Phase I/II Study of SRP-4053 in DMD Patients

PHASE1/PHASE2CompletedINTERVENTIONAL
Enrollment

39

Participants

Timeline

Start Date

January 13, 2015

Primary Completion Date

March 25, 2019

Study Completion Date

March 25, 2019

Conditions
Duchenne Muscular Dystrophy
Interventions
DRUG

Placebo

SRP-4053 placebo-matching solution for IV infusion.

DRUG

SRP-4053

SRP-4053 (golodirsen) solution for IV infusion.

Trial Locations (5)

75013

Institute de Myologie, Paris

02116

Boston Children's Hospital, Boston

00168

Policlinico Universitario A Gemelli, Rome

WC1N 3JH

Great Ormond Street Hospital for Children NHS Foundation Trust, London

Unknown

Newcastle University Hospital, Newcastle

All Listed Sponsors
collaborator

Institut de Myologie, France

OTHER

collaborator

Consultants for Research in Imaging and Spectroscopy

OTHER

collaborator

Great Ormond Street Hospital for Children NHS Foundation Trust

OTHER

collaborator

Catholic University of the Sacred Heart

OTHER

collaborator

Royal Holloway University

OTHER

collaborator

SYSNAV

INDUSTRY

collaborator

University College, London

OTHER

collaborator

University of Newcastle Upon-Tyne

OTHER

lead

Sarepta Therapeutics, Inc.

INDUSTRY

NCT02310906 - Phase I/II Study of SRP-4053 in DMD Patients | Biotech Hunter | Biotech Hunter