Disease: Thalassemia Genetic Diseases, Inborn Hematologic Diseases Hemoglobinopathies, (NCT03655678)

Disease info:

Beta thalassemia is a blood disorder that reduces the production of hemoglobin. Hemoglobin is the iron-containing protein in red blood cells that carries oxygen to cells throughout the body.

In people with beta thalassemia, low levels of hemoglobin lead to a lack of oxygen in many parts of the body.

People with beta thalassemia are at an increased risk of developing abnormal blood clots.

Frequency:
Beta thalassemia is a fairly common blood disorder worldwide. Thousands of infants with beta thalassemia are born each year. Beta thalassemia occurs most frequently in people from Mediterranean countries, North Africa, the Middle East, India,Central Asia
Official title:
A Phase 1/2 Study of the Safety and Efficacy of a Single Dose of Autologous CRISPR-Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (hHSPCs) in Subjects With Transfusion-Dependent β-Thalassemia
Who:

No information

Locations:

United States, California

United States, New York

United States, Tennessee

Canada, Toronto

Canada, Vancouver

Germany, Regensburg

Germany, Tuebingen

Italy, Rome

United Kingdom, London

 

Study start:
Sep. 14, 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 up to 12 subjects 18 to 35 years of age with transfusion-dependent β-thalassemia (TDT). 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: May. 14, 2020