OBS17003
Research type
Research Study
Full title
A prospective observational study to describe clinical outcomes of alglucosidase alfa treatment in patients ≤6 months of age with infantile-onset Pompe disease (IOPD)
IRAS ID
294580
Contact name
James Davison
Contact email
Sponsor organisation
Sanofi
Clinicaltrials.gov Identifier
109569, IND
Duration of Study in the UK
3 years, 0 months, 19 days
Research summary
The Sponsor has developed alglucosidase alfa as a long-term ERT for patients with a confirmed diagnosis of Pompe disease. Alglucosidase alfa is globally approved for the treatment of Pompe disease, based on its effectiveness in prolonging invasive ventilation-free survival in infants and ability to stabilize respiratory function in children and adults. It is the current standard of care treatment for Pompe disease. Since its initial approval in 2006, there have been improvements in the diagnosis and management of IOPD. However, there is variability in real-world care depending on practice at treating sites. For example, a subset of pediatric patients have been reported who experience prolonged ventilation-free survival while receiving alglucosidase alfa but have slow declines in gross motor function and mobility starting at 18 24 months of age. Some pediatric and adult patients receiving alglucosidase alfa continue to have decline in respiratory function although slower than prior to treatment, while others do not show any effect of treatment. Therefore, it is imperative
to better understand the impact of site-specific real-life practices on the effects of alglucosidase alfa and on patient outcomes.The Sponsor is developing a new treatment for Pompe disease, avalglucosidase alfa, which has shown promising results in late-onset Pompe disease and will be assessed in a planned Phase 3 study in IOPD. It is therefore necessary to have more up-to-date data on current standard of care practice in the management of IOPD, this study will provide information on outcomes in patients with IOPD receiving alglucosidase alfa. This is a noninterventional observational study of current site-specific routine practice in IOPD treatment, to assess effectiveness and safety of intravenous alglucosidase alfa ERT in pediatric patients ≤6 months. This observational study will provide valuable results on the standard of care in IOPD, and on site-related routine treatment.
REC name
North East - Newcastle & North Tyneside 1 Research Ethics Committee
REC reference
21/NE/0094
Date of REC Opinion
1 Jul 2021
REC opinion
Further Information Favourable Opinion