Study of DISC-0974 (RALLY-MF) in Participants With Myelofibrosis or Myelodysplastic Syndrome and … (NCT05320198) | Clinical Trial Compass
RecruitingPhase 1/2
Study of DISC-0974 (RALLY-MF) in Participants With Myelofibrosis or Myelodysplastic Syndrome and Anemia
United States150 participantsStarted 2022-06-06
Plain-language summary
This phase 1b/2a open-label study will assess the safety, tolerability, pharmacokinetics and pharmacodynamics of DISC-0974 as well as categorize the effects on anemia response in subjects with myelofibrosis or myelodysplastic syndrome and anemia.
Who can participate
Age range18 Years
SexALL
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
✓. Age 18 years or older at the time of signing the informed consent form (ICF).
✓. For Phase 1b: DIPSS score of 3 to 4 (intermediate 2 risk) or ≥5 (high-risk) primary MF, post PV MF, and/or post ET MF, as confirmed in the most recent local bone marrow biopsy report, according to WHO 2016 criteria.55
✓. Washout of at least 28 days prior to Screening of the following treatments:
✓. Androgens
✓. EPO
✓. Cladribine
✓. Immunomodulators (lenalidomide, thalidomide)
✓. Luspatercept/sotatercept
Exclusion criteria
✕. Hereditary hemochromatosis
✕. Hemoglobinopathy or intrinsic RBC defect associated with anemia
✕. Total splenectomy
✕. Hematopoietic cell transplant within the past 2 years or graft vs host disease requiring immunosuppression
✕. Current anemia from iron deficiency, vitamin B12 or folate deficiency, infection, or bleeding
✕. Active immune-mediated hemolytic anemia
What they're measuring
1
Incidence of treatment-emergent adverse events (Phase 1b only)
Timeframe: up to 225 days
2
Incidence of clinically abnormal vital signs (Phase 1b only)
Timeframe: up to 225 days
3
Incidence of clinically abnormal physical exam (Phase 1b only)
Timeframe: up to 225 days
4
Incidence of clinically abnormal electrocardiograms (Phase 1b only)
Timeframe: up to 225 days
5
Incidence of abnormal laboratory test results (Phase 1b only)
Timeframe: up to 225 days
6
Transfusion-dependent (TD) high cohort: transfusion independence, 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 (Phase 2 only)
Timeframe: up to 225 days
7
TD low cohort: transfusion independence, 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 (Phase 2 only)
. Symptomatic bleeding, unrelated to surgery, in a critical area or organ and/or bleeding causing a decrease in Hgb of ≥2 g/dL or leading to transfusion of ≥2 units of RBCs in the 6 months prior to Screening
✕. Major surgery within 8 weeks prior to Screening or incomplete recovery from any previous surgery
Non-transfusion-dependent (nTD) cohort: anemia response, 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 (Phase 2 only)