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Enzyme Replacement Therapy

Cipaglucosidase Alfa + Miglustat for Pompe Disease (ROSSELLA Trial)

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
Cohort 2: Male or female subjects aged 0 to <6 months at Day 1 with documentation of IOPD genotype, hypertrophic cardiomyopathy at the time of diagnosis, and are ERT-naïve.
Be younger than 18 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to 5 years
Awards & highlights
No Placebo-Only Group
Pivotal Trial

Summary

This trial will study the safety, how well it works, and side effects of an experimental drug called cipaglucosidase alfa/miglustat in children with a disease called classic infantile-onset Pompe disease.

Who is the study for?
This trial is for children and teens aged 0 to <18 with Infantile-Onset Pompe Disease (IOPD). Participants must have seen benefits from previous treatment without major safety issues, or be new to treatment. They should not require invasive ventilation, have certain immune responses, or conditions affecting motor function. Girls in the trial cannot be pregnant or breastfeeding.
What is being tested?
The study tests Cipaglucosidase alfa/Miglustat's safety and effectiveness in kids who've had enzyme replacement therapy (ERT) and those who haven't. It's an open-label Phase 3 trial, meaning everyone knows what treatment they're getting, focusing on how the body processes the drugs and their impact on IOPD.
What are the potential side effects?
Possible side effects include reactions similar to allergies during infusion of the drug, digestive disturbances due to Miglustat, potential changes in blood sugar levels because of Cipaglucosidase alfa's role in breaking down glycogen storage disease like Pompe Disease.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
Select...
I am under 6 months old, have IOPD with heart issues, and haven't had enzyme replacement therapy.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to 5 years
This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 5 years for reporting.

Treatment Details

Awards & Highlights

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

Trial Design

2Treatment groups
Experimental Treatment
Group I: Cohort 2: Cipaglucosidase Alfa/Miglustat in ERT-naïve pediatric IOPD subjectsExperimental Treatment2 Interventions
Pediatric IOPD subjects \<6 months
Group II: Cohort 1: Cipaglucosidase Alfa/Miglustat in ERT-experienced pediatric IOPD subjectsExperimental Treatment2 Interventions
Pediatric IOPD subjects 6 months to \<18 years experiencing clinical decline
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Miglustat
2020
Completed Phase 4
~210

Find a Location

Who is running the clinical trial?

Amicus TherapeuticsLead Sponsor
54 Previous Clinical Trials
2,691 Total Patients Enrolled

Media Library

Cipaglucosidase alfa (Enzyme Replacement Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT04808505 — Phase 3
Pompe disease Research Study Groups: Cohort 1: Cipaglucosidase Alfa/Miglustat in ERT-experienced pediatric IOPD subjects, Cohort 2: Cipaglucosidase Alfa/Miglustat in ERT-naïve pediatric IOPD subjects
Pompe disease Clinical Trial 2023: Cipaglucosidase alfa Highlights & Side Effects. Trial Name: NCT04808505 — Phase 3
Cipaglucosidase alfa (Enzyme Replacement Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04808505 — Phase 3
~22 spots leftby Apr 2027
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