Phase 1 Beta-Thalassemia Clinical Trials
4 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 1–4 of 4 trials
Recruiting
Phase 1Phase 2
ALS20-101 Lentiviral Gene Therapy for Beta Thalassemia
Beta-Thalassemia
Children's Hospital of Philadelphia12 enrolled1 locationNCT06364774
Recruiting
Phase 1
Hematopoietic Stem Cell BCL11A Enhancer Gene Editing for Severe β-Hemoglobinopathies
Beta-ThalassemiaSickle Cell DiseaseSickle Cell Anemia (HbSS, or HbSβ-thalassemia0)+1 more
Daniel Bauer10 enrolled1 locationNCT06647979
Recruiting
Phase 1Phase 2
An Open-label Study of a Gene Therapy Product (Vebeglogene Autotemcel) in Transfusion Dependent Beta-Thalassemia
Beta-Thalassemia
Lantu Biopharma6 enrolled2 locationsNCT06308159
Recruiting
Phase 1
A Multiple Ascending Dose Study of 9MW3011 in Patients With Non-transfusion-dependent β-thalassemia
Beta-Thalassemia
Mabwell (Shanghai) Bioscience Co., Ltd.40 enrolled2 locationsNCT06772766