Phase 1 Severe Combined Immunodeficiency, X-Linked Clinical Trials

2 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 1–2 of 2 trials

Recruiting

Phase 1Phase 2

Phase I/II Trial of Lentiviral Gene Transfer for SCID-X1 With Low Dose Targeted Busulfan Conditioning

Gene TherapySevere Combined Immunodeficiency, X-Linked

David Williams12 enrolled4 locationsNCT03311503

Recruiting

Phase 1

Lentiviral Gene Therapy for X-linked Severe Combined Immunodeficiency

Severe Combined Immunodeficiency, X-Linked

Great Ormond Street Hospital for Children NHS Foundation Trust5 enrolled1 locationNCT03601286