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Study of Safety and Efficacy of Genome-edited Hematopoietic Stem and Progenitor Cells in Sickle Cell Disease (SCD)

Sickle Cell Disease Clinical Trial

Official title:
A First-in-patient Phase I/II Clinical Study to Investigate the Safety, Tolerability and Efficacy of Genome-edited Hematopoietic Stem and Progenitor Cells in Subjects With Severe Complications of Sickle Cell Disease

Clinical Trial Summary

This study is evaluating a genome-edited, autologous, hematopoietic stem and progenitor cell (HSPC) product - OTQ923 to reduce the biologic activity of BCL11A, increasing fetal hemoglobin (HbF) and reducing complications of sickle cell disease.

Clinical Trial Description

CADPT03A12101 is a multicenter, multi-part, first-in-human, proof-of-concept, open label non-randomized, clinical study in Sickle Cell Disease (SCD) subjects. This study includes apheresis of mobilized hematopoietic stem and progenitor cells (HSPCs), ex vivo CRISPR/Cas9-mediated genome editing and expansion, followed by myeloablative conditioning and autologous hematopoietic stem cell transplant (HSCT) with follow-up for a minimum of one year and up to two years. The study is divided into the following parts: - Part A - Adult subjects dosed with OTQ923. - Part B - Assessment of OTQ923 in pediatric patients. Part B will not be opened. ;

Study Design

Related Conditions & MeSH terms

Clinical Trial Details
NCT number NCT04443907
Study type Interventional
Source Novartis
Contact
Status Active, not recruiting
Phase Phase 1
Start date August 26, 2020
Completion date September 18, 2026
View Details
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