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Lentiviral Gene Therapy for SCID-X1
NCT03601286Phase 1Recruiting
- Sponsor
- Great Ormond Street Hospital
- Start
- Dec 2018
- Primary completion
- Aug 2026
- Modality
- Gene Editing, Gene Silencing, Stem Cell Therapies
- Countries
- 1
01
Overview
Phase 1, open-label, single-arm trial of autologous CD34+ cells transduced with G2SCID vector in X-linked severe combined immunodeficiency (X-SCID), sponsored by Great Ormond Street Hospital. Primary endpoints: Event-free survival at 1 year, CD3+ T cell count at 1 year and 1 more. Started Dec 2018; status: recruiting.
02
Drugs
autologous CD34+ cells transduced with G2SCID vector
03
Conditions
X-linked severe combined immunodeficiency (X-SCID)
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