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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)

This study is currently recruiting participants. (see Contacts and Locations)
Verified March 2017 by Sanofi
Sponsor:
Information provided by (Responsible Party):
Sanofi
ClinicalTrials.gov Identifier:
NCT02782741
First received: May 23, 2016
Last updated: April 5, 2017
Last verified: March 2017
  Purpose

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 Intervention Phase
Glycogen Storage Disease Type II-Pompe's Disease
Drug: GZ402666
Drug: alglucosidase alfa (GZ419829)
Phase 3

Study Type: Interventional
Study Design: Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: 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

Resource links provided by NLM:


Further study details as provided by Sanofi:

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

Secondary Outcome Measures:
  • Change from baseline in maximal inspiratory pressure in upright position [ Time Frame: Baseline to 12 months ]
  • Change from baseline in maximal expiratory pressure in upright position [ Time Frame: Baseline to 12 months ]
  • Change from baseline in six-minute walk test scores [ Time Frame: Baseline to 12 months ]
  • Change from baseline in hand-held dynamometry measurement [ Time Frame: Baseline to 12 months ]
  • Change from baseline in Quick Motor Function Test scores [ Time Frame: Baseline to 12 months ]
  • Change from baseline in 12- Item Short-form health survey scores [ Time Frame: Baseline to 12 months ]
  • Number of participants with adverse events [ Time Frame: Baseline to 12 months ]

Estimated Enrollment: 96
Study Start Date: October 2016
Estimated Study Completion Date: October 2020
Estimated Primary Completion Date: October 2020 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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

Detailed Description:
The duration of the study per patient will be up to 3 years that will consist of a 14-day screening period, a 49-week blinded treatment period, a 96-week open-label treatment period, and a 4-week post-treatment observation period. Legally minor patients, as defined by local regulation, may be aged 18 years and older.
  Eligibility

Ages Eligible for Study:   3 Years and older   (Child, Adult, Senior)
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.

  Contacts and Locations
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, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT02782741

Contacts
Contact: For site information, send an email with site number to Contact-Us@sanofi.com

  Show 74 Study Locations
Sponsors and Collaborators
Sanofi
Investigators
Study Director: Clinical Sciences & Operations Sanofi
  More Information

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 )
Study First Received: May 23, 2016
Last Updated: April 5, 2017
Individual Participant Data  
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

ClinicalTrials.gov processed this record on April 28, 2017