Myelodysplastic SyndromesPhase 2NCT07738510
A Study to Evaluate Ofirnoflast in Adults With Very Low- to Intermediate-risk Myelodysplastic Syndromes Requiring Red Blood Cell Transfusions
A Phase 2 study testing Ofirnoflast in people with Myelodysplastic Syndromes and Anemia, run by Halia Therapeutics, Inc..
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Myelodysplastic Syndromes, Anemia
- What is being tested?
- Ofirnoflast
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 70 participants
- Where
- No sites listed yet
- Study dates
- Nov 2026to Dec 2028
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
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- 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.
Who cannot join
- 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.
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- 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.
Trial locations and contacts
No sites are listed yet. Contact the study team or a site near you to ask about taking part.
About this study
Official title: 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.
- Conditions
- Myelodysplastic Syndromes, Anemia
- Being tested
- Ofirnoflast
- Keywords
- Ofirnoflast, HT-6184, Myelodysplastic syndromes, MDS
- Medical subject headings
- Myelodysplastic Syndromes, Anemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
OfirnoflastDrug
Also called HT-6184
tablet
| Study group | Role | What they receive |
|---|---|---|
| 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). Receives: 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). Receives: 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). Receives: 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). Receives: 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). Receives: 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). Receives: Drug: Ofirnoflast |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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
Measured: Weeks 1-24
Secondary outcomes (6)
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
Measured: Weeks 1-24
Evaluate the overall hematologic response to ofirnoflast
The hematologic response to ofirnoflast will be evaluated by proportion of participants achieving overall response
Measured: Weeks 1-24
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
Measured: Weeks 1-24
Evaluate the hematologic improvement in participants treated with ofirnoflast
The hematologic improvement will be evaluated by assessment of red blood cells
Measured: Weeks 1-24
Evaluate the hematologic improvement in participants treated with ofirnoflast
The hematologic improvement will be evaluated by assessment of platelets
Measured: Weeks 1-24
Evaluate the hematologic improvement in participants treated with ofirnoflast
The hematologic improvement will be evaluated by assessment of neutrophils
Measured: Weeks 1-24
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted31 Jul 2026
- Study startNov 2026 (estimated)
- Primary completionOct 2028 (estimated)
- Study completionDec 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Myelodysplastic SyndromesStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 70 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Halia Therapeutics, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Parexel
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07738510
- Sponsor's protocol ID
- HT-6184-MDS-002
- Status last verified
- Sep 2026
- First submitted
- 27 Jul 2026
- Last updated
- 2 Oct 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 2 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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
- ClinicalTrials.gov ID
- NCT07738510
- Acronym / sponsor ID
- HT-6184-MDS-002
- Conditions
- Myelodysplastic Syndromes, Anemia
- Keywords
- Ofirnoflast, HT-6184, Myelodysplastic syndromes, MDS
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 70 participants (estimated)
- Lead sponsor
- Halia Therapeutics, Inc.Industry (pharma, biotech, devices)
- Collaborators
- Parexel
- Study start
- Nov 2026 (estimated)
- Primary completion
- Oct 2028 (estimated)
- Study completion
- Dec 2028 (estimated)
- First posted
- 31 Jul 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 6
| Arm | Type | Receives |
|---|---|---|
| Part 1: Low dose ofirnoflast | Experimental | Drug: OfirnoflastParticipants 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). |
| Part 1: Medium-low dose ofirnoflast | Experimental | Drug: OfirnoflastParticipants 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). |
| Part 1: Medium-high dose ofirnoflast | Experimental | Drug: OfirnoflastParticipants 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). |
| Part 1: High dose ofirnoflast Optional | Experimental | Drug: OfirnoflastParticipants 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). |
| Part 2: First Dose ofirnoflast Selected | Experimental | Drug: OfirnoflastTwenty 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). |
| Part 2: Second Dose ofirnoflast Selected | Experimental | Drug: OfirnoflastTwenty 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). |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (11 listed)
- 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
Key exclusion criteria (19 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| OfirnoflastAlso: HT-6184tablet | Drug | 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 |
Primary outcome
Dose selection for dose optimization
Time frame: Days 1-28 of each Part 1 Cohort
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
Time frame: Weeks 1-24
The efficacy will be evaluated by proportion of participants achieving red blood cell transfusion independence for at least 16 consecutive weeks
6 secondary outcomes
Assess safety and tolerability of ofirnoflast across dose levels evaluated
Time frame: Weeks 1-24
Evaluate the overall hematologic response to ofirnoflast
Time frame: Weeks 1-24
Evaluate the duration of response in participants achieving transfusion independence with ofirnoflast
Time frame: Weeks 1-24
Evaluate the hematologic improvement in participants treated with ofirnoflast
Time frame: Weeks 1-24
Evaluate the hematologic improvement in participants treated with ofirnoflast
Time frame: Weeks 1-24
Evaluate the hematologic improvement in participants treated with ofirnoflast
Time frame: Weeks 1-24
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 2 Oct 2026.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Ofirnoflast in people with Myelodysplastic Syndromes and Anemia, run by Halia Therapeutics, Inc..
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 11 inclusion and 19 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: opening soon, as of the sponsor's last update on 2 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Associate Medical Director (385-355-4315, monitor@haliatx.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Dose selection for dose optimization (time frame: Days 1-28 of each Part 1 Cohort). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 2 Oct 2026.