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Trial record 4 of 4 for:    neoGAA

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 : April 12, 2018
Sponsor:
Information provided by (Responsible Party):
Sanofi

May 23, 2016
May 25, 2016
April 12, 2018
October 19, 2016
November 5, 2021   (Final data collection date for primary outcome measure)
Change from baseline in percent predicted forced vital capacity (%FVC) in upright position [ Time Frame: Baseline to 12 months ]
Same as current
Complete list of historical versions of study NCT02782741 on ClinicalTrials.gov Archive Site
  • Change from baseline in six-minute walk test scores [ Time Frame: Baseline to 12 months ]
  • 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 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 ]
  • 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 ]
Not Provided
Not Provided
 
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
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

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).

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.
Interventional
Phase 3
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Glycogen Storage Disease Type II-Pompe's Disease
  • Drug: 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 Name: Myozyme; Lumizyme
  • Experimental: GZ402666
    GZ402666 administered intravenously every 2 weeks
    Intervention: Drug: GZ402666
  • Active Comparator: alglucosidase alfa
    alglucosidase alfa administered intravenously every 2 weeks
    Intervention: Drug: alglucosidase alfa (GZ419829)
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruiting
96
86
November 5, 2021
November 5, 2021   (Final data collection date for primary outcome measure)

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.

Sexes Eligible for Study: All
3 Years and older   (Child, Adult, Senior)
No
Contact: For site information, send an email with site number to Contact-Us@sanofi.com
Argentina,   Australia,   Austria,   Belgium,   Canada,   Czechia,   Denmark,   France,   Germany,   Italy,   Japan,   Korea, Republic of,   Mexico,   Netherlands,   Poland,   Russian Federation,   Spain,   Sweden,   Switzerland,   Taiwan,   Turkey,   United Kingdom,   United States
Colombia,   Czech Republic
 
NCT02782741
EFC14028
2016‐000942‐77 ( EudraCT Number )
U1111-1178-4806 ( Other Identifier: UTN )
Yes
Not Provided
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
Sanofi
Sanofi
Not Provided
Study Director: Clinical Sciences & Operations Sanofi
Sanofi
April 2018

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