FOR-DMD

Principal Investigator: Russell Butterfield
Keywords: Duchenne Muscular Dystrophy , prednisone , deflazacort , DMD Department: Pediatric Administration
IRB Number: 00063692 Co Investigator:  
Specialty: Neurology, Neurology, Neurology
Sub Specialties: Neuromuscular Diseases, Neuromuscular Pathology, Muscular Dystrophy
Recruitment Status: Recruiting

Contact Information

Becky  Crockett
bcrockett@genetics.utah.edu
801-585-1676

Brief Summary

This investigator-initiated Phase III trial compares three steroid regimens for boys with Duchenne muscular dystrophy in regard to functional outcome and subject/parent satisfaction.  The long-term objective of this trial is to identify the optimum steroid regimen for boys with Duchenne muscular dystrophy.

The primary objective is to compare three most commonly in use corticosteroid regimens in regard to functional outcome and subject/parent satisfaction.  The primary outcome variable is a multivariate (3-dimensional) measure, comprising two dimensions of function (time to rise from the floor, forced vital capacity) and on of satisfaction (global treatment satisfaction).  The null hypotheses states that the three corticosteroid regimens do not differ from one another with regard to any of the three dimensions of outcome while the alternative hypothesis states that they differ (in the same direction) with regard to at least one of the dimensions of outcome.  
 

The secondary objectives are to compare the three corticosteroid regimens with regard to:

  • Tolerability
  • Adverse Events
  • Secondary Functional Outcomes and Disease Milestones
  • Quality of Life
  • Cardiac Function

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

Inclusion Criteria

Inclusion Criteria:

1. Evidence of signed and dated informed consent form indicating that the subject and his parents or guardain have been informed about al pertinent aspects of the study.

2. Confirmed diagnosis of Duchenne muscular dystrophy defined as: male with clinical signs compatible with DMD AND confirmed DMD mutation in the dystrophin gene (out of frame deletion or duplication or point mutation).

3. Age > 4 years and < 8 years.

4. Ability to rise independently from floor, from supine to standing as assessed at screening visit.

5. Willingness and ability to comply with scheduled visits, drug administration plan and study procedures (including laboratory tests, NSAA, 6MST, ECG, Echo, wrist X-Ray, DXA, PedsQL and TSQM questionnaires) as assessed by the site investigator at the end of the screening period.

6. Ability to maintain reproducible FVC measurements.  Boys must have reproducible measurements of FVC.  The boy will be observed to insure complete understanding of the instructions and that he has given maximal effort.  If the values continue to increase, the boy may be learning and testing will continue.  If necessary, beyond the 3 required trials until the boy reaches a plateau.  The evaluator will use his/her expert judgement as to whether or not the boy can produce, and will likely be able to continue to produce, a reliable FVC measurement.

 

 

Exclusion Criteria

 

Exclusion Criteria:

1. History of major renal or hepatic impairment, immunosuppression or other contraindications to corticosteroid therapy.

2. History of chronic systemic fungal or viral infections.  Acute bacterial infection (including TB) would exclude from enrollment until the infection had been appropriately treated and resolved.

3. Diabetes mellitus.

4. Idiopathic hypercalcuria.

5. Lack of chicken pox immunity and refusal to undergo immunization.

6. Evidence of symptomatic cardiomyopathy at screening assessment.  Asymptomatic cardiac abnormality on investigation would not be an exclusion.

7. Current or previous treatment (greater than four consecutive weeks of oral therapy) with corticosteroids or other immunosuppressive treatments for DMD or other recurrent indications (e.g. asthma).

8. Inability to take tablets, as assessed by the site investigator by the end of the screening period.

9. Allergy/sensitivity to study drugs or other formulations including lactose and/or sucrose intolerance.

10. Severe behavioral problems, including severe autism.

11. Previous or ongoing medical condition, medical history, physical findings or laboratory abnormalities that could affect safety, make it unlikely that treatment and follow-up will be correctly completed or impair the assessment of study results, in the judgement of the site investigator.

12. Weight of less than 13 kilograms.

13. Exposure to any investigational drug currently or within 3 months prior to start of study treatment.