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Avalglucosidase Alfa for Pompe Disease (Baby-COMET Trial)
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.
Baby-COMET Trial Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowBaby-COMET Trial Timeline
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Baby-COMET Trial Design
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Who is running the clinical trial?
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- I am experiencing breathing difficulties and may be using a ventilator.I have been diagnosed with infantile-onset Pompe disease.You have participated in a clinical trial for avalglucosidase alfa before.I have received treatment for Pompe disease, like enzyme therapy.I have been diagnosed with cardiomyopathy based on specific heart muscle measurements.My legal guardian can give consent for me.You have a serious health condition that is not related to Pompe disease.You need to have a CRIM status test results before joining the study.
- Group 1: Avalglucosidase alfa
- 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.
- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
Frequently Asked Questions
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."
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."
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."
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."
For this particular research, are elderly patients being sought out?
"This trial is enrolling infants who are less than one year old."
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."
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."
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