The primary objective of this study is to two part. First, dose selection for dose optimization based on safety of ofirnoflast. Second, to evaluate the efficacy of ofirnoflast administered in the study population.
The secondary objectives of this study are to evaluate the extended hematologic response to ofirnoflast, to assess the safety and tolerability of ofirnoflast during dose optimization, and to evaluate hematologic improvement with ofirnoflast treatment.
At least 18 years of age at the time of signing informed consent.
Capable of giving signed informed consent
Documented diagnosis of very low-, low-, or intermediate-risk MDS
Documented diagnosis of anemia
Anemia due to other causes (e.g., iron deficiency).
Low platelet count
Low neutrophil count
History of hemoglobinopathies, intrinsic RBC membrane/enzyme defects, or hemolytic anemia.
Parallel
Ofirnoflast
Drugtablet
6
Treatment groupsSee each treatment group below.
Dose selection for dose optimization
Incidence of dose-limiting toxicities during the 28-day DLT observation window at each dose level to determine the dose levels acceptable for advancement to Part 2.
Evaluate the efficacy of ofirnoflast in to low- to intermediate-risk MDS who are transfusion dependent
The efficacy will be evaluated by proportion of participants achieving red blood cell transfusion independence for at least 16 consecutive weeks
Assess safety and tolerability of ofirnoflast across dose levels evaluated
The safety and tolerability of ofirnoflast will be evaluated based on the incidence and severity of TEAEs
Evaluate the overall hematologic response to ofirnoflast
The hematologic response to ofirnoflast will be evaluated by proportion of participants achieving overall response
Evaluate the duration of response in participants achieving transfusion independence with ofirnoflast
The duration of hematologic response to ofirnoflast will be evaluated by the duration of transfusion independence greater than or equal to 16 consecutive weeks
Evaluate the hematologic improvement in participants treated with ofirnoflast
The hematologic improvement will be evaluated by assessment of red blood cells
Click on the lead sponsor to view all of their trials.
Halia Therapeutics, Inc.
Lead sponsor
Parexel
Collaborator
This trial is not recruiting at the moment. You can still explore other options:
