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Clinical Trials in the UK / NCT04808505
Recruiting Phase 3

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

NCT04808505 · tracked via the Priya Life Science UK tracker
Sponsor
Amicus Therapeutics
Phase
Phase 3
Started
2023-07-18
Last updated
2026-01-22

Condition(s) studied

Glycogen Storage Disease Type II Infantile Onset

Investigational drug(s) / intervention(s)

Cipaglucosidase alfaMiglustat

Cipaglucosidase alfa: Sterile lyophilized powder intravenous (IV) infusion

Miglustat: 65 mg oral capsules

Study 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

Sex
ALL
Min age
Max age
17 Years
Healthy volunteers
No
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)

Primary outcome measure(s)

Trial sites (14)

FacilityCityRegionStatus
University of Florida Clinical Research Center Gainesville Florida Recruiting
The Emory Clinic Atlanta Georgia Recruiting
Duke University Early Phase Research Unit Durham North Carolina Recruiting
Cincinnati Children's Hospital Medical Center Cincinnati Ohio Recruiting
UPMC Hospital of Pittsburgh Pittsburgh Pennsylvania Recruiting
University of Utah, Clinical and Translational Sciences Institute Salt Lake City Utah Recruiting
Universitätsklinikum Gießen und Marburg GmbH, Zentrum fur Kinderheilkunde und Jugendmedizin Abteilung fur Kinderneurologic, Sozialpadiatric und Epileptologie Giessen Germany Recruiting
Universitätsklinikum Heidelberg - Pädiatrisches Klinisch-Pharmakologisches Studienzentrum (paedKliPS) Heidelberg Germany Recruiting
SphinCS GmbH Höchheim Germany Recruiting
Universitätsklinikum Münster Klinik für Kinder- und Jugendmedizin Albert-Schweitzer-Campus 1 Münster Germany Recruiting
AOU Federico II Naples Italy Recruiting
Erasmus MC, Sophia Kinderziekenhuis Rotterdam Netherlands Recruiting
National Taiwan University Hospital Taipei Taiwan Withdrawn
Great Ormond Street Hospital for Children NHS Foundation Trust London United Kingdom Recruiting

More Amicus Therapeutics trials in the UK

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT04808505 on ClinicalTrials.gov ↗ ← All trials in the UK