Phase 1 Bone Marrow Failure Clinical Trials

3 recruitingPhase 1

What is a Phase 1 trial?

Phase 1 trials test a new treatment in a small group of people for the first time. The primary goal is to evaluate safety, determine a safe dosage range, and identify side effects. These studies typically enroll 20 to 80 participants.

Showing 1–3 of 3 trials

Recruiting

Phase 1

Imatinib to Increase RUNX1 Activity in Participants With Germline RUNX1 Deficiency

Inherited Bone Marrow Failure SyndromeFamilial Platelet Disorder With Predisposition to Myeloid Malignancies

National Cancer Institute (NCI)75 enrolled1 locationNCT06090669

Recruiting

Phase 1Phase 2

Reduced Intensity Conditioning and Familial HLA-Mismatched BMT for Non-Malignant Disorders

HemoglobinopathiesBone Marrow Failure SyndromesMetabolic Disorders+3 more

Washington University School of Medicine29 enrolled4 locationsNCT03128996

Recruiting

Phase 1Phase 2

Clinical Study of UCB Combined With UC-MSCs in the Treatment of Bone Marrow Failure Disorders

Bone Marrow Failure Disorders

Shandong Qilu Stem Cells Engineering Co., Ltd.100 enrolled8 locationsNCT05794425