NCT05320198 · Disc Medicine, Inc
Study of DISC-0974 (RALLY-MF) in Participants With Myelofibrosis or Myelodysplastic Syndrome and Anemia
What this study is about
This phase 1b/2a where both patients and doctors know the treatment given study will assess the safety, tolerability, how the drug moves through the body and how the drug affects the body of DISC-0974 as well as categorize the effects on anemia response in subjects with myelofibrosis or myelodysplastic syndrome and anemia.
View original scientific description
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.
Interventions
DRUG
DISC-0974
DISC-0974 is administered subcutaneously.
Primary outcome measures
Safety and Tolerability of DISC-0974 (Phase 1b only)
Time frame: 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)
Time frame: 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)
Time frame: From Day 1 to the end of treatment on Day 169
Assessed by physical examinations
Safety and Tolerability of DISC-0974 (Phase 1b only)
Time frame: 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)
Time frame: 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)
Time frame: 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)
Time frame: 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)
Time frame: 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)
Time frame: 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.
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
Inclusion criteria
- for Participants with MF and Anemia Participants are eligible for the study if all of the following criteria apply: 1. Age 18 years or older at the time of signing the informed consent form (ICF). 2. 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 For Phase 2: In addition to the criteria above, DIPSS score of ≥2 (intermediate 1 risk) may also be included. 3. Washout of at least 28 days prior to Screening of the following treatments: 1. Androgens 2. EPO 3. Cladribine 4. Immunomodulators (lenalidomide, thalidomide) 5. Luspatercept/sotatercept 6. Systemic corticosteroids are permitted for non-hematological conditions if stable or decreasing dose for ≥28 days prior to Screening and receiving an equivalent to ≤10 mg prednisone for the 28 days immediately prior to Screening. Sc
Where
- Gilbert, Arizona
- Duarte, California
- Irvine, California
- Los Angeles, California
- San Francisco, California
- Aurora, Colorado
- Jacksonville, Florida
- Miami, Florida
- Tampa, Florida
- Atlanta, Georgia
- Ann Arbor, Michigan
- Rochester, Minnesota
And 12 more locations — see the full list below.
Related conditions & keywords
Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced Aug 12, 2026 · Source of record for eligibility and locations