Disease: Sickle Cell Disease, Hematological Diseases, Hemoglobinopathies, (NCT03745287)

Disease info:

Sickle cell disease is a group of disorders that affects hemoglobin, the molecule in red blood cells that delivers oxygen to cells throughout the body. People with this disorder have atypical hemoglobin molecules called hemoglobin S, which can distort red blood cells into a sickle, or crescent, shape.

Frequency:
Sickle cell disease is the most common inherited blood disorder in the United States, affecting 70,000 to 80,000 Americans. The disease is estimated to occur in 1 in 500 African Americans and 1 in 1,000 to 1,400 Hispanic Americans.
Official title:
A Phase 1/2 Study to Evaluate the Safety and Efficacy of a Single Dose of Autologous CRISPR-Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (CTX001) in Subjects With Severe Sickle Cell Disease
Who:

No information

Locations:

United States, California

United States, Illinois

United States, New York

United States, Pennsylvania

United States, Tennessee

United States, Texas

Belgium, Brussels

Canada, Toronto

Germany, Regensburg

Italy, Rome

 

 

Study start:
Nov. 27, 2018
Enrollment:
45 participants
Gene editing method:
CRISPR-Cas9
Gene:
BCL11A
Vector:
No information
IndicatorIndicator
IND Enabling Pre-clinical
Phase I Safety
Phase II Safety and Dosing
Phase III Safety and Efficacy

Status: Active recruiting

Description

This is a single-arm, open-label, multi-site, single-dose Phase 1/2 study in subjects 18 to 35 years of age with severe sickle cell disease (SCD). The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (hHSPCs) using CTX001.

Last updated: Apr. 25, 2020