Evaluation of Efficacy and Safety of a Single Dose of Exa-cel Subjects Severe SCD βS/βC Genotype
Research type
Research Study
Full title
A Phase 3 Study to Evaluate Efficacy and Safety of a Single Dose of Exa-cel in Subjects with Severe Sickle Cell Disease, βS/βC Genotype
IRAS ID
1007551
Contact name
Nina Liang
Contact email
Sponsor organisation
Vertex Pharmaceuticals Incorporated
ISRCTN Number
N/A
Research summary
This is a Phase 3, single-dose, open-label study in adult and adolescent participants with severe Sickle Cell Disease.
This study is being done to learn more about the safety and effectiveness of the drug exa-cel in patients with severe Sickle Cell Disease. The study drug is created by changing the DNA (material that makes up genes) in blood stem cells (gene therapy).
Ex-acel is a one-time infusion. To create it blood will be collected to obtain stem cells through a process called “mobilisation” and "apheresis". The blood stem cells will be collected and edited at a central facility. The Study Product is created using a technology called "CRISPR/Cas9", which stands for "Clustered Regularly Interspaced Short Palindromic Repeats/CRISPR associated 9 nuclease”.
The study will include participants aged 12-35 years inclusive.
The study will be conducted in 4 stages:
Stage 1: Screening (approximately 2-4 months) Screening tests are performed to see if participant can be in the study.
Stage 2: Blood stem cell collection and Study Product manufacture (approx 4 months/may be longer) Participants will receive a medication called plerixafor in order to release the blood stem cells from participant's bone marrow into bloodstream (a process called mobilisation) so that they can be collected through a process called apheresis. The cells collected from participant's bloodstream will be used to create the Study Product.
Stage 3: Chemotherapy conditioning and Study Product infusion (approximately 1 month) Participant will be admitted to the hospital at the study site to prepare participant's body to receive the Study Product.
Stage 4: Follow-up (approx 2 years after receiving Study Product).
Following completion of the study, participants will be enrolled in a follow up study for 15 years after dosing.
As of 01 June 2022, a total of 78 people who are 12 to 35 years of age have received exa-cel across 2 studies. To date, exa-cel has been generally safe and well-tolerated.REC name
London - West London & GTAC Research Ethics Committee
REC reference
23/LO/0280
Date of REC Opinion
3 Oct 2023
REC opinion
Further Information Favourable Opinion