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NCT04808505PHASE3Recruiting

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

Amicus Therapeutics

Start Date

7/18/2023

Completion Date

7/1/2027

Summary

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.

Eligibility Criteria

Age Range: No minimum to 17 years

Inclusion Criteria: Cohort 1: 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 Cohort 2: 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 Long-term Extension (Cohort 1 or Cohort 2): 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. Exclusion Criteria: Cohort 1 and Cohort 2, unless specified 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)

Interventions

BIOLOGICAL

Cipaglucosidase alfa

DRUG

Miglustat

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Conditions

Glycogen Storage Disease Type II Infantile Onset

Locations

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

Universitätsklinikum Gießen und Marburg GmbH, Zentrum fur Kinderheilkunde und Jugendmedizin Abteilung fur Kinderneurologic, Sozialpadiatric und Epileptologie

Giessen, 35392

Germany

Universitätsklinikum Heidelberg - Pädiatrisches Klinisch-Pharmakologisches Studienzentrum (paedKliPS)

Heidelberg, 69120

Germany

SphinCS GmbH

Höchheim, 65239

Germany

Universitätsklinikum Münster Klinik für Kinder- und Jugendmedizin Albert-Schweitzer-Campus 1

Münster, 48149

Germany

AOU Federico II

Naples, 80131

Italy

Erasmus MC, Sophia Kinderziekenhuis

Rotterdam, 3015 GD

Netherlands

National Taiwan University Hospital

Taipei, 100

Taiwan

Great Ormond Street Hospital for Children NHS Foundation Trust

London, WC1N3JH

United Kingdom