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Enzyme Replacement Therapy

Avalglucosidase Alfa for Pompe Disease (Mini-COMET Trial)

Phase 2

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

For participants in Stage 1: The participant has documented evidence of clinical decline in at least 1 of the following parameters related to Pompe Disease and not related to intercurrent illness as assessed by the Investigator: respiratory function, motor skills, and/or cardiac parameters.

The participant has cardiomyopathy at the time of diagnosis: i.e., left ventricular mass index (LVMI) equivalent to mean age specific LVMI plus 2 standard deviations.

Timeline

Screening 3 weeks

Treatment Varies

Follow Up cohort 1: pre-dose; at end of infusion; and at 2, 4, 6, and 8 hours after end of infusion on day 1 (week 1) and week 25 cohort 2 & 3: pre-dose; at end of infusion; and at 2, 4, 6, 8, and 12 to 16 hours after end of infusion on day 1 (week 1) and week 25

Awards & highlights

Mini-COMET Trial Summary

This trial will compare the safety and effectiveness of two treatments for infantile-onset Pompe disease, a disease that affects the muscles.

Who is the study for?

This trial is for children under 18 with infantile-onset Pompe disease who've been on alglucosidase alfa for at least 6 months. They must show signs of the disease worsening or not improving enough, and be able to follow the study plan. Pregnant or breastfeeding females can't join, nor those allergic to the study drugs or on certain other medications.Check my eligibility

What is being tested?

The trial tests avalglucosidase alfa's safety and effectiveness in kids previously treated with alglucosidase alfa for Pompe Disease. It compares how both drugs work in the body (pharmacokinetics) and their impact on symptoms like heart size, muscle function, breathing, and eyelid drooping.See study design

What are the potential side effects?

Possible side effects include allergic reactions similar to those from current treatments like infusion-related responses. Since it's a new drug being tested against an existing one, unexpected side effects may also occur.

Mini-COMET Trial Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I have Pompe Disease and my breathing, movement, or heart health is getting worse.

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I have been diagnosed with cardiomyopathy, with my heart's size larger than typical for my age.

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I have Pompe Disease and my breathing, movement, or eyelid drooping hasn't improved.

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I have been diagnosed with a GAA enzyme deficiency.

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I have been on a stable dose of alglucosidase alfa for at least 6 months.

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I am not pregnant, not breastfeeding, and have a negative pregnancy test.

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I am under 18 years old.

Mini-COMET Trial Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ cohort 1: pre-dose; at end of infusion; and at 2, 4, 6, and 8 hours after end of infusion on day 1 (week 1) and week 25 cohort 2 and 3: pre-dose; at end of infusion; and at 2, 4, 6, 8, and 12 to 16 hours after end of infusion on day 1 (week 1) and week 25

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~cohort 1: pre-dose; at end of infusion; and at 2, 4, 6, and 8 hours after end of infusion on day 1 (week 1) and week 25 cohort 2 and 3: pre-dose; at end of infusion; and at 2, 4, 6, 8, and 12 to 16 hours after end of infusion on day 1 (week 1) and week 25

This trial's timeline: 3 weeks for screening, Varies for treatment, and cohort 1: pre-dose; at end of infusion; and at 2, 4, 6, and 8 hours after end of infusion on day 1 (week 1) and week 25 cohort 2 and 3: pre-dose; at end of infusion; and at 2, 4, 6, 8, and 12 to 16 hours after end of infusion on day 1 (week 1) and week 25 for reporting.

Treatment Details

Study Objectives

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

Primary outcome measures

PAP: Number of Participants With Infusion-associated Reactions (IARs)

PAP: Number of Participants With Treatment-emergent Adverse Events (TEAEs), Serious Treatment-emergent Adverse Events, and Adverse Event of Special Interest (AESI)

Secondary outcome measures

ETP: Number of Participants With Infusion-associated Reactions

ETP: Number of Participants With Treatment Emergent Adverse Events, Serious Treatment Emergent Adverse Events, and Adverse Event of Special Interest

PAP: Change From Baseline in Creatine Kinase Value at Week 25

+15 more

Side effects data

From 2023 Phase 3 trial • 101 Patients • NCT02782741

24%

Nasopharyngitis

24%

Back Pain

22%

Headache

20%

Influenza

18%

Fatigue

16%

Pain In Extremity

14%

Fall

12%

Diarrhoea

12%

Arthralgia

12%

Nausea

10%

Myalgia

10%

Contusion

10%

Dizziness

Upper Respiratory Tract Infection

Pruritus

Vomiting

Non-Cardiac Chest Pain

Dyspepsia

Paraesthesia

Erythema

Cystitis

Muscle Spasms

Urticaria

Oedema Peripheral

Dysuria

Vitamin D Deficiency

Dyspnoea

Urinary Tract Infection

Abdominal Pain Upper

Pain

Alanine Aminotransferase Increased

Cough

Oropharyngeal Pain

Influenza Like Illness

Pyrexia

Rash

Insomnia

Chills

Tremor

Toothache

Ocular Hyperaemia

Peripheral Swelling

Depression

Renal Colic

Calculus Urinary

Hydronephrosis

Hypoventilation

Respiratory Failure

Nasal Congestion

Syncope

Breast Cyst

Pneumonia

Abdominal Pain

Hypertension

100%

80%

60%

40%

20%

Study treatment Arm

PAP: Avalglucosidase Alfa

PAP: Alglucosidase Alfa

Open-label: Avalglucosidase Alfa

Open-label: Alglucosidase Alfa-PAP Then Avalglucosidase Alfa

Mini-COMET Trial Design

4Treatment groups

Experimental Treatment

Group I: Cohort 3b: Alglucosidase Alfa in PAPExperimental Treatment2 Interventions

After determination of the highest tolerated avalglucosidase alfa dose in Cohort 1 and Cohort 2 (after at least 5 participants in each Cohort 1 and Cohort 2 had received the 7th dose of avalglucosidase alfa or completed Week 13 with a minimum of 6 infusions), participants received alglucosidase alfa at their current stable dose (defined as dose [between 20 mg/kg qow and 40 mg/kg weekly as per physician] administered regularly for a minimum of 6 months immediately prior to entry in this study) IV infusion for 25 weeks in PAP. After PAP, participants received avalglucosidase alfa 40mg/kg IV infusion qow from Week 26 up to Week 371 in ETP.

Group II: Cohort 3a: Avalglucosidase Alfa 40 mg//kgExperimental Treatment1 Intervention

Group III: Cohort 2: Avalglucosidase Alfa 40 mg/kgExperimental Treatment1 Intervention

Avalglucosidase alfa 40 mg/kg IV infusion qow for 25 weeks in the PAP, followed by same treatment from Week 26 up to Week 371 in ETP.

Group IV: Cohort 1: Avalglucosidase Alfa 20 mg/kgExperimental Treatment1 Intervention

Avalglucosidase alfa, 20 mg/kg intravenous (IV) infusion every other week (qow) for 25 weeks in the Primary Analysis Period (PAP), followed by same treatment from Week 26 up to Week 371 in extension treatment period (ETP).

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Avalglucosidase alfa (GZ402666)

2016

Completed Phase 3

~110

Alglucosidase alfa (GZ419829)

2016

Completed Phase 3

~110

0 / 7Eligibility criteria met

Find a Location

Who is running the clinical trial?

Genzyme, a Sanofi CompanyLead Sponsor

524 Previous Clinical Trials

85,608 Total Patients Enrolled

Clinical Sciences & OperationsStudy DirectorSanofi

857 Previous Clinical Trials

2,019,277 Total Patients Enrolled

Media Library

alglucosidase alfa GZ419829 (Enzyme Replacement Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT03019406 — Phase 2

Pompe Disease Research Study Groups: Cohort 1: Avalglucosidase Alfa 20 mg/kg, Cohort 2: Avalglucosidase Alfa 40 mg/kg, Cohort 3a: Avalglucosidase Alfa 40 mg//kg, Cohort 3b: Alglucosidase Alfa in PAP

Pompe Disease Clinical Trial 2023: alglucosidase alfa GZ419829 Highlights & Side Effects. Trial Name: NCT03019406 — Phase 2

alglucosidase alfa GZ419829 (Enzyme Replacement Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT03019406 — Phase 2

Frequently Asked Questions

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

Is the recruitment period for this investigation still open?

"The trial is no longer seeking participants, as it was first published on the 12th of October 2017 and last updated on March 5th 2022. If you're looking for other studies to take part in, there are presently 23 medical trials recruiting patients with glycogen storage disease type II and 7 clinical investigations actively enrolling folks who need avalglucosidase alfa GZ402666 treatment."

Answered by AI

Has the FDA sanctioned avalglucosidase alfa GZ402666?

"Due to the medication's Phase 2 status, our team has assigned this drug a safety rating of 2. This implies some evidence exists for its security but no data demonstrating efficacy is currently available."

Answered by AI

Is this a pioneering clinical trial?

"Since 2016, avalglucosidase alfa GZ402666 has been under investigation. Initially undertaken by Genzyme (a Sanofi Company), the initial clinical trial involved a cohort of 100 participants which was enough to secure Phase 3 approval. Currently, there are 7 studies of this medication taking place across 26 nations and 54 cities."

Answered by AI

Would I meet the criteria to join this research endeavor?

"To participate in this investigation, patients need to have glycogen storage disease type ii and must be between 6 months old and 17 years of age. The study is aiming to recruit 22 participants."

Answered by AI

Are applicants under 35 years old considered for inclusion in this trial?

"This trial is seeking candidates aged between half a year and 17 years old."

Answered by AI

Are there any related investigations looking into avalglucosidase alfa GZ402666?

"Avaglucosidase alfa GZ402666 was first researched in 2016 at Site 2760002. Currently, there are 30 finished studies and 7 ongoing ones, a few of which take place in Seattle, Washington."

Answered by AI

What is the participant count for this research endeavor?

"As of now, this experiment is not enrolling any more patients. It was initially posted on October 12th 2017 with the latest update being May 3rd 2022. If you are in search for alternative trials, 23 studies related to glycogen storage disease type II and 7 investigations concerning avalglucosidase alfa GZ402666 are actively recruiting participants."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

A Study to Assess Safety and Efficacy of Avalglucosidase Alfa Administered Every Other Week in Pediatric Patients With Infantile-onset Pompe Disease Previously Treated With Alglucosidase Alfa

2017. "A Study to Assess Safety and Efficacy of Avalglucosidase Alfa Administered Every Other Week in Pediatric Patients With Infantile-onset Pompe Disease Previously Treated With Alglucosidase Alfa". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT03019406.

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