BMN 701 Phase 3 in rhGAA Exposed Subjects With Late Onset Pompe Disease (INSPIRE Study)

ClinicalTrials.gov ID: NCT01924845

Public ClinicalTrials.gov record NCT01924845. 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.

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ClinicalTrials.gov public records Last synced May 20, 2026, 1:05 AM EDT

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 3 Switchover Study of the Efficacy and Safety of BMN 701 (GILT-tagged Recombinant Human GAA) and Long-Term Study for Extended Treatment in rhGAA Exposed Subjects With Late-onset Pompe Disease

Study identification

NCT ID: NCT01924845
Recruitment status: Terminated
Study type: Interventional
Phase: Phase 3
Lead sponsor: BioMarin Pharmaceutical; Industry
Enrollment: 24 participants

Conditions and interventions

Conditions

Late-onset Pompe Disease

Interventions

BMN 701 Drug

Drug

Eligibility (public fields only)

Age range: 18 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: Mar 31, 2014
Primary completion: Sep 11, 2016
Completion: Sep 11, 2016
Last update posted: Jun 13, 2018

2014 – 2016

United States locations

U.S. sites: 8
U.S. states: 8
U.S. cities: 8

Facility	City	State	ZIP	Site status
Neuromuscular Research Centre	Phoenix	Arizona	85028	—
University of California, Irvine	Orange	California	92868	—
University of Florida	Gainesville	Florida	32610	—
University of Kansas Medical Center	Kansas City	Kansas	66160	—
Washington University	St Louis	Missouri	36110	—
Duke University Medical Center	Durham	North Carolina	27710	—
The Ohio State University - Wexner Medical Center	Columbus	Ohio	43210	—
University of Utah	Salt Lake City	Utah	84132	—

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 14 non-U.S. sites.

About this trial record page

What this page shows: Public field values for ClinicalTrials.gov record NCT01924845, 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 13, 2018 · Synced May 20, 2026

Open the official record

The complete protocol, eligibility criteria, and contact information for NCT01924845 live on ClinicalTrials.gov.

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