Phase 1 Thalassemia Clinical Trials

12 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.

Phase 1 Thalassemia Clinical Trials

What is a Phase 1 trial?

Partial Stem Cell Transplant for Sickle Cell Disease From Matched Donors

Study to Evaluate the Safety and Tolerability of Escalating Doses of Fostamatinib in Subjects With Stable Sickle Cell Disease

ALS20-101 Lentiviral Gene Therapy for Beta Thalassemia

Hematopoietic Stem Cell BCL11A Enhancer Gene Editing for Severe β-Hemoglobinopathies

An Open-label Study of a Gene Therapy Product (Vebeglogene Autotemcel) in Transfusion Dependent Beta-Thalassemia

T-Cell Depleted Alternative Donor Bone Marrow Transplant for Sickle Cell Disease (SCD) and Other Anemias

Β-Thalassemia Treatment with KL003 Cell Injection

A Multiple Ascending Dose Study of 9MW3011 in Patients With Non-transfusion-dependent β-thalassemia

Evaluating Safety and Efficacy of Lentiviral-transduced CD34+ HSCs in Β-thalassaemia Patients.

A Phase 1 Study of Gene-modified Autologous Hematopoietic Stem Cell (BD211) Treating β-thalassemia Major

Thal-Fabs: Reduced Toxicity Conditioning for High Risk Thalassemia

A Study Evaluating the Safety and Efficacy of the GMCN-508A Drug Product in Transfusion-dependent α-Thalassemia Participants