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High-dose Prednisone in Duchenne Muscular Dystrophy

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ClinicalTrials.gov Identifier: NCT00110669
Recruitment Status : Completed
First Posted : May 13, 2005
Last Update Posted : October 27, 2011
Sponsor:
Information provided by:
Cooperative International Neuromuscular Research Group

Study Description
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Brief Summary:
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 months.

Condition or disease Intervention/treatment Phase
Duchenne Muscular Dystrophy Drug: Prednisone Phase 3

Detailed Description:
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.
Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 64 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Triple (Participant, Care Provider, Investigator)
Primary Purpose: Treatment
Official Title: A Randomized Study of Daily vs. High-dose Weekly Prednisone Therapy in Duchenne Muscular Dystrophy
Study Start Date : January 2004
Actual Primary Completion Date : December 2007
Actual Study Completion Date : February 2008

Resource links provided by the National Library of Medicine

Drug Information available for: Prednisone

Arms and Interventions
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Arm Intervention/treatment
Active Comparator: High Dose Prednisone

Subjects who are randomized to the high-dose prednisone arm of the study will receive the following starting dose:

•Prednisone at 10.0 mg/kg/wk (divided into two doses given on Saturday and Sunday)

 Drug: Prednisone
Prednisone and dummy preparations for this study will be obtained from Frank's Pharmacy in Ocala, FL and will be supplied as a tablet containing 2.5mg, 5mg, 10mg, 20mg or 50mg Prednisone. Inactive "dummy" pills of similar look/taste will be supplied to maintain blinding.
Active Comparator: Daily Prednisone

Subjects who are randomized to the daily prednisone arm of the study will receive the following starting dose:

•Prednisone at 0.75 mg/kg/d

 Drug: Prednisone
Prednisone and dummy preparations for this study will be obtained from Frank's Pharmacy in Ocala, FL and will be supplied as a tablet containing 2.5mg, 5mg, 10mg, 20mg or 50mg Prednisone. Inactive "dummy" pills of similar look/taste will be supplied to maintain blinding.


Outcome Measures
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Primary Outcome Measures :
  1. Quantitative muscle strength will be measured using the CINRG Quantitative Measurement System (CQMS) [ Time Frame: February 2008 ]
  2. 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 ]

Secondary Outcome Measures :
  1. 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 ]
  2. 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 ]

Eligibility Criteria
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Ages Eligible for Study:   4 Years to 10 Years   (Child)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
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
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00110669


Locations
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United States, District of Columbia
Children's National Medical Center
Washington, District of Columbia, United States, 20010
Sponsors and Collaborators
Cooperative International Neuromuscular Research Group
Investigators
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Study Chair: Diana Escolar, MD Children's National Research Institute
More Information
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Additional Information:

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Responsible Party: Study Chair, CINRG
ClinicalTrials.gov Identifier: NCT00110669    
Other Study ID Numbers: CNMC0601
First Posted: May 13, 2005    Key Record Dates
Last Update Posted: October 27, 2011
Last Verified: October 2011
Keywords provided by Cooperative International Neuromuscular Research Group:
Muscular dystrophy, Duchenne and Beckers
Beckers Muscular dystrophy
Additional relevant MeSH terms:
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Muscular Dystrophies
Muscular Dystrophy, Duchenne
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Genetic Diseases, X-Linked
 Prednisone
Anti-Inflammatory Agents
Glucocorticoids
Hormones
Hormones, Hormone Substitutes, and Hormone Antagonists
Physiological Effects of Drugs
Antineoplastic Agents, Hormonal
Antineoplastic Agents