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Enzyme Replacement Therapy

Avalglucosidase Alfa for Pompe Disease (Baby-COMET Trial)

Phase 3
Recruiting
Research Sponsored by Sanofi
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Participants must have confirmed diagnosis of infantile-onset Pompe disease with specific genetic and enzymatic criteria
Participants must have cardiomyopathy at the time of diagnosis as per specified left ventricular mass index (LVMI) criteria
Must not have
Participants with symptoms of respiratory insufficiency, including any ventilation use (invasive or noninvasive) at the time of enrollment
Participant received any Pompe disease specific treatment, e.g. enzyme-replacement gene therapy (ERT)
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to 5 years
Awards & highlights
No Placebo-Only Group
Pivotal Trial

Summary

This trialwill assess safety & effectiveness of a drug for up to 4 years in people newly diagnosed with a rare genetic disorder. Visits every other week & potentially every week.

Who is the study for?
This trial is for babies with infantile-onset Pompe disease who haven't been treated before. They must have a confirmed diagnosis, known CRIM status, and cardiomyopathy. Babies can't join if they've been in avalglucosidase alfa trials, have major birth defects unrelated to Pompe disease, other serious diseases, or are on ventilation.
What is being tested?
The study tests the effectiveness and safety of a drug called avalglucosidase alfa in babies with Pompe Disease over up to 4 years. It includes regular visits every week or two for doses and monitoring how well the treatment works and its effects on the body.
What are the potential side effects?
While specific side effects aren't listed here, typical reactions may include immune responses to the medication (like allergies), potential organ-specific issues due to enzyme replacement therapy (ERT), infusion-related reactions, and general discomfort.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I have been diagnosed with infantile-onset Pompe disease.
Select...
I have been diagnosed with cardiomyopathy based on specific heart muscle measurements.
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My legal guardian can give consent for me.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
Select...
I am experiencing breathing difficulties and may be using a ventilator.
Select...
I have received treatment for Pompe disease, like enzyme 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

1Treatment groups
Experimental Treatment
Group I: Avalglucosidase alfaExperimental Treatment1 Intervention
Administered intravenously every 2 weeks

Find a Location

Who is running the clinical trial?

SanofiLead Sponsor
2,216 Previous Clinical Trials
4,047,614 Total Patients Enrolled
Genzyme, a Sanofi CompanyLead Sponsor
527 Previous Clinical Trials
185,732 Total Patients Enrolled
Clinical Sciences & OperationsStudy DirectorSanofi
876 Previous Clinical Trials
2,021,640 Total Patients Enrolled

Media Library

avalglucosidase alfa (Enzyme Replacement Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT04910776 — Phase 3
Pompe Disease Research Study Groups: Avalglucosidase alfa
Pompe Disease Clinical Trial 2023: avalglucosidase alfa Highlights & Side Effects. Trial Name: NCT04910776 — Phase 3
avalglucosidase alfa (Enzyme Replacement Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04910776 — Phase 3
~0 spots leftby Dec 2024
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