RALLY-MF: A Phase 1b/2 Open-label Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Clinical Activity of DISC-0974 in Participants with Myelofibrosis or Myelodysplastic Syndrome and AnemiaA Phase 1b/2a Open-label Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of DISC-0974 in Participants with Myelofibrosis and Anemia RALLY-MF
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What's the purpose of this trial?

Researchers want to find the highest dose of DISC-0974 to use in people with myelofibrosis and anemia. Myelofibrosis happens when bone marrow cells called fibroblasts make too much fibrous (scar) tissue. The bone marrow is not able to make enough blood cells, which leads to anemia. Anemia is a reduced number of red blood cells, and it causes tiredness and shortness of breath.

DISC-0974 helps lower levels of a hormone called hepcidin, which controls iron levels. People with myelofibrosis often have high hepcidin levels and low iron levels, leading to anemia. By lowering hepcidin levels, DISC-0974 may treat your anemia. DISC-0974 is given as a subcutaneous (under the skin) injection.

This trial is currently open and accepting patients.

You may be able to join this trial if you:

The following criteria is a partial list of reasons why patients may be eligible to participate in this clinical trial. Further evaluation with a medical professional is required.

To join this study, there are a few conditions. You must:

  • Have myelofibrosis with anemia.
  • Have been on a stable dose or completed prior myelofibrosis treatments at least 4 weeks before getting DISC-0974.
  • Be able to walk and do routine activities for more than half the time you are awake.
  • Be age 18 or older.

Additional Trial Information

Phase 1/2

Enrollment: 150 patients (estimated)

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Published Results

A Phase 1b Study of DISC-0974, an Anti-Hemojuvelin Antibody, in Patients with Myelofibrosis and Anemia

November 05, 2024

At the time of data cut, 34 participants were enrolled at 5 dose levels: 14 mg (n=1), 28 mg (n=7), 50 mg (n=12), 75 mg (n=8), and 100 mg (n=6). Treatment with DISC-0974 resulted in meaningful and sustained hepcidin reductions with mean change from baseline (mean 88.2 ng/mL; range [8.7, 374.7]) reaching over 80% across treated participants. This corresponded to iron mobilization at all dose levels.

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Trial Locations

All Trial Locations

View all clinical trial locations sorted by state.

Florida

Mayo Clinic (Jacksonville)

Jacksonville, FL

Open and Accepting

Michigan

University of Michigan Comprehensive Cancer Center Rogel Cancer Center

Ann Arbor, MI

Open and Accepting

Minnesota

Mayo Clinic (Rochester)

Rochester, MN

Open and Accepting

Missouri

Alvin J. Siteman Cancer Center Washington University Medical Campus

St. Louis, MO

Open and Accepting

New Jersey

Memorial Sloan Kettering Basking Ridge

Basking Ridge, NJ

Open and Accepting

Memorial Sloan Kettering Monmouth

Middletown, NJ

Open and Accepting

Memorial Sloan Kettering Bergen

Montvale, NJ

Open and Accepting

New York

Memorial Sloan Kettering Cancer Center

New York, NY

Open and Accepting

Memorial Sloan Kettering Commack

Commack, NY

Open and Accepting

Mount Sinai Hospital Tisch Cancer Institute

New York, NY

Open and Accepting

Memorial Sloan Kettering Nassau

Uniondale, NY

Open and Accepting

Memorial Sloan Kettering Westchester

West Harrison, NY

Open and Accepting

North Carolina

Wake Forest Baptist - Comprehensive Cancer Center Atrium Health

Winston-Salem, NC

Open and Accepting

Ohio

Cleveland Clinic - Taussig Cancer Center Taussig Cancer Institute

Cleveland, OH

Open and Accepting

Oregon

Oregon Health and Science University OHSU Knight Cancer Institute

Portland, OR

Open and Accepting

Pennsylvania

Abramson Cancer Center - University of Pennsylvania (UPENN) University of Pennsylvania

Philadelphia, PA

Open and Accepting

Texas

MD Anderson Cancer Center The University of Texas

Houston, TX

Open and Accepting

Washington

Fred Hutchinson Cancer Research Center University of Washington Cancer Consortium / SCCA

Seattle, WA

Open and Accepting

Wisconsin

Medical College of Wisconsin Froedtert Hospital

Milwaukee, WI

Open and Accepting
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