Study of DISC-0974 for Myelofibrosis or Myelodysplastic Syndrome and Anemia
This study is testing a drug called DISC-0974 for people with myelofibrosis (a bone marrow disorder) or myelodysplastic syndrome (a group of disorders where blood cells don't mature properly) who also have anemia (low red blood cell count). The main goals are to see how safe DISC-0974 is and how well people tolerate it. Researchers will also look at how the drug affects your body and if it helps improve blood counts. You could be eligible if you are 18 years or older and have certain types of myelofibrosis with anemia. The current recruitment status is unclear.
- Study design
- This is an open-label study, meaning you and your doctors will know you are receiving DISC-0974. It is planned to include 150 participants.
- What's involved
- Not specified in the trial record.
- Compensation
- Not stated in the trial record.
- Follow-up
- Safety and tolerability will be measured from Day 1 to the end of treatment on Day 169.
AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.
Study of DISC-0974 (RALLY-MF) in Participants With Myelofibrosis or Myelodysplastic Syndrome and Anemia
At a glance
Conditions
Where it's being run
30 sites across 17 statesStudy leadership
- Will Savage, MD PhD · STUDY_DIRECTOR · Disc Medicine
Who to contact
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What this trial measures
- Safety and Tolerability of DISC-0974 (Phase 1b only)From Day 1 to the end of treatment on Day 169
Assessed by treatment-emergent adverse events
- Safety and Tolerability of DISC-0974 (Phase 1b only)From Day 1 to the end of treatment on Day 169
Assessed by vital signs through blood pressure (mmHg), heart rate (bpm), respiration rate (breaths/min), and temperature (°C)
- Safety and Tolerability of DISC-0974 (Phase 1b only)From Day 1 to the end of treatment on Day 169
Assessed by physical examinations
- Safety and Tolerability of DISC-0974 (Phase 1b only)From Day 1 to the end of treatment on Day 169
Assessed by electrocardiogram (ECG) parameters: heart rate, PR interval, QRS duration, QRS axis, QT interval, and QTcF interval)
- Safety and Tolerability of DISC-0974 assessed through blood testing (Phase 1b only)From Day 1 to the end of treatment on Day 169
The blood testing will include a hematology panel, blood chemistry panel, serology/virology panel, biomarker assessments, PK assessments, and Anti-Drug Antibodies
- Safety and Tolerability of DISC-0974 assessed through urine testing (Phase 1b only)From Day 1 to the end of treatment on Day 169
The urine testing will include a urinalysis
- Transfusion-dependent (TD) high cohort: transfusion independence (Phase 2 only)From Day 1 to the end of treatment on Day 169
Defined as the absence of packed red blood cell (PRBC) transfusions over any rolling 12-week interval during the treatment period with a minimum hemoglobin (Hgb) of 7 g/dL. Participants meeting this criterion will be considered to have a major response to treatment.
- TD low cohort: transfusion independence (Phase 2 only)From Day 1 to the end of treatment on Day 169
Defined as the absence of PRBC transfusions over any rolling 16-week interval during the treatment period with a minimum Hgb of 7 g/dL. Participants meeting this criterion will be considered to have a major response to treatment.
- Non-transfusion-dependent (nTD) cohort: anemia response (Phase 2 only)From Day 1 to the end of treatment on Day 169
Defined as the composite of the absence of transfusions over any rolling 12-week period and a concomitant mean Hgb increase of ≥1.5 g/dL over baseline. Participants meeting this criterion will be considered to have a major response to treatment.