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Study to Compare the Efficacy and Safety of Enzyme Replacement Therapies Avalglucosidase Alfa and Alglucosidase Alfa Administered Every Other Week in Patients With Late-onset Pompe Disease Who Have Not Been Previously Treated for Pompe Disease

NCT02782741 · Status: COMPLETED · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 101

Last updated 2024-04-04

Study results available
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Summary

Primary Objective:

To determine the effect of avalglucosidase alfa 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 avalglucosidase alfa treatment on functional endurance (6-minute walk test, inspiratory muscle strength (maximum inspiratory pressure), expiratory muscle strength (maximum expiratory pressure), lower extremity muscle strength (hand-held dynamometry), motor function (Quick Motor Function Test), and health-related quality of life (Short Form-12).

Conditions

  • Glycogen Storage Disease Type II;Pompe's Disease

Interventions

DRUG

Avalglucosidase alfa (GZ402666)

Pharmaceutical form: powder for concentrate for solution for infusion Route of administration: intravenous

DRUG

Alglucosidase alfa (GZ419829)

Pharmaceutical form: powder for concentrate for solution for infusion Route of administration: intravenous

Sponsors & Collaborators

  • Genzyme, a Sanofi Company

    lead INDUSTRY

Principal Investigators

  • Clinical Sciences & Operations · Sanofi

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
PARALLEL

Eligibility

Min Age
3 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2016-11-02
Primary Completion
2020-03-19
Completion
2023-05-31
FDA Drug
Yes

Countries

  • United States
  • Argentina
  • Australia
  • Austria
  • Belgium
  • Brazil
  • Canada
  • Czechia
  • Denmark
  • France
  • Germany
  • Hungary
  • Italy
  • Japan
  • Mexico
  • Netherlands
  • Poland
  • Portugal
  • Russia
  • South Korea
  • Spain
  • Switzerland
  • Taiwan
  • Turkey (Türkiye)
  • United Kingdom

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT02782741 on ClinicalTrials.gov