Disease: Thalassemia Genetic Diseases, Inborn Hematologic Diseases Hemoglobinopathies

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:
The disorder affects all genders but is more prevalent in certain ethnicities and age groups. In the United States, thalassemia’s prevalence is approximately 1 in 272,000 or 1,000 people.
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

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.