Clinical Trial: A Study to Assess Safety and Efficacy of NeoGAA Administered Every Other Week in Pediatric Patients With Infantile-onset Pompe Disease Previously Treated With Alglucosidase Alfa

Study Status: Not yet recruiting
Recruit Status: Not yet recruiting
Study Type: Interventional

Official Title: An Open-label Ascending Dose Cohort Study to Assess the Safety, Pharmacokinetics, and Preliminary Efficacy of neoGAA (GZ402666) in Patients With Infantile-onset Pompe Disease Treated With Alglucosidas

Brief Summary:

Primary Objective:

To evaluate the safety profile of neoGAA in patients with infantile-onset Pompe disease (IOPD) previously treated with alglucosidase alfa.

Secondary Objective:

To characterize the pharmacokinetic profile of neoGAA and to evaluate the preliminary efficacy of neoGAA in comparison to alglucosidase alfa.


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 25-week treatment period, a 120-week extension period, and a 4-week post-treatment observation period.
Sponsor: Genzyme, a Sanofi Company

Current Primary Outcome: Number of participants with adverse events [ Time Frame: Baseline to 6 months ]

Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Assessment of pharmacokinetic parameter: maximum concentration (Cmax) [ Time Frame: Baseline to 6 months ]
  • Assessment of pharmacokinetic parameter: area under curve (AUC) [ Time Frame: Baseline to 6 months ]
  • Change from baseline in Gross Motor Function Measure-88 Test [ Time Frame: Baseline to 6 months ]
  • Change from revised Gross Motor Function Classification System score [ Time Frame: Baseline to 6 months ]
  • Change from baseline in Pompe specific Pediatric Evaluation of Disability Inventory (Pompe PEDI) Functional Skills Scale: Mobility Domain Test score (normative standard score) [ Time Frame: Baseline to 6 months ]
  • Change from baseline in Pompe specific Pediatric Evaluation of Disability Inventory (Pompe PEDI) Functional Skills Scale: Mobility Domain Test score (scaled score) [ Time Frame: Baseline to 6 months ]
  • Change from baseline in Quick Motor Function Test scores [ Time Frame: Baseline to 6 months ]
  • Change from baseline in Left Ventricular Mass Index [ Time Frame: Baseline to 6 months ]
  • Change from baseline in Left Ventricular Mass Index Z score [ Time Frame: Baseline to 6 months ]
  • Change from baseline in Eyelid position measurements: Interpalpebral fissure distance (IPFD) [ Time Frame: Baseline to 6 months ]
  • Change from baseline in Eyelid position measurements: Margin reflex distance-1 (MRD-1) [ Time Frame: Baseline to 6 months ]
  • Change from baseline in Eyelid position measurements: Margin pupil distance (MPD) [ Time Frame: Baseline to 6 months ]
  • Change from baseline in Creatine kinase value [ Time Frame: Baseline to 6 months ]


Original Secondary Outcome: Same as current

Information By: Sanofi

Dates:
Date Received: December 20, 2016
Date Started: July 2017
Date Completion: January 2022
Last Updated: April 24, 2017
Last Verified: April 2017