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 location...
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 ERT-experienced and ERT-naïve pediatric subjects with IOPD.

Eligibility
Participation Requirements
Sex: All
Maximum Age: 17
Healthy Volunteers: No
View:

• Cohort 1:

• Male or female subjects who are aged 6 mo to < 18 years on Day 1

• Prior to initiation of study drug subject must have documentation of IOPD genotype, CRIM status, and no or low measurable GAA enzyme activity

• 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 on a modified dose and regimen, at least 3 months prior to enrollment

• Subjects must have experienced a clinical decline on their current approved rhGAA dose and frequency

• Cohort 2:

• Male or female subjects who are aged 0 to <12 month at Day 1

• Prior to initiation of study drug subject must have documentation of IOPD genotype, CRIM status, and no or low measurable GAA enzyme activity

• Subject must have had hypertrophic cardiomyopathy at the time of diagnosis

• Subject is ERT-naive

Locations
United States
Florida
University of Florida Clinical Research Center
Recruiting
Gainesville
Contact Information
Primary
For Site
PompeSiteInfo@amicusrx.com
215-921-7600
Backup
For Patient
patientadvocacy@amicusrx.com
215-921-7600
Time Frame
Start Date: December 2022
Estimated Completion Date: February 2025
Participants
Target number of participants: 36
Treatments
Experimental: Cohort 1: Cipaglucosidase Alfa/Miglustat in ERT-experienced pediatric IOPD subjects
Weekly co-administration in pediatric IOPD subjects 6 months to <18 years experiencing clinical decline
Experimental: Cohort 2: Cipaglucosidase Alfa/Miglustat in ERT-naïve pediatric IOPD subjects
Weekly co-administration in pediatric IOPD subjects <12 months
Related Therapeutic Areas
Sponsors
Leads: Amicus Therapeutics

This content was sourced from clinicaltrials.gov

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