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Gene therapy hope for boys with duchenne muscular dystrophy

NCT ID NCT04281485

Disease control Sponsor: Pfizer Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing This study
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Jul 23, 2026 · Updated 2 times

Summary

This study tests a gene therapy called PF-06939926 in 114 boys with Duchenne muscular dystrophy (DMD), a severe muscle-wasting disease. Two-thirds receive the gene therapy, while one-third get a placebo, but can switch to the real treatment after one year. The main goal is to see if it improves muscle function and dystrophin levels over 52 weeks.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
gene therapy (PF-06939926)
What this could lead to
If successful, this gene therapy could slow or improve muscle function in boys with Duchenne muscular dystrophy, offering a new treatment option.
What could go wrong
This is an early Phase 3 trial with only 114 participants, so results may not apply to all. Gene therapy carries risks like immune reactions, and long-term effects are unknown.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

114 people

The number who actually took part.

Started

Nov 2020

Expected to finish

Apr 2039

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

4 to 7 years

Sex

Male participants only

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Key inclusion criteria: 1. Confirmed diagnosis of Duchenne muscular dystrophy by prior genetic testing 2. Receiving a stable daily dose (at least 0.5 mg/kg/day prednisone or prednisolone, or at least 0.75 mg/kg/day deflazacort) for at least 3 months prior to Screening 3. Ambulatory, as assessed by protocol-specified criteria Key exclusion criteria: 1. Positive test performed by Pfizer for neutralizing antibodies to AAV9 2. Any treatment designed to increase dystrophin expression within 6 months prior to screening (e.g., Translarna™, EXONDYS 51™, VYONDYS 53™) 3. Any prior treatment with gene therapy 4. Any non-healed injury that may impact functional testing (eg NSAA) 5. Abnormality in specified laboratory tests, including blood counts, liver and kidney function 6. Any of the following genetic abnormalities in the dystrophin gene: 1. Any mutation (exon deletion, exon duplication, insertion, or point mutation) affecting any exon between exon 9 and exon 13, inclusive; OR 2. A deletion that affects both exon 29 and exon 30;OR 3. A deletion that affects any exons between 56-71, inclusive.

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Conditions

The condition(s) this trial relates to.

Duchenne muscular dystrophy

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Duchenne muscular dystrophy

Contacts and locations

Locations

  • Alberta Children's Hospital

    Calgary, Alberta, T3B 6A8, Canada

  • Alder Hey Children's NHS Foundation Trust

    Liverpool, Merseyside, L12 2AP, United Kingdom

  • Arkansas Children's

    Little Rock, Arkansas, 72202, United States

  • Arkansas Children's Hospital

    Little Rock, Arkansas, 72202, United States

  • CHU de Nantes- Hotel Dieu

    Nantes, 44093, France

  • Charité - Universitätsmedizin Berlin

    Berlin, 13353, Germany

  • Children's Hospital - London Health Sciences Centre

    London, Ontario, N6A 4G5, Canada

  • Childrens Hospital of Eastern Ontario

    Ottawa, Ontario, K1H8L1, Canada

  • Fondazione Policlinico Universitario Agostino Gemelli IRCCS

    Roma, 00168, Italy

  • Great Ormond Street Institute of Child Health

    London, WCIN 1EH, United Kingdom

  • Hadassah University Medical Center, Ein Kerem

    Jerusalem, 91120, Israel

  • Hopital Necker

    Paris, 75015, France

  • Hospital Sant Joan de Déu

    Esplugues de Llobregat, Barcelona, 08950, Spain

  • Hospital Universitari i Politecnic La Fe de Valencia

    Valencia, 46026, Spain

  • Hospital Universitario Vall d'Hebron

    Barcelona, 08035, Spain

  • Hyogo College of Medicine College Hospital

    Nishinomiya, Hyōgo, 663-8501, Japan

  • IRCCS Ospedale Pediatrico Bambino Gesù

    Rome, 00165, Italy

  • Inselspital, University Children's Hospital Berne

    Bern, 3010, Switzerland

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

    Fairway, Kansas, 66205, United States

  • KU Clinical Research Center - Clinical and Translational Science Unit (CTSU) - Rainbow

    Kansas City, Kansas, 66160, United States

  • Kaohsiung Medical University Chung-Ho Memorial Hospital

    Kaohsiung City, 807, Taiwan

  • Lenox Baker Children's Hospital

    Durham, North Carolina, 27705, United States

  • Nagoya City University Hospital

    Nagoya, Aichi-ken, 467-8602, Japan

  • National Center of Neurology and Psychiatry

    Tokyo, 187-8551, Japan

  • National Taiwan University Hospital

    Taipei, 100, Taiwan

  • Pediatric Cardiology

    Prairie Village, Kansas, 66208, United States

  • Perth Children's Hospital

    Nedlands, Western Australia, 6009, Australia

  • Primary Childrens Hospital

    Salt Lake City, Utah, 84113, United States

  • Pusan National University Yangsan Hospital

    Yangsan, Gyeongsangnam-do, 50612, South Korea

  • Saint Petersburg State Paediatric Medical University

    Saint Petersburg, 194100, Russia

  • Samsung Medical Center

    Seoul, 06351, South Korea

  • Schneider Children's Medical Center of Israel

    Petach Tikvah, 4920235, Israel

  • Seattle Children's

    Seattle, Washington, 98105, United States

  • Seoul National University Hospital

    Seoul, 03080, South Korea

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

    Yekaterinburg, 620134, Russia

  • Taipei Veterans General Hospital

    Taipei, 11217, Taiwan

  • The Children's Hospital at Westmead

    Westmead, New South Wales, 2145, Australia

  • The Children's Hospital of Philadelphia

    Philadelphia, Pennsylvania, 19104, United States

  • The Hospital For Sick Children

    Toronto, Ontario, M5G 1X8, Canada

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

    Newcastle upon Tyne, England, NE1 4LP, United Kingdom

  • The Royal Children's Hospital Melbourne

    Parkville, Victoria, 3052, Australia

  • UCLA Medical Center

    Los Angeles, California, 90095, United States

  • UZ Gent

    Ghent, 9000, Belgium

  • UZ leuven

    Leuven, 3000, Belgium

  • Universitaets-Kinderspital Zuerich

    Zurich, 8008, Switzerland

  • Universitatsklinikum Essen

    Essen, 45147, Germany

  • University of Florida

    Gainesville, Florida, 32610, United States

  • University of Iowa Hospitals and Clinics

    Iowa City, Iowa, 52242, United States

  • University of Kansas Hospital - Investigational Pharmacy

    Kansas City, Kansas, 66160, United States

  • University of Kansas Hospital - Pediatric and Pediatric ICU - Operating Room

    Kansas City, Kansas, 66160, United States

  • University of Kansas Medical Center

    Kansas City, Kansas, 66160, United States

  • University of Utah Clinical Neurosciences Center

    Salt Lake City, Utah, 84132, United States

  • University of Utah Hospital

    Salt Lake City, Utah, 84132, United States

More trials for these conditions

Other studies related to the condition(s) this trial covers.