Facioscapulohumeral Muscular Dystrophy
Conditions
Keywords
FSHD, Antioxidants, Oxidative stress, Muscle function, Quality of life, Activity, (FSHD;4q35)
Brief summary
On the basis of published data and the investigators' results indicating that oxidative stress may contribute to the peripheral skeletal muscle dysfunction in patients with FSHD, the investigators propose a study to test whether or not an antioxidant supplementation has a therapeutic interest for patients with FSHD. Their results have important implications for the successful implementation of rational antioxidant therapy in FSHD in which cell loss could be linked to oxidative stress.
Detailed description
This study will compare the effect of the therapeutic interest of an antioxidant supplementation on the functional deficits and the molecular muscle abnormalities into two groups of patients affected by FSHD, one treated with the antioxidant supplementation during 17 weeks. The antioxidant by capsule consisted of:Vitamin E (400 mg /day), Selenium (200 µg/day in the form of selenomethionine), Vitamin C (500 mg/day), Zinc (25 mg/day in the form of gluconate). The second one treated with a placebo during 17 weeks. Patients will be assigned to intervention groups by chance, and neither physician, nor patient, will know which product is administrated (study in double blind).
Interventions
PROCEDURETaking of blood
Taking venous blood samples to analyse oxidant stress
DIETARY_SUPPLEMENTneedle biopsy of the vastus lateralis muscle
T0 needle biopsy of the vastus lateralis muscle (analysis of oxidative stress and inflammatory markers)During 17 weeks supplementation by Vit E, C , Zn Se After 17 weeks: veinous blood samples and needle biopsy
DIETARY_SUPPLEMENTVit C Vit E Zn Se
T0 venous blood samples and needle biopsy of the vastus lateralis muscle During 17 weeks vit E 400mg/d, Se 200µg/d, Vit C 500mg/day, Zn 25 mg/d After the supplementation of 17 weeks:venous blood samples and needle biopsy of the vastus lateralis muscle
DIETARY_SUPPLEMENTPlacebo Vit E Placebo Vit C Zn Se
venous blood samples and needle biopsy
Sponsors
Hospital Clinical Research Project 2010
Association Amis FSH France
FSH Dutch Fondation The Netherland
University Hospital, Montpellier
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
SUPPORTIVE_CARE
Masking
DOUBLE (Subject, Investigator)
Eligibility
Sex/Gender
ALL
Age
18 Years to 60 Years
Healthy volunteers
No
Inclusion criteria
* FSHD patients will be recruited on the basis of: * The number of repeat units (4 to 9) * FSHD patients with a positive family history for FSHD * Not confined to a wheelchair * No smokers * No associated co-morbidity (cardiac or pulmonary disease, diabetes etc.) * No Medications or nutritional supplementation (vitamins and/or antioxidants) at the time of the study * No HIV positive
Exclusion criteria
* No consent form
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Improvement of muscle effort tolerance after antioxidant supplementation | duration study 3 years | 17-weeks evaluation of an antioxidant supplementation in order to modulate or delay oxidative insult that could be useful to maintain FSHD muscle function. At T0, each patient will perform functionnal evaluation (Exercise Tolerance (2 minutes walking test), and/or Maximal Volontary Contraction and/or Endurance of quadriceps muscles). |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Changes in inflammatory and oxidative stress parameters after antioxidant supplementation | duration study 3 years | T0 evaluations spirometry, electrocardiogram, holter,Magnetic Resonance imaging of the both thighs,estimation muscle oxygen consumption during acute exercise . T7 venous blood samples (oxidative stress inflammatory markers)needle biopsy of the vastus lateralis muscle (oxidative stress inflammatory markers). analysis oxidative stress on muscle biochemical analyses.T7 patient receives placebo or antioxidant supplementation corresponding randomly-assigned trial. After 17 weeks supplementation the subjects will obtain venous blood samples and needle biopsy. |
| Changes in muscular function after antioxidant supplementation | duration study 3 years | T0 evaluations spirometry, electrocardiogram, holter Magnetic Resonance imaging of the both thighs,estimation muscle oxygen consumption . T7 venous blood samples (oxidative stress inflammatory markers)needle biopsy of the vastus lateralis muscle (oxidative stress inflammatory markers). This one-week lap's time is needed to avoid potential confounding effects of exercise-induced oxidative stress on muscle biochemical analyses.T7 patient receives placebo or antioxidant supplementation corresponding randomly-assigned trial. After 17 weeks supplementation venous blood samples and needle biopsy. |
Countries
France
Outcome results
None listed