RECRUITING

A Study to Evaluate the Safety, Efficacy, PK, PD and Immunogenicity of Cipaglucosidase Alfa/Miglustat in IOPD Subjects Aged 0 to <18

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Conditions

Glycogen Storage Disease Type II Infantile Onset

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

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.

Official Title

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

Quick Facts

Study Start:2023-07-18
Study Completion:2027-04
Study Type:Not specified
Phase:Not Applicable
Enrollment:Not specified
Status:RECRUITING

Study ID

NCT04808505

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:Not specified to 17 Years
Sexes Eligible for Study:ALL
Accepts Healthy Volunteers:No
Standard Ages:CHILD
Inclusion CriteriaExclusion Criteria
  1. 1. Male or female subjects who are aged 6 months to \< 18 years on Day 1
  2. 2. Subject must have documentation of IOPD genotype
  3. 3. Subject must have had hypertrophic cardiomyopathy at the time of diagnosis
  4. 4. 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
  5. 5. 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
  6. 6. Subjects must have experienced a clinical decline on their current rhGAA dose and frequency
  7. 1. Male or female subjects who are aged 0 to \<6 months at Day 1
  8. 2. Subject must have documentation of IOPD genotype
  9. 3. Subject must have had hypertrophic cardiomyopathy at the time of diagnosis
  10. 4. Subject is ERT-naïve
  11. 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.
  1. 1. Subject requires invasive ventilation (eg, tracheostomy)
  2. 2. Subject is CRIM negative and has not received prophylactic immunomodulation (Cohort 1); Subject is CRIM negative and will not be receiving prophylactic immunomodulation (Cohort 2)
  3. 3. Subject has a history of life-threatening IARs/hypersensitivity (eg, anaphylaxis and severe cutaneous reactions) to ERT (eg, alglucosidase alfa, cipaglucosidase alfa, miglustat) or other iminosugars, or to any of the excipients, where rechallenge was unsuccessful
  4. 4. Subject has prior history of illness or condition known to affect motor function
  5. 5. Female subject is pregnant (or intends to get pregnant) or breastfeeding at screening (Cohort 1)

Contacts and Locations

Study Contact

For Site
CONTACT
215-921-7600
PompeSiteInfo@amicusrx.com
For Patient
CONTACT
215-921-7600
patientadvocacy@amicusrx.com

Study Locations (Sites)

University of Florida Clinical Research Center
Gainesville, Florida, 32610
United States
The Emory Clinic
Atlanta, Georgia, 30322
United States
Duke University Early Phase Research Unit
Durham, North Carolina, 27710
United States
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 54229
United States
UPMC Hospital of Pittsburgh
Pittsburgh, Pennsylvania, 15224
United States
University of Utah, Clinical and Translational Sciences Institute
Salt Lake City, Utah, 84108
United States

Collaborators and Investigators

Sponsor: Amicus Therapeutics

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2023-07-18
Study Completion Date2027-04

Study Record Updates

Study Start Date2023-07-18
Study Completion Date2027-04

Terms related to this study

Additional Relevant MeSH Terms

  • Glycogen Storage Disease Type II Infantile Onset