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Safety and Efficacy of Avalglucosidase Alfa in Patients With Non-classic Pompe Disease Aged ≥ 5 Years

NCT07652814 · Status: ACTIVE_NOT_RECRUITING · Phase: PHASE4 · Type: INTERVENTIONAL · Enrollment: 6

Last updated 2026-06-17

No results posted yet for this study

Summary

The goal of this clinical trial is to study the efficacy and safety of treatment with avalglucosidase alfa in patients with late onset Pompe disease that previously deteriorated on alglucosidase alfa.

The main question it aims to answer is:

* Is switching to avalglucosidase alfa in late-onset Pompe patients deteriorating on alglucosidase alfa safe?
* Is switching to avalglucosidase alfa in late-onset Pompe patients deteriorating on alglucosidase alfa potentially more effective?

Participants will switch to biweekly avalglucosidase alfa infusions (instead of alglucosidase alfa infusions) and perform assessment for:

* Efficacy: muscle strength and function, pulmonary function, patient-reported outcomes.
* Safety: Adverse events assessment, physical examination, clinical laboratory evaluations, vital signs, ECGs and immunogenicity assessments.
* Pharmacokinetic assessments during 2 avalglucosidase alfa infusions.

Conditions

  • Glycogen Storage Disease Type II Late Onset
  • Pompe Disease (Late-onset)
  • Pompe Disease Late-Onset
  • Pompe's Disease Juvenile Onset
  • Pompe Disease

Interventions

DRUG

Avalglucosidase Alfa

Enzyme replacement therapy

Sponsors & Collaborators

  • Sanofi

    collaborator INDUSTRY

  • Iris Plug

    lead OTHER

Principal Investigators

  • Nadine van der Beek, MD PHD · Erasmus Medical Center

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
5 Years
Max Age
55 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2022-10-13
Primary Completion
2028-03-09
Completion
2028-03-09

Countries

  • Netherlands

Study Locations

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Entities

Diseases
Companies

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 NCT07652814 on ClinicalTrials.gov