Avalglucosidase Alfa for Pompe Disease
(Mini-COMET Trial)
Trial Summary
What is the purpose of this trial?
This is a multi-stage, phase 2, open-label, multicenter, multinational, ascending dose cohort, repeated intravenous (IV) infusion study of avalglucosidase alfa in pediatric patients with Infantile-Onset Pompe Disease (IOPD) who have been previously treated with alglucosidase alfa for a minimum of 6 months immediately prior to study entry and have demonstrated clinical decline or unsatisfactory clinical response.
Research Team
Clinical Sciences & Operations
Principal Investigator
Sanofi
Eligibility Criteria
This trial is for children under 18 with infantile-onset Pompe disease who've been on alglucosidase alfa for at least 6 months. They must show signs of the disease worsening or not improving enough, and be able to follow the study plan. Pregnant or breastfeeding females can't join, nor those allergic to the study drugs or on certain other medications.Inclusion Criteria
Exclusion Criteria
Timeline
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive avalglucosidase alfa or alglucosidase alfa via IV infusion every other week for 25 weeks
Extension Treatment
Participants continue to receive avalglucosidase alfa IV infusion every other week
Follow-up
Participants are monitored for safety and effectiveness after treatment
Treatment Details
Interventions
- alglucosidase alfa GZ419829
- avalglucosidase alfa GZ402666
Find a Clinic Near You
Who Is Running the Clinical Trial?
Genzyme, a Sanofi Company
Lead Sponsor
David Meeker
Genzyme, a Sanofi Company
Chief Executive Officer since 2011
MD from the University of Vermont Medical School, Advanced Management Program at Harvard Business School
Jean-Paul Kress
Genzyme, a Sanofi Company
Chief Medical Officer since 2015
MD from Faculte Necker-Enfants Malades, Paris