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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

Timeline

Screening 3 weeks

Treatment Varies

Follow Up at 12 and 18 months of age

Awards & highlights

Baby-COMET 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.Check my eligibility

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.See study design

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.

Baby-COMET Trial 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.

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I have been diagnosed with cardiomyopathy based on specific heart muscle measurements.

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My legal guardian can give consent for me.

Baby-COMET Trial Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ at 12 and 18 months of age

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~at 12 and 18 months of age

This trial's timeline: 3 weeks for screening, Varies for treatment, and at 12 and 18 months of age 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 participants who are alive and free of invasive ventilation at Week 52

Secondary outcome measures

Change from baseline to Week 52 in Alberta Infant Motor Scale (AIMS) score

Change from baseline to Week 52 in body length Z-scores

Change from baseline to Week 52 in body length percentiles

+18 more

Baby-COMET Trial Design

1Treatment groups

Experimental Treatment

Group I: Avalglucosidase alfaExperimental Treatment1 Intervention

Administered intravenously every 2 weeks

0 / 3Eligibility criteria met

Find a Location

Who is running the clinical trial?

SanofiLead Sponsor

2,163 Previous Clinical Trials

3,512,462 Total Patients Enrolled

Genzyme, a Sanofi CompanyLead Sponsor

524 Previous Clinical Trials

85,612 Total Patients Enrolled

Clinical Sciences & OperationsStudy DirectorSanofi

857 Previous Clinical Trials

2,019,281 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

Frequently Asked Questions

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

Are we still able to sign new patients up for this experiment?

"That is correct, the listing on clinicaltrials.gov does show that this study is still open to enrolling patients. The trial was first posted September 1st, 2021 and updated October 27th, 2022. There are 18 positions available for patients at 2 different locations."

Answered by AI

Are there any safety concerns you can share about Avalglucosidase alfa?

"There is some efficacy data and multiple safety studies for Avalglucosidase alfa, so it was given a score of 3."

Answered by AI

Who meets the prerequisites to participate in this experiment?

"This study is for infants 0 days to 12 months old who have been diagnosed with acid maltase deficiency. There are currently 18 slots available for participants."

Answered by AI

How does Avalglucosidase alfa compare to other treatments?

"Avalglucosidase alfa is being trialled in 4 studies at the moment, 2 of which are large-scale Phase 3 clinical trials. The 106 research sites for Avalglucosidase alfa are mostly located in Rotterdam and Oregon."

Answered by AI

For this particular research, are elderly patients being sought out?

"This trial is enrolling infants who are less than one year old."

Answered by AI

Is this a trial for an innovative new treatment?

"Currently, there are 4 Avalglucosidase alfa trials being conducted in 24 cities and 26 countries. The first study was held in 2016 and completed Phase 3 drug approval in the same year. It is important to note that this study was sponsored by Genzyme, a subsidiary of Sanofi, and 100 patients were involved. Since 2016, 1 more study has been carried out."

Answered by AI

How many subjects are enrolled in this experiment?

"The latest information from clinicaltrials.gov suggests that this trial is still recruiting patients. The study began on September 1st, 2021 and will continue collecting data until October 27th, 2022. They need 18 more participants across 2 sites to complete the research goals."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

Clinical Study for Treatment-naïve IOPD Babies to Evaluate Efficacy and Safety of ERT With Avalglucosidase Alfa

2021. "Clinical Study for Treatment-naïve IOPD Babies to Evaluate Efficacy and Safety of ERT With Avalglucosidase Alfa". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT04910776.

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