Can a daily pill ease anemia in bone marrow disorders?
NCT ID NCT07738510
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 Hide 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.
Get updates
Get notified about this study
Sign up to get updates when this study changes or when new studies for Anemia are added.
Genom att skicka in godkänner du våra Användarvillkor
Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
How to take part
Only the study team decides who joins. These are the ways to reach them.
-
The official record
ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.
-
A doctor treating you
A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Can adding venetoclax make donor stem cell transplants safer for High-Risk blood cancers?
- Can starting anemia drug sooner keep MDS patients off transfusions?
- Blood cancer registry tracks thousands to map disease course
- New drug combination targets Hard-to-Treat blood cancers
- Can IV iron boost blood levels and daily energy for mothers before and after delivery?
- Can a calming drug make cord blood transplants safer?