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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 (COMET)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT02782741
Recruitment Status : Active, not recruiting
First Posted : May 25, 2016
Last Update Posted : August 13, 2019
Sponsor:
Information provided by (Responsible Party):
Sanofi

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).


Condition or disease Intervention/treatment Phase
Glycogen Storage Disease Type II;Pompe's Disease Drug: avalglucosidase alfa(GZ402666) Drug: alglucosidase alfa (GZ419829) Phase 3

Detailed Description:
The duration of the study per patient will be up to approximately 6 years that will consist of a 14-day screening period (may be extended up to 8 weeks in pre-specified situations), a 49-week blinded treatment period (except for the subgroup of pediatric patients aged 3 to <18 years enrolling directly in the open-label long-term follow-up phase), a 240-week open-label treatment period, and a 4-week post-treatment observation period.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 102 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official 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
Actual Study Start Date : October 2016
Estimated Primary Completion Date : March 2020
Estimated Study Completion Date : September 2024


Arm Intervention/treatment
Experimental: avalglucosidase alfa (GZ402666)
Administered intravenously every 2 weeks
Drug: avalglucosidase alfa(GZ402666)
Pharmaceutical form: powder for concentrate for solution for infusion Route of administration: intravenous

Active Comparator: alglucosidase alfa (GZ419829)
Administered intravenously every 2 weeks
Drug: alglucosidase alfa (GZ419829)
Pharmaceutical form: powder for concentrate for solution for infusion Route of administration: intravenous
Other Names:
  • Myozyme
  • Lumizyme




Primary Outcome Measures :
  1. Change from baseline in percent predicted forced vital capacity (%FVC) in upright position [ Time Frame: Baseline to 12 months ]

Secondary Outcome Measures :
  1. Change from baseline in six-minute walk test scores [ Time Frame: Baseline to 49 weeks ]
  2. Change from baseline in maximal inspiratory pressure in upright position [ Time Frame: Baseline to 49 weeks ]
  3. Change from baseline in maximal expiratory pressure in upright position [ Time Frame: Baseline to 49 weeks ]
  4. Change from baseline in hand-held dynamometry measurement [ Time Frame: Baseline to 49 weeks ]
  5. Change from baseline in Quick Motor Function Test scores [ Time Frame: Baseline to 49 weeks ]
  6. Change from baseline in 12- Item Short-form health survey scores [ Time Frame: Baseline to 49 weeks ]
  7. Number of participants with adverse events [ Time Frame: Baseline to 49 weeks and up to 6 years ]


Information from the National Library of Medicine

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Ages Eligible for Study:   3 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.

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.


Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02782741


  Hide Study Locations
Locations
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United States, Arizona
Investigational Site Number 8400015
Phoenix, Arizona, United States, 85013
United States, California
Investigational Site Number 8400020
Los Angeles, California, United States, 90095
Investigational Site Number 8400011
Orange, California, United States, 92868
Investigational Site Number 8400017
Stanford, California, United States, 94305
United States, Florida
Investigational Site Number 8400016
Gainesville, Florida, United States, 32610
United States, Georgia
Investigational Site Number 8400007
Decatur, Georgia, United States, 30033
United States, Illinois
Investigational Site Number 8400023
Chicago, Illinois, United States, 60611
United States, Iowa
Investigational Site Number 8400002
Iowa City, Iowa, United States, 52242
United States, Kansas
Investigational Site Number 8400012
Kansas City, Kansas, United States, 66160-7321
United States, Massachusetts
Investigational Site Number 8400010
Boston, Massachusetts, United States, 02114
United States, Michigan
Investigational Site Number 8400001
Detroit, Michigan, United States, 48201
United States, Minnesota
Investigational Site Number 8400019
Minneapolis, Minnesota, United States, 55455
United States, New York
Investigational Site Number 8400026
Great Neck, New York, United States, 11020
Investigational Site Number 8400008
Valhalla, New York, United States, 10595
United States, North Carolina
Investigational Site Number 8400006
Durham, North Carolina, United States, 27710
United States, Ohio
Investigational Site Number 8400009
Cincinnati, Ohio, United States, 45267-0542
United States, Oregon
Investigational Site Number 8400014
Portland, Oregon, United States, 97239
United States, Pennsylvania
Investigational Site Number 8400004
Hershey, Pennsylvania, United States, 17033
Investigational Site Number 8400025
Pittsburgh, Pennsylvania, United States, 15213
United States, Utah
Investigational Site Number 8400018
Salt Lake City, Utah, United States, 84132
United States, Virginia
Investigational Site Number 8400005
Fairfax, Virginia, United States, 22030
United States, West Virginia
Investigational Site Number 8400024
Morgantown, West Virginia, United States, 26506
Argentina
Investigational Site Number 0320001
Caba, Argentina, C1181ACH
Australia
Investigational Site Number 0360001
Auchenflower, Australia, 4066
Austria
Investigational Site Number 0400001
Wien, Austria, 1090
Belgium
Investigational Site Number 0560003
Bruxelles, Belgium, 1070
Investigational Site Number 0560001
Leuven, Belgium, 3000
Brazil
Investigational Site Number 0760004
Brasilia, Brazil, 71625-009
Investigational Site Number 0760001
Sao Paulo, Brazil, 04037-002
Canada
Investigational Site Number 1240003
Hamilton, Canada, L8N 3Z5
Investigational Site Number 1240002
Montreal, Canada, H3A 2B4
Colombia
Investigational Site Number 1700001
Bogotá, Colombia
Czechia
Investigational Site Number 2030001
Praha 2, Czechia, 12808
Denmark
Investigational Site Number 2080003
København Ø, Denmark, 2100
France
Investigational Site Number 2500008
Angers, France, 49933
Investigational Site Number 2500007
Bordeaux, France
Investigational Site Number 2500011
Brest Cedex 2, France, 29609
Investigational Site Number 2500004
Bron Cedex, France, 69677
Investigational Site Number 2500010
Clermont Ferrand, France, 63003
Investigational Site Number 2500005
Lille, France, 59037
Investigational Site Number 2500006
Marseille Cedex 5, France, 13385
Investigational Site Number 2500001
Paris Cedex 13, France, 75651
Germany
Investigational Site Number 2760006
Bochum, Germany, 44789
Investigational Site Number 2760001
Mainz, Germany, 55131
Investigational Site Number 2760003
München, Germany, 80336
Investigational Site Number 2760002
Münster, Germany, 48149
Hungary
Investigational Site Number 3480001
Budapest, Hungary, 1083
Italy
Investigational Site Number 3800006
Brescia, Italy, 25123
Investigational Site Number 3800001
Messina, Italy, 98125
Investigational Site Number 3800002
Milano, Italy, 20122
Investigational Site Number 3800007
Napoli, Italy, 80131
Investigational Site Number 3800003
Torino, Italy, 10126
Japan
Investigational Site Number 3920003
Izumi-Shi, Japan
Investigational Site Number 3920002
Kodaira-Shi, Japan
Korea, Republic of
Investigational Site Number 4100001
Seoul, Korea, Republic of, 03080
Investigational Site Number 4100002
Seoul, Korea, Republic of, 06273
Mexico
Investigational Site Number 4840001
Guadalajara, Mexico, 44950
Netherlands
Investigational Site Number 5280001
Rotterdam, Netherlands, 3015 GE
Poland
Investigational Site Number 6160001
Warszawa, Poland, 02-097
Portugal
Investigational Site Number 6200001
Braga, Portugal, 4710-243
Russian Federation
Investigational Site Number 6430001
Moscow, Russian Federation, 125367
Spain
Investigational Site Number 7240002
Barcelona, Spain, 08025
Investigational Site Number 7240003
Esplugues De Llobregat, Spain, 08950
Switzerland
Investigational Site Number 7560002
Zürich, Switzerland, 8091
Taiwan
Investigational Site Number 1580001
Taipei, Taiwan, 10043
Investigational Site Number 1580002
Taoyuan County, Taiwan, 33305
Turkey
Investigational Site Number 7920001
Ankara, Turkey, 06100
Investigational Site Number 7920002
Istanbul, Turkey, 34390
United Kingdom
Investigational Site Number 8260005
Birmingham, United Kingdom, B15 2GW
Investigational Site Number 8260002
Cambridge, United Kingdom, CB2 OQQ
Investigational Site Number 8260001
London, United Kingdom, NW3 2QG
Investigational Site Number 8260004
Newcastle Upon Tyne, United Kingdom, NE1 4LP
Investigational Site Number 8260003
Salford, United Kingdom, M6 8HD
Sponsors and Collaborators
Sanofi
Investigators
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Study Director: Clinical Sciences & Operations Sanofi

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Responsible Party: Sanofi
ClinicalTrials.gov Identifier: NCT02782741     History of Changes
Other Study ID Numbers: EFC14028
2016‐000942‐77
First Posted: May 25, 2016    Key Record Dates
Last Update Posted: August 13, 2019
Last Verified: August 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
Plan Description: Individual participant data (IPD) and supporting clinical documents are available for request at clinicalstudydatarequest.com. While making information available we continue to protect the privacy of the participants in our clinical trials and to remove commercially confidential information (CCI). Details on Data Sharing criteria and process for requesting access can be found at this web address: clinicalstudydatarequest.com

Additional relevant MeSH terms:
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Glycogen Storage Disease Type II
Glycogen Storage Disease
Lysosomal Storage Diseases, Nervous System
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Carbohydrate Metabolism, Inborn Errors
Lysosomal Storage Diseases
Metabolic Diseases