The FOR DMD study is an international, multi-centre study which will compare the benefits and side effects of the three most widely prescribed steroid treatments in children with Duchenne muscular dystrophy (DMD).
The FOR DMD study will compare
- daily prednisone (0.75 mg/kg/day)
- intermittent prednisone (0.75 mg/kg/day, 10 days on, 10 days off)
- daily deflazacort (0.9 mg/kg/day).
All three dosages are commonly used in boys with Duchenne muscular dystrophy and have shown to be beneficial.
- Confirmed diagnosis of DMD (confirmed DMD mutation in the dystrophin gene (out of frame deletion or duplication or point mutation)
- Age > 4 years and < 8 years
- Ability to rise independently from floor, from supine to standing
- Willingness and ability to comply with scheduled visits, drug administration plan and study procedures (including laboratory tests, North Star Ambulatory Assessment, 6-Minute Walk Test, ECG, Echo, wrist X-ray, DEXA, PedsQL and Treatment Satisfaction Questionnaire for Medication) as assessed by the site investigator at the end of the screening period
- Ability to maintain reproducible FVC measurements as assessed by the site investigator at the end of the screening period
- Current or previous treatment (> 4 weeks) with oral corticosteroids
- Inability to take tablets (as assessed by the site investigator by the end of the screening period)
- Major renal or hepatic diseases, immunosuppression, diabetes mellitus, idiopathic hypercalcuria
- Lack of chicken pox immunity and refusal to undergo immunization
- Severe behavioural problems, including severe autism
- Symptomatic cardiomyopathy
- Allergy/sensitivity to corticosteroids, intolerance to lactose and/or sucrose
- Weight of less than 13 kilograms
- Previous (within 3 months) or current participation in another clinical trial with an investigational drug
Study design and duration:
300 DMD boys will take part in the FOR DMD study trial.
The boy will be randomly (like tossing a coin) assigned to one of the three treatment regimens (0.75 mg/kg/d prednisone; 0.75 mg/kg/d prednisone for 10 days alternating with 10 days off; or 0.9 mg/kg/d deflazacort). There is no placebo group in the FOR DMD study and all participants will receive one of the three active treatment.
The overall time that a child will stay in the study will be from a minimum of 3 years and a maximum of 5 years, depending on when he is enrolled within the study timeline. All boys will receive the study drug for a minimum of three years. Children who are enrolled and complete the three years of treatment first will continue the study drug until all participants finish the 36-month treatment period. Therefore each boy will be in the study a minimum of 36 months (three years) if he is recruited toward the end of the study and a maximum of 60 months (five years) if he is recruited very early in the course of the study.