# Safety/Tolerability/Pharmacokinetic (PK)/Pharmacodynamics (PD) Study of BMN701 in Patients With Late-Onset Pompe Disease

> **NCT01230801** · PHASE1,PHASE2 · COMPLETED · sponsor: **BioMarin Pharmaceutical** · enrollment: 22 (actual)

## Conditions studied

- Pompe Disease

## Interventions

- **BIOLOGICAL:** BMN 701

## Key facts

- **NCT ID:** NCT01230801
- **Lead sponsor:** BioMarin Pharmaceutical
- **Sponsor class:** INDUSTRY
- **Phase:** PHASE1,PHASE2
- **Study type:** INTERVENTIONAL
- **Status:** COMPLETED
- **Start date:** 2011-01-17
- **Primary completion:** 2013-03-06
- **Final completion:** 2013-03-06
- **Target enrollment:** 22 (ACTUAL)
- **Last updated:** 2018-06-11


## Primary source

ClinicalTrials.gov registry: https://clinicaltrials.gov/study/NCT01230801

## Citation

> US National Library of Medicine, ClinicalTrials.gov registration NCT01230801, "Safety/Tolerability/Pharmacokinetic (PK)/Pharmacodynamics (PD) Study of BMN701 in Patients With Late-Onset Pompe Disease". Retrieved via AI Analytics 2026-06-02 from https://api.ai-analytics.org/clinical/NCT01230801. Licensed CC0.

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