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Congenital amegakaryocytic thrombocytopenia

MONDO:0800451

Also known as: congenital amegakaryocytic thrombocytopenia

8 clinical trials for this condition and its sub-types.

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Sub-types

Congenital amegakaryocytic thrombocytopenia 1 (3)

Broader categories

Disease (680) Hereditary disease (176) Hematologic disorder (170) Thrombocytopenia (70) Blood platelet disease (17) Human disease (14) Disease of genetic or genomic mechanism (2) Inherited thrombocytopenia (2) Disease by body system or component (0) Disease by etiologic mechanism (0)

Trials to join now! 4 Not yet recruiting 1 Not yet finished but already full! 1 Completed 2

New drug combo aims to make bone marrow transplants safer for kids

Disease control Ongoing

This pilot study tests a fludarabine-based drug regimen to prepare children with bone marrow failure syndromes for a bone marrow transplant from a matched sibling donor. The goal is to help the donor cells successfully take root while reducing serious side effects. The study incl…

Phase: EARLY_PHASE1 • Sponsor: Children's Hospital of Philadelphia • Aim: Disease control

Last updated Jun 27, 2026 12:39 UTC