FOR-DMD - Finding the Optimal steroid Regimen for Duchenne Muscular Dystrophy

 

Frequently Asked Questions (FAQs) about this study:


What stage is this research?

Recruitment ended in September, 2016. 

 

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 also 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 underway and information will be shared on ClinicalTrials.gov (NCT01603407), as it becomes available.

 

Who was eligible to be in this study?

• To be in this study your boy must have a confirmed diagnosis of Duchenne by genetic test. He must be between 3 and 7 years old and not previously treated with steroids except by inhaler or as an ointment.

 

Where will this study take place?

This study will take place in at least 36 muscle clinics in the US, Canada, UK, Germany and Italy and other countries may also be included later. Please visit 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 will happen 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 your boy is enrolled. At each visit your boy will be assessed to monitor benefits and side effects of corticosteroids. We hope that many boys will be able to find a muscle clinic that is participating in the study reasonably close to where they live and that the study visits will take the place of their routine follow up.  We expect participants will be in the study for 3-5 years.

 

Will I have access to the drug once the study has ended?

• Yes, your doctor will discuss treatment options at the end of the study to decide the best steroid treatment plan for your boy.

Will participating in this study prevent my boy from taking part in other clinical trials?

• We are aware that trials of other potential new therapies may start during the course of the study. As steroid treatment is part of the normal standard of care in Duchenne we do not believe that being in this study would prevent your boy from being in another study later if there were one that he were eligible for. For example, if your boy is/will be participating in Summit Pharmaceuticals' or Bristol-Myers Suibb's Duchenne trials, he may also be able to participate in FOR-DMD at the same time. Moreover, although we hope that the majority of boys will finish the whole trial, as with any clinical study, you are entitled to withdraw at any time if you no longer wish to participate.

 

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.

 

Where can I learn more about this study?

• You can learn more about this study at www.for-dmd.org and www.clinicaltrials.gov (NCT01603407). You can also contact the FOR-DMD US Project Manager, Kimberly Hart, at telephone 585-275-3767 or email This email address is being protected from spambots. You need JavaScript enabled to view it.

• You can view the PPMD webinar on the FOR-DMD study: https://www.youtube.com/watch?v=W7B24zpBrDw 

 

(Updated 4/28/2017)