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Trial details imported from ClinicalTrials.gov

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




Registration number
NCT02782741
Ethics application status
Date submitted
23/05/2016
Date registered
25/05/2016

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
Country [1] 0 0
United States of America
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Arizona
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California
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Florida
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United States of America
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Georgia
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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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Argentina
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Caba
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Austria
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Wien
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Belgium
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Bruxelles
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Belgium
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Leuven
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Brazil
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Brasilia
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Brazil
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Sao Paulo
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Canada
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Hamilton
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Canada
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Praha 2
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Mainz
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Germany
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Hungary
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Budapest
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Brescia
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Messina
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Milano
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Italy
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Napoli
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Italy
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Torino
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Japan
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Kodaira-Shi
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Korea, Republic of
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Seoul
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Mexico
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Rotterdam
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Warszawa
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Portugal
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Braga
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Russian Federation
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Moscow
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Barcelona
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Zürich
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Taipei
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Turkey
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Ankara
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Istanbul
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Birmingham
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Cambridge
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London
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Newcastle Upon Tyne
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United Kingdom
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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
Trial website
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

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Yes
What data in particular will be shared?
Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org
When will data be available (start and end dates)?
Available to whom?
Available for what types of analyses?
How or where can data be obtained?


What supporting documents are/will be available?




Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results are available at https://clinicaltrials.gov/study/NCT02782741