RP-L201

BIOLOGICAL2 trials

Sponsors

Rocket Pharmaceuticals Inc.

Conditions

Leukocyte Adhesion Defect - Type I

Phase 1

A Clinical Trial to Evaluate the Safety and Efficacy of RP-L201 in Subjects With Leukocyte Adhesion Deficiency-I

CompletedNCT03812263

Rocket Pharmaceuticals Inc.Leukocyte Adhesion Defect - Type I

Start: 2019-08-30End: 2023-09-12Updated: 2023-11-15

A Gene Therapy Trial to Evaluate the Safety and Efficacy of RP-L201 in Subjects With Leukocyte Adhesion Deficiency-I

WithdrawnNCT03825783

Rocket Pharmaceuticals Inc.Leukocyte Adhesion Defect - Type I

Start: 2019-04-15End: 2021-10-31Updated: 2021-12-07

Related Papers

Autologous Ex Vivo Lentiviral Gene Therapy for Severe Leukocyte Adhesion Deficiency-I Achieves Durable Immune Reconstitution and Reduction of Infection-Related Morbidity: Updated 3.5-5.5-Year Results from a Phase I/II Study

Journal of Human Immunity2026-07-14

Lentiviral Gene Therapy for Severe Leukocyte Adhesion Deficiency Type 1

New England Journal of Medicine2025-05-017 citations

Interim Results from an Ongoing Phase 1/2 Study of Lentiviral-mediated <i>Ex-vivo</i> Gene Therapy for Pediatric Patients with Severe Leukocyte Adhesion Deficiency-I (LAD-I)

Blood2022-11-152 citations

A Phase 1/2 Study of Lentiviral-Mediated Ex-Vivo Gene Therapy for Pediatric Patients with Severe Leukocyte Adhesion Deficiency-I (LAD-I): Interim Results

Blood2021-11-056 citations

A Phase 1/2 Study of Lentiviral-Mediated <i>Ex-Vivo</i> Gene Therapy for Pediatric Patients with Severe Leukocyte Adhesion Deficiency-I (LAD-I): Results from Phase 1

Blood2020-11-0512 citations