High-dose Prednisone in Duchenne Muscular Dystrophy

• Register: ClinicalTrials.gov
• Last refreshed on: 19 February 2015
• Main ID: NCT00110669
• Date of registration: 12/05/2005
• Prospective Registration: No
• Primary sponsor: Cooperative International Neuromuscular Research Group
• Public title: High-dose Prednisone in Duchenne Muscular Dystrophy
• Scientific title: A Randomized Study of Daily vs. High-dose Weekly Prednisone Therapy in Duchenne Muscular Dystrophy
• Date of first enrolment: January 2004
• Target sample size: 64
• Recruitment status: Completed
• URL: http://clinicaltrials.gov/show/NCT00110669
• Study type: Interventional
• Study design: Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Caregiver, Investigator), Primary Purpose: Treatment
• Phase: Phase 3

Countries of recruitment

India, United States

Contacts

• Name: Diana Escolar, MD
• Affiliation: Children's Research Institute

Key inclusion & exclusion criteria

Inclusion Criteria:

- 4 to 10 years of age

- Ambulant

- Confirmed DMD Diagnosis

- Steroid naive

- Evidence of muscle weakness by MRC score or clinical functional evaluation

- Ability to provide reproducible QMT bicep score

Exclusion Criteria:

- History of significant concomitant illness or significant impairment of renal or
hepatic function, or other contraindication to steroid therapy

- Symptomatic DMD carrier

- Positive PPD

- Lack of prior exposure to chickenpox or immunization

- Use of carnitine, glutamine, Coenzyme Q10, other amino acids or any herbal
medications within the last 3 months

- History of symptomatic cardiomyopathy

- Prior attainment of quota for the age group in which the patient belongs



Age minimum: 4 Years
Age maximum: 10 Years
Gender: Male

Health Condition(s) or Problem(s) studied

Duchenne Muscular Dystrophy

Intervention(s)

Drug: Prednisone

Primary Outcome(s)

Primary strength endpoints will be quantitative myometry (QMT) scores of the upper and lower extremities, consisting of paired flexor/extensor groups. [Time Frame: February 2008]

Quantitative muscle strength will be measured using the CINRG Quantitative Measurement System (CQMS) [Time Frame: February 2008]

Secondary Outcome(s)

Side-effect profiles will assessed by monitoring side-effects, including differences in growth (height and weight), calculated weight/height ratio, bone density, cataract formation, blood glucose, blood pressure and behavioral changes. [Time Frame: February 2008]

Secondary strength endpoints will include individual QMT scores of elbow and knee flexors and extensors and hand grip, manual muscle testing scores, which will be measured using the Medical Research Council's (MRC) muscle strength scoring method. [Time Frame: February 2008]

Secondary ID(s)

CNMC0601

Source(s) of Monetary Support

Please refer to primary and secondary sponsors