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

NCT07738510 | Not Yet Recruiting Phase 2 FDA Drug

• 1 other identifier: HT-6184-MDS-002
• Study Type: interventional
• Target: 70
• Locations: 0 countries
• Sites: N/A

Brief Summary

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.

Conditions

Myelodysplastic Syndromes; Anemia

Keywords

Ofirnoflast, HT-6184, Myelodysplastic syndromes, MDS

Outcome Measures

Primary Outcomes (2)

• 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.

  Days 1-28 of each Part 1 Cohort

• 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

  Weeks 1-24

Secondary Outcomes (6)

• Assess safety and tolerability of ofirnoflast across dose levels evaluated

  Weeks 1-24

• Evaluate the overall hematologic response to ofirnoflast

  Weeks 1-24

• Evaluate the duration of response in participants achieving transfusion independence with ofirnoflast

  Weeks 1-24

• Evaluate the hematologic improvement in participants treated with ofirnoflast

  Weeks 1-24

• Evaluate the hematologic improvement in participants treated with ofirnoflast

  Weeks 1-24

Study Arms (6)

Part 1: Low dose ofirnoflast

EXPERIMENTAL

Participants receive low dose ofirnoflast, up to 6 participants total. 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

Part 1: Medium-low dose ofirnoflast

EXPERIMENTAL

Participants receive medium-low dose ofirnoflast, up to 6 participants total. 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

Part 1: Medium-high dose ofirnoflast

EXPERIMENTAL

Participants receive medium-high dose ofirnoflast, up to 6 participants total. 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

Part 1: High dose ofirnoflast Optional

EXPERIMENTAL

Participants receive high dose ofirnoflast, up to 6 participants total. 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

Part 2: First Dose ofirnoflast Selected

EXPERIMENTAL

Twenty participants receive the first selected dose of ofirnoflast, dose to be determined during Part 1 of study. 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

Part 2: Second Dose ofirnoflast Selected

EXPERIMENTAL

Twenty participants receive the second dose of ofirnoflast selected, dose to be determined during Part 1 of study. 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

tablet

Also known as: HT-6184

Part 1: High dose ofirnoflast Optional; Part 1: Low dose ofirnoflast; Part 1: Medium-high dose ofirnoflast; Part 1: Medium-low dose ofirnoflast; Part 2: First Dose ofirnoflast Selected; Part 2: Second Dose ofirnoflast Selected

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
• 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.

You may not qualify if:

• 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.

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): November 1, 2026
• Primary Completion (Estimated): October 1, 2028
• Study Completion (Estimated): December 1, 2028
• Last Updated: October 2, 2026

Record last verified: 2026-09