External comparator for alglucosidase alfa in IOPD
Research type
Research Study
Full title
A retrospective, observational study of disease management and clinical outcomes associated with alglucosidase alfa treatment among patients with infantile-onset Pompe disease (IOPD)
IRAS ID
294971
Contact name
Pronabesh DasMahapatra
Contact email
Sponsor organisation
Sanofi Genzyme
Clinicaltrials.gov Identifier
21BI03, Study registration number at Chief Investigator's organization
Duration of Study in the UK
0 years, 6 months, 18 days
Research summary
This a retrospective, observational study of disease management and clinical outcomes associated with alglucosidase alfa treatment among patients with infantile-onset Pompe disease (IOPD) - a rare, inherited and progressively debilitating disorder of multiple bodily systems. The study is a chart review, so research teams will only be abstracting data into secure study database.
The primary goal of this research is to inform on the real-world clinical care and all associated outcomes. The main benefit of this study is to help understand the IOPD's natural history, standard treatment practices and its results.
The study is planned in approximately 8 sites across the United States and Europe.It is planned to enroll about 20 paediatric study patients with confirmed diagnosis of IOPD before or at 26 weeks of age who meet the required eligibility criteria. The consenting procedure will be done for all parents of those patients. There is no formal study duration for enrolled patients.
REC name
North East - Tyne & Wear South Research Ethics Committee
REC reference
21/NE/0098
Date of REC Opinion
12 May 2021
REC opinion
Favourable Opinion