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Trial record 6 of 93 for:    "Duchenne muscular dystrophy"
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High-dose Prednisone in Duchenne Muscular Dystrophy

This study has been completed.
Sponsor:
Information provided by:
Cooperative International Neuromuscular Research Group
ClinicalTrials.gov Identifier:
NCT00110669
First received: May 12, 2005
Last updated: October 26, 2011
Last verified: October 2011
History of Changes
  Purpose

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 Intervention Phase
Duchenne Muscular Dystrophy
 Drug: Prednisone
 Phase 3

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
Official Title: A Randomized Study of Daily vs. High-dose Weekly Prednisone Therapy in Duchenne Muscular Dystrophy

Resource links provided by NLM:

Drug Information available for: Prednisone
U.S. FDA Resources

Further study details as provided by Cooperative International Neuromuscular Research Group:

Primary Outcome Measures:
  • Quantitative muscle strength will be measured using the CINRG Quantitative Measurement System (CQMS) [ Time Frame: February 2008 ] [ Designated as safety issue: No ]
  • 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 ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • 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 ] [ Designated as safety issue: No ]
  • 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 ] [ Designated as safety issue: Yes ]

Enrollment: 64
Study Start Date: January 2004
Study Completion Date: February 2008
Primary Completion Date: December 2007 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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.

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.

  Eligibility

Ages Eligible for Study:   4 Years to 10 Years
Genders 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
Please refer to this study by its ClinicalTrials.gov identifier: NCT00110669

Locations
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
Study Chair: Diana Escolar, MD Children's Research Institute
  More Information

Additional Information:
CINRG public website  This link exits the ClinicalTrials.gov site

No publications provided

Responsible Party: Study Chair, CINRG
ClinicalTrials.gov Identifier: NCT00110669     History of Changes
Other Study ID Numbers: CNMC0601
Study First Received: May 12, 2005
Last Updated: October 26, 2011
Health Authority: United States: Institutional Review Board

Keywords provided by Cooperative International Neuromuscular Research Group:
Muscular dystrophy, Duchenne and Beckers
Beckers Muscular dystrophy

Additional relevant MeSH terms:
Muscular Dystrophy, Duchenne
Muscular Dystrophies
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, X-Linked
Genetic Diseases, Inborn
Prednisone
 Glucocorticoids
Hormones
Hormones, Hormone Substitutes, and Hormone Antagonists
Physiological Effects of Drugs
Pharmacologic Actions
Antineoplastic Agents, Hormonal
Antineoplastic Agents
Therapeutic Uses
Anti-Inflammatory Agents

ClinicalTrials.gov processed this record on May 23, 2013