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A Phase I/II Study of BMN053 in Subjects With Duchenne Muscular Dystrophy (DMD)

NCT01957059 · Status: TERMINATED · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 9

Last updated 2017-12-08

No results posted yet for this study

Summary

The purpose of the study is to see whether BMN053 is safe and effective to use as medication for Duchenne muscular dystrophy (DMD) patients with a mutation around location 53 in the DNA for the dystrophin protein.

Conditions

Interventions

DRUG

Regimen Selection Phase Group 2

All doses of BMN053 will be administered as IV infusions. The proposed doses are as follows: • 3 mg/kg

DRUG

Regimen Selection Phase Group 3

All doses of BMN053 will be administered as IV infusions. The proposed doses are as follows: • 4-6 mg/kg

DRUG

Treatment Phase Group 4

All doses of PRO053 will be administered as IV infusions. The proposed doses will be decided upon completion of the Regimen Selection Phase of Groups 2 and 3

DRUG

Regimen Selection Phase Group 1 (COMPLETED)

All doses of BMN053 have been administered as subcutaneous injections.

DRUG

Dosing Extension

All doses of PRO053 will be administered as IV infusions. The proposed doses will be decided upon completion of the Regimen Selection Phase of Groups 2 and 3 and the Treatment Phase Group 4.

Sponsors & Collaborators

Principal Investigators

  • V. Straub, Prof. · Institute of Genetic Medicine, Newcastle University, UK

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
PARALLEL

Eligibility

Min Age
5 Years
Max Age
18 Years
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2013-06-30
Primary Completion
2016-08-03
Completion
2016-08-03

Countries

  • Belgium
  • France
  • Italy
  • Netherlands
  • United Kingdom

Study Locations

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Entities

Diseases
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 NCT01957059 on ClinicalTrials.gov