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Trial record 1 of 1 for:    NCT02782741
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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)

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ClinicalTrials.gov Identifier: NCT02782741
Recruitment Status : Active, not recruiting
First Posted : May 25, 2016
Last Update Posted : October 14, 2020
Sponsor:
Information provided by (Responsible Party):
Sanofi

Study Description
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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.
Study Design
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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
Actual Primary Completion Date : March 19, 2020
Estimated Study Completion Date : September 2024

Resource links provided by the National Library of Medicine


Arms and Interventions
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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


Outcome Measures
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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 ]

Eligibility Criteria
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Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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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.

Contacts and Locations
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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


Locations
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Sponsors and Collaborators
Sanofi
Investigators
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Study Director: Clinical Sciences & Operations Sanofi
More Information
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Responsible Party: Sanofi
ClinicalTrials.gov Identifier: NCT02782741    
Other Study ID Numbers: EFC14028
2016-000942-77
First Posted: May 25, 2016    Key Record Dates
Last Update Posted: October 14, 2020
Last Verified: October 2020
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