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Enzyme Replacement Therapy

Cipaglucosidase Alfa + Miglustat for Pompe Disease (ROSSELLA Trial)

Q: Does this research project have any historical precedent?

Cipaglucosidase alfa (ATB200) has been studied since 2016. The initial clinical trial was completed in 2016 and was sponsored by Amicus Therapeutics. Following the first trial in 2016, which involved 32, Cipaglucosidase alfa (ATB200) received its Phase 1 &#x26; 2 drug approval. Today there are 6 active trials for Cipaglucosidase alfa (ATB200) across 39 cities and 24 countries.

Q: Are there still vacancies in this experiment for test subjects?

From what is stated on clinicaltrials.gov, this trial is still looking for patients to enroll. The 8/1/2022 date marks when the trial was first posted, with the most recent update being 1/21/2022.

Q: How many people are willing to participate in this research?

That is correct. The website clinicaltrials.gov has listed this study as currently seeking participants. This study was originally posted on August 1st, 2020 and was last updated on January 21st, 2020. They are looking for a total of 22 individuals across 1 location.

Q: Has the FDA given ATB200 the green light?

Cipaglucosidase alfa (ATB200) is a Phase 3 trial medication, meaning that while there is data supporting its efficacy, there is also extensive safety data. Power team rates it a 3 on a scale of 1 to 3.

Q: Are there any previous cases of research done on Cipaglucosidase alfa (ATB200)?

There are 6 ongoing clinical trials researching Cipaglucosidase alfa (ATB200), 4 of which are in Phase 3. Of the 98 study locations for Cipaglucosidase alfa (ATB200), several are based in Houston, <a href="https://www.withpower.com/clinical-trials/texas">Texas</a>.

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 104 weeks

Awards & highlights

ROSSELLA 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.Check my eligibility

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.See study design

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.

ROSSELLA Trial 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.

ROSSELLA Trial Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 104 weeks

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~104 weeks

This trial's timeline: 3 weeks for screening, Varies for treatment, and 104 weeks for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Proportion of subjects with infusion-associated reactions (IARs)

ROSSELLA 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

0 / 1Eligibility criteria met

Find a Location

Who is running the clinical trial?

Amicus TherapeuticsLead Sponsor

54 Previous Clinical Trials

2,688 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

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Does this research project have any historical precedent?

"Cipaglucosidase alfa (ATB200) has been studied since 2016. The initial clinical trial was completed in 2016 and was sponsored by Amicus Therapeutics. Following the first trial in 2016, which involved 32, Cipaglucosidase alfa (ATB200) received its Phase 1 & 2 drug approval. Today there are 6 active trials for Cipaglucosidase alfa (ATB200) across 39 cities and 24 countries."

Answered by AI

Are there still vacancies in this experiment for test subjects?

"From what is stated on clinicaltrials.gov, this trial is still looking for patients to enroll. The 8/1/2022 date marks when the trial was first posted, with the most recent update being 1/21/2022."

Answered by AI

How many people are willing to participate in this research?

"That is correct. The website clinicaltrials.gov has listed this study as currently seeking participants. This study was originally posted on August 1st, 2020 and was last updated on January 21st, 2020. They are looking for a total of 22 individuals across 1 location."

Answered by AI

Has the FDA given ATB200 the green light?

"Cipaglucosidase alfa (ATB200) is a Phase 3 trial medication, meaning that while there is data supporting its efficacy, there is also extensive safety data. Power team rates it a 3 on a scale of 1 to 3."

Answered by AI

Are there any previous cases of research done on Cipaglucosidase alfa (ATB200)?

"There are 6 ongoing clinical trials researching Cipaglucosidase alfa (ATB200), 4 of which are in Phase 3. Of the 98 study locations for Cipaglucosidase alfa (ATB200), several are based in Houston, Texas."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

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

2023. "A Study to Evaluate the Safety, Efficacy, PK, PD and Immunogenicity of Cipaglucosidase Alfa/Miglustat in IOPD Subjects Aged 0 to <18". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT04808505.

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