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

Summary

The primary purpose of the Phase I portion of the study is to determine the safety profile and preliminary evidence of efficacy associated with infusion of autologous gene-corrected hematopoietic stem cells.

Timeline

Start

2019-04-15

Primary completion

2021-10

Completion

2021-10

Outcome

Outcome not reported

Stopped (Enrollment): “Patients were enrolled under global Phase 1/2 protocol RP-L201-0318.”

Drugs

Indications