Trial Profile

Base Editing Hematopoietic Stem Cell Gene Therapy for CD40L-HyperIgM Syndrome-Single Patient Study

Status: Recruiting

Phase of Trial: Phase I/II

Latest Information Update: 25 Jun 2025

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At a glance

Drugs Alemtuzumab (Primary) ; Busulfan (Primary) ; Palifermin (Primary) ; Sirolimus (Primary)
Indications Type 1 hyper-IgM immunodeficiency syndrome
Focus Adverse reactions; Therapeutic Use

Most Recent Events

22 Jun 2025 Planned initiation date (estimated date for recruitment of the first subject) changed from 2 Jun 2025 to 26 Jun 2025.
28 May 2025 Planned initiation date (estimated date for recruitment of the first subject) changed from 30 May 2025 to 2 Jun 2025.
25 May 2025 Planned initiation date changed from 28 May 2025 to 30 May 2025.

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