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Study to Evaluate the Safety of AT2220 (Duvoglustat) in Pompe Disease

NCT00688597 · Status: TERMINATED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 3

Last updated 2025-09-11

Study results available
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Summary

The main purpose of this study was to determine the safety and tolerability of 3 different doses of duvoglustat (AT2220) in participants affected by Pompe disease. The study also evaluated the effects of duvoglustat on functional parameters in Pompe disease.

Conditions

  • Pompe Disease

Interventions

DRUG

Duvoglustat

Powder in a bottle for dissolution in water for oral administration

Sponsors & Collaborators

  • Amicus Therapeutics

    lead INDUSTRY

Principal Investigators

  • Medical Monitor Clinical Research · Amicus Therapeutics

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
PARALLEL

Eligibility

Min Age
18 Years
Max Age
74 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2008-12-08
Primary Completion
2009-12-14
Completion
2009-12-14

Countries

  • United States

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 NCT00688597 on ClinicalTrials.gov