Safety/Tolerability/Pharmacokinetic (PK)/Pharmacodynamics (PD) Study of BMN701 in Patients With Late-Onset Pompe Disease
Public ClinicalTrials.gov record NCT01230801. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
A Phase 1/2 Open-label Study of the Safety, Tolerability, Pharmacokinetics, Pharmacodynamic and Preliminary Efficacy of BMN 701 (GILT-tagged Recombinant Human GAA) in Patients With Late-onset Pompe Disease
Study identification
- NCT ID
- NCT01230801
- Recruitment status
- Completed
- Study type
- Interventional
- Phase
- Phase 1, Phase 2
- Lead sponsor
- BioMarin Pharmaceutical
- Industry
- Enrollment
- 22 participants
Conditions and interventions
Conditions
Interventions
- BMN 701 Biological
Biological
Eligibility (public fields only)
- Age range
- 13 Years and older
- Sex
- All
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Jan 16, 2011
- Primary completion
- Mar 5, 2013
- Completion
- Mar 5, 2013
- Last update posted
- Jun 10, 2018
2011 – 2013
United States locations
- U.S. sites
- 3
- U.S. states
- 3
- U.S. cities
- 3
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| Univ of California San Diego School of Medicine | La Jolla | California | 92103-8765 | — |
| University of Florida College of Medicine | Gainesville | Florida | 32610 | — |
| University of Kansas Medical Center | Kansas City | Kansas | 66160 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
Non-U.S. locations
This page focuses on the U.S. directory. The official record also lists 7 non-U.S. sites.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT01230801, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Jun 10, 2018 · Synced Jul 22, 2026
Related: full search, browse by condition, browse by drug or therapy, browse by sponsor, browse by U.S. city.
Open the official record
The complete protocol, eligibility criteria, and contact information for NCT01230801 live on ClinicalTrials.gov.