A Study to Evaluate Ofirnoflast in Adults With Very Low- to Intermediate-risk Myelodysplastic Syndromes Requiring Red Blood Cell Transfusions

What this study involves

The registry does not currently label this study as recruiting. Ask about its status before planning a screening visit.

Study type

Interventional

Phase

Phase 2

Age range

18 years - Not specified

Sex eligible

All sexes

Healthy volunteers

Not accepted

Participants

70 Estimated

These are starting points, not an eligibility decision. The research team must review the full requirements.

About the research

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.

Who may be able to participate?

The list below is the sponsor’s full registry wording. It can include conditions, prior treatment, test results and other requirements. A match in search does not mean you qualify.

Inclusion Criteria:

• 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 Criteria:

• 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.
• 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.
• Diagnosis of MPN, CMML, or overlap MDS/MPN per WHO classification.
• Any condition or concomitant treatment that may impair absorption of orally administered study intervention.
• Uncontrolled infection or severe organ dysfunction.
• 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.
• History of stem cell, bone marrow, or solid organ transplant.
• Known hypersensitivity to ofirnoflast or its excipients.
• Severe renal or hepatic impairment
• Inability to swallow tablets.
• Participation in another interventional clinical study within 90 days prior to first dose.
• QTcF \>480 ms.
• Prior treatment with ofirnoflast.
• 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.

Treatment, groups and participation

The registry may describe treatments and study duration without a complete visit calendar. Ask the study team for the exact schedule, procedures, risks, costs and alternatives. Keep your usual care plan unless your treating clinician tells you otherwise.

Assignment

Random assignment

Masking

No masking

What researchers are measuring

Outcome measures describe what is being studied. They are not proof that a treatment works.

Primary measures

Secondary measures

Research locations (1)

The overall study status and a local site’s status can differ. Confirm that location’s availability before traveling.

1 locations

Study team and timeline

Lead sponsor

Halia Therapeutics, Inc.

Study start

2026-11 Estimated

Primary completion

2028-10 Estimated

Study completion

2028-12 Estimated

First posted

Jul 31, 2026 Actual

Last updated

Nov 10, 2026 Actual

Official study contacts