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

NCT04808505 · Status: RECRUITING · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 36

Last updated 2026-01-22

No results posted yet for this 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.

Conditions

  • Glycogen Storage Disease Type II Infantile Onset

Interventions

BIOLOGICAL

Cipaglucosidase alfa

Sterile lyophilized powder intravenous (IV) infusion

DRUG

Miglustat

65 mg oral capsules

Sponsors & Collaborators

  • Amicus Therapeutics

    lead INDUSTRY

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
PARALLEL

Eligibility

Max Age
17 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2023-07-18
Primary Completion
2027-07-31
Completion
2027-07-31
FDA Drug
Yes

Countries

  • United States
  • Germany
  • Italy
  • Netherlands
  • Taiwan
  • United Kingdom

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04808505 on ClinicalTrials.gov