NCT04091737View on ClinicalTrials.gov

CSL200 Gene Therapy in Adults With Severe Sickle Cell Disease

PHASE1TerminatedINTERVENTIONAL
Enrollment

1

Participants

Timeline

Start Date

October 2, 2019

Primary Completion Date

May 5, 2021

Study Completion Date

May 5, 2021

Conditions
Anemia, Sickle Cell
Interventions
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"

Trial Locations (1)

91010

City of Hope Medical Center, Duarte

Sponsors

Lead Sponsor

CSL Behring
All Listed Sponsors
lead

CSL Behring

INDUSTRY

NCT04091737 - CSL200 Gene Therapy in Adults With Severe Sickle Cell Disease | Biotech Hunter | Biotech Hunter