Ofirnoflast for Adults With Lower-Risk Myelodysplastic Syndromes Who Need Red Blood Cell Transfusions

Trial details

Who runs it

Halia Therapeutics, Inc.

Trial code

NCT07738510

Record updated

10 November 2026

Original description

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.Read full original descriptionShow less

A Phase 2b, Multicenter, Open-Label, Dose-Escalation, and Randomized Dose-Optimization Study of Ofirnoflast (HT-6184) for the Treatment of Myelodysplastic Syndromes in Adults With Very Low- to Intermediate-Risk Myelodysplastic Syndromes Requiring Red Blood Cell Transfusions

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.

Inclusion: 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; Documented low serum erythropoietin level; Relapsed or refractory disease after 1 to 3 prior lines of therapy for lower-risk MDS; Willing to provide a bone marrow aspirate at Screening; Eastern Cooperative Group Performance Status (ECOG PS) of 0, 1, or 2; Life expectancy of more than 6 months at screening; Participants of childbearing potential must have a negative pregnancy test at screening (serum) and Day 1 (urine); Participants and partners must use contraception consistent with local regulations and protocol-defined criteria during the intervention period and for at least 30 days after the last dose; periodic abstinence and withdrawal are not acceptable methods.

Exclusion: Prior history of AML, secondary MDS, or other malignancy (except non-melanoma skin cancer or in situ cervical/breast carcinoma) unless disease-free for >1 year; Any condition or concomitant treatment that may impair absorption of orally administered study intervention; Concomitant intercurrent illness or condition that, per investigator judgment, would compromise safe participation (e.g., uncontrolled hypertension, uncontrolled seizure, unstable angina, new-onset/exacerbated cardiac arrhythmia); Prior treatment with disease-modifying agents (e.g., hypomethylating agents) or immunosuppressive therapy, except prior lenalidomide (permitted); Treatment with cytotoxic chemotherapy or experimental agents within 4 weeks prior to first dose; Participation in another interventional clinical study within 90 days prior to first dose; Prior treatment with ofirnoflast; History of hemoglobinopathies, intrinsic RBC membrane/enzyme defects, or hemolytic anemia; Uncontrolled infection or severe organ dysfunction; History of stem cell, bone marrow, or solid organ transplant; Known hypersensitivity to ofirnoflast or its excipients; Severe renal or hepatic impairment; Medications or food products that are strong inhibitors, substrates, and inducers of CYP3A4/5, CYP2C9, P-glycoprotein (P-gp) and/or Breast Cancer Resistance Protein (BCRP) may cause drug-drug interactions and should be avoided; Low platelet count; Low neutrophil count; Anemia due to other causes (e.g., iron deficiency); Diagnosis of MPN, CMML, or overlap MDS/MPN per WHO classification; Inability to swallow tablets; QTcF >480 ms.

ClinicalTrials.gov NCT07738510