# An Exploratory Study of the Safety and Efficacy of Prophylactic Immunomodulatory Treatment in Myozyme-naive Cross-Reacting Immunologic Material (CRIM[-]) Patients With Infantile-Onset Pompe Disease

> **NCT00701129** · PHASE4 · COMPLETED · sponsor: **Genzyme, a Sanofi Company** · enrollment: 4 (actual)

## Conditions studied

- Pompe Disease
- Glycogen Storage Disease Type II

## Interventions

- **BIOLOGICAL:** Alglucosidase Alfa
- **DRUG:** Methotrexate
- **DRUG:** Rituximab

## Key facts

- **NCT ID:** NCT00701129
- **Lead sponsor:** Genzyme, a Sanofi Company
- **Sponsor class:** INDUSTRY
- **Phase:** PHASE4
- **Study type:** INTERVENTIONAL
- **Status:** COMPLETED
- **Start date:** 2009-10
- **Primary completion:** 2013-03
- **Final completion:** 2013-03
- **Target enrollment:** 4 (ACTUAL)
- **Last updated:** 2014-05-13


## Primary source

ClinicalTrials.gov registry: https://clinicaltrials.gov/study/NCT00701129

## Citation

> US National Library of Medicine, ClinicalTrials.gov registration NCT00701129, "An Exploratory Study of the Safety and Efficacy of Prophylactic Immunomodulatory Treatment in Myozyme-naive Cross-Reacting Immunologic Material (CRIM[-]) Patients With Infantile-Onset Pompe Disease". Retrieved via AI Analytics 2026-06-12 from https://api.ai-analytics.org/clinical/NCT00701129. Licensed CC0.

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