Study to Evaluate the Safety and Efficacy of PF-06939926 for the Treatment of Duchenne Muscular Dystrophy

PHASE3Active, not recruitingINTERVENTIONAL
Enrollment

114

Participants

Timeline

Start Date

November 5, 2020

Primary Completion Date

May 15, 2024

Study Completion Date

April 15, 2039

Conditions
Duchenne Muscular Dystrophy
Interventions
GENETIC

PF-06939926

PF-06939926 will be administered as a single IV infusion at Year 1 for Cohort 1.

OTHER

Placebo

Placebo will be administered as a single IV infusion at Year 1 for Cohort 2.

OTHER

Placebo

Placebo will be administered as a single IV infusion at Year 2 for Cohort 1.

GENETIC

PF-06939926

PF-06939926 will be administered as a single IV infusion at Year 2 for Cohort 2

Trial Locations (51)

100

National Taiwan University Hospital, Taipei

807

Kaohsiung Medical University Chung-Ho Memorial Hospital, Kaohsiung City

2145

The Children's Hospital at Westmead, Westmead

3000

UZ leuven, Leuven

3010

Inselspital, University Children's Hospital Berne, Bern

3052

The Royal Children's Hospital Melbourne, Parkville

6009

Perth Children's Hospital, Nedlands

8008

Universitaets-Kinderspital Zuerich, Zurich

9000

UZ Gent, Ghent

11217

Taipei Veterans General Hospital, Taipei

13353

Charité - Universitätsmedizin Berlin, Berlin

19104

The Children's Hospital of Philadelphia, Philadelphia

27705

Lenox Baker Children's Hospital, Durham

32610

University of Florida, Gainesville

44093

CHU de Nantes- Hotel Dieu, Nantes

45147

Universitatsklinikum Essen, Essen

46026

Hospital Universitari i Politecnic La Fe de Valencia, Valencia

50612

Pusan National University Yangsan Hospital, Yangsan

52242

University of Iowa Hospitals and Clinics, Iowa City

66160

KU Clinical Research Center - Clinical and Translational Science Unit (CTSU) - Rainbow, Kansas City

University of Kansas Hospital - Investigational Pharmacy, Kansas City

University of Kansas Hospital - Pediatric and Pediatric ICU - Operating Room, Kansas City

University of Kansas Medical Center, Kansas City

66205

KU Clinical Research Center - Clinical and Translational Science Unit (CTSU) - Fairway, Fairway

66208

Pediatric Cardiology, Prairie Village

72202

Arkansas Children's Hospital, Little Rock

Arkansas Children's, Little Rock

75015

Hopital Necker, Paris

84132

University of Utah Clinical Neurosciences Center, Salt Lake City

90095

UCLA Medical Center, Los Angeles

91120

Hadassah University Medical Center, Ein Kerem, Jerusalem

98105

Seattle Children's, Seattle

194100

Saint Petersburg State Paediatric Medical University, Saint Petersburg

620134

State Autonomous Healthcare Institution of Sverdlovsk Region Children's City Clinical Hospital No 9, Yekaterinburg

4920235

Schneider Children's Medical Center of Israel, Petach Tikvah

T3B 6A8

Alberta Children's Hospital, Calgary

N6A 4G5

Children's Hospital - London Health Sciences Centre, London

K1H8L1

Childrens Hospital of Eastern Ontario, Ottawa

M5G 1X8

The Hospital For Sick Children, Toronto

00168

Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Roma

00165

IRCCS Ospedale Pediatrico Bambino Gesù, Rome

467-8602

Nagoya City University Hospital, Nagoya

663-8501

Hyogo College of Medicine College Hospital, Nishinomiya

187-8551

National Center of Neurology and Psychiatry, Tokyo

03080

Seoul National University Hospital, Seoul

06351

Samsung Medical Center, Seoul

08950

Hospital Sant Joan de Déu, Esplugues de Llobregat

08035

Hospital Universitario Vall d'Hebron, Barcelona

NE1 4LP

The Newcastle upon Tyne Hospitals NHS Foundation Trust, Royal Victoria Infirmary, Newcastle upon Tyne

L12 2AP

Alder Hey Children's NHS Foundation Trust, Liverpool

WCIN 1EH

Great Ormond Street Institute of Child Health, London

Sponsors

Lead Sponsor

All Listed Sponsors
lead

Pfizer

INDUSTRY

NCT04281485 - Study to Evaluate the Safety and Efficacy of PF-06939926 for the Treatment of Duchenne Muscular Dystrophy | Biotech Hunter | Biotech Hunter