RALLY-MF

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

What's the purpose of the 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.

Trial status

Accepting patients

Phase

Phase 1/2

Enrollment

150

Last Updated

Participating Centers

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Experimental Treatments

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DISC-0974 is a monoclonal antibody that may help to increase iron levels in patients with certain blood disorders and cancers.

Arms / Cohorts

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Accepting patients

Exploratory MDS Cohort

Published Results

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

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.

2024-11-05T00:00:00Z Read more

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