Evaluate Ofirnoflast in Adults With Very Low- to Intermediate-risk Myelodysplastic Syndromes Requ… (NCT07738510) | Clinical Trial Compass
Not Yet RecruitingPhase 2
Evaluate Ofirnoflast in Adults With Very Low- to Intermediate-risk Myelodysplastic Syndromes Requiring Transfusions
50 participantsStarted 2026-10
Plain-language 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.
Who can participate
Age range
18 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
. 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).
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1This trial is testing a drug called ofirnoflast that I haven't heard of before — can you tell me what class of drug it is and how it's thought to help with myelodysplastic syndromes and transfusion-dependent anemia?
2Since this is a Phase 2 trial and the primary focus is still assessing safety, what does that mean about how much is already known regarding risks and side effects of ofirnoflast compared to treatments I might already be eligible for?
3The trial isn't recruiting yet — do you have a sense of when it might open, and given how uncertain that timeline is, would it make more sense to start a standard treatment now rather than waiting?
4My current situation requires regular blood transfusions — would enrolling in this trial affect my ability to continue receiving transfusions if ofirnoflast doesn't adequately improve my blood counts?
5Are there currently approved or standard-of-care options for very low- to intermediate-risk MDS with transfusion dependence that I should consider first before deciding whether a Phase 2 trial like this one is the right path for me?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
. 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).