Experimental drug for rare bone marrow disease shows early promise, but study halted
NCT ID NCT04409080
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study tested an experimental drug called REGN7257 in 17 people with severe aplastic anemia, a rare condition where the bone marrow stops making enough blood cells. The drug was given to patients whose disease did not get better or came back after standard immune-suppressing treatments. The main goal was to check safety and see if the drug could improve blood cell counts, but the study was stopped early.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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Cleveland Clinic Foundation
Cleveland, Ohio, 44195, United States
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Ewha Womans University Medical Centre
Seoul, Seoul Capital Area, 07985, South Korea
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Gachon University Gil Hospital
Incheon, Gyeonggi-do, 21565, South Korea
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Hopital Saint-Louis - APHP
Paris, Île-de-France Region, 75010, France
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King's College Hospital, London
London, SE5 9RS, United Kingdom
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Samsung Medical Center
Seoul, Seoul Capital Area, 06351, South Korea
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Seoul National University Hospital
Seoul, Seoul Capital Area, 03080, South Korea
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St James's University Hospital
Leeds, West Yorkshire, LS97TF, United Kingdom
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The Catholic University of Korea, Seoul St. Marys Hospital
Seoul, Seoul Capital Area, 06591, South Korea
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The University of Texas MD Anderson Cancer Center
Houston, Texas, 77030, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Experimental drug targets bone marrow failure
- Simpler stem cell collection could ease donor burden in Life-Threatening anemia
- New drug cocktail aims to free aplastic anemia patients from transfusions
- Aplastic anemia drug combo study pulled before starting
- New drug combo shows promise for rare blood disorder
- New stem cell infusion could boost transplant success in rare blood disorder