An Open-label Study of the Safety, Pharmacokinetics, Efficacy, Pharmacodynamics, and Immunogenicity of Cipaglucosidase Alfa/Miglustat in Pediatric Subjects Aged 0 to < 18 Years With Late-onset Pompe Disease
Who is this study for? Pediatric patients with Pompe disease
What treatments are being studied? Cipaglucosidase Alfa+Miglustat
Status: Recruiting
Location: See all (23) locations...
Intervention Type: Biological, Drug
Study Type: Interventional
Study Phase: Phase 3
SUMMARY
This is a Phase 3, open-label, multicenter study to evaluate the safety, PK, efficacy, PD, and immunogenicity of Cipaglucosidase Alfa/Miglustat treatment in enzyme replacement therapy (ERT)-experienced and ERT-naïve pediatric subjects with Pompe disease, aged 0 to \< 18 years
Eligibility
Participation Requirements
Sex: All
Maximum Age: 17
Healthy Volunteers: f
View:
• Male or female subjects (ERT-naïve \[have never received a dose of rhGAA\] or ERT-experienced \[have received rhGAA every 2 weeks for at least 6 months immediately before enrollment, and if ERT dosage has been modified, must have been on the modified dosage for at least 3 months before enrollment\]) diagnosed with LOPD who are aged 12 to \<18 years at screening (Cohort 1 only) or aged 0 months to \< 12 years at screening (Cohort 2 only)
• Subject weighs ≤ 115 kg. (Cohort 1 Only)
• Subject must have a diagnosis of LOPD based on documentation as defined in study protocol
• If of reproductive potential and if sexually active, female and male subjects agree to use a highly effective method of contraception throughout the duration of the study and for up to 90 days after their last dose of Cipaglucosidase Alfa/Miglustat
• Subject has a sitting forced vital capacity (FVC) ≥ 30% of the predicted value for healthy Adolescents at screening (Cohort 1 only)
• Subject (aged 12 to \<18 years; Cohort 1) performs one 6-Minute Walk Test (6MWT) (≥ 75 meters) at screening that is valid, as determined by the clinical evaluator, or subject (aged ≥ 5 to \< 12 years; Cohort 2) performs one 6MWT (≥ 40 meters) at screening that is valid, as determined by the clinical evaluator
Locations
United States
Arkansas
Arkansas Children's Hospital
WITHDRAWN
Little Rock
Arizona
Neuromuscular Research Center
WITHDRAWN
Phoenix
California
UCSF Benioff Children's Hospital
WITHDRAWN
Oakland
Florida
University of Florida Clinical Research Center
RECRUITING
Gainesville
Wolfson Children's Hospital
RECRUITING
Jacksonville
Georgia
Woodruff Memorial Research Building
NOT_YET_RECRUITING
Atlanta
Michigan
Infusion Associates
WITHDRAWN
Grand Rapids
Minnesota
Mayo Clinic
WITHDRAWN
Rochester
Missouri
St. Louis Children's Hospital
RECRUITING
St Louis
North Carolina
Duke University Medical Center
RECRUITING
Durham
Ohio
Cincinnati Children's Hospital Medical Center
RECRUITING
Cincinnati
Pennsylvania
UPMC Children's Hospital of Pittsburgh
RECRUITING
Pittsburgh
Utah
University of Utah, Clinical and Translational Sciences Institute
RECRUITING
Salt Lake City
Virginia
Lysosomal and Rare Disorders Research and Treatment Center, Inc.