SMD Clinical Trial
Official title:
A Phase I-II Trial of Acadesine in IPSS High and Int-2 SMD, LAM With 20-30% Marrow Blasts and CMML Type 2 Not Responding to Azacitidine or Decitabine for at Least 6 Courses or Relapsing After a Response
A phase I-II trial of Acadesine in IPSS high and int 2 myelodysplastic syndromes, acute
myeloid leukemia with 20-30% marrow blasts and chronic myelomonocytic leukemia type 2 not
responding to Azacitidine or Decitabine for at least 6 courses or relapsing after a
response:
Patients will receive 6 treatment cycles unless disease progression, transformation, or
unacceptable toxicity occurs, or the patient refuses to continue participating in the study.
Efficacy will be assessed at the end of the 2nd, 4th and 6th cycles. After 6 cycles,
patients demonstrating a response (CR, PR, marrow CR, or HI) will be able to continue with
cycles of Acadesine (at the same dose as in the preceding cycles, depending on their cohort)
until progression.
Primary objectives
Phase I:
To determine the maximal tolerated dose (MTD) and dose limiting toxicities (DLTs) of
increasing doses of IV Acadesine administered on D1, D3, D5, D8, D10 and D12 of 28 to 56
day-courses
Phase II:
To confirm safety and hematological toxicity in 18 additional patients
Secondary objectives:
Phase I:
- To determine response rates, as defined by the 2006 modified IWG criteria,
- To evaluate response duration, time to IPSS progression, and loss of RBC transfusion
independence in these patients.
- To evaluate hospitalization duration, rates of rehospitalization for non-hematological
toxicities, severe bleeding or febrile neutropenia.
Phase II:
To determine
- response rate as defined by the 2006 modified IWG criteria
- toxicity profile and safety
- response duration
- rate of progression to AML
- overall survival
;
Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment