CSL200 Gene Therapy in Adults With Severe Sickle Cell Disease

Summary

This is a phase 1 pilot study of CSL200 in adult subjects with severe sickle cell disease. The primary objectives of this study are to evaluate the safety of the following: collection of CD34+ hematopoietic stem / progenitor cells by apheresis after mobilization with plerixafor, reduced intensity conditioning with melphalan, and administration of CSL200.

Timeline

Start

2019-10-02

Primary completion

2021-05-05

Completion

2021-05-05

Outcome

Outcome not reported

Stopped: “Unanticipated delays, not for safety reasons”

Drugs

Indications