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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, 4 tagged with Congenital amegakaryocytic thrombocytopenia itself.

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Part of

↑ Inherited thrombocytopenia (29)

Sub-types of Congenital amegakaryocytic thrombocytopenia

Congenital amegakaryocytic thrombocytopenia 1 3 trials

Including sub-types (8) Tagged with Congenital amegakaryocytic thrombocytopenia (4)

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

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…

Early phase 1 • Sponsor: Children's Hospital of Philadelphia • Aim: Disease control

Last updated Jun 27, 2026 12:39 UTC