Geneva, May 20 -- International Clinical Trials Registry received information related to the study (NCT06959771) titled 'Base Editing Hematopoietic Stem Cell Gene Therapy for CD40L-HyperIgM Syndrome: Single Patient Study' on May 6.
Study Type: Interventional
Study Design:
Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
Primary Sponsor: National Institute of Allergy and Infectious Diseases (NIAID)
Condition:
CD40L-HyperIgM Syndrome
Intervention:
Drug: Busulfan
Drug: Palifermin
Drug: Sirolimus
Drug: Alemtuzumab
Recruitment Status: Recruiting
Phase: Phase 1/Phase 2
Date of First Enrollment: May 14, 2025
Target Sample Size: 1
Countries of Recruitment:
Unite...