Clinical Research Directory
Browse clinical research sites, groups, and studies.
Study to Evaluate the Safety and Efficacy of PF-06939926 for the Treatment of Duchenne Muscular Dystrophy
Sponsor: Pfizer
Summary
The study will evaluate the safety and efficacy of gene therapy in boys with DMD. It is a randomized, double-blind, placebo-controlled study with two thirds of participants assigned to gene therapy. The one third of participants who are randomized to the placebo arm will have an opportunity for treatment with gene therapy at the beginning of the second year.
Official title: A PHASE 3, MULTICENTER, RANDOMIZED, DOUBLE-BLIND, PLACEBO CONTROLLED STUDY TO EVALUATE THE SAFETY AND EFFICACY OF PF 06939926 FOR THE TREATMENT OF DUCHENNE MUSCULAR DYSTROPHY
Key Details
Gender
MALE
Age Range
4 Years - 7 Years
Study Type
INTERVENTIONAL
Enrollment
114
Start Date
2020-11-05
Completion Date
2039-04-15
Last Updated
2026-03-02
Healthy Volunteers
No
Conditions
Interventions
PF-06939926
PF-06939926 will be administered as a single IV infusion at Year 1 for Cohort 1.
Placebo
Placebo will be administered as a single IV infusion at Year 1 for Cohort 2.
Placebo
Placebo will be administered as a single IV infusion at Year 2 for Cohort 1.
PF-06939926
PF-06939926 will be administered as a single IV infusion at Year 2 for Cohort 2
Locations (53)
Arkansas Children's Hospital
Little Rock, Arkansas, United States
Arkansas Children's
Little Rock, Arkansas, United States
UCLA Medical Center
Los Angeles, California, United States
University of Florida
Gainesville, Florida, United States
University of Iowa Hospitals and Clinics
Iowa City, Iowa, United States
KU Clinical Research Center - Clinical and Translational Science Unit (CTSU) - Fairway
Fairway, Kansas, United States
KU Clinical Research Center - Clinical and Translational Science Unit (CTSU) - Rainbow
Kansas City, Kansas, United States
University of Kansas Hospital - Investigational Pharmacy
Kansas City, Kansas, United States
University of Kansas Hospital - Pediatric and Pediatric ICU - Operating Room
Kansas City, Kansas, United States
University of Kansas Medical Center
Kansas City, Kansas, United States
Pediatric Cardiology
Prairie Village, Kansas, United States
Lenox Baker Children's Hospital
Durham, North Carolina, United States
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
Primary Childrens Hospital
Salt Lake City, Utah, United States
University of Utah Clinical Neurosciences Center
Salt Lake City, Utah, United States
University of Utah Hospital
Salt Lake City, Utah, United States
Seattle Children's
Seattle, Washington, United States
The Children's Hospital at Westmead
Westmead, New South Wales, Australia
The Royal Children's Hospital Melbourne
Parkville, Victoria, Australia
Perth Children's Hospital
Nedlands, Western Australia, Australia
UZ Gent
Ghent, Belgium
UZ leuven
Leuven, Belgium
Alberta Children's Hospital
Calgary, Alberta, Canada
Children's Hospital - London Health Sciences Centre
London, Ontario, Canada
Childrens Hospital of Eastern Ontario
Ottawa, Ontario, Canada
The Hospital For Sick Children
Toronto, Ontario, Canada
CHU de Nantes- Hotel Dieu
Nantes, France
Hopital Necker
Paris, France
Charité - Universitätsmedizin Berlin
Berlin, Germany
Universitatsklinikum Essen
Essen, Germany
Hadassah University Medical Center, Ein Kerem
Jerusalem, Israel
Schneider Children's Medical Center of Israel
Petach Tikvah, Israel
Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Roma, Italy
IRCCS Ospedale Pediatrico Bambino Gesù
Rome, Italy
Nagoya City University Hospital
Nagoya, Aichi-ken, Japan
Hyogo College of Medicine College Hospital
Nishinomiya, Hyōgo, Japan
National Center of Neurology and Psychiatry
Tokyo, Japan
Saint Petersburg State Paediatric Medical University
Saint Petersburg, Russia
State Autonomous Healthcare Institution of Sverdlovsk Region Children's City Clinical Hospital No 9
Yekaterinburg, Russia
Pusan National University Yangsan Hospital
Yangsan, Gyeongsangnam-do, South Korea
Seoul National University Hospital
Seoul, South Korea
Samsung Medical Center
Seoul, South Korea
Hospital Sant Joan de Déu
Esplugues de Llobregat, Barcelona, Spain
Hospital Universitario Vall d'Hebron
Barcelona, Spain
Hospital Universitari i Politecnic La Fe de Valencia
Valencia, Spain
Inselspital, University Children's Hospital Berne
Bern, Switzerland
Universitaets-Kinderspital Zuerich
Zurich, Switzerland
Kaohsiung Medical University Chung-Ho Memorial Hospital
Kaohsiung City, Taiwan
National Taiwan University Hospital
Taipei, Taiwan
Taipei Veterans General Hospital
Taipei, Taiwan
The Newcastle upon Tyne Hospitals NHS Foundation Trust, Royal Victoria Infirmary
Newcastle upon Tyne, England, United Kingdom
Alder Hey Children's NHS Foundation Trust
Liverpool, Merseyside, United Kingdom
Great Ormond Street Institute of Child Health
London, United Kingdom