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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/ct2/show/NCT02782741




Registration number
NCT02782741
Ethics application status
Date submitted
23/05/2016
Date registered
25/05/2016
Date last updated
4/04/2024

Titles & IDs
Public title
Study to Compare the Efficacy and Safety of Enzyme Replacement Therapies Avalglucosidase Alfa and Alglucosidase Alfa Administered Every Other Week in Patients With Late-onset Pompe Disease Who Have Not Been Previously Treated for Pompe Disease
Scientific title
A Phase 3 Randomized, Multicenter, Multinational, Double-blinded Study Comparing the Efficacy and Safety of Repeated Biweekly Infusions of Avalglucosidase Alfa (neoGAA, GZ402666) and Alglucosidase Alfa in Treatment naïve Patients With Late-onset Pompe Disease
Secondary ID [1] 0 0
2016-000942-77
Secondary ID [2] 0 0
EFC14028
Universal Trial Number (UTN)
Trial acronym
COMET
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Glycogen Storage Disease Type II;Pompe's Disease 0 0
Condition category
Condition code
Metabolic and Endocrine 0 0 0 0
Metabolic disorders
Metabolic and Endocrine 0 0 0 0
Other metabolic disorders
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Avalglucosidase alfa (GZ402666)
Treatment: Drugs - Alglucosidase alfa (GZ419829)

Experimental: avalglucosidase alfa (GZ402666) - Administered intravenously every 2 weeks

Active Comparator: alglucosidase alfa (GZ419829) - Administered intravenously every 2 weeks


Treatment: Drugs: Avalglucosidase alfa (GZ402666)
Pharmaceutical form: powder for concentrate for solution for infusion Route of administration: intravenous

Treatment: Drugs: Alglucosidase alfa (GZ419829)
Pharmaceutical form: powder for concentrate for solution for infusion Route of administration: intravenous
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
PAP: Change From Baseline in Percent Predicted FVC in Upright Position at Week 49
Timepoint [1] 0 0
Baseline, Week 49
Secondary outcome [1] 0 0
PAP: Change From Baseline in Total Distance Walked During Six-minute Walk Test (6MWT) at Week 49
Timepoint [1] 0 0
Baseline, Week 49
Secondary outcome [2] 0 0
PAP: Change From Baseline in Percent Predicted Maximal Inspiratory Pressure (MIP) in Upright Position at Week 49
Timepoint [2] 0 0
Baseline, Week 49
Secondary outcome [3] 0 0
PAP: Change From Baseline in Percent Predicted Maximal Expiratory Pressure (MEP) in Upright Position at Week 49
Timepoint [3] 0 0
Baseline, Week 49
Secondary outcome [4] 0 0
PAP: Change From Baseline in Lower Extremity Muscle Strength at Week 49 as Assessed by Hand-Held Dynamometry (HHD)
Timepoint [4] 0 0
Baseline, Week 49
Secondary outcome [5] 0 0
PAP: Change From Baseline in Quick Motor Function Test (QMFT) Total Scores at Week 49
Timepoint [5] 0 0
Baseline, Week 49
Secondary outcome [6] 0 0
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
Timepoint [6] 0 0
Baseline, Week 49
Secondary outcome [7] 0 0
PAP: Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Infusion-Associated Reactions (IARs)
Timepoint [7] 0 0
From Baseline up to Week 49
Secondary outcome [8] 0 0
Open-label Period: Number of Participants With TEAEs and IARs
Timepoint [8] 0 0
Week 50 to 289 in open-label long-term period
Secondary outcome [9] 0 0
PAP: Percentage of Participants With Treatment-Emergent Antidrug Antibodies (ADA) Response
Timepoint [9] 0 0
From Baseline up to Week 49

Eligibility
Key inclusion criteria
Inclusion criteria :

- 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.
Minimum age
3 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
Exclusion criteria:

- The participant is <3 years of age.

- The participant has known Pompe specific cardiac hypertrophy.

- The participant is wheelchair dependent.

- The participant is not able to ambulate 40 meters (approximately 130 feet) without
stopping and without an assistive device.

- The participant requires invasive-ventilation (non-invasive ventilation is allowed).

- The participant is not able to successfully perform repeated forced vital capacity
(FVC) measurements in upright position of greater than or equal to 30% predicted and
less than or equal to 85% predicted.

- The participant (and participant's legal guardian if participant is legally minor as
defined by local regulation) is (are) not able to comply with the clinical protocol.

- The participant has had previous treatment with alglucosidase alfa or any
investigational therapy for Pompe disease.

- The participant has prior or current use of immune tolerance induction therapy.

- The participant, if female and of childbearing potential, has a positive pregnancy
test (beta-human chorionic gonadotropin) at baseline.

The above information is not intended to contain all considerations relevant to a
participant's potential participation in a clinical trial.

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
2/11/2016
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
31/05/2023
Sample size
Target
Accrual to date
Final
101
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Investigational Site Number 0360001 - Auchenflower
Recruitment postcode(s) [1] 0 0
4066 - Auchenflower
Recruitment outside Australia
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United States of America
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Arizona
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California
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Florida
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Georgia
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Illinois
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Salford

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Genzyme, a Sanofi Company
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Primary Objective:

To determine the effect of avalglucosidase alfa treatment on respiratory muscle strength
measured by percent (%) predicted forced vital capacity (FVC) in the upright position, as
compared to alglucosidase alfa.

Secondary Objective:

To determine the safety and effect of avalglucosidase alfa treatment on functional endurance
(6-minute walk test, inspiratory muscle strength (maximum inspiratory pressure), expiratory
muscle strength (maximum expiratory pressure), lower extremity muscle strength (hand-held
dynamometry), motor function (Quick Motor Function Test), and health-related quality of life
(Short Form-12).
Trial website
https://clinicaltrials.gov/ct2/show/NCT02782741
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Clinical Sciences & Operations
Address 0 0
Sanofi
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries