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Safety/Tolerability/Pharmacokinetic (PK)/Pharmacodynamics (PD) Study of BMN701 in Patients With Late-Onset Pompe Disease

NCT01230801 · Status: COMPLETED · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 22

Last updated 2018-06-11

Study results available
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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

  • Pompe Disease

Interventions

BIOLOGICAL

BMN 701

GILT-tagged recombinant human GAA

Sponsors & Collaborators

Principal Investigators

  • Medical Monitor · BioMarin Pharmaceutical

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
13 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2011-01-17
Primary Completion
2013-03-06
Completion
2013-03-06
FDA Drug
Yes

Countries

  • United States
  • Australia
  • France
  • Germany
  • United Kingdom

Study Locations

More Related Trials

Entities

Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT01230801 on ClinicalTrials.gov