Duchenne muscular dystrophy: double-blind randomized trial to find optimum steroid regimen - FOR-DMD

niversity of Rochester0 sites300 target enrollmentSeptember 20, 2012

ConditionsDuchenne muscular dystrophy (DMD)MedDRA version: 14.1Level: PTClassification code 10013801Term: Duchenne muscular dystrophySystem Organ Class: 10010331 - Congenital, familial and genetic disorders Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]

DrugsDeflazacort Prednisone

Overview

Phase: Not Applicable
Intervention: Not specified
Conditions: Duchenne muscular dystrophy (DMD)
Sponsor: niversity of Rochester
Enrollment: 300
Status: Active, not recruiting
Last Updated: 13 years ago

Overview Investigators Eligibility Criteria Outcomes Similar Trials

Overview

Brief Summary

No summary available.

Registry

who.int

Start Date

September 20, 2012

End Date

TBD

Last Updated

13 years ago

Study Type

Interventional clinical trial of medicinal product

Sex

Male

Investigators

Sponsor

niversity of Rochester

Eligibility Criteria

Inclusion Criteria

•1\) Evidence of signed and dated informed consent form indicating that the subject and his parents or guardian (according to local legislation) have been informed about all pertinent aspects of the study. The child might be asked to give his assent, possibly in writing, if considered intellectually capable, in line with the legal requirements in the participating countries and with the permission of the parent(s)/guardian(s). 2\) Confirmed diagnosis of Duchenne muscular dystrophy defined as: Male with proximal weakness AND a confirmed DMD mutation in the dystrophin gene (out of frame deletion OR point mutation OR duplication) OR absent/\< 3% dystrophin on muscle biopsy (by immunohistochemistry or Western blot). 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, 6MWT, 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 judgment as to whether or not the boy can produce, and will likely be able to continue to produce, a reliable FVC measurement.
•Are the trial subjects under 18? yes
•Number of subjects for this age range: 0
•F.1\.2 Adults (18\-64 years) no
•F.1\.2\.1 Number of subjects for this age range 0
•F.1\.3 Elderly (\>\=65 years) no
•F.1\.3\.1 Number of subjects for this age range 0

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 capsules, as assessed by the site investigator by the end of the screening period. 9\) Allergy/sensitivity to study drugs or their 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 judgment 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.