Ignite Creation Date:
2025-12-24 @ 2:59 PM
Ignite Modification Date:
2026-03-06 @ 9:33 AM
Study NCT ID:
NCT01957059
Status:
TERMINATED
Last Update Posted:
2017-12-08
First Post:
2013-07-02
Is Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
A Phase I/II Study of BMN053 in Subjects With Duchenne Muscular Dystrophy (DMD)
Sponsor:
BioMarin Pharmaceutical
Organization:
Study Overview
Official Title:
A Phase I/II, Open-label, Dose Escalating With 48 Week Treatment Study to Assess the Safety and Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of BMN053 (Previously Known as PRO053) in Subjects With Duchenne Muscular Dystrophy.
Status:
TERMINATED
Status Verified Date:
2017-12
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief 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.
Detailed Description:
A Phase I/II, open-label, dose escalating with 48-week treatment study to assess the safety and tolerability, pharmacokinetics, pharmacodynamics and efficacy of BMN 053 (previously known as PRO053) in subjects with Duchenne muscular dystrophy
Study Oversight
Has Oversight DMC:
True
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?: