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 States, Australia150 participantsStarted 2022-06-06
Plain-language summary
This phase 1b/2a open-label study will evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics, and clinical activity of DISC-0974 as well as categorize the effects on hematologic response in participants with myelofibrosis or myelodysplastic syndrome and anemia.
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
. Age 18 years or older at the time of signing the informed consent form (ICF).
. For Phase 1b: Dynamic International Prognostic Scoring System (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 World Health Organization (WHO) 2016 criteria.
. Washout of at least 28 days prior to Screening of the following treatments:
. Androgens
. EPO
. Cladribine
. Immunomodulators (lenalidomide, thalidomide)
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.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
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.
What they're measuring
1
Safety and Tolerability of DISC-0974 (Phase 1b only)
Timeframe: From Day 1 to the end of treatment on Day 169
2
Safety and Tolerability of DISC-0974 (Phase 1b only)
Timeframe: From Day 1 to the end of treatment on Day 169
3
Safety and Tolerability of DISC-0974 (Phase 1b only)
Timeframe: From Day 1 to the end of treatment on Day 169
4
Safety and Tolerability of DISC-0974 (Phase 1b only)
Timeframe: From Day 1 to the end of treatment on Day 169
5
Safety and Tolerability of DISC-0974 assessed through blood testing (Phase 1b only)
Timeframe: From Day 1 to the end of treatment on Day 169
6
Safety and Tolerability of DISC-0974 assessed through urine testing (Phase 1b only)
Timeframe: From Day 1 to the end of treatment on Day 169
7
Transfusion-dependent (TD) high cohort: transfusion independence (Phase 2 only)
. 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
. 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
Timeframe: From Day 1 to the end of treatment on Day 169