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Can a daily pill ease anemia in bone marrow disorders?

NCT ID NCT07738510

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting This study
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jul 31, 2026 · Last updated Jul 31, 2026

Summary

This trial tests an experimental oral drug, ofirnoflast, in adults with lower-risk myelodysplastic syndromes (MDS) who have anemia and need regular red blood cell transfusions. The goal is to see if the drug can reduce or eliminate transfusion needs and improve blood counts. Participants will receive one of several doses of the drug daily, and the study will compare doses to find the most effective and safe one for future testing.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
ofirnoflast (HT-6184), an oral drug taken once daily
What this could lead to
If successful, this could lead to a new oral treatment that reduces or eliminates the need for red blood cell transfusions in people with lower-risk myelodysplastic syndromes.
What could go wrong
This is an early-stage (phase 2b) trial with only 50 participants, so results may not hold up in larger studies. The drug may cause side effects or may not improve blood counts enough to make a real difference.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

About 50 people

The number the study aims to enrol. It can still change while the study runs.

Expected to start

Oct 2026

An estimate. Start dates often move.

Expected to finish

Dec 2028

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 years and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: 1. At least 18 years of age at the time of signing informed consent. 2. Capable of giving signed informed consent 3. Documented diagnosis of very low-, low-, or intermediate-risk MDS 4. Documented diagnosis of anemia 5. Relapsed or refractory disease after 1 to 3 prior lines of therapy for lower-risk MDS 6. Willing to provide a bone marrow aspirate at Screening. 7. Life expectancy of more than 6 months at screening. 8. Participants of childbearing potential must have a negative pregnancy test at screening (serum) and Day 1 (urine). 9. 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: 1. Anemia due to other causes (e.g., iron deficiency). 2. Known clinically significant anemia due to iron, vitamin B12, or folate deficiency; autoimmune or hereditary hemolytic anemia; or gastrointestinal bleeding. 3. History of hemoglobinopathies, intrinsic RBC membrane/enzyme defects, or hemolytic anemia. 4. 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. 5. Diagnosis of MPN, CMML, or overlap MDS/MPN per WHO classification. 6. Any condition or concomitant treatment that may impair absorption of orally administered study intervention. 7. Uncontrolled infection or severe organ dysfunction. 8. 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). 9. Prior treatment with disease-modifying agents (e.g., hypomethylating agents) or immunosuppressive therapy, except prior lenalidomide (permitted). 10. Treatment with cytotoxic chemotherapy or experimental agents within 4 weeks prior to first dose. 11. History of stem cell, bone marrow, or solid organ transplant. 12. Known hypersensitivity to ofirnoflast or its excipients. 13. Severe renal or hepatic impairment 14. Inability to swallow tablets. 15. Participation in another interventional clinical study within 90 days prior to first dose. 16. QTcF \>480 ms. 17. Prior treatment with ofirnoflast.

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Conditions

The condition(s) this trial relates to.

anemia myelodysplastic syndrome Myelodysplastic Syndromes

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

How to take part

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  1. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  2. A doctor treating you

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More trials for these conditions

Other studies related to the condition(s) this trial covers.