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Trial record 2 of 2 for:    COMET pompe

Study to Compare the Efficacy and Safety of Enzyme Replacement Therapies neoGAA and Alglucosidase Alfa Administered Every Other Week in Patients With Late-onset Pompe Disease Who Have Not Been Previously Treated for Pompe Disease (COMET)

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ClinicalTrials.gov Identifier: NCT02782741
Recruitment Status : Recruiting
First Posted : May 25, 2016
Last Update Posted : December 10, 2018
Sponsor:
Information provided by (Responsible Party):
Sanofi

Brief Summary:

Primary Objective:

To determine the effect of neoGAA (GZ402666) 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 neoGAA 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: GZ402666 Drug: alglucosidase alfa (GZ419829) Phase 3

Detailed Description:
The duration of the study per patient will be up to 3 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, a 96-week open-label treatment period, and a 4-week post-treatment observation period.

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 96 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 NeoGAA (GZ402666) and Alglucosidase Alfa in Treatment-naïve Patients With Late-onset Pompe Disease
Actual Study Start Date : October 19, 2016
Estimated Primary Completion Date : February 4, 2022
Estimated Study Completion Date : February 4, 2022


Arm Intervention/treatment
Experimental: GZ402666
GZ402666 administered intravenously every 2 weeks
Drug: GZ402666

Pharmaceutical form: powder for concentrate for solution for infusion

Route of administration: intravenous


Active Comparator: alglucosidase alfa
alglucosidase alfa administered intravenously every 2 weeks
Drug: alglucosidase alfa (GZ419829)

Pharmaceutical form: powder for concentrate for solution for infusion

Route of administration: intravenous

Other Name: 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 12 months ]
  2. Change from baseline in maximal inspiratory pressure in upright position [ Time Frame: Baseline to 12 months ]
  3. Change from baseline in maximal expiratory pressure in upright position [ Time Frame: Baseline to 12 months ]
  4. Change from baseline in hand-held dynamometry measurement [ Time Frame: Baseline to 12 months ]
  5. Change from baseline in Quick Motor Function Test scores [ Time Frame: Baseline to 12 months ]
  6. Change from baseline in 12- Item Short-form health survey scores [ Time Frame: Baseline to 12 months ]
  7. Number of participants with adverse events [ Time Frame: Baseline to 12 months ]


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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.
  • The patient (and patient's legal guardian if patient is legally minor as defined by local regulation) must have the ability to comply with the clinical protocol.
  • The patient, if female and of childbearing potential, must have a negative pregnancy test (beta-human chorionic gonadotropin) at baseline.

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 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 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


Contacts
Contact: Trial Transparency email recommended (Toll free number for US & Canada) 800-633-1610 ext 1 then # Contact-Us@sanofi.com

  Hide Study Locations
Locations
United States, Arizona
Investigational Site Number 8400015 Recruiting
Phoenix, Arizona, United States, 85013
United States, California
Investigational Site Number 8400020 Recruiting
Los Angeles, California, United States, 90095
Investigational Site Number 8400011 Recruiting
Orange, California, United States, 92868
Investigational Site Number 8400017 Recruiting
Stanford, California, United States, 94305
United States, Florida
Investigational Site Number 8400016 Recruiting
Gainesville, Florida, United States, 32610
United States, Georgia
Investigational Site Number 8400007 Recruiting
Decatur, Georgia, United States, 30033
United States, Illinois
Investigational Site Number 8400023 Recruiting
Chicago, Illinois, United States, 60611
United States, Iowa
Investigational Site Number 8400002 Recruiting
Iowa City, Iowa, United States, 52242
United States, Kansas
Investigational Site Number 8400012 Recruiting
Kansas City, Kansas, United States, 66160-7321
United States, Massachusetts
Investigational Site Number 8400010 Recruiting
Boston, Massachusetts, United States, 02114
United States, Michigan
Investigational Site Number 8400001 Recruiting
Detroit, Michigan, United States, 48201
United States, Minnesota
Investigational Site Number 8400019 Recruiting
Minneapolis, Minnesota, United States, 55455
United States, Missouri
Investigational Site Number 8400013 Recruiting
Saint Louis, Missouri, United States, 63110
United States, New York
Investigational Site Number 8400026 Recruiting
Great Neck, New York, United States, 11020
Investigational Site Number 8400021 Recruiting
New York, New York, United States, 10016
Investigational Site Number 8400008 Recruiting
Valhalla, New York, United States, 10595
United States, North Carolina
Investigational Site Number 8400006 Recruiting
Durham, North Carolina, United States, 27710
United States, Ohio
Investigational Site Number 8400009 Recruiting
Cincinnati, Ohio, United States, 45267-0542
United States, Oregon
Investigational Site Number 8400014 Recruiting
Portland, Oregon, United States, 97239
United States, Pennsylvania
Investigational Site Number 8400004 Recruiting
Hershey, Pennsylvania, United States, 17033
Investigational Site Number 8400025 Recruiting
Pittsburgh, Pennsylvania, United States, 15213
United States, Texas
Investigational Site Number 8400022 Recruiting
Dallas, Texas, United States, 75246
United States, Utah
Investigational Site Number 8400018 Recruiting
Salt Lake City, Utah, United States, 84132
United States, Virginia
Investigational Site Number 8400005 Recruiting
Fairfax, Virginia, United States, 22030
United States, West Virginia
Investigational Site Number 8400024 Recruiting
Morgantown, West Virginia, United States, 26506
Argentina
Investigational Site Number 0320001 Recruiting
Caba, Argentina, C1181ACH
Australia
Investigational Site Number 0360001 Recruiting
Auchenflower, Australia, 4066
Austria
Investigational Site Number 0400001 Recruiting
Wien, Austria, 1090
Belgium
Investigational Site Number 0560003 Recruiting
Bruxelles, Belgium, 1070
Investigational Site Number 0560001 Recruiting
Leuven, Belgium, 3000
Bulgaria
Investigational Site Number 1000001 Recruiting
Sofia, Bulgaria, 1431
Canada
Investigational Site Number 1240003 Recruiting
Hamilton, Canada, L8N 3Z5
Investigational Site Number 1240002 Recruiting
Montreal, Canada, H3A 2B4
Colombia
Investigational Site Number 1700001 Recruiting
Bogotá, Colombia
Czechia
Investigational Site Number 2030001 Recruiting
Praha 2, Czechia, 12808
Denmark
Investigational Site Number 2080002 Recruiting
Aarhus C, Denmark, 8000
Investigational Site Number 2080003 Recruiting
København Ø, Denmark, 2100
France
Investigational Site Number 2500008 Recruiting
Angers, France, 49933
Investigational Site Number 2500011 Recruiting
Brest Cedex 2, France, 29609
Investigational Site Number 2500003 Recruiting
Bron Cedex, France, 69677
Investigational Site Number 2500004 Recruiting
Bron Cedex, France, 69677
Investigational Site Number 2500010 Recruiting
Clermont Ferrand, France, 63003
Investigational Site Number 2500005 Recruiting
Lille, France, 59037
Investigational Site Number 2500006 Recruiting
Marseille Cedex 5, France, 13385
Investigational Site Number 2500002 Recruiting
Nice Cedex 1, France, 06002
Investigational Site Number 2500001 Recruiting
Paris Cedex 13, France, 75651
Germany
Investigational Site Number 2760006 Recruiting
Bochum, Germany, 44789
Investigational Site Number 2760005 Recruiting
Bochum, Germany, 44791
Investigational Site Number 2760004 Recruiting
Hamburg, Germany, 20246
Investigational Site Number 2760001 Recruiting
Mainz, Germany, 55131
Investigational Site Number 2760003 Recruiting
München, Germany, 80336
Investigational Site Number 2760002 Recruiting
Münster, Germany, 48149
Italy
Investigational Site Number 3800006 Recruiting
Brescia, Italy, 25123
Investigational Site Number 3800001 Recruiting
Messina, Italy, 98125
Investigational Site Number 3800002 Recruiting
Milano, Italy, 20122
Investigational Site Number 3800007 Recruiting
Napoli, Italy, 80131
Investigational Site Number 3800004 Recruiting
Padova, Italy, 35128
Investigational Site Number 3800005 Recruiting
Roma, Italy, 00168
Investigational Site Number 3800003 Recruiting
Torino, Italy, 10126
Japan
Investigational Site Number 3920003 Recruiting
Izumi-Shi, Japan
Investigational Site Number 3920002 Recruiting
Kodaira-Shi, Japan
Korea, Republic of
Investigational Site Number 4100004 Recruiting
Daejeon, Korea, Republic of, 35015
Investigational Site Number 4100001 Recruiting
Seoul, Korea, Republic of, 03080
Investigational Site Number 4100002 Recruiting
Seoul, Korea, Republic of, 06273
Investigational Site Number 4100003 Recruiting
Yangsan-Si, Korea, Republic of
Mexico
Investigational Site Number 4840001 Recruiting
Guadalajara, Mexico, 44950
Netherlands
Investigational Site Number 5280001 Recruiting
Rotterdam, Netherlands, 3015 GE
Poland
Investigational Site Number 6160001 Recruiting
Warszawa, Poland, 02-097
Portugal
Investigational Site Number 6200001 Recruiting
Braga, Portugal, 4710-243
Russian Federation
Investigational Site Number 6430001 Recruiting
Moscow, Russian Federation, 125367
Spain
Investigational Site Number 7240002 Recruiting
Barcelona, Spain, 08025
Investigational Site Number 7240003 Recruiting
Esplugues De Llobregat, Spain, 08950
Investigational Site Number 7240001 Recruiting
Madrid, Spain, 28046
Investigational Site Number 7240005 Recruiting
Valencia, Spain, 46026
Sweden
Investigational Site Number 7520001 Recruiting
Uppsala, Sweden, 751 85
Switzerland
Investigational Site Number 7560002 Recruiting
Zürich, Switzerland, 8091
Taiwan
Investigational Site Number 1580001 Recruiting
Taipei, Taiwan, 10043
Investigational Site Number 1580002 Recruiting
Taoyuan County, Taiwan, 33305
Turkey
Investigational Site Number 7920001 Recruiting
Ankara, Turkey, 06100
Investigational Site Number 7920002 Recruiting
Istanbul, Turkey, 34390
United Kingdom
Investigational Site Number 8260005 Recruiting
Birmingham, United Kingdom, B15 2TH
Investigational Site Number 8260002 Recruiting
Cambridge, United Kingdom, CB2 OQQ
Investigational Site Number 8260001 Recruiting
London, United Kingdom, NW3 2QG
Investigational Site Number 8260004 Recruiting
Newcastle Upon Tyne, United Kingdom, NE1 4LP
Sponsors and Collaborators
Sanofi
Investigators
Study Director: Clinical Sciences & Operations Sanofi

Responsible Party: Sanofi
ClinicalTrials.gov Identifier: NCT02782741     History of Changes
Other Study ID Numbers: EFC14028
2016‐000942‐77 ( EudraCT Number )
U1111-1178-4806 ( Other Identifier: UTN )
First Posted: May 25, 2016    Key Record Dates
Last Update Posted: December 10, 2018
Last Verified: December 2018
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:
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