A Study to Assess Safety and Efficacy of Avalglucosidase Alfa Administered Every Other Week in Pediatric Patients With Infantile-onset Pompe Disease Previously Treated With Alglucosidase Alfa (Mini-COMET)
A Study to Assess Safety and Efficacy of Avalglucosidase Alfa Administered Every Other Week in Pediatric Patients With Infantile-onset Pompe Disease Previously Treated With Alglucosidase Alfa (Mini-COMET)
Brief Summary:
Primary Objective:
- To evaluate the safety profile of avalglucosidase alfa in participants with infantile-onset Pompe disease previously treated with alglucosidase alfa.
Secondary Objective:
- To characterize the pharmacokinetic profile of avalglucosidase alfa and to evaluate the preliminary efficacy of avalglucosidase alfa in comparison to alglucosidase alfa.
Detailed Description:
The duration of the study for each participant will be up to approximately 7 years, and consists of a 14-day screening period, that may be extended to up to 4 weeks in pre-specified situations. This is followed by a 25-week treatment period and an up to 346-week treatment extension period and a 4-week post-treatment observation period. Cohort 1 and 2 (Cohort 1: avalglucosidase alfa 20 milligrams per kilogram [mg/kg], Cohort 2: avalglucosidase alfa 40 mg/kg) will be non-randomized and Cohort 3 (Cohort 3a: avalglucosidase alfa 40 mg/kg [maximum tolerated dose] and Cohort 3b: alglucosidase alfa) will be randomized.
Study Type: Interventional (Clinical Trial)
Actual Enrollment: 22 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: An Open-label Ascending Dose Cohort Study to Assess the Safety, Pharmacokinetics, and Preliminary Efficacy of Avalglucosidase Alfa (NeoGAA, GZ402666) in Patients With Infantile-onset Pompe Disease Treated With Alglucosidase Alfa Who Demonstrate Clinical Decline or Sub-optimal Clinical Response
Actual Study Start Date: October 12, 2017
Actual Primary Completion Date: September 30, 2019
Estimated Study Completion Date: December 2024
Arm:
- Experimental: Cohort 1: Avalglucosidase Alfa 20 mg/kg
- Experimental: Cohort 2: Avalglucosidase Alfa 40 mg/kg
- Experimental: Cohort 3a: Avalglucosidase Alfa 40 mg//kg
- Experimental: Cohort 3b: Alglucosidase Alfa in PAP
| Category | Value |
|---|---|
| Study type(s) | Interventional |
| Actual enrolment | 22 |
| Actual Study start date | 12 October 2017 |
| Estimated study completion date | 01 December 2024 |