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

Phase 1

Completed

• Conditions: Pompe Disease
• Interventions: Biological: BMN 701

• Registration Number: NCT01230801
• Lead Sponsor: BioMarin Pharmaceutical

Brief 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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2010-10-27
• Study First Submitted QC: 2010-10-28
• Study First Posted: 2010-10-29
• Study Start: 2011-01-17
• Primary Completion: 2013-03-06
• Study Completion: 2013-03-06
• Results First Submitted: 2018-03-19
• Status Verified: 2018-05
• Results First Submitted QC: 2018-05-08
• Last Update Submitted: 2018-05-08
• Results First Posted: 2018-06-11
• Last Update Posted: 2018-06-11

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 22

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
BMN 701	BMN 701	IV infusion

Primary Outcome Measures

Name	Time	Method
Number of Participants With Adverse Events	24 weeks	Number of Participants with Adverse Events as a Measure of Safety and Tolerability

Secondary Outcome Measures

Name	Time	Method
Change From Baseline in Six Minutes Walk Test	Baseline up to 24 weeks	Change from Baseline in Six Minutes Walk Test. The 6MWT measured the maximum distance the subject could walk on a flat, hard surface in a period of 6 minutes

Trial Locations

Locations (10)

Royal Adelaide Hospital, SA Pathology; 🇦🇺 Adelaide, Adelaide, SA, Australia

Old Queen Elizabeth Hospital, Department of Medicine; 🇬🇧 Birmingham, United Kingdom

Salford Royal Hospital NHS Trust; 🇬🇧 Salford, United Kingdom

Royal Free Hospital; 🇬🇧 London, United Kingdom

Univ of California San Diego School of Medicine; 🇺🇸 La Jolla, California, United States

Hôpital de I´Archet- Centre Hospitalier Universitaire Nice; 🇫🇷 Nice, France

Hôpital Pitié-Salpêtrière; 🇫🇷 Paris Cedex 13, France

Zentrum für Kinder- und Jugenmedizin; 🇩🇪 Mainz, Rheinland-pfalz, Germany

University of Florida College of Medicine; 🇺🇸 Gainesville, Florida, United States

University of Kansas Medical Center; 🇺🇸 Kansas City, Kansas, United States