 (0.00%)  0 0/16 (0.00%)  0
Neutropenia  1  16/32 (50.00%)  16 1/16 (6.25%)  1 4/16 (25.00%)  4
Thrombocytopenia  1  13/32 (40.63%)  13 1/16 (6.25%)  1 1/16 (6.25%)  1
Gastrointestinal disorders       
Nausea  1  27/32 (84.38%)  27 3/16 (18.75%)  3 6/16 (37.50%)  6
Anorexia  1  7/32 (21.88%)  7 2/16 (12.50%)  2 1/16 (6.25%)  1
Diarrhea  1  9/32 (28.13%)  9 0/16 (0.00%)  0 3/16 (18.75%)  3
Constipation  1  6/32 (18.75%)  6 2/16 (12.50%)  2 1/16 (6.25%)  1
Mucositis  1  6/32 (18.75%)  6 1/16 (6.25%)  1 1/16 (6.25%)  1
Taste Alteration  1  0/32 (0.00%)  0 1/16 (6.25%)  1 0/16 (0.00%)  0
Emesis  1  16/32 (50.00%)  16 0/16 (0.00%)  0 0/16 (0.00%)  0
General disorders       
Fatigue  1  15/32 (46.88%)  15 2/16 (12.50%)  2 0/16 (0.00%)  0
Headache  1  11/32 (34.38%)  11 2/16 (12.50%)  2 3/16 (18.75%)  3
Abdominal Pain  1  0/32 (0.00%)  0 0/16 (0.00%)  0 1/16 (6.25%)  1
Dizziness  1  5/32 (15.63%)  5 0/16 (0.00%)  0 0/16 (0.00%)  0
Infections and infestations       
Neutropenic Fever  1  0/32 (0.00%)  0 0/16 (0.00%)  0 2/16 (12.50%)  2
Metabolism and nutrition disorders       
Elevated ALT  1  16/32 (50.00%)  16 1/16 (6.25%)  1 3/16 (18.75%)  3
Elevated AST  1  22/32 (68.75%)  22 1/16 (6.25%)  1 1/16 (6.25%)  1
Hyperbilirrubinemia  1  13/32 (40.63%)  13 0/16 (0.00%)  0 4/16 (25.00%)  4
Iron Overload  1  0/32 (0.00%)  0 1/16 (6.25%)  1 0/16 (0.00%)  0
Elevated Alkaline Phosphatase  1  6/32 (18.75%)  6 0/16 (0.00%)  0 0/16 (0.00%)  0
Elevated Amylase  1  2/32 (6.25%)  2 0/16 (0.00%)  0 0/16 (0.00%)  0
Musculoskeletal and connective tissue disorders       
Myalgia  1  6/32 (18.75%)  6 0/16 (0.00%)  0 0/16 (0.00%)  0
Musculoskeletal Other  1  0/32 (0.00%)  0 1/16 (6.25%)  1 1/16 (6.25%)  1
Nervous system disorders       
Insomnia  1  0/32 (0.00%)  0 0/16 (0.00%)  0 1/16 (6.25%)  1
Renal and urinary disorders       
Acute Renal Failure  1  4/32 (12.50%)  4 0/16 (0.00%)  0 0/16 (0.00%)  0
Elevated Creatinine  1  8/32 (25.00%)  8 1/16 (6.25%)  1 1/16 (6.25%)  1
Urinary Frequency  1  0/32 (0.00%)  0 0/16 (0.00%)  0 1/16 (6.25%)  1
Skin and subcutaneous tissue disorders       
Dermatology/Skin  1  0/32 (0.00%)  0 1/16 (6.25%)  1 0/16 (0.00%)  0
Petechiae  1  0/32 (0.00%)  0 1/16 (6.25%)  1 0/16 (0.00%)  0
Pruritus  1  9/32 (28.13%)  9 0/16 (0.00%)  0 0/16 (0.00%)  0
Skin Rash  1  18/32 (56.25%)  18 1/16 (6.25%)  1 1/16 (6.25%)  1
Palmoplantar Dysesthesia  1  7/32 (21.88%)  7 0/16 (0.00%)  0 0/16 (0.00%)  0
Indicates events were collected by systematic assessment
1
Term from vocabulary, CTCAE (3.0)
Certain Agreements
All Principal Investigators ARE employed by the organization sponsoring the study.
Results Point of Contact
Name/Title: Hagop Kantarjian, MD /Professor
Organization: The University of Texas MD Anderson Cancer Center
Phone: 713-792-7026
Responsible Party: M.D. Anderson Cancer Center
ClinicalTrials.gov Identifier: NCT00299156     History of Changes
Other Study ID Numbers: 2005-0536
First Submitted: March 3, 2006
First Posted: March 6, 2006
Results First Submitted: August 19, 2015
Results First Posted: September 18, 2015
Last Update Posted: November 3, 2015