* Baby-COMET
Research type
Research Study
Full title
An Open-label, Multinational, Multicenter, Intravenous Infusion Study of the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of avalglucosidase alfa in Treatment naïve Pediatric Participants Less than or Equal to 6 Months of Age with Infantile-Onset Pompe Disease (IOPD)
IRAS ID
294578
Contact name
James Edward Davison
Contact email
Sponsor organisation
Sanofi-aventis recherche & développement
Eudract number
2020-004686-39
Clinicaltrials.gov Identifier
Clinicaltrials.gov Identifier
109569, IND; U1111-1246-6645, WHO UTN
Duration of Study in the UK
4 years, 4 months, 3 days
Research summary
The Sponsor has developed alglucosidase alfa as long-term enzyme-replacement therapy (ERT) for participants with a confirmed diagnosis of Pompe disease. Alglucosidase alfa treatment is globally approved for the treatment of Pompe disease based on its efficacy to prolong invasive ventilator-free survival in infants and its ability to stabilize respiratory function in children and adults with the disease. Despite the clear benefits of alglucosidase alfa, data suggest that there is room for improvement.
The Sponsor has initiated development of avalglucosidase alfa in an effort to address this unmet need. Four studies evaluated avalglucosidase alfa in participants with LOPD or IOPD (ACT14132). The ACT14132 study was the first exposure of avalglucosidase alfa in the IOPD population and showed that it is safe and effective and can stabilize or improve clinical outcomes in IOPD participants who showed incomplete response to alglucosidase alfa.
Study EFC14462 will be the first exposure of avalglucosidase alfa in treatment-naive participants with IOPD who are less than or equal to 6 months of age. The primary objective of this Phase 3, open-label study is to determine the effect of avalglucosidase alfa treatment on survival and invasive ventilator-free survival of IOPD participants less than or equal to 6 months of age after 52 weeks of treatment. Considering the rarity of the pediatric population presenting with the infantile-onset form of Pompe disease, a head-to-head (avalglucosidase alfa versus alglucosidase alfa) study to demonstrate statistical significance is not feasible and no active control is planned for study EFC14462. The highest dose previously evaluated in participants with IOPD (40 mg/kg qow) will be used to treat the most vulnerable participants aged less than or equal to 6 months of age with the most profound enzyme deficiency by providing a high-dose recombinant ERT to halt disease progression as soon as possible for better outcomes.
REC name
North West - Liverpool Central Research Ethics Committee
REC reference
22/NW/0260
Date of REC Opinion
14 May 2021
REC opinion
Further Information Favourable Opinion