FOR-DMD: Phase 3 study to find the optimal steroid treatment for Duchenne

Finding the optimal steroid treatment for Duchenne muscular dystrophy

Questions and Answers

What stage is this research?

The study is ongoing (SUBJECT FOLLOW-UP VISITS THROUGH NOVEMBER, 2019) but recruitment ended in September, 2016.

Updates:

Subjects enrolled in FOR-DMD are able to participate concurrently in Summit’s SMTC11005 trial or Italfarmaco’s Givinostat study. Participants should check with their FOR-DMD investigator or study coordinator if any other Duchenne trial the might want to enter has a concurrent participation agreement in place with the FOR-DMD study.

What is the goal or purpose of the FOR-DMD study?

This study will look at the benefits and side effects of the three most widely prescribed steroid treatments for Duchenne muscular dystrophy. The type of steroids commonly prescribed for Duchenne are called corticosteroids. Corticosteroids are a type of drug similar to natural hormones produced by the adrenal glands that reduce inflammation and suppress the immune response. They are often prescribed to boys with Duchenne. These steroids may have an effect on stabilizing or even improving muscle strength for a period of time but not all boys respond to treatment. The main steroid that is used is called prednisone. Deflazacort is also used in some countries. These are not “anabolic steroids” which is what athletes use illegally to build up muscle – these do not have an effect in Duchenne. Sometimes they are referred to as ‘glucocorticoids’.

We will compare three different treatment groups:

  • Daily prednisone
  • Daily deflazacort
  • Intermittent prednisone (10 days on / 10 days off).

The study is randomised (your boy’s treatment group will be decided randomly, as in drawing names from a hat or tossing a coin) and double-blind which means that neither participants nor their doctors will know which group the boy is in (until the study is completed).

All three steroid treatments are commonly used in boys with Duchenne and have been shown to be beneficial. Benefits include an increase in the length of time that the boys can continue to walk, reduction in the development of curvature of the spine, a longer time of adequate breathing, and possibly protection against the development of heart problems.

However, we do not yet know which steroid treatment has the most benefit and most tolerable side effects. Therefore, this is a trial of present day steroid use which is needed because the practice of prescribing steroids varies a lot across doctors. This means that patients may not be getting the best possible treatment and management of side effects. All boys in this study will be receiving treatment with steroids and will be managed as per the recognized standards of care.

Analyses of the baseline data are currently underway and information will be shared on ClinicalTrials.gov (NCT01603407), as it becomes available.

Who was eligible to be in this study?

This study was open to boys with a confirmed diagnosis of Duchenne by genetic test, ages 3 – 7 years old and NOT previously treated with steroids except by inhaler or as an ointment.

Where does this study take place?

This study is taking place in numerous muscle clinics in the US, Canada, UK, Germany and Italy. Please visit www.clinicaltrials.gov for a complete list of all study sites. The principal investigators are Dr. R Griggs at the University of Rochester, NY and Prof. K Bushby at Newcastle University, UK.

What are participants doing during the study?

After the screening period, participants will visit the study site at 3 months and then every 6 months after that. There will be a total of around 8-13 visits depending on when enrollment begins. At each visit boys will be assessed to monitor benefits and side effects of corticosteroids. We expect that most boys will be in the study for 3-5 years.

Who is funding this study?

This study is funded by the US National Institutes of Health (NINDS). Parent Project Muscular Dystrophy and the MDA have also provided funding for this study. Funding to complete the study has also been provided by PTC Therapeutics AND SAREPTA.

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