NCT04091737

CSL200 Gene Therapy in Adults With Severe Sickle Cell Disease

A Phase 1 Pilot Study to Evaluate the Safety and Feasibility of Gene Therapy With CSL200 (Autologous Enriched CD34+ Cell Fraction That Contains CD34+ Cells Transduced With Lentiviral Vector Encoding Human γ-GlobinG16D and Short-Hairpin RNA734) in Adult Subjects With Severe Sickle Cell Disease

• Status: Terminated
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 1 (actual)
• Sponsor: CSL Behring · Industry
• Sex: All
• Age: 18 Years – 45 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Anemia, Sickle Cell

Interventions

Type	Name	Description
BIOLOGICAL	Autologous enriched CD34+ cell fraction that contains CD34+ cells transduced with lentiviral vector encoding human γ-globinG16D and short-hairpin RNA734	* Cryopreserved formulated autologous enriched CD34+ cell fraction that contains CD34+ cells transduced with lentiviral vector encoding human γ-globinG16D and short-hairpin RNA734 in a bag for infusion * Plerixafor to mobilize hematopoietic stem cells prior to each apheresis * Single dose melphalan before administration of CSL200

Timeline

• Start date: 2019-10-02
• Primary completion: 2021-05-05
• Completion: 2021-05-05
• First posted: 2019-09-17
• Last updated: 2021-06-18

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study