An Open-label Study to Evaluate the Safety, Efficacy, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of Cipaglucosidase Alfa/Miglustat in Both ERT-experienced and ERT-naïve Pediatric Subjects With Infantile-onset Pompe Disease Aged 0 to < 18 Years
Who is this study for? Pediatric patients with infantile-onset Pompe disease aged 0 to < 18 years
What treatments are being studied? Cipaglucosidase alfa+Miglustat
Status: Recruiting
Location: See all (16) locations...
Intervention Type: Drug, Biological
Study Type: Interventional
Study Phase: Phase 3
SUMMARY
This is a Phase 3, open-label, multicenter study to evaluate the safety, efficacy, PK, PD, and immunogenicity of cipaglucosidase alfa/miglustat treatment in ERT-experienced and ERT-naïve pediatric subjects with IOPD.
Eligibility
Participation Requirements
Sex: All
Maximum Age: 17
Healthy Volunteers: f
View:
⁃ Cohort 1:
• Male or female subjects who are aged 6 months to \< 18 years on Day 1
• Subject must have documentation of IOPD genotype
• Subject must have had hypertrophic cardiomyopathy at the time of diagnosis
• Subject must have received ERT for at least 6 months immediately before enrollment. For subjects whose ERT dosage has been modified, the subject must have been on the modified dosage and regimen for at least 3 months before enrollment
• Subjects aged ≥ 12 to \< 18 years must perform one valid 6-minute walk test (6MWT) (≥ 75 meters) at screening; Subjects aged ≥ 5 to \< 12 years must perform one valid 6MWT (≥ 40 meters) at screening; Subjects aged 18 months to \< 5 years must be ambulatory and assessed to be likely to be able to perform 6MWT (≥ 40 meters) when they turn 5 years old
• Subjects must have experienced a clinical decline on their current rhGAA dose and frequency
⁃ Cohort 2:
• Male or female subjects who are aged 0 to \<6 months at Day 1
• Subject must have documentation of IOPD genotype
• Subject must have had hypertrophic cardiomyopathy at the time of diagnosis
• Subject is ERT-naïve
⁃ Long-term Extension (Cohort 1 or Cohort 2):
⁃ 1\. Subject must have, in the opinion of the investigator, benefited from therapy with cipaglucosidase alfa/miglustat during the 104-week primary treatment period with no significant safety concerns.
Locations
United States
Florida
University of Florida Clinical Research Center
RECRUITING
Gainesville
Georgia
The Emory Clinic
RECRUITING
Atlanta
North Carolina
Duke University Early Phase Research Unit
RECRUITING
Durham
Ohio
Cincinnati Children's Hospital Medical Center
RECRUITING
Cincinnati
Pennsylvania
UPMC Hospital of Pittsburgh
RECRUITING
Pittsburgh
Utah
University of Utah, Clinical and Translational Sciences Institute
RECRUITING
Salt Lake City
Other Locations
France
Hôpital Raymond Poincaré, Neurologie et réanimation pédiatriques
NOT_YET_RECRUITING
Garches
Germany
Universitätsklinikum Gießen und Marburg GmbH, Zentrum fur Kinderheilkunde und Jugendmedizin Abteilung fur Kinderneurologic, Sozialpadiatric und Epileptologie