MYH9 disorders

(No active clinical trials at this time)

Wiskott-Aldrich Syndrome/XLT

Baby Detect: Genomic Newborn Screening
Belgium
https://www.clinicaltrials.gov/study/NCT05687474?cond=Wiskott-Aldrich%20Syndrome&aggFilters=status:not%20rec&rank=1
Actively recruiting

Gray Platelet Syndrome

(No active clinical trials at this time)

Platelet Storage Pool Disorders

Study of Chediak-Higashi Syndrome
USA
https://www.clinicaltrials.gov/study/NCT00005917?cond=Platelet%20Storage%20Pool%20&aggFilters=status:not%20rec&rank=2
Actively recruiting

Clinical and Basic Investigations into Hermansky-Pudlak Syndrome
USA
https://www.clinicaltrials.gov/study/NCT00001456?cond=Platelet%20Storage%20Pool%20&aggFilters=status:not%20rec&rank=3
Actively recruiting

Bernard-Soulier Syndrome

(No active clinical trials at this time)

Congenital Amegakaryocytic Thrombocytopenia

Treosulfan-Based Conditioning Regimen Before a Blood or Bone Marrow Transplant for the Treatment of Bone Marrow Failure Diseases (BMT CTN 1904)
USA
https://www.clinicaltrials.gov/study/NCT04965597?cond=Congenital%20Amegakaryocytic%20Thrombocytopenia&aggFilters=status:not%20rec&rank=1
Actively recruiting

RUNX1

Protocol Title: Safety and Feasibility of Autologous CD34+ Hematopoietic Stem Cells Mobilization and Apheresis in Participants with RUNX1 Familial Platelet Disorder
USA
https://www.clinicaltrials.gov/study/NCT06414889?cond=RUNX1&rank=2
Actively recruiting

Low-Dose Sirolimus to Increase Hematopoietic Function in Patients with RUNX1 Familial Platelet Disorder
USA
https://www.clinicaltrials.gov/study/NCT06261060?cond=RUNX1&rank=3
Actively recruiting

Imatinib to Increase RUNX1 Activity in Participants with Germline RUNX1 Deficiency
USA
https://www.clinicaltrials.gov/study/NCT06090669?cond=RUNX1&rank=4
Actively recruiting

Longitudinal Studies of Patient With FPDMM
USA
https://www.clinicaltrials.gov/study/NCT03854318?cond=RUNX1&rank=7
Actively recruiting