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Gene Therapy for Leukocyte Adhesion Deficiency-I (LAD-I)
NCT03825783Phase 1Withdrawn
- Sponsor
- Rocket Pharmaceuticals, Inc.
- Start
- Apr 2019
- Primary completion
- Oct 2021
- Modality
- Gene Editing, Gene Silencing, Stem Cell Therapies
- Countries
- 1
01
Overview
Phase 1, open-label, single-arm trial of marnetegragene autotemcel in Leukocyte Adhesion Deficiency Type 1 (LAD1), sponsored by Rocket Pharmaceuticals, Inc. Primary endpoints: Number of participants with treatment-related adverse events at 2 years and Survival following infusion of RP-L201 at 2 years. Planned start Apr 2019; status: withdrawn.
02
Drugs
marnetegragene autotemcel
03
Conditions
Leukocyte Adhesion Deficiency Type 1 (LAD1)
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