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Cyclophosphamide (NCT03520647)

Haplo-identical Transplantation for Severe Aplastic Anemia, Hypo-plastic MDS and PNH Using Peripheral Blood Stem Cells and Post-transplant Cyclophosphamide for GVHD Prophylaxis: a one-minute explainerNCT035206471 min

Cyclophosphamide trial for Severe Aplastic Anemia (SAA) (NCT03520647). This is a short film with English narration. The transcript is on this page, and the written brief is separate.

What the film says

Read the transcript

This trial is for patients aged 4–60 with selected severe aplastic anemia, MDS arising after aplastic anemia, or PNH. Prior-treatment requirements apply. A suitable fully matched donor is unavailable; a partly matched relative and backup graft source are required.

The idea: use the relative’s blood stem cells after chemotherapy and radiation. Cyclophosphamide after transplantation aims to prevent donor cells attacking recipients. Researchers measure survival without significant chronic graft-versus-host disease. Monitoring near NIH is required.

This phase 2 study plans 56 patients. NHLBI leads it at NIH in Bethesda, the sole recruiting US site.

Public record; not medical advice.

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