A study to investigate the efficacy and safety of OTL-203 compared with SoC in subjects with MPS-IH
Research type
Research Study
Full title
A multi-center, randomized, active controlled clinical trial to evaluate the efficacy and safety of OTL-203 in subjects with mucopolysaccharidosis type I, Hurler syndrome (MPS-IH) compared to standard of care with allogeneic hematopoietic stem cell transplantation (allo-HSCT)
IRAS ID
1008060
Contact name
Liz Joule
Contact email
Sponsor organisation
Orchard Therapeutics (Europe) Ltd
Research summary
This Phase 3 study aims to compare a new gene therapy, called OTL-203, with a standard treatment called “allogeneic hematopoietic stem cell transplantation” (allo-HSCT), to find out which is better for the treatment of mucopolysaccharidosis Type I, Hurler syndrome in children aged >28 days to <30 months old.
Allo-HSCT uses healthy bone marrow stem cells from another person to replace defective stem cells and has been used by doctors for many years, although it is not considered an approved drug by medicines regulators.
Participants in this study will be randomised (like flipping a coin) into one of the two treatment groups, OTL-203 or allo-HSCT, and will have a 1 in 2 (or 50%) chance of receiving either treatment.
The study is planned to run in 1 UK hospital and a further 5 hospitals in the EU and USA and comprises a pre-treatment phase of around 3 months followed by a treatment phase, where participants will be given drugs to prepare them for treatment before they receive OTL-203 or allo-HSCT, and a follow-up phase. Participation in this study will last around 5 years. The study sponsor will also seek permission from parents/caregivers to follow participants who receive OTL-203 in this study for a total of 15 years after treatment in a separate study to monitor the long-term effects.
Participants and their parents/caregivers will need to attend multiple hospital visits during the study, where clinic staff will perform procedures including: physical examination, vital signs; tests of hearing, sight, motor skills and brain functioning; brain and spine scans using X-rays and magnetic resonance imaging; liver and spleen ultrasounds; tests looking at the health of the heart; blood, urine and bone marrow sample collection; lumbar puncture; and collection of information from their medical record and through questionnaires. Participants' parents/caregivers may also receive telephone calls from the study team to check the health of participants during the study.REC name
London - West London & GTAC Research Ethics Committee
REC reference
23/LO/0489
Date of REC Opinion
7 Dec 2023
REC opinion
Further Information Favourable Opinion