Long Term Follow Up- MLD
Research type
Research Study
Full title
Long-term Efficacy and Safety follow-up of MLD Patients treated with atidarsagene autotemcel
IRAS ID
317067
Contact name
Alan Richardson
Contact email
Sponsor organisation
Orchard Therapeutics (Europe) Limited
Duration of Study in the UK
15 years, 0 months, 1 days
Research summary
MLD is a rare inherited disorder caused by pathogenic mutations in the arylsulfatase A (ARSA) gene leading to an accumulation of unwanted substances that can lead to degeneration of the protective fatty layer of the nervous system. Symptoms of this disease vary but it typically includes difficulty talking and walking, seizures and behavioral and personality changes.
Currently, no effective treatment is available to reverse the deterioration and loss of function that MLD causes. Libmeldy is the first medicinal product approved for the treatment of MLD.
Patients are being invited to participate in this study as they have MLD and have received, or are due to receive gene therapy treatment with Libmeldy (also known as OTL-200 and atidarsagene autotemcel).
This is a long term follow up, observational study which means that only information and samples generated from visits that are part of the participants regular medical care will be collected. No study drug or other experimental treatment/ procedures will be administered.
There are 2 groups in this study. Group 1 participants will be those that have received Libmeldy as part of a previous research study, and Group 2 participants will be those who received treatment with Libmeldy or are due to receive this commercially outside of a research study.
Approximately 72 participants with MLD will be invited to participate from all over the world.
Participation in this study is expected to last up to 15 years after the first Libmeldy treatment and visits will be scheduled at 3 months, 6 months, and 1, 2 and 3 years after Libmeldy for Group 2 and then at least once every year for all participants following this.
The aim of this study is to learn more about the long-term safety of Libmeldy and how well it works for the treatment of MLD.REC name
London - Central Research Ethics Committee
REC reference
22/PR/1117
Date of REC Opinion
14 Nov 2022
REC opinion
Further Information Favourable Opinion