At a glance
ClinicalIndex Comparison RecordPhase 2Completed· 22 enrolled
Drug / intervention
BMN 701biological
Likely dose
Not stated in record
Structured eligibility isn't available for this trial yet — see the full criteria in the Eligibility tab below.
Standardized by ClinicalIndex from the ClinicalTrials.gov record · verify against the source.
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
In Brief
A Phase 2 clinical trial evaluating BMN 701 for Pompe Disease. Completed, enrolled 22 participants across 10 sites in 5 countries.
Detailed Summary
A Phase 1/2, open-label, multicenter, multiple dose escalation study of BMN 701 administered by intravenous infusion every 2 weeks over a 24-week treatment period to patients with late-onset Pompe disease.
Study Details
Study Typeinterventional
Allocation--
Masking--
Primary Purpose--
ConditionsPompe Disease
CountriesAustralia, France, Germany, United Kingdom, United States
Collaborators--
Timeline
Phase 2CompletedFinished
2011201220132014201520162017201820192020202120222023202420252026
First PostedOct 2010
Enrollment StartJan 2011
Primary CompletionMar 2013
TodayJul 2026
First PostedOct 29, 2010
Enrollment StartJan 17, 2011
Primary CompletionMar 6, 2013
TodayJul 2, 2026
Enrollment to primary: 2.1 yearsPosted 15.7 years ago
Interventions
BMN 701biological
GILT-tagged recombinant human GAA