High-dose Prednisone in Duchenne Muscular Dystrophy

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

Tracking Information

• First Submitted Date: May 12, 2005
• First Posted Date: May 13, 2005
• Last Update Posted Date: October 27, 2011
• Study Start Date: January 2004
• Actual Primary Completion Date: December 2007 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: January 2, 2008)

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

Original Primary Outcome Measures (submitted: June 23, 2005)

• Quantitative muscle strength will be measured using the Pediatric Quantitative Measurement System (PQMS)
• Primary strength endpoints will be quantitative myometry (QMT) scores of the upper and lower extremities, consisting of paired flexor/extensor groups.

Current Secondary Outcome Measures (submitted: January 2, 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 ]
• 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 ]

• Original Secondary Outcome Measures (submitted: June 23, 2005): 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.
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: High-dose Prednisone in Duchenne Muscular Dystrophy
• Official Title: A Randomized Study of Daily vs. High-dose Weekly Prednisone Therapy in Duchenne Muscular Dystrophy
• 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.
• 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 Type: Interventional
• Study Phase: Phase 3
• Study Design: Allocation: Randomized; Intervention Model: Parallel Assignment; Masking: Triple (Participant, Care Provider, Investigator); Primary Purpose: Treatment
• Condition: Duchenne Muscular Dystrophy

Intervention

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.

Study Arms

• 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)

  Intervention: Drug: Prednisone
• 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

  Intervention: Drug: Prednisone

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Completed
• Actual Enrollment (submitted: January 2, 2008): 64
• Original Enrollment (submitted: June 23, 2005): 140
• Actual Study Completion Date: February 2008
• Actual Primary Completion Date: December 2007 (Final data collection date for primary outcome measure)

Eligibility 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

Sex/Gender

• Sexes Eligible for Study: Male

• Ages: 4 Years to 10 Years (Child)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: United States
• Removed Location Countries: India

Administrative Information

• NCT Number: NCT00110669
• Other Study ID Numbers: CNMC0601
• Has Data Monitoring Committee: Yes
• U.S. FDA-regulated Product: Not Provided
• IPD Sharing Statement: Not Provided
• Current Responsible Party: Study Chair, CINRG
• Original Responsible Party: Not Provided
• Current Study Sponsor: Cooperative International Neuromuscular Research Group
• Original Study Sponsor: Same as current
• Collaborators: Not Provided

Investigators

• Study Chair: Diana Escolar, MD; Children's National Research Institute

• PRS Account: Cooperative International Neuromuscular Research Group
• Verification Date: October 2011