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Treatment Frequency Reduction in Pompe Disease

NCT06575829 · Status: NOT_YET_RECRUITING · Phase: PHASE4 · Type: INTERVENTIONAL · Enrollment: 10

Last updated 2024-08-28

No results posted yet for this study

Summary

The aim of this study is to assess if dosing frequency reduction of alglucosidase alfa 20 mg/kg once every 2 weeks to once every 4 weeks is safe and does not lead to increased progression of disease in a selected group of patients with late-onset Pompe disease.

Conditions

  • Pompe Disease (Late-onset)
  • GAA Deficiency
  • Glycogen Storage Disease Type II
  • Acid Maltase Deficiency

Interventions

DRUG

Algucosidase alfa 20 mg/kg once every 4 weeks instead of once every 2 weeks

The interval of ERT with alglucosidase alfa will be extended from once every 2 weeks to once every 4 weeks. The dose of 20 mg/kg per infusion remains the same.

Sponsors & Collaborators

Principal Investigators

  • Pieter A. van Doorn, Prof. dr. · Erasmus Medical Center

  • Nadine A.M.E. van der Beek, Dr. · Erasmus Medical Center

  • Tim Preijers, Dr. · Erasmus Medical Center

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
50 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2024-10-01
Primary Completion
2026-12-31
Completion
2027-12-31

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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 NCT06575829 on ClinicalTrials.gov