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 (14) 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: No
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
Arizona
Neuromuscular Research Center
Recruiting
Phoenix
California
UCSF Benioff Children's Hospital
Recruiting
Oakland
Florida
University of Florida Clinical Research Center
Recruiting
Gainesville
Michigan
Infusion Associates
Recruiting
Grand Rapids
Minnesota
Mayo Clinic
Recruiting
Rochester
Missouri
St. Louis Children's Hospital
Recruiting
Saint Louis
North Carolina
Duke University Medical Center
Recruiting
Durham
Virginia
Lysosomal and Rare Disorders Research and Treatment Center, Inc.
Recruiting
Fairfax
Other Locations
Australia
Women's and Children's Hospital
Not yet recruiting
North Adelaide
Canada
University of Calgary
Recruiting
Calgary
Japan
Gunma University Hospital
Recruiting
Gunma
Tohoku University Hospital
Recruiting
Miyagi
Tokyo Women's Medical University
Recruiting
Tokyo
Taiwan
National Taiwan University Hospital
Recruiting
Taipei
Contact Information
Primary
For Site
PompeSiteInfo@amicusrx.com
215-921-7600
Backup
For Patient
patientadvocacy@amicusrx.com
215-921-7600
Time Frame
Start Date: February 13, 2020
Estimated Completion Date: June 2026
Participants
Target number of participants: 22
Treatments
Experimental: Cipaglucosidase Alfa (ATB200)/Miglustat(AT2221)
Participants received Cipaglucosidase Alfa (ATB200) co-administered with Miglustat (AT2221) capsule
Related Therapeutic Areas
Sponsors
Leads: Amicus Therapeutics

This content was sourced from clinicaltrials.gov

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