Actively Recruiting
A Study to Evaluate the Safety, Efficacy, PK, PD and Immunogenicity of Cipaglucosidase Alfa/Miglustat in IOPD Subjects Aged 0 to <18
Led by Amicus Therapeutics · Updated on 2026-01-22
36
Participants Needed
14
Research Sites
206 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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
Official Title
A Study to Evaluate the Safety, Efficacy, PK, PD and Immunogenicity of Cipaglucosidase Alfa/Miglustat in IOPD Subjects Aged 0 to <18
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Male or female subjects aged 6 months to under 18 years on Day 1 (Cohort 1)
- Documentation of infantile-onset Pompe disease genotype
- History of hypertrophic cardiomyopathy at diagnosis
- Received enzyme replacement therapy (ERT) for at least 6 months prior to enrollment with stable dosage for at least 3 months if modified (Cohort 1)
- Subjects aged 12 to under 18 years must complete a valid 6-minute walk test (6MWT) of at least 75 meters at screening
- Subjects aged 5 to under 12 years must complete a valid 6MWT of at least 40 meters at screening
- Subjects aged 18 months to under 5 years must be ambulatory and likely able to perform a 6MWT of at least 40 meters when they turn 5 years old
- Experienced clinical decline on current rhGAA dose and frequency (Cohort 1)
- Male or female subjects aged 0 to under 6 months on Day 1 (Cohort 2)
- ERT-naive (Cohort 2)
- For long-term extension, subjects must have benefited from cipaglucosidase alfa/miglustat therapy during the 104-week primary treatment period with no significant safety concerns
You will not qualify if you...
- Requires invasive ventilation such as tracheostomy
- CRIM negative subjects without prophylactic immunomodulation (Cohort 1) or not receiving prophylactic immunomodulation (Cohort 2)
- History of life-threatening infusion-associated reactions or hypersensitivity to ERT, miglustat, or related substances with unsuccessful rechallenge
- Prior illness or condition known to affect motor function
- Female subjects who are pregnant, intend to get pregnant, or are breastfeeding at screening (Cohort 1)
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 14 locations
1
University of Florida Clinical Research Center
Gainesville, Florida, United States, 32610
Actively Recruiting
2
The Emory Clinic
Atlanta, Georgia, United States, 30322
Actively Recruiting
3
Duke University Early Phase Research Unit
Durham, North Carolina, United States, 27710
Actively Recruiting
4
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States, 54229
Actively Recruiting
5
UPMC Hospital of Pittsburgh
Pittsburgh, Pennsylvania, United States, 15224
Actively Recruiting
6
University of Utah, Clinical and Translational Sciences Institute
Salt Lake City, Utah, United States, 84108
Actively Recruiting
7
Universitätsklinikum Gießen und Marburg GmbH, Zentrum fur Kinderheilkunde und Jugendmedizin Abteilung fur Kinderneurologic, Sozialpadiatric und Epileptologie
Giessen, Germany, 35392
Actively Recruiting
8
Universitätsklinikum Heidelberg - Pädiatrisches Klinisch-Pharmakologisches Studienzentrum (paedKliPS)
Heidelberg, Germany, 69120
Actively Recruiting
9
SphinCS GmbH
Höchheim, Germany, 65239
Actively Recruiting
10
Universitätsklinikum Münster Klinik für Kinder- und Jugendmedizin Albert-Schweitzer-Campus 1
Münster, Germany, 48149
Actively Recruiting
11
AOU Federico II
Naples, Italy, 80131
Actively Recruiting
12
Erasmus MC, Sophia Kinderziekenhuis
Rotterdam, Netherlands, 3015 GD
Actively Recruiting
13
National Taiwan University Hospital
Taipei, Taiwan, 100
Withdrawn
14
Great Ormond Street Hospital for Children NHS Foundation Trust
London, United Kingdom, WC1N3JH
Actively Recruiting
Research Team
F
For Site
CONTACT
F
For Patient
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
2
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