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

ClinicalTrials.gov ID: NCT04808505

Public ClinicalTrials.gov record NCT04808505. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.

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ClinicalTrials.gov public records Last synced May 20, 2026, 1:05 AM EDT

Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.

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

Study identification

NCT ID: NCT04808505
Recruitment status: Recruiting
Study type: Interventional
Phase: Phase 3
Lead sponsor: Amicus Therapeutics; Industry
Enrollment: 36 participants

Conditions and interventions

Conditions

Glycogen Storage Disease Type II Infantile Onset

Interventions

Cipaglucosidase alfa Biological
Miglustat Drug

Biological · Drug

Eligibility (public fields only)

Age range: Up to 17 Years
Sex: All
Healthy volunteers: Healthy volunteers not accepted

This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.

Study timeline

Start date: Jul 17, 2023
Primary completion: Jun 30, 2027
Completion: Jun 30, 2027
Last update posted: Jan 21, 2026

2023 – 2027

United States locations

U.S. sites: 6
U.S. states: 6
U.S. cities: 6

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

Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.

Non-U.S. locations

This page focuses on the U.S. directory. The official record also lists 8 non-U.S. sites.

About this trial record page

What this page shows: Public field values for ClinicalTrials.gov record NCT04808505, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
What this page does not do: No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
Where the data comes from: Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
Last refresh: Last update posted Jan 21, 2026 · Synced May 20, 2026

Open the official record

The complete protocol, eligibility criteria, and contact information for NCT04808505 live on ClinicalTrials.gov.

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