Safety and Efficacy Evaluation of Repeat neoGAA Dosing in Late Onset Pompe Disease Patients.
- Conditions
- Glycogen Storage Disease Type II (GSD II)Pompe DiseaseAcid Maltase Deficiency
- Interventions
- Registration Number
- NCT01898364
- Lead Sponsor
- Genzyme, a Sanofi Company
- Brief Summary
Primary Objective:
To evaluate the safety and tolerability of neoGAA in treatment naïve and alglucosidase alfa treated late-onset Pompe disease patients.
Secondary Objective:
To evaluate the pharmacokinetics, pharmacodynamics of neoGAA in treatment naïve and alglucosidase alfa treated late-onset Pompe disease patients.
To evaluate the effect of neoGAA on exploratory efficacy endpoints in treatment naïve and alglucosidase alfa treated late-onset Pompe disease patients.
- Detailed Description
Screening: within 90 days Period of treatment: 24 weeks (including 13 bi-weekly infusions) Post treatment evaluation visit: 2 weeks after last neoGAA infusion (at Week 27) End of study visit: 4 weeks after last neoGAA infusion (at Week 29) Total duration: approximately 41 weeks
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 24
Not provided
Not provided
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description GZ402666 (neoGAA) Group 1 - 10 mg GZ402666 Intravenous infusion of 10mg neoGAA to treatment naïve late onset Pompe disease patients once every other week for a total of 24 weeks. GZ402666 (neoGAA) Group 2 - 10 mg GZ402666 Intravenous infusion of 10mg neoGAA once every other week for a total of 24 weeks to late onset Pompe disease patients previously treated with alglucoside alfa. GZ402666 (neoGAA) Group 2 - 20 mg GZ402666 Intravenous infusion of 20mg neoGAA once every other week for a total of 24 weeks to late onset Pompe disease patients previously treated with alglucoside alfa. GZ402666 (neoGAA) Group 1 - 20 mg GZ402666 Intravenous infusion of 20mg neoGAA to treatment naïve late onset Pompe disease patients once every other week for a total of 24 weeks. GZ402666 (neoGAA) Group 1 - 5 mg GZ402666 Intravenous infusion of 5mg neoGAA to treatment naïve late onset Pompe disease patients once every other week for a total of 24 weeks GZ402666 (neoGAA) Group 2 - 5 mg GZ402666 Intravenous infusion of 5mg neoGAA once every other week for a total of 24 weeks to late onset Pompe disease patients previously treated with alglucoside alfa.
- Primary Outcome Measures
Name Time Method Vital signs screening/baseline to Week 25 Adverse events screening/baseline to Week 25 Laboratory assessments including hematology, biochemistry and urinalysis screening/baseline to Week 25
- Secondary Outcome Measures
Name Time Method Electrocardiogram screening/baseline, Week 1, Week 13, Week 25 Cmax Week 1, Week 13, Week 25 t1/2 Week 1, Week 13, Week 25 Skeletal muscle magnetic resonance images for qualitative and quantitative muscle degenerative assessments. screening/baseline, Week 27 Quality of life assessments screening/baseline, Week 13, Week 25 AUC Week 1, Week 13, Week 25 Skeletal muscle glycogen content screening/baseline, Week 27 Immunogenicity assessments screening/baseline to Week 29 Functional assessments including 6 Minute Walk Test (6MWT) screening/baseline, Week 13, Week 25 Functional Assessment includes - pulmonary function testing (PFT) endpoints, Gait, Stair, Gower's Maneuver, Chair (GSGC), Gross Motor Function Measure-88 (GMFM-88), Quick Motor Function Test (QMFT), hand-held dynamometer testing, Pediatric Quality of Life Inventory Multidimensional Fatigue Scale (PedsQL)
Urinary Hex4 screening/baseline to Week 25
Trial Locations
- Locations (17)
Investigational Site Number 250001
🇫🇷Marseille, France
Investigational Site Number 208001
🇩🇰København Ø, Denmark
Investigational Site Number 250003
🇫🇷Nice, France
Investigational Site Number 250002
🇫🇷Paris, France
Investigational Site Number 276003
🇩🇪Mainz, Germany
Investigational Site Number 276001
🇩🇪München, Germany
Investigational Site Number 276002
🇩🇪Münster, Germany
Investigational Site Number 826003
🇬🇧Newcastle Upon Tyne, United Kingdom
Investigational Site Number 840002
🇺🇸Durham, North Carolina, United States
Investigational Site Number 840009
🇺🇸Dallas, Texas, United States
Investigational Site Number 840001
🇺🇸Kansas City, Kansas, United States
Investigational Site Number 528001
🇳🇱Rotterdam, Netherlands
Investigational Site Number 840008
🇺🇸Saint Louis, Missouri, United States
Investigational Site Number 840010
🇺🇸Jacksonville, Florida, United States
Investigational Site Number 840003
🇺🇸Fairfax, Virginia, United States
Investigational Site Number 056001
🇧🇪Leuven, Belgium
Investigational Site Number 840006
🇺🇸Phoenix, Arizona, United States