← Back to Search

Enzyme Replacement Therapy

Cipaglucosidase Alfa + Miglustat for Pompe Disease

Phase 3
Recruiting
Research Sponsored by Amicus Therapeutics
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Subject has a sitting forced vital capacity (FVC) ≥ 30% of the predicted value for healthy Adolescents at screening (Cohort 1 only)
Subject must have a diagnosis of LOPD based on documentation as defined in study protocol
Must not have
Subject has evidence of moderate to severe hypertrophic cardiomyopathy aligning with classic IOPD
Female subject is pregnant or breast-feeding at screening
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 52 weeks
Awards & highlights
Pivotal Trial
No Placebo-Only Group

Summary

This trial will study the safety and effectiveness of Cipaglucosidase Alfa/Miglustat in treating Pompe disease in children who have either received enzyme replacement therapy before or have not.

Who is the study for?
This trial is for pediatric patients with Pompe disease, aged from birth to less than 18 years. It includes those who have previously received enzyme replacement therapy and those who haven't. Participants must meet certain breathing capacity criteria, agree to use contraception if of reproductive age, and not exceed a weight limit (for older children).
What is being tested?
The study tests the safety and effectiveness of Cipaglucosidase Alfa/Miglustat in treating Pompe disease in children. This Phase 3 trial involves both experienced and new patients to enzyme replacement therapy, assessing how the body processes the drug, its efficacy, pharmacodynamics, and potential immune response.
What are the potential side effects?
While specific side effects are not listed here, common ones for treatments like Cipaglucosidase Alfa/Miglustat may include allergic reactions to ingredients, gastrointestinal symptoms such as nausea or diarrhea, fatigue or headaches. The exact side effects will be monitored throughout the study.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
Select...
My lung function is at least 30% of what is expected for a healthy person my age.
Select...
I have been diagnosed with Late-Onset Pompe Disease (LOPD).
Select...
I am diagnosed with LOPD and fall within the specified age range for my cohort.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
Select...
I have a severe thickening of my heart muscle.
Select...
I am currently pregnant or breastfeeding.
Select...
I have a history of conditions like Guillain-Barre syndrome that affect my movement.
Select...
I have received gene therapy before.
Select...
I need a ventilator for more than 6 hours a day when I'm awake.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~52 weeks
This trial's timeline: 3 weeks for screening, Varies for treatment, and 52 weeks for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Secondary study objectives
Assessment of pharmacokinetic parameters

Awards & Highlights

Pivotal Trial
The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.
No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups
Experimental Treatment
Group I: Cipaglucosidase Alfa (ATB200)/Miglustat(AT2221)Experimental Treatment2 Interventions
Participants received Cipaglucosidase Alfa (ATB200) co-administered with Miglustat (AT2221) capsule
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Cipaglucosidase Alfa
2018
Completed Phase 3
~130
Miglustat
2020
Completed Phase 4
~210

Find a Location

Who is running the clinical trial?

Amicus TherapeuticsLead Sponsor
54 Previous Clinical Trials
2,705 Total Patients Enrolled

Media Library

Cipaglucosidase Alfa (Enzyme Replacement Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT03911505 — Phase 3
Pompe Disease Research Study Groups: Cipaglucosidase Alfa (ATB200)/Miglustat(AT2221)
Pompe Disease Clinical Trial 2023: Cipaglucosidase Alfa Highlights & Side Effects. Trial Name: NCT03911505 — Phase 3
Cipaglucosidase Alfa (Enzyme Replacement Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT03911505 — Phase 3
~5 spots leftby Jun 2026
Unbiased ResultsWe believe in providing patients with all the options.
Your Data Stays Your DataWe only share your information with the clinical trials you're trying to access.
Verified Trials OnlyAll of our trials are run by licensed doctors, researchers, and healthcare companies.
Back to top