- Conditions
- Inherited Bone Marrow Failure Syndrome, Familial Platelet Disorder With Predisposition to Myeloid Malignancies
- Interventions
- imatinib, TruSight Oncology
- Drug · Device
- Lead sponsor
- National Cancer Institute (NCI)
- NIH
- Eligibility
- 18 Years to 120 Years
- Enrollment
- 75 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2023 – 2027
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
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Showing 1–4
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- Conditions
- RUNX1 Familial Platelet Disorder
- Interventions
- G-CSF (filgrastim or biosimilar), Apheresis, Plerixafor
- Drug · Procedure
- Lead sponsor
- M.D. Anderson Cancer Center
- Other
- Eligibility
- 18 Years to 75 Years
- Enrollment
- 4 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2024 – 2027
- U.S. locations
- 1
- States / cities
- Houston, Texas
- Conditions
- Inherited Hematological Diseases, Rare Diseases, FPDMM
- Interventions
- Not listed
- Lead sponsor
- National Human Genome Research Institute (NHGRI)
- NIH
- Eligibility
- 1 Day to 100 Years
- Enrollment
- 1,000 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2019 – 2099
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Familial Platelet Disorder, Hematopoietic
- Interventions
- Sirolimus
- Drug
- Lead sponsor
- M.D. Anderson Cancer Center
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 10 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2028
- U.S. locations
- 1
- States / cities
- Houston, Texas