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

> **NCT00515398** · — · COMPLETED · sponsor: **Amicus Therapeutics** · enrollment: 30 (actual)

## Conditions studied

- Pompe Disease
- Glycogen Storage Disease Type II

## Interventions

- **OTHER:** Observation

## Key facts

- **NCT ID:** NCT00515398
- **Lead sponsor:** Amicus Therapeutics
- **Sponsor class:** INDUSTRY
- **Phase:** —
- **Study type:** OBSERVATIONAL
- **Status:** COMPLETED
- **Start date:** 2007-08
- **Primary completion:** 2008-01
- **Final completion:** 2008-01
- **Target enrollment:** 30 (ACTUAL)
- **Last updated:** 2008-06-05


## Primary source

ClinicalTrials.gov registry: https://clinicaltrials.gov/study/NCT00515398

## Citation

> US National Library of Medicine, ClinicalTrials.gov registration NCT00515398, "A Study to Evaluate the Effects of Pharmacological Chaperones in Cells From Patients With Pompe Disease". Retrieved via AI Analytics 2026-05-27 from https://api.ai-analytics.org/clinical/NCT00515398. Licensed CC0.

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*[Clinical trials dataset](/datasets/clinical-trials) · CC0 1.0*