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A Study to Evaluate the Effects of Pharmacological Chaperones in Cells From Patients With Pompe Disease

NCT00515398 · Status: COMPLETED · Type: OBSERVATIONAL · Enrollment: 30

Last updated 2008-06-05

No results posted yet for this study

Summary

The purpose of this study is to see how molecules called pharmacological chaperones affect the cells of patients with Pompe disease. The study will last 1 or 2 visits which will include a blood collection, urine collection, and two skin biopsies. Information will also be collected from the medical records about disease history and diagnosis. Patients will not receive any study medication.

Conditions

  • Pompe Disease
  • Glycogen Storage Disease Type II

Interventions

OTHER

Observation

Ex vivo administration of AT2220

Sponsors & Collaborators

  • Amicus Therapeutics

    lead INDUSTRY

Principal Investigators

  • Irina Kline, MD · Amicus Therapeutics

Eligibility

Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2007-08-31
Primary Completion
2008-01-31
Completion
2008-01-31

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 NCT00515398 on ClinicalTrials.gov