High-Dose Prednisone in Duchenne Muscular Dystrophy
Information source: Cooperative International Neuromuscular Research Group
Information obtained from ClinicalTrials.gov on June 20, 2008
Link to the current ClinicalTrials.gov record.
Condition(s) targeted: Duchenne Muscular Dystrophy
Intervention: Prednisone (Drug)
Phase: Phase 3
Sponsored by: Cooperative International Neuromuscular Research Group
Official(s) and/or principal investigator(s):
Diana Escolar, MD, Study Chair, Affiliation: Childrens Research Institute
This study will help to determine whether a high-dose weekly course of prednisone therapy is
safer than and at least as effective as daily dose therapy for people with Duchenne muscular
dystrophy (DMD). Boys who are enrolled in this study should not have taken carnitine, other
amino acids, creatine, glutamine, Coenzyme Q10 or any herbal medicines within the last three
months. There will be a two-visit screening to take place in one week to ensure a
reproducible manual muscle test. The subject will then be randomized and put into either the
daily or weekly regimen. The duration of the study is twelve 28-day treatment cycles
(approximately 12 months) with follow-up visits at month one, three and then every three
Official title: A Randomized Study of Daily vs. High-Dose Weekly Prednisone Therapy in Duchenne Muscular Dystrophy
Study design: Treatment, Randomized, Double Blind (Subject, Caregiver, Investigator), Placebo Control, Parallel Assignment, Safety/Efficacy Study
Quantitative muscle strength will be measured using the CINRG Quantitative Measurement System (CQMS)
Primary strength endpoints will be quantitative myometry (QMT) scores of the upper and lower extremities, consisting of paired flexor/extensor groups.
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.
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.
Duchenne muscular dystrophy (DMD) is the most common lethal inherited disorder worldwide.
Despite the exponential increase in our understanding of the disorder since the discovery and
characterization of the causative gene and its product dystrophin in 1987, current
therapeutic management remains largely supportive. Awaiting a final genetic cure to be
available in the future, further investments in developing better drug therapies for DMD
remain important. The effect of a high dose prednisone regimen will be evaluated in
comparison to a daily dose regimen in a multi-center, randomized, double-blind
placebo-controlled 4-arm study. Ambulant children aged 4-10 years with an established DMD
diagnosis will be studied. Patients will undergo 2 screening evaluations within 1 week.
Patients will be randomized into treatment groups on the second screening visit, followed by
a 12-month treatment period. During the treatment period, patients will be evaluated at
monthly intervals. The primary endpoints are percentage change in average muscle strength
score and QMT performance for specific muscle groups. Secondary endpoints include timed
function tests, functional grades for arms and legs, and pulmonary function tests.
Minimum age: 4 Years.
Maximum age: 10 Years.
- 4 to 10 years of age
- Confirmed DMD Diagnosis
- Steroid naive
- Evidence of muscle weakness by MRC score or clinical functional evaluation
- Ability to provide reproducible QMT bicep score
- 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
Locations and Contacts
Sundaram Medical Foundation, Chennai, India
UC Davis, Sacramento, California, United States
Children's National Medical Center, Washington, District of Columbia 20010, United States
Children's Hospital of Pittsburgh, Pittsburgh, Pennsylvania, United States
University of Tennessee, Memphis, Tennessee, United States
CINRG public website
Starting date: January 2004
Ending date: February 2008
Last updated: June 9, 2008