Clinical Trial: 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
Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional
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-naï
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).
Detailed Summary: 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.
Sponsor: Sanofi
Current Primary Outcome: Change from baseline in percent predicted forced vital capacity (%FVC) in upright position [ Time Frame: Baseline to 12 months ]
Original Primary Outcome: Same as current
Current Secondary Outcome:
- 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 ]
Original Secondary Outcome:
- 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 ]
Information By: Sanofi
Dates:
Date Received: May 23, 2016
Date Started: October 2016
Date Completion: October 2020
Last Updated: May 18, 2017
Last Verified: May 2017