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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 transplant cocktail aims to cut complications in bone marrow failure

Disease control Completed

This phase 2 trial tested a combination of treosulfan, fludarabine, and rabbit antithymocyte globulin before a bone marrow transplant in 40 people with bone marrow failure diseases like Diamond-Blackfan anemia and Shwachman-Diamond syndrome. The goal was to see if this conditioni…

Phase 2 • Sponsor: Fred Hutchinson Cancer Center • Aim: Disease control

Last updated Jun 27, 2026 11:00 UTC