Trials / Completed
CompletedNCT01230801
Safety/Tolerability/Pharmacokinetic (PK)/Pharmacodynamics (PD) Study of BMN701 in Patients With Late-Onset Pompe Disease
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
- Status
- Completed
- Phase
- Phase 1 / Phase 2
- Study type
- Interventional
- Enrollment
- 22 (actual)
- Sponsor
- BioMarin Pharmaceutical · Industry
- Sex
- All
- Age
- 13 Years
- Healthy volunteers
- Not accepted
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.
Conditions
Interventions
| Type | Name | Description |
|---|---|---|
| BIOLOGICAL | BMN 701 | GILT-tagged recombinant human GAA |
Timeline
- Start date
- 2011-01-17
- Primary completion
- 2013-03-06
- Completion
- 2013-03-06
- First posted
- 2010-10-29
- Last updated
- 2018-06-11
- Results posted
- 2018-06-11
Locations
10 sites across 5 countries: United States, Australia, France, Germany, United Kingdom
Regulatory
- FDA-regulated drug study
Source: ClinicalTrials.gov record NCT01230801. Inclusion in this directory is not an endorsement.