Extension Study for Patients Who Have Participated in a BMN 701 Study
Public ClinicalTrials.gov record NCT01435772. 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 Long-Term Study for Extended BMN 701 Treatment of Patients With Pompe Disease Who Have Participated in a BMN 701 Study
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
This is a Phase 2 open-label, multiple dose study of BMN 701 administered by IV infusion every 2 weeks (qow) to patients with late-onset Pompe disease.
Study identification
- NCT ID
- NCT01435772
- Recruitment status
- Terminated
- Study type
- Interventional
- Phase
- Phase 2
- Enrollment
- 21 participants
Conditions and interventions
Conditions
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
- Aug 14, 2011
- Primary completion
- Sep 8, 2016
- Completion
- Sep 8, 2016
- Last update posted
- May 21, 2018
2011 – 2016
United States locations
- U.S. sites
- 4
- U.S. states
- 3
- U.S. cities
- 4
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| Univ of California San Diego School of Medicine | San Diego | California | 92103 | — |
| University of Florida College of Medicine | Gainesville | Florida | 32610 | — |
| Tampa General Hospital | Tampa | Florida | 33606 | — |
| 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 8 non-U.S. sites.
Recently updated Pompe Disease trials
Other public records listing Pompe Disease. Sorted by last update posted; newest first. Not a recommendation.
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About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT01435772, 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 May 21, 2018 · Synced Sep 10, 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 NCT01435772 live on ClinicalTrials.gov.