# A Study of the Safety and Efficacy of rhGAA in Patients With Infantile-onset Pompe Disease

> **NCT00059280** · PHASE2,PHASE3 · COMPLETED · sponsor: **Genzyme, a Sanofi Company** · enrollment: 16 (actual)

## Conditions studied

- Glycogen Storage Disease Type II

## Interventions

- **BIOLOGICAL:** Myozyme

## Key facts

- **NCT ID:** NCT00059280
- **Lead sponsor:** Genzyme, a Sanofi Company
- **Sponsor class:** INDUSTRY
- **Phase:** PHASE2,PHASE3
- **Study type:** INTERVENTIONAL
- **Status:** COMPLETED
- **Start date:** 2003-04
- **Primary completion:** 2005-06
- **Final completion:** 2005-09
- **Target enrollment:** 16 (ACTUAL)
- **Last updated:** 2014-02-05


## Primary source

ClinicalTrials.gov registry: https://clinicaltrials.gov/study/NCT00059280

## Citation

> US National Library of Medicine, ClinicalTrials.gov registration NCT00059280, "A Study of the Safety and Efficacy of rhGAA in Patients With Infantile-onset Pompe Disease". Retrieved via AI Analytics 2026-06-02 from https://api.ai-analytics.org/clinical/NCT00059280. Licensed CC0.

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