Active not recruitingPHASE3INTERVENTIONAL

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

This research study is looking into a new gene therapy called PF-06939926 for Duchenne muscular dystrophy (DMD) in boys aged 4 to 7. The main goals are to check if the treatment is safe and if it helps improve how well boys can move. Participants will be split into groups; two-thirds will receive the gene therapy at the start, while the other third will first receive a 'dummy' treatment (placebo) and then the gene therapy after one year. The gene therapy is given through a drip into a vein. All boys will be followed for many years to see how they get on. The study covers all costs related to your involvement, including travel.

At a glance

Status

Active not recruiting

Phase

PHASE3

Sponsor

Pfizer

Enrolment target

114

Start

05 Nov 2020

Estimated completion

15 Apr 2039

Duchenne Muscular Dystrophy

What is this study about?

This study is about a new approach to treating Duchenne muscular dystrophy, a condition that causes muscles to weaken over time. Researchers are testing a special treatment called 'gene therapy,' which aims to help the body make a missing protein essential for healthy muscles. The specific gene therapy being studied is called PF-06939926.

The main purpose of this study is to find out if this gene therapy is safe for boys with Duchenne muscular dystrophy and if it can help improve their muscle strength and movement. About 99 boys will take part. To make sure the results are as clear as possible, the study uses a method where some boys receive the active gene therapy, and others receive a 'placebo' – which looks just like the gene therapy but contains no active medicine. This helps researchers understand the true effects of the treatment.

Two out of three participants will receive the gene therapy at the beginning of the study. The remaining one third will receive the placebo first, and then get the active gene therapy after one year, provided it's still considered safe. Everyone involved in the study – the participants, their families, and the medical team – won't know who is getting the active treatment and who is getting the placebo at the start. This is called 'double-blind' and helps ensure the study results are fair and unbiased.

Key takeaways

This study is testing a new gene therapy (PF-06939926) for Duchenne muscular dystrophy.
It's for boys aged 4 to 7 who can still walk and are taking daily steroids.
Two out of three boys will get the active treatment first; others get a placebo then the active treatment a year later.
The treatment is given as a drip into a vein and involves long-term monitoring for up to 15 years.
All study-related costs, including travel, are covered.
The study aims to check if the therapy is safe and improves movement.

Who may be eligible?

This study is for boys with Duchenne muscular dystrophy who are between 4 and 7 years old (up to their 8th birthday). To join, they must have a confirmed diagnosis of DMD through a genetic test and be able to walk. Also, they need to have been taking a daily steroid medication (like prednisolone or deflazacort) for at least three months before the study starts, and plan to continue taking it for the first two years of the study.

There are some reasons why a boy might not be able to join. For example, if they have certain antibodies in their blood that might stop the gene therapy from working effectively, as identified by a specific test for the study. Also, if they have had other treatments to increase muscle protein in the last six months, or any other gene therapy before, they won't be able to participate. Certain genetic changes in their DMD gene or other health issues found in blood tests could also mean they can't join.

It's important that any injuries that could affect movement tests are fully healed. If you think your son might be a good fit, it's best to discuss all these points with the study team.

Quick self-check

Is your son a boy aged between 4 and 7 years old?
Does he have a confirmed diagnosis of Duchenne muscular dystrophy by a genetic test?
Is he currently able to walk?
Has he been taking daily steroid medication for at least 3 months?
Has he NOT had any other gene therapy before?
Has he NOT had any other treatments to increase muscle protein in the last 6 months?

This is a guide only — the research team will confirm whether you can take part.

What does participation involve?

If your son takes part in this study, he will receive either the gene therapy or a placebo through a drip into a vein, which will take up to two hours. For the first year, some boys will receive the active treatment, and others the placebo. After one year, those who received the placebo will then receive the active gene therapy, if it's still safe to do so. You and the study doctors won't know which treatment he gets at the start.

Throughout the study, there will be regular visits to the study clinic for assessments. These will involve checking your son's health, taking blood samples, and conducting tests to measure his walking and movement abilities. The main assessment to see how well the treatment is working will happen at 52 weeks (about one year).

After receiving the gene therapy, all participants will be followed very carefully for a long period – up to 15 years – to monitor their health and any long-term effects. The study covers all costs for the medication, all medical tests, and visits to the study sites. They will also help with your travel costs for attending these visits.

Potential risks and benefits

Taking part in any clinical study has potential benefits and risks. The potential benefit of this study is that the gene therapy could improve muscle function and slow down the progression of Duchenne muscular dystrophy, although this is not guaranteed and is what the study aims to find out. As with any new medicine, there are potential side effects, and these will be carefully monitored by the study team. They will explain all known or potential risks to you in detail before you decide to participate. You have the right to withdraw your child from the study at any time, for any reason, without it affecting his medical care.

Locations (53)

Arkansas Children's Hospital
Little Rock, United States
Arkansas Children's
Little Rock, United States
UCLA Medical Center
Los Angeles, United States
University of Florida
Gainesville, United States
University of Iowa Hospitals and Clinics
Iowa City, United States
KU Clinical Research Center - Clinical and Translational Science Unit (CTSU) - Fairway
Fairway, United States
KU Clinical Research Center - Clinical and Translational Science Unit (CTSU) - Rainbow
Kansas City, United States
University of Kansas Hospital - Investigational Pharmacy
Kansas City, United States
University of Kansas Hospital - Pediatric and Pediatric ICU - Operating Room
Kansas City, United States
University of Kansas Medical Center
Kansas City, United States
Pediatric Cardiology
Prairie Village, United States
Lenox Baker Children's Hospital
Durham, United States

+41 more sites — see the official record for the full list.

Common questions

What is gene therapy?

Gene therapy is a new type of treatment that tries to fix the root cause of a disease like Duchenne muscular dystrophy by introducing healthy genetic material into the body's cells.

What is a placebo?

A placebo is a 'dummy' treatment that looks exactly like the real treatment but doesn't contain any active medicine. It helps researchers fairly compare the effects of the actual drug.

How is the treatment given?

The gene therapy, or placebo, is given through a special drip into a vein, similar to how fluids or medicines are sometimes given in hospital. This usually takes up to two hours.

How long will my son be in the study?

After your son receives the gene therapy, he will be followed by the study team for a very long time – up to 15 years – to monitor his health and any long-term effects.

Will I have to pay for anything?

No, all study treatment, medical tests, visits, and reasonable travel costs related to the study will be covered. This study is free to participants.