Trial Outcomes & Findings for Oral Clofarabine Study in Patients With Myelodysplastic Syndrome (NCT NCT00299156)

Last Updated: 2015-11-03

Results Overview

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.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

65 participants

Primary outcome timeframe

After 3 courses of treatment, up to 24 weeks.

Results posted on

2015-11-03

Participant Flow

Recruitment Period: March 2006 to July 2012. All patients registered at The University of Texas M.D. Anderson Cancer Center.

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.

Participant milestones

Participant milestones
Measure	Oral Clofarabine 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.	Randomized, Oral Clofarabine 10mg Arm A: 10 mg tablets once a day for 5 days in a row and repeated every 4-8 week cycle.	Randomized, Oral Clofarabine 20mg Arm B: 20 mg tablets once a day for 5 days in a row and repeated every 4-8 week cycle.
Period 1 (Non-Randomized) STARTED	32	0	0
Period 1 (Non-Randomized) COMPLETED	32	0	0
Period 1 (Non-Randomized) NOT COMPLETED	0	0	0
Period 2 (Randomized) STARTED	0	17	16
Period 2 (Randomized) COMPLETED	0	16	16
Period 2 (Randomized) NOT COMPLETED	0	1	0

Reasons for withdrawal

Reasons for withdrawal
Measure	Oral Clofarabine 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.	Randomized, Oral Clofarabine 10mg Arm A: 10 mg tablets once a day for 5 days in a row and repeated every 4-8 week cycle.	Randomized, Oral Clofarabine 20mg Arm B: 20 mg tablets once a day for 5 days in a row and repeated every 4-8 week cycle.
Period 2 (Randomized) Screen Failure	0	1	0

Baseline Characteristics

Oral Clofarabine Study in Patients With Myelodysplastic Syndrome

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Oral Clofarabine n=32 Participants 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.	Randomized, Oral Clofarabine 10mg n=17 Participants Arm A: 10 mg tablets once a day for 5 days in a row and repeated every 4-8 week cycle.	Randomized, Oral Clofarabine 20mg n=16 Participants Arm B: 20 mg tablets once a day for 5 days in a row and repeated every 4-8 week cycle.	Total n=65 Participants Total of all reporting groups
Age, Continuous	70.0 years n=5 Participants	67.5 years n=7 Participants	72.5 years n=5 Participants	70.0 years n=4 Participants
Sex: Female, Male Female	9 Participants n=5 Participants	7 Participants n=7 Participants	7 Participants n=5 Participants	23 Participants n=4 Participants
Sex: Female, Male Male	23 Participants n=5 Participants	10 Participants n=7 Participants	9 Participants n=5 Participants	42 Participants n=4 Participants
Region of Enrollment United States	32 participants n=5 Participants	17 participants n=7 Participants	16 participants n=5 Participants	65 participants n=4 Participants

PRIMARY outcome

Timeframe: After 3 courses of treatment, up to 24 weeks.

Outcome measures

Outcome measures
Measure	Oral Clofarabine n=32 Participants 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.	Randomized, Oral Clofarabine 10mg n=16 Participants Arm A: 10 mg tablets once a day for 5 days in a row and repeated every 4-8 week cycle.	Randomized, Oral Clofarabine 20mg n=16 Participants Arm B: 20 mg tablets once a day for 5 days in a row and repeated every 4-8 week cycle.
Participants With a Complete Remission (CR) Complete Remission	8 Participants	3 Participants	0 Participants
Participants With a Complete Remission (CR) Partial Remission	0 Participants	0 Participants	0 Participants
Participants With a Complete Remission (CR) Hematologic Improvement	3 Participants	1 Participants	3 Participants
Participants With a Complete Remission (CR) Clinical Benefit	3 Participants	0 Participants	0 Participants

Adverse Events

Oral Clofarabine

Serious events: 16 serious events

Other events: 32 other events

Deaths: 0 deaths

Randomized, Oral Clofarabine 10mg

Serious events: 8 serious events

Other events: 16 other events

Deaths: 0 deaths

Randomized, Oral Clofarabine 20mg

Serious events: 12 serious events

Other events: 16 other events

Deaths: 0 deaths

Serious adverse events

Other adverse events

Additional Information

Hagop Kantarjian, MD /Professor

The University of Texas MD Anderson Cancer Center

Phone: 713-792-7026

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place

Serious adverse events
Measure	Oral Clofarabine n=32 participants at risk 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.	Randomized, Oral Clofarabine 10mg n=16 participants at risk Arm A: 10 mg tablets once a day for 5 days in a row and repeated every 4-8 week cycle.	Randomized, Oral Clofarabine 20mg n=16 participants at risk Arm B: 20 mg tablets once a day for 5 days in a row and repeated every 4-8 week cycle.
General disorders Death	12.5% 4/32 • Number of events 4 • 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. The first participant of the phase II portion of this study started treatment in August 2009.	6.2% 1/16 • Number of events 1 • 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. The first participant of the phase II portion of this study started treatment in August 2009.	0.00% 0/16 • 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. The first participant of the phase II portion of this study started treatment in August 2009.
Respiratory, thoracic and mediastinal disorders Respiratory Failure	3.1% 1/32 • Number of events 1 • 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. The first participant of the phase II portion of this study started treatment in August 2009.	0.00% 0/16 • 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. The first participant of the phase II portion of this study started treatment in August 2009.	0.00% 0/16 • 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. The first participant of the phase II portion of this study started treatment in August 2009.
Renal and urinary disorders Renal Failure	6.2% 2/32 • Number of events 2 • 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. The first participant of the phase II portion of this study started treatment in August 2009.	0.00% 0/16 • 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. The first participant of the phase II portion of this study started treatment in August 2009.	0.00% 0/16 • 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. The first participant of the phase II portion of this study started treatment in August 2009.
General disorders Tumor Lysis Syndrome	3.1% 1/32 • Number of events 1 • 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. The first participant of the phase II portion of this study started treatment in August 2009.	0.00% 0/16 • 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. The first participant of the phase II portion of this study started treatment in August 2009.	0.00% 0/16 • 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. The first participant of the phase II portion of this study started treatment in August 2009.
Nervous system disorders Cerebrovascular Ischemia	3.1% 1/32 • Number of events 1 • 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. The first participant of the phase II portion of this study started treatment in August 2009.	0.00% 0/16 • 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. The first participant of the phase II portion of this study started treatment in August 2009.	0.00% 0/16 • 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. The first participant of the phase II portion of this study started treatment in August 2009.
Infections and infestations Infection	21.9% 7/32 • Number of events 8 • 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. The first participant of the phase II portion of this study started treatment in August 2009.	6.2% 1/16 • Number of events 1 • 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. The first participant of the phase II portion of this study started treatment in August 2009.	18.8% 3/16 • Number of events 4 • 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. The first participant of the phase II portion of this study started treatment in August 2009.
Blood and lymphatic system disorders Gastrointestinal Hemorrhage	6.2% 2/32 • Number of events 3 • 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. The first participant of the phase II portion of this study started treatment in August 2009.	0.00% 0/16 • 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. The first participant of the phase II portion of this study started treatment in August 2009.	6.2% 1/16 • Number of events 1 • 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. The first participant of the phase II portion of this study started treatment in August 2009.
Blood and lymphatic system disorders Vaginal Hemorrhage	3.1% 1/32 • Number of events 1 • 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. The first participant of the phase II portion of this study started treatment in August 2009.	0.00% 0/16 • 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. The first participant of the phase II portion of this study started treatment in August 2009.	0.00% 0/16 • 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. The first participant of the phase II portion of this study started treatment in August 2009.
Cardiac disorders Cardiac Arrythmia	3.1% 1/32 • Number of events 1 • 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. The first participant of the phase II portion of this study started treatment in August 2009.	0.00% 0/16 • 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. The first participant of the phase II portion of this study started treatment in August 2009.	0.00% 0/16 • 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. The first participant of the phase II portion of this study started treatment in August 2009.
Cardiac disorders Supraventricular Tachycardia	3.1% 1/32 • Number of events 1 • 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. The first participant of the phase II portion of this study started treatment in August 2009.	0.00% 0/16 • 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. The first participant of the phase II portion of this study started treatment in August 2009.	0.00% 0/16 • 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. The first participant of the phase II portion of this study started treatment in August 2009.
Gastrointestinal disorders Dehydration	3.1% 1/32 • Number of events 1 • 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. The first participant of the phase II portion of this study started treatment in August 2009.	0.00% 0/16 • 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. The first participant of the phase II portion of this study started treatment in August 2009.	0.00% 0/16 • 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. The first participant of the phase II portion of this study started treatment in August 2009.
Gastrointestinal disorders Diarrhea	3.1% 1/32 • Number of events 1 • 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. The first participant of the phase II portion of this study started treatment in August 2009.	0.00% 0/16 • 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. The first participant of the phase II portion of this study started treatment in August 2009.	0.00% 0/16 • 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. The first participant of the phase II portion of this study started treatment in August 2009.
Gastrointestinal disorders Nausea	3.1% 1/32 • Number of events 1 • 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. The first participant of the phase II portion of this study started treatment in August 2009.	0.00% 0/16 • 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. The first participant of the phase II portion of this study started treatment in August 2009.	0.00% 0/16 • 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. The first participant of the phase II portion of this study started treatment in August 2009.
General disorders Abdominal Pain	3.1% 1/32 • Number of events 1 • 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. The first participant of the phase II portion of this study started treatment in August 2009.	0.00% 0/16 • 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. The first participant of the phase II portion of this study started treatment in August 2009.	0.00% 0/16 • 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. The first participant of the phase II portion of this study started treatment in August 2009.
General disorders Fever	3.1% 1/32 • Number of events 1 • 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. The first participant of the phase II portion of this study started treatment in August 2009.	6.2% 1/16 • Number of events 1 • 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. The first participant of the phase II portion of this study started treatment in August 2009.	12.5% 2/16 • Number of events 2 • 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. The first participant of the phase II portion of this study started treatment in August 2009.
Infections and infestations Fungal Pneumonia	3.1% 1/32 • Number of events 1 • 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. The first participant of the phase II portion of this study started treatment in August 2009.	0.00% 0/16 • 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. The first participant of the phase II portion of this study started treatment in August 2009.	0.00% 0/16 • 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. The first participant of the phase II portion of this study started treatment in August 2009.
Infections and infestations Pneumonia	3.1% 1/32 • Number of events 1 • 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. The first participant of the phase II portion of this study started treatment in August 2009.	0.00% 0/16 • 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. The first participant of the phase II portion of this study started treatment in August 2009.	12.5% 2/16 • Number of events 3 • 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. The first participant of the phase II portion of this study started treatment in August 2009.
Nervous system disorders Ataxia	3.1% 1/32 • Number of events 1 • 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. The first participant of the phase II portion of this study started treatment in August 2009.	0.00% 0/16 • 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. The first participant of the phase II portion of this study started treatment in August 2009.	0.00% 0/16 • 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. The first participant of the phase II portion of this study started treatment in August 2009.
Nervous system disorders Syncope	3.1% 1/32 • Number of events 1 • 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. The first participant of the phase II portion of this study started treatment in August 2009.	0.00% 0/16 • 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. The first participant of the phase II portion of this study started treatment in August 2009.	0.00% 0/16 • 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. The first participant of the phase II portion of this study started treatment in August 2009.
Renal and urinary disorders Elevated Creatinine	3.1% 1/32 • Number of events 1 • 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. The first participant of the phase II portion of this study started treatment in August 2009.	0.00% 0/16 • 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. The first participant of the phase II portion of this study started treatment in August 2009.	0.00% 0/16 • 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. The first participant of the phase II portion of this study started treatment in August 2009.
Cardiac disorders Pericardial Effusion	0.00% 0/32 • 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. The first participant of the phase II portion of this study started treatment in August 2009.	0.00% 0/16 • 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. The first participant of the phase II portion of this study started treatment in August 2009.	6.2% 1/16 • Number of events 1 • 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. The first participant of the phase II portion of this study started treatment in August 2009.
Gastrointestinal disorders Constipation	0.00% 0/32 • 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. The first participant of the phase II portion of this study started treatment in August 2009.	6.2% 1/16 • Number of events 1 • 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. The first participant of the phase II portion of this study started treatment in August 2009.	0.00% 0/16 • 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. The first participant of the phase II portion of this study started treatment in August 2009.
General disorders Bone Pain	0.00% 0/32 • 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. The first participant of the phase II portion of this study started treatment in August 2009.	6.2% 1/16 • Number of events 1 • 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. The first participant of the phase II portion of this study started treatment in August 2009.	0.00% 0/16 • 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. The first participant of the phase II portion of this study started treatment in August 2009.
General disorders Chest Pain	0.00% 0/32 • 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. The first participant of the phase II portion of this study started treatment in August 2009.	0.00% 0/16 • 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. The first participant of the phase II portion of this study started treatment in August 2009.	12.5% 2/16 • Number of events 2 • 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. The first participant of the phase II portion of this study started treatment in August 2009.
General disorders Fatigue	0.00% 0/32 • 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. The first participant of the phase II portion of this study started treatment in August 2009.	0.00% 0/16 • 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. The first participant of the phase II portion of this study started treatment in August 2009.	6.2% 1/16 • Number of events 1 • 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. The first participant of the phase II portion of this study started treatment in August 2009.
General disorders Pain	0.00% 0/32 • 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. The first participant of the phase II portion of this study started treatment in August 2009.	0.00% 0/16 • 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. The first participant of the phase II portion of this study started treatment in August 2009.	6.2% 1/16 • Number of events 1 • 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. The first participant of the phase II portion of this study started treatment in August 2009.
Infections and infestations Cellulitis	0.00% 0/32 • 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. The first participant of the phase II portion of this study started treatment in August 2009.	6.2% 1/16 • Number of events 1 • 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. The first participant of the phase II portion of this study started treatment in August 2009.	6.2% 1/16 • Number of events 1 • 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. The first participant of the phase II portion of this study started treatment in August 2009.
Infections and infestations E. Coli in Urine and Blood	0.00% 0/32 • 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. The first participant of the phase II portion of this study started treatment in August 2009.	0.00% 0/16 • 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. The first participant of the phase II portion of this study started treatment in August 2009.	6.2% 1/16 • Number of events 1 • 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. The first participant of the phase II portion of this study started treatment in August 2009.
Infections and infestations Hemolytic Strep	0.00% 0/32 • 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. The first participant of the phase II portion of this study started treatment in August 2009.	0.00% 0/16 • 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. The first participant of the phase II portion of this study started treatment in August 2009.	6.2% 1/16 • Number of events 1 • 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. The first participant of the phase II portion of this study started treatment in August 2009.
Infections and infestations Herpetic Mouth Sores	0.00% 0/32 • 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. The first participant of the phase II portion of this study started treatment in August 2009.	6.2% 1/16 • Number of events 1 • 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. The first participant of the phase II portion of this study started treatment in August 2009.	0.00% 0/16 • 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. The first participant of the phase II portion of this study started treatment in August 2009.
Infections and infestations Neutropenic Fever	0.00% 0/32 • 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. The first participant of the phase II portion of this study started treatment in August 2009.	25.0% 4/16 • Number of events 6 • 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. The first participant of the phase II portion of this study started treatment in August 2009.	43.8% 7/16 • Number of events 8 • 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. The first participant of the phase II portion of this study started treatment in August 2009.
Infections and infestations Pseudomonas	0.00% 0/32 • 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. The first participant of the phase II portion of this study started treatment in August 2009.	6.2% 1/16 • Number of events 2 • 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. The first participant of the phase II portion of this study started treatment in August 2009.	0.00% 0/16 • 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. The first participant of the phase II portion of this study started treatment in August 2009.
Nervous system disorders Altered Mental Status	0.00% 0/32 • 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. The first participant of the phase II portion of this study started treatment in August 2009.	0.00% 0/16 • 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. The first participant of the phase II portion of this study started treatment in August 2009.	6.2% 1/16 • Number of events 1 • 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. The first participant of the phase II portion of this study started treatment in August 2009.

Other adverse events
Measure	Oral Clofarabine n=32 participants at risk 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.	Randomized, Oral Clofarabine 10mg n=16 participants at risk Arm A: 10 mg tablets once a day for 5 days in a row and repeated every 4-8 week cycle.	Randomized, Oral Clofarabine 20mg n=16 participants at risk Arm B: 20 mg tablets once a day for 5 days in a row and repeated every 4-8 week cycle.
General disorders Fatigue	46.9% 15/32 • Number of events 15 • 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. The first participant of the phase II portion of this study started treatment in August 2009.	12.5% 2/16 • Number of events 2 • 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. The first participant of the phase II portion of this study started treatment in August 2009.	0.00% 0/16 • 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. The first participant of the phase II portion of this study started treatment in August 2009.
Gastrointestinal disorders Nausea	84.4% 27/32 • Number of events 27 • 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. The first participant of the phase II portion of this study started treatment in August 2009.	18.8% 3/16 • Number of events 3 • 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. The first participant of the phase II portion of this study started treatment in August 2009.	37.5% 6/16 • Number of events 6 • 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. The first participant of the phase II portion of this study started treatment in August 2009.
Gastrointestinal disorders Anorexia	21.9% 7/32 • Number of events 7 • 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. The first participant of the phase II portion of this study started treatment in August 2009.	12.5% 2/16 • Number of events 2 • 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. The first participant of the phase II portion of this study started treatment in August 2009.	6.2% 1/16 • Number of events 1 • 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. The first participant of the phase II portion of this study started treatment in August 2009.
General disorders Headache	34.4% 11/32 • Number of events 11 • 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. The first participant of the phase II portion of this study started treatment in August 2009.	12.5% 2/16 • Number of events 2 • 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. The first participant of the phase II portion of this study started treatment in August 2009.	18.8% 3/16 • Number of events 3 • 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. The first participant of the phase II portion of this study started treatment in August 2009.
Musculoskeletal and connective tissue disorders Myalgia	18.8% 6/32 • Number of events 6 • 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. The first participant of the phase II portion of this study started treatment in August 2009.	0.00% 0/16 • 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. The first participant of the phase II portion of this study started treatment in August 2009.	0.00% 0/16 • 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. The first participant of the phase II portion of this study started treatment in August 2009.
Renal and urinary disorders Acute Renal Failure	12.5% 4/32 • Number of events 4 • 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. The first participant of the phase II portion of this study started treatment in August 2009.	0.00% 0/16 • 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. The first participant of the phase II portion of this study started treatment in August 2009.	0.00% 0/16 • 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. The first participant of the phase II portion of this study started treatment in August 2009.
Gastrointestinal disorders Diarrhea	28.1% 9/32 • Number of events 9 • 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. The first participant of the phase II portion of this study started treatment in August 2009.	0.00% 0/16 • 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. The first participant of the phase II portion of this study started treatment in August 2009.	18.8% 3/16 • Number of events 3 • 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. The first participant of the phase II portion of this study started treatment in August 2009.
General disorders Abdominal Pain	0.00% 0/32 • 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. The first participant of the phase II portion of this study started treatment in August 2009.	0.00% 0/16 • 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. The first participant of the phase II portion of this study started treatment in August 2009.	6.2% 1/16 • Number of events 1 • 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. The first participant of the phase II portion of this study started treatment in August 2009.
Metabolism and nutrition disorders Elevated ALT	50.0% 16/32 • Number of events 16 • 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. The first participant of the phase II portion of this study started treatment in August 2009.	6.2% 1/16 • Number of events 1 • 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. The first participant of the phase II portion of this study started treatment in August 2009.	18.8% 3/16 • Number of events 3 • 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. The first participant of the phase II portion of this study started treatment in August 2009.
Metabolism and nutrition disorders Elevated AST	68.8% 22/32 • Number of events 22 • 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. The first participant of the phase II portion of this study started treatment in August 2009.	6.2% 1/16 • Number of events 1 • 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. The first participant of the phase II portion of this study started treatment in August 2009.	6.2% 1/16 • Number of events 1 • 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. The first participant of the phase II portion of this study started treatment in August 2009.
Metabolism and nutrition disorders Hyperbilirrubinemia	40.6% 13/32 • Number of events 13 • 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. The first participant of the phase II portion of this study started treatment in August 2009.	0.00% 0/16 • 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. The first participant of the phase II portion of this study started treatment in August 2009.	25.0% 4/16 • Number of events 4 • 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. The first participant of the phase II portion of this study started treatment in August 2009.
Gastrointestinal disorders Constipation	18.8% 6/32 • Number of events 6 • 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. The first participant of the phase II portion of this study started treatment in August 2009.	12.5% 2/16 • Number of events 2 • 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. The first participant of the phase II portion of this study started treatment in August 2009.	6.2% 1/16 • Number of events 1 • 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. The first participant of the phase II portion of this study started treatment in August 2009.
Renal and urinary disorders Elevated Creatinine	25.0% 8/32 • Number of events 8 • 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. The first participant of the phase II portion of this study started treatment in August 2009.	6.2% 1/16 • Number of events 1 • 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. The first participant of the phase II portion of this study started treatment in August 2009.	6.2% 1/16 • Number of events 1 • 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. The first participant of the phase II portion of this study started treatment in August 2009.
Skin and subcutaneous tissue disorders Dermatology/Skin	0.00% 0/32 • 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. The first participant of the phase II portion of this study started treatment in August 2009.	6.2% 1/16 • Number of events 1 • 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. The first participant of the phase II portion of this study started treatment in August 2009.	0.00% 0/16 • 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. The first participant of the phase II portion of this study started treatment in August 2009.
General disorders Dizziness	15.6% 5/32 • Number of events 5 • 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. The first participant of the phase II portion of this study started treatment in August 2009.	0.00% 0/16 • 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. The first participant of the phase II portion of this study started treatment in August 2009.	0.00% 0/16 • 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. The first participant of the phase II portion of this study started treatment in August 2009.
Blood and lymphatic system disorders Edema	12.5% 4/32 • Number of events 4 • 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. The first participant of the phase II portion of this study started treatment in August 2009.	0.00% 0/16 • 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. The first participant of the phase II portion of this study started treatment in August 2009.	0.00% 0/16 • 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. The first participant of the phase II portion of this study started treatment in August 2009.
Nervous system disorders Insomnia	0.00% 0/32 • 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. The first participant of the phase II portion of this study started treatment in August 2009.	0.00% 0/16 • 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. The first participant of the phase II portion of this study started treatment in August 2009.	6.2% 1/16 • Number of events 1 • 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. The first participant of the phase II portion of this study started treatment in August 2009.
Metabolism and nutrition disorders Iron Overload	0.00% 0/32 • 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. The first participant of the phase II portion of this study started treatment in August 2009.	6.2% 1/16 • Number of events 1 • 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. The first participant of the phase II portion of this study started treatment in August 2009.	0.00% 0/16 • 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. The first participant of the phase II portion of this study started treatment in August 2009.
Gastrointestinal disorders Mucositis	18.8% 6/32 • Number of events 6 • 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. The first participant of the phase II portion of this study started treatment in August 2009.	6.2% 1/16 • Number of events 1 • 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. The first participant of the phase II portion of this study started treatment in August 2009.	6.2% 1/16 • Number of events 1 • 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. The first participant of the phase II portion of this study started treatment in August 2009.
Musculoskeletal and connective tissue disorders Musculoskeletal Other	0.00% 0/32 • 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. The first participant of the phase II portion of this study started treatment in August 2009.	6.2% 1/16 • Number of events 1 • 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. The first participant of the phase II portion of this study started treatment in August 2009.	6.2% 1/16 • Number of events 1 • 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. The first participant of the phase II portion of this study started treatment in August 2009.
Skin and subcutaneous tissue disorders Petechiae	0.00% 0/32 • 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. The first participant of the phase II portion of this study started treatment in August 2009.	6.2% 1/16 • Number of events 1 • 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. The first participant of the phase II portion of this study started treatment in August 2009.	0.00% 0/16 • 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. The first participant of the phase II portion of this study started treatment in August 2009.
Skin and subcutaneous tissue disorders Pruritus	28.1% 9/32 • Number of events 9 • 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. The first participant of the phase II portion of this study started treatment in August 2009.	0.00% 0/16 • 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. The first participant of the phase II portion of this study started treatment in August 2009.	0.00% 0/16 • 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. The first participant of the phase II portion of this study started treatment in August 2009.
Skin and subcutaneous tissue disorders Skin Rash	56.2% 18/32 • Number of events 18 • 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. The first participant of the phase II portion of this study started treatment in August 2009.	6.2% 1/16 • Number of events 1 • 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. The first participant of the phase II portion of this study started treatment in August 2009.	6.2% 1/16 • Number of events 1 • 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. The first participant of the phase II portion of this study started treatment in August 2009.
Gastrointestinal disorders Taste Alteration	0.00% 0/32 • 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. The first participant of the phase II portion of this study started treatment in August 2009.	6.2% 1/16 • Number of events 1 • 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. The first participant of the phase II portion of this study started treatment in August 2009.	0.00% 0/16 • 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. The first participant of the phase II portion of this study started treatment in August 2009.
Renal and urinary disorders Urinary Frequency	0.00% 0/32 • 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. The first participant of the phase II portion of this study started treatment in August 2009.	0.00% 0/16 • 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. The first participant of the phase II portion of this study started treatment in August 2009.	6.2% 1/16 • Number of events 1 • 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. The first participant of the phase II portion of this study started treatment in August 2009.
Metabolism and nutrition disorders Elevated Alkaline Phosphatase	18.8% 6/32 • Number of events 6 • 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. The first participant of the phase II portion of this study started treatment in August 2009.	0.00% 0/16 • 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. The first participant of the phase II portion of this study started treatment in August 2009.	0.00% 0/16 • 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. The first participant of the phase II portion of this study started treatment in August 2009.
Skin and subcutaneous tissue disorders Palmoplantar Dysesthesia	21.9% 7/32 • Number of events 7 • 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. The first participant of the phase II portion of this study started treatment in August 2009.	0.00% 0/16 • 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. The first participant of the phase II portion of this study started treatment in August 2009.	0.00% 0/16 • 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. The first participant of the phase II portion of this study started treatment in August 2009.
Gastrointestinal disorders Emesis	50.0% 16/32 • Number of events 16 • 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. The first participant of the phase II portion of this study started treatment in August 2009.	0.00% 0/16 • 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. The first participant of the phase II portion of this study started treatment in August 2009.	0.00% 0/16 • 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. The first participant of the phase II portion of this study started treatment in August 2009.
Blood and lymphatic system disorders Gastrointestinal Hemorrhage	9.4% 3/32 • Number of events 3 • 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. The first participant of the phase II portion of this study started treatment in August 2009.	0.00% 0/16 • 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. The first participant of the phase II portion of this study started treatment in August 2009.	0.00% 0/16 • 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. The first participant of the phase II portion of this study started treatment in August 2009.
Infections and infestations Neutropenic Fever	0.00% 0/32 • 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. The first participant of the phase II portion of this study started treatment in August 2009.	0.00% 0/16 • 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. The first participant of the phase II portion of this study started treatment in August 2009.	12.5% 2/16 • Number of events 2 • 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. The first participant of the phase II portion of this study started treatment in August 2009.
Blood and lymphatic system disorders Neutropenia	50.0% 16/32 • Number of events 16 • 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. The first participant of the phase II portion of this study started treatment in August 2009.	6.2% 1/16 • Number of events 1 • 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. The first participant of the phase II portion of this study started treatment in August 2009.	25.0% 4/16 • Number of events 4 • 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. The first participant of the phase II portion of this study started treatment in August 2009.
Blood and lymphatic system disorders Thrombocytopenia	40.6% 13/32 • Number of events 13 • 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. The first participant of the phase II portion of this study started treatment in August 2009.	6.2% 1/16 • Number of events 1 • 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. The first participant of the phase II portion of this study started treatment in August 2009.	6.2% 1/16 • Number of events 1 • 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. The first participant of the phase II portion of this study started treatment in August 2009.
Metabolism and nutrition disorders Elevated Amylase	6.2% 2/32 • Number of events 2 • 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. The first participant of the phase II portion of this study started treatment in August 2009.	0.00% 0/16 • 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. The first participant of the phase II portion of this study started treatment in August 2009.	0.00% 0/16 • 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. The first participant of the phase II portion of this study started treatment in August 2009.