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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

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 52 weeks

Awards & highlights

Study 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).Check my eligibility

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.See study design

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

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My lung function is at least 30% of what is expected for a healthy person my age.

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I have been diagnosed with Late-Onset Pompe Disease (LOPD).

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I am diagnosed with LOPD and fall within the specified age range for my cohort.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 52 weeks

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

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

Primary outcome measures

Incidence of treatment-emergent adverse events (TEAEs) from baseline

Secondary outcome measures

Assessment of pharmacokinetic parameters

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

0 / 3Eligibility criteria met

Find a Location

Who is running the clinical trial?

Amicus TherapeuticsLead Sponsor

54 Previous Clinical Trials

2,702 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

Frequently Asked Questions

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

Does the age limit for this clinical trial extend to people who are under 20 years old?

"This study is looking for pediatric patients that are up to 17 years old."

Answered by AI

Is there previous research to support Miglustat's efficacy?

"Duke University Medical Center was the first to study miglustat back in 2016 and, since then, 12 more studies have completed. Right now, there are 6 active clinical trials taking place; a couple of these are based in Gainesville, Florida."

Answered by AI

Do a majority of hospitals in North America participate in this research?

"Currently, this clinical trial is being conducted at 9 different sites. If you are interested in enrolling, please choose the location closest to you from the following list of cities: Gainesville, Phoenix, Oakland and 6 others."

Answered by AI

Are there any available slots for willing participants?

"From what is posted on clinicaltrials.gov, it seems that this study is still enrolling patients. The original posting was on February 13th, 2020 and there was a recent update on July 14th, 2020."

Answered by AI

What makes this research unique compared to other projects like it?

"Amicus Therapeutics sponsored the first trial for Miglustat in 2016. The Phase 1 & 2 drug approval process was completed with the aid of 32 patients. Since then, 12 more studies have been completed in 24 countries and 39 cities."

Answered by AI

When did Miglustat receive FDA clearance?

"There is previous clinical evidence backing up Miglustat's safety, so it was given a 3."

Answered by AI

What are the qualifying factors for participants in this experiment?

"This trial is looking for 22 people with acid maltase deficiency, between the ages of 0 and 17. Patients must also meet the following criteria: Male or female subjects (ERT-naïve [have never received a dose of rhGAA] or ERT-experienced [have received rhGAA every 2 weeks for at least 6 months immediately before enrollment, and if ERT dosage has been modified, must have been on the modified dosage for at least 3 months before enrollment]) diagnosed with LOPD who are aged 12 to <18 years at screening (Cohort 1 only) or aged 0 months to < 12"

Answered by AI

Recent research and studies

clinicaltrials.govStudy

ZIP Study-OL Study of Safety, PK, Efficacy, PD, Immunogenicity of ATB200/AT2221 in Pediatrics Aged 0 to < 18 y.o. w/LOPD

2020. "ZIP Study-OL Study of Safety, PK, Efficacy, PD, Immunogenicity of ATB200/AT2221 in Pediatrics Aged 0 to < 18 y.o. w/LOPD". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT03911505.

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