Evaluate Ofirnoflast in Adults With Very Low- to Intermediate-risk Myelodysplastic Syndromes Requiring Transfusions

NCT07738510 | Not Yet Recruiting Phase 2 DMC FDA Drug

• 1 other identifier: HT-6184-MDS-002
• Study Type: interventional
• Target: 50
• Locations: 0 countries
• Sites: N/A

Brief Summary

The primary objective of this study is to evaluate the efficacy and safety of ofirnoflast administered orally once daily in adults with very low- to intermediate-risk myelodysplastic syndromes (MDS) who are transfusion-dependent and have failed one to three prior therapies, in order to identify the optimal dose for continuation into a Phase 3 study. The secondary objectives of this study are to evaluate the extended hematologic response to ofirnoflast, to assess the safety and tolerability of ofirnoflast during the dose-selection phase, and to evaluate hematologic improvement with ofirnoflast treatment.

Conditions

Myelodysplastic Syndromes; Anemia

Keywords

Ofirnoflast, HT-6184, Myelodysplastic syndromes, MDS

Outcome Measures

Primary Outcomes (4)

• Assess safety of ofirnoflast

  The safety and tolerability of ofirnoflast will be evaluated based on the incidence of treatment-emergent adverse events (TEAEs)

  Weeks 1-24

• Assess safety of ofirnoflast

  The safety and tolerability of ofirnoflast will be evaluated based on the incidence dose modifications

  Weeks 1-24

• Assess safety of ofirnoflast

  The safety and tolerability of ofirnoflast will be evaluated based on the pharmacokinetic exposure data

  Weeks 1-24

• Assess the hematologic response to ofirnoflast

  The hematologic response to ofirnoflast will be evaluated by the duration of time the participants do not require red blood cell transfusions

  Weeks 1-24

Secondary Outcomes (4)

• Assess the extended hematologic response to ofirnoflast

  Weeks 1-24

• Assess the hematologic improvement on orfirnoflast

  Weeks 1-24

• Assess the hematologic improvement on orfirnoflast

  Weeks 1-24

• Assess the hematologic improvement on orfirnoflast

  Weeks 1-24

Study Arms (2)

Arm 1: Ofirnoflast

EXPERIMENTAL

Participants receive ofirnoflast once daily, including the 5 participants enrolled in the Phase A safety lead-in cohort. Treatment continues through the 24-Week Initial Treatment Period and, for participants demonstrating clinical benefit, the Extension Period (up to an additional 24 weeks).

Drug: Ofirnoflast

Arm 2: Ofirnoflast

EXPERIMENTAL

Participants randomized to this arm in Phase B receive ofirnoflast once daily. Treatment continues through the 24-Week Initial Treatment Period and, for participants demonstrating clinical benefit, the Extension Period (up to an additional 24 weeks).

Drug: Ofirnoflast

Interventions

Ofirnoflast DRUG

once daily.

Also known as: HT-6184

Arm 1: Ofirnoflast; Arm 2: Ofirnoflast

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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
• 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.
• 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.

You may not qualify if:

• Anemia due to other causes (e.g., iron deficiency).
• Known clinically significant anemia due to iron, vitamin B12, or folate deficiency; autoimmune or hereditary hemolytic anemia; or gastrointestinal bleeding.
• 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.

Sponsors & Collaborators

• Halia Therapeutics, Inc.: lead
• Parexel: collaborator

MeSH Terms

Conditions

Myelodysplastic Syndromes; Anemia

Condition Hierarchy (Ancestors)

Bone Marrow Diseases; Hematologic Diseases; Hemic and Lymphatic Diseases

Central Study Contacts

Associate Medical Director

CONTACT

385-355-4315; monitor@haliatx.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 27, 2026
• First Posted: July 31, 2026
• Study Start (Estimated): October 1, 2026
• Primary Completion (Estimated): October 1, 2028
• Study Completion (Estimated): December 1, 2028
• Last Updated: July 31, 2026

Record last verified: 2026-07