Clinical Trial

Disease: Sickle Cell Disease, SCD, (NCT06287086)

Disease info:

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

The production of haemoglobin A, which is the principle type of haemoglobin in humans, is governed by 3 genes: HBA1, HBA2, and HBB. Each haemoglobin A molecule consists of two alpha and two beta chains, and mutations in either of the HBA or the HBB genes may result in abnormal haemoglobin molecules with reduced or diminshed function. Sickle cell diseaase arises from a single point mutation in the 6th codon of the beta-globin gene (HBB), which results in a valine instead of a glutamic acid in the haemoglobin beta-chain.

Abnormal haemoglobin ultimately leads to anaemia as well as other symptoms, depending on the exact mutations present. Diseases caused by defective haemoglobin fall into a larger category of diseases known as the "haemoglobinopathies" which also include the thalassemias, a related group of diseases that are characterised by reduced or deficient rather than abnormal haemoglobin. 

Frequency:
Sickle cell disease affects approximately 100,000 individuals in the USA and more than 3 million worldwide.
Official title:
Clinical Study on the Safety and Efficacy of a Single Intravenous Dose of CRISPR/Cas9-Edited Autologous CD34+ Hematopoietic Stem/Progenitor Cells (BRL-101) in the Treatment of Sickle Cell Disease
Who:

Contact

Name: Jun shi, PhD

Phone: 13920307855

Email: shijun@ihcams.ac.cn

Sponsor:

Bioray Laboratories

Partners:

Hospital of Hematology, Chinese Academy of Medical Sciences (Institute of Hematology, Chinese Academy of Medical Sciences)

Locations:

No information provided

Study start:
May. 14, 2024
Enrollment:
5 participants
Gene editing method:
CRISPR-Cas9
Type of edit:
Gene knock-down
Gene:
BCL11A
Delivery method:
- Ex-vivo
IND Enabling Pre-clinical
Phase I Safety
Phase II Safety and Dosing
Phase III Safety and Efficacy

Status: Not yet recruiting

Description

This clinical trial is a single-arm, single-dose, single center, open-label study without dose escalation. The proposed dose is ≥ 3 × 106 CD34 + cells/kg administered as a single intravenous infusion. The primary objective is to explore the safety of the study drug in SCD. Myeloablative conditioning and administration for the remaining subjects can only be started after the first subject completes dosing and safety observation and assessment.

Last updated: Mar. 11, 2024
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