A myostatin inhibitor for the treatment of Duchenne muscular dystrophy

Start Date
01/10/2014
Expected end Date
01/02/2018
Location
UK, North America, Japan
Status
Recruiting

What is the aim of the trial?

Myostatin is a natural protein that inhibits muscle growth. The aim of this trial is to investigate the safety, tolerability and efficacy of the compound PF-06252616. This is an antibody that blocks myostatin. This way it is hoped that the muscle mass and strength of people with Duchenne muscular dystrophy may be increased.

Who can be involved in the trial?

The trial is enrolling boys aged six to nine years old with Duchenne muscular dystrophy and who are still able to walk. Participants must be receiving glucocorticosteroids for a minimum of six months before enrolling. If participants have kidney, liver or heart problems, use certain medications or have participated in other clinical trials they may be ineligible for the trial.

For full inclusion and exclusion criteria, please see ‘Further trial details’ below.

What happens during the trial?

The trial will last 96 weeks. The participants will be divided in three groups. During the first 48 weeks two of the groups will receive the drug PF-06252616, while the third group will receive a placebo. During the second 48 weeks, the group that received the placebo will receive the drug, and the other two groups will either receive the drug or a placebo. Therefore one group will have received the drug throughout the 96 weeks whereas the other groups will first receive the drug then the placebo and vice versa. The whole assignment will be made randomly and neither the participants nor the clinicians will know who is receiving the placebo and who is receiving the drug.

Both the drug and the placebo will be given to the participants as a monthly injection. Participants will be closely monitored through laboratory tests, heart monitoring, physical examinations, X-rays and MRI. The efficacy of the drug will be tested by tests such as lung function, four stair climb, six minute walk and muscle strength.

Where is the study taking place?

The study is taking place at the Institute of Genetic Medicine and Royal Victoria Infirmary in Newcastle. The trial is also taking place in five states in the USA, two states in Canada and two cities in Japan.

For exact details of the trial locations, please see ‘Further trial details’ below.

How could the results of the trial benefit patients?

The trial is exploring whether the drug has the potential to increase muscle mass and function. If the results of this phase 2 trial are positive PF-06252616 may be a treatment option for boys with Duchenne muscular dystrophy regardless of their mutation as well as for people with other muscle-wasting conditions.

Trial results:

The results of this trial are expected to be available in 2018.

Contact Details:

Pfizer CT.gov Call Center

Tel: 1-800-718-1021

Who is funding this study?

  • Pfizer

Official name of the Trial:

A Phase 2 Study to Evaluate the Safety, Efficacy, Pharmacokinetics and Pharmacodynamics of PF-06252616 in Duchenne Muscular Dystrophy.

Trial study number:

NCT02310763

Further trial details:

For further information on the trial and detailed inclusion and exclusion please click on the link below. Sometimes these details can be quite technical. If you have any questions please discuss this with your clinician or contact the clinical trial organisers.

https://clinicaltrials.gov/ct2/show/NCT02310763?term=PF-06252616&rank=2

Background information and related links:

Myostatin is a natural protein that inhibits muscle growth. The aim of this trial is to investigate the safety, tolerability and efficacy of the compound PF-06252616. This is an antibody that blocks myostatin. This way it is hoped that the muscle mass and strength of people with Duchenne muscular dystrophy may be increased.

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