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Gene Correction in Autologous CD34+ Hematopoietic Stem Cells (HbS to HbA) to Treat Severe Sickle Cell Disease — RESTORE  

Sickle Cell Disease Clinical Trial

Official title:
A Phase I/II Study of Nula-cel in Autologous CD34+ Hematopoietic Stem Cells to Convert HbS to HbA for Treating Severe Sickle Cell Disease

Clinical Trial Summary

This study is a first-in-human, single-arm, open-label Phase I/II study of nula-cel in approximately 15 participants, diagnosed with severe Sickle Cell Disease. The primary objective is to evaluate safety of the treatment in this patient population, as well as preliminary efficacy and pharmacodynamic data.

Clinical Trial Description

Participants diagnosed with severe SCD will receive nula-cel via IV infusion following myeloablative conditioning in an autologous HSCT setting. ;

Study Design

Related Conditions & MeSH terms

Clinical Trial Details
NCT number NCT04819841
Study type Interventional
Source Kamau Therapeutics
Contact Jenni Herber
Phone 303-437-7356
Email jherber@kamautx.com
Status Recruiting
Phase Phase 1/Phase 2
Start date November 15, 2021
Completion date July 31, 2027
View Details
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