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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

Tracking Information
First Submitted Date  ICMJE May 12, 2005
First Posted Date  ICMJE May 13, 2005
Last Update Posted Date October 27, 2011
Study Start Date  ICMJE January 2004
Actual Primary Completion Date December 2007   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (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  ICMJE
 (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.
Change History Complete list of historical versions of study NCT00110669 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (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  ICMJE
 (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  ICMJE High-dose Prednisone in Duchenne Muscular Dystrophy
Official Title  ICMJE 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  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Triple (Participant, Care Provider, Investigator)
Primary Purpose: Treatment
Condition  ICMJE Duchenne Muscular Dystrophy
Intervention  ICMJE 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  ICMJE
  • 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

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: January 2, 2008)
64
Original Enrollment  ICMJE
 (submitted: June 23, 2005)
140
Actual Study Completion Date  ICMJE February 2008
Actual Primary Completion Date December 2007   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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  ICMJE
Sexes Eligible for Study: Male
Ages  ICMJE 4 Years to 10 Years   (Child)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries India
 
Administrative Information
NCT Number  ICMJE NCT00110669
Other Study ID Numbers  ICMJE CNMC0601
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Study Chair, CINRG
Study Sponsor  ICMJE Cooperative International Neuromuscular Research Group
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Chair: Diana Escolar, MD Children's Research Institute
PRS Account Cooperative International Neuromuscular Research Group
Verification Date October 2011

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP