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Trial details imported from ClinicalTrials.gov

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




Registration number
NCT02782741
Ethics application status
Date submitted
23/05/2016
Date registered
25/05/2016
Date last updated
21/07/2020

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
Change from baseline in percent predicted forced vital capacity (%FVC) in upright position
Timepoint [1] 0 0
Baseline to 12 months
Secondary outcome [1] 0 0
Change from baseline in six-minute walk test scores
Timepoint [1] 0 0
Baseline to 49 weeks
Secondary outcome [2] 0 0
Change from baseline in maximal inspiratory pressure in upright position
Timepoint [2] 0 0
Baseline to 49 weeks
Secondary outcome [3] 0 0
Change from baseline in maximal expiratory pressure in upright position
Timepoint [3] 0 0
Baseline to 49 weeks
Secondary outcome [4] 0 0
Change from baseline in hand-held dynamometry measurement
Timepoint [4] 0 0
Baseline to 49 weeks
Secondary outcome [5] 0 0
Change from baseline in Quick Motor Function Test scores
Timepoint [5] 0 0
Baseline to 49 weeks
Secondary outcome [6] 0 0
Change from baseline in 12- Item Short-form health survey scores
Timepoint [6] 0 0
Baseline to 49 weeks
Secondary outcome [7] 0 0
Number of participants with adverse events
Timepoint [7] 0 0
Baseline to 49 weeks and up to 6 years

Eligibility
Key inclusion criteria
Inclusion criteria :

- The patient has confirmed GAA enzyme deficiency from any tissue source and/or 2
confirmed GAA gene mutations.

- The patient 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 patients as defined by local regulation. If the patient is legally
minor, signed written consent shall be obtained from parent(s)/legal guardian and
assent obtained from patients, if applicable.
Minimum age
3 Years
Maximum age
No limit
Gender
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
Exclusion criteria:

- The patient is <3 years of age.

- The patient has known Pompe specific cardiac hypertrophy.

- The patient is wheelchair dependent.

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

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

- The patient is not able to successfully perform repeated forced vital capacity (FVC)
measurements in upright position of =30% predicted and =85% predicted.

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

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

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

- The patient, 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 patient'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
Active, not recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
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
Country [1] 0 0
United States of America
State/province [1] 0 0
Arizona
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State/province [2] 0 0
California
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State/province [3] 0 0
Florida
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United States of America
State/province [4] 0 0
Georgia
Country [5] 0 0
United States of America
State/province [5] 0 0
Illinois
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Iowa
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Kansas
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Massachusetts
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Michigan
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Minnesota
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New York
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North Carolina
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Ohio
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Oregon
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Pennsylvania
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Utah
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Virginia
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West Virginia
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Caba
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Wien
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Bruxelles
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Belgium
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Leuven
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Brasilia
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Brasília
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Hamilton
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Brescia
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Messina
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Newcastle Upon Tyne
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Salford

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Sanofi
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 [6MWT], inspiratory muscle strength (maximum inspiratory pressure [MIP]),
expiratory muscle strength (maximum expiratory pressure [MEP]), lower extremity muscle
strength (hand-held dynamometry [HHD]), motor function (Quick Motor Function Test [QMFT]),
and health-related quality of life (SF-12).
Trial website
https://clinicaltrials.gov/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

Summary results
Other publications