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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.

Must not have

Female participant of childbearing potential not protected by highly effective contraceptive method of birth control and/or who is unwilling or unable to be tested for pregnancy.

The participant has high antibody titer to alglucosidase alfa.

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 8 years and 9 months

Awards & highlights

No Placebo-Only Group

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.

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.

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.

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.

Select...

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.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

Select...

I am using effective birth control or am willing to be tested for pregnancy.

Select...

I have high antibody levels against alglucosidase alfa.

Select...

I am not on any medication that is not allowed in the study.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 8 years and 9 months

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 8 years and 9 months

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 8 years and 9 months for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

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 study objectives

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

Pain

Vitamin D Deficiency

Dysuria

Urinary Tract Infection

Dyspnoea

Abdominal Pain Upper

Alanine Aminotransferase Increased

Cough

Oropharyngeal Pain

Influenza Like Illness

Pyrexia

Rash

Insomnia

Peripheral Swelling

Tremor

Ocular Hyperaemia

Renal Colic

Depression

Toothache

Chills

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

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

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