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

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.

Conditions

Glycogen Storage Disease Type II Infantile Onset

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Study Overview

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Study Details

Study overview

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.

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

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

Condition
Glycogen Storage Disease Type II Infantile Onset
Intervention / Treatment

-

Contacts and Locations

Gainesville

University of Florida Clinical Research Center, Gainesville, Florida, United States, 32610

Atlanta

The Emory Clinic, Atlanta, Georgia, United States, 30322

Durham

Duke University Early Phase Research Unit, Durham, North Carolina, United States, 27710

Cincinnati

Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio, United States, 54229

Pittsburgh

UPMC Hospital of Pittsburgh, Pittsburgh, Pennsylvania, United States, 15224

Salt Lake City

University of Utah, Clinical and Translational Sciences Institute, Salt Lake City, Utah, United States, 84108

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.

For general information about clinical research, read Learn About Studies.

Eligibility Criteria

  • 1. Male or female subjects who are aged 6 months to \< 18 years on Day 1
  • 2. Subject must have documentation of IOPD genotype
  • 3. Subject must have had hypertrophic cardiomyopathy at the time of diagnosis
  • 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. 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. Subjects must have experienced a clinical decline on their current rhGAA dose and frequency
  • 1. Male or female subjects who are aged 0 to \<6 months at Day 1
  • 2. Subject must have documentation of IOPD genotype
  • 3. Subject must have had hypertrophic cardiomyopathy at the time of diagnosis
  • 4. Subject is ERT-naïve
  • 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. Subject requires invasive ventilation (eg, tracheostomy)
  • 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. 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. Subject has prior history of illness or condition known to affect motor function
  • 5. Female subject is pregnant (or intends to get pregnant) or breastfeeding at screening (Cohort 1)

Ages Eligible for Study

to 17 Years

Sexes Eligible for Study

ALL

Accepts Healthy Volunteers

No

Collaborators and Investigators

Amicus Therapeutics,

Study Record Dates

2027-04