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)
ClinicalTrials.gov ID: NCT03019406
Sponsor: Genzyme, a Sanofi Company
Information provided by: Sanofi (Genzyme, a Sanofi Company) (Responsible Party)
Last Update Posted: 2024-09-19
Brief Summary:
This is a multi-stage, phase 2, open-label, multicenter, multinational, ascending dose cohort, repeated intravenous (IV) infusion study of avalglucosidase alfa in pediatric patients with Infantile-Onset Pompe Disease (IOPD) who have been previously treated with alglucosidase alfa for a minimum of 6 months immediately prior to study entry and have demonstrated clinical decline or unsatisfactory clinical response.
Detailed Description:
The duration of the study for each participant will be up to 8 years and 9 months that will consist 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 a 430-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.
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
Intervention / Treatment:
- Drug: Avalglucosidase alfa (GZ402666)
- Drug: Alglucosidase alfa (GZ419829)
| Category | Value |
|---|---|
| Study Start (Actual) |
2017-10-12
|
| Primary Completion (Actual) |
2019-09-30
|
| Study Completion (Estimated) |
2026-12-30
|
| Enrollment (Actual) | 22 |
| Study Type | Interventional |
| Phase | Phase 3 |
| Other Study ID Numbers |
ACT14132
2016-003475-21 (EudraCT Number) U1111-1179-4616 (Registry Identifier) (REGISTRY: ICTRP) |