Working...
ClinicalTrials.gov
ClinicalTrials.gov Menu

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

Tracking Information
First Submitted Date  ICMJE May 23, 2016
First Posted Date  ICMJE May 25, 2016
Last Update Posted Date August 13, 2019
Actual Study Start Date  ICMJE October 2016
Estimated Primary Completion Date March 2020   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: May 23, 2016)
Change from baseline in percent predicted forced vital capacity (%FVC) in upright position [ Time Frame: Baseline to 12 months ]
Original Primary Outcome Measures  ICMJE Same as current
Change History Complete list of historical versions of study NCT02782741 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: May 28, 2019)
  • Change from baseline in six-minute walk test scores [ Time Frame: Baseline to 49 weeks ]
  • Change from baseline in maximal inspiratory pressure in upright position [ Time Frame: Baseline to 49 weeks ]
  • Change from baseline in maximal expiratory pressure in upright position [ Time Frame: Baseline to 49 weeks ]
  • Change from baseline in hand-held dynamometry measurement [ Time Frame: Baseline to 49 weeks ]
  • Change from baseline in Quick Motor Function Test scores [ Time Frame: Baseline to 49 weeks ]
  • Change from baseline in 12- Item Short-form health survey scores [ Time Frame: Baseline to 49 weeks ]
  • Number of participants with adverse events [ Time Frame: Baseline to 49 weeks and up to 6 years ]
Original Secondary Outcome Measures  ICMJE
 (submitted: May 23, 2016)
  • 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 ]
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE 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
Official Title  ICMJE 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
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).

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 Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Condition  ICMJE Glycogen Storage Disease Type II;Pompe's Disease
Intervention  ICMJE
  • Drug: avalglucosidase alfa(GZ402666)
    Pharmaceutical form: powder for concentrate for solution for infusion Route of administration: intravenous
  • Drug: alglucosidase alfa (GZ419829)
    Pharmaceutical form: powder for concentrate for solution for infusion Route of administration: intravenous
    Other Names:
    • Myozyme
    • Lumizyme
Study Arms  ICMJE
  • Experimental: avalglucosidase alfa (GZ402666)
    Administered intravenously every 2 weeks
    Intervention: Drug: avalglucosidase alfa(GZ402666)
  • Active Comparator: alglucosidase alfa (GZ419829)
    Administered intravenously every 2 weeks
    Intervention: Drug: alglucosidase alfa (GZ419829)
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Active, not recruiting
Actual Enrollment  ICMJE
 (submitted: July 18, 2019)
102
Original Estimated Enrollment  ICMJE
 (submitted: May 23, 2016)
86
Estimated Study Completion Date  ICMJE September 2024
Estimated Primary Completion Date March 2020   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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.

Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 3 Years and older   (Child, Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Argentina,   Australia,   Austria,   Belgium,   Brazil,   Canada,   Colombia,   Czechia,   Denmark,   France,   Germany,   Hungary,   Italy,   Japan,   Korea, Republic of,   Mexico,   Netherlands,   Poland,   Portugal,   Russian Federation,   Spain,   Switzerland,   Taiwan,   Turkey,   United Kingdom,   United States
Removed Location Countries Bulgaria,   Czech Republic,   Sweden
 
Administrative Information
NCT Number  ICMJE NCT02782741
Other Study ID Numbers  ICMJE EFC14028
2016‐000942‐77
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE
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
Responsible Party Sanofi
Study Sponsor  ICMJE Sanofi
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Clinical Sciences & Operations Sanofi
PRS Account Sanofi
Verification Date August 2019

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP