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Enzyme Replacement Therapy

Avalglucosidase alfa (GZ402666) for Pompe Disease (COMET Trial)

Phase 3
Waitlist Available
Research Sponsored by Genzyme, a Sanofi Company
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
The participant has confirmed acid alpha-glucosidase (GAA) enzyme deficiency from any tissue source and/or 2 confirmed GAA gene mutations.
The participant must provide signed, informed consent prior to performing any study related procedures. Consent of a legally authorized guardian(s) is (are) required for legally minor participant as defined by local regulation. If the participant is legally minor, signed written consent shall be obtained from parent(s)/legal guardian and assent obtained from participants, if applicable.
Timeline
Screening 3 weeks
Treatment Varies
Follow Up week 50 to 145 in open-label long-term period
Awards & highlights

COMET Trial Summary

This trial is looking at the effects of a drug called avalglucosidase alfa on people with a disease called Pompe. The trial will measure how well the drug works by looking at how it affects the patients' respiratory muscle strength and their ability to walk. The trial will also look at the safety of the drug and how it affects the patients' quality of life.

Eligible Conditions
  • Pompe Disease

COMET Trial Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
Select...
You have a confirmed deficiency in the GAA enzyme or have two confirmed mutations in the GAA gene.
Select...
You or your legal guardian must sign a document saying that you understand what the study involves and agree to participate. If you are under 18, your parent or legal guardian will also need to sign this document, and you may need to give your own agreement as well.

COMET Trial Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~week 50 to 145 in open-label long-term period
This trial's timeline: 3 weeks for screening, Varies for treatment, and week 50 to 145 in open-label long-term period for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Vital capacity
Secondary outcome measures
Therapeutic procedure
PAP: Change From Baseline in 12-item Short-form Health Survey (SF-12): Physical Component Summary (PCS) and Mental Component Summary (MCS) Scores at Week 49
PAP: Change From Baseline in Lower Extremity Muscle Strength at Week 49 as Assessed by Hand-held Dynamometry (HHD)
+6 more

Side effects data

From 2023 Phase 3 trial • 100 Patients • NCT02782741
24%
Nasopharyngitis
24%
Back Pain
22%
Headache
18%
Fatigue
18%
Influenza
16%
Pain In Extremity
14%
Fall
12%
Diarrhoea
12%
Nausea
10%
Myalgia
10%
Contusion
10%
Arthralgia
10%
Dizziness
8%
Upper Respiratory Tract Infection
8%
Pruritus
8%
Vomiting
6%
Non-Cardiac Chest Pain
6%
Dyspepsia
6%
Paraesthesia
6%
Erythema
6%
Influenza Like Illness
6%
Cystitis
6%
Muscle Spasms
6%
Urticaria
6%
Oedema Peripheral
4%
Pain
4%
Alanine Aminotransferase Increased
4%
Cough
4%
Oropharyngeal Pain
4%
Abdominal Pain Upper
4%
Pyrexia
4%
Rash
2%
Renal Colic
2%
Calculus Urinary
2%
Hydronephrosis
2%
Dyspnoea
2%
Hypoventilation
2%
Respiratory Failure
2%
Nasal Congestion
2%
Syncope
2%
Breast Cyst
2%
Pneumonia
2%
Abdominal Pain
2%
Musculoskeletal Pain
2%
Hypertension
100%
80%
60%
40%
20%
0%
Study treatment Arm
PAP: Avalglucosidase Alfa
PAP: Alglucosidase Alfa
Open-label Period: Avalglucosidase Alfa
Open-label Period: Alglucosidase Alfa in PAP Then Avalglucosidase Alfa in Open-label

COMET Trial Design

2Treatment groups
Experimental Treatment
Active Control
Group I: avalglucosidase alfa (GZ402666)Experimental Treatment1 Intervention
Administered intravenously every 2 weeks
Group II: alglucosidase alfa (GZ419829)Active Control1 Intervention
Administered intravenously every 2 weeks
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Avalglucosidase alfa (GZ402666)
2016
Completed Phase 3
~100

Find a Location

Who is running the clinical trial?

Genzyme, a Sanofi CompanyLead Sponsor
524 Previous Clinical Trials
85,542 Total Patients Enrolled
Clinical Sciences & OperationsStudy DirectorSanofi
857 Previous Clinical Trials
2,018,855 Total Patients Enrolled

Frequently Asked Questions

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

Are there any historical precedence for Avalglucosidase alfa (GZ402666) in medical research?

"Avalglucosidase alfa (GZ402666) was first studied in 2014, and there have been 30 completed clinical trials to date. 8 of these studies are ongoing, with a majority taking place in Boston, Massachusetts."

Answered by AI

Across how many distinct geographical areas is this experiment being conducted?

"There are 26 total investigational sites for this clinical trial, with Site Number 8400010 in Boston, Site Number 8400016 in Gainesville, and Site Number 8400018 in Salt Lake City being a few of them."

Answered by AI

What other trials are similar to this one?

"Since 2014, Avalglucosidase alfa (GZ402666) has been studied in clinical trials. The first trial was completed in 2014 and was sponsored by Genzyme, a Sanofi company. After the initial study, which involved 19 patients, Avalglucosidase alfa (GZ402666) received approval for Phase 2 and 3 clinical trials. Today, there are 8 active trials for Avalglucosidase alfa (GZ402666) in 57 cities across 26 countries."

Answered by AI

Has the FDA greenlit Avalglucosidase alfa (GZ402666)?

"Avalglucosidase alfa (GZ402666) has been given a score of 3 for safety. This is due to the fact that it is a Phase 3 trial, meaning that there is both supportive data for efficacy and rounds of data affirming its safety."

Answered by AI
Recent research and studies
~12 spots leftby Mar 2025