High-dose Prednisone in Duchenne Muscular Dystrophy - Full Text View

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:

Genetics Home Reference related topics: Duchenne and Becker muscular dystrophy

MedlinePlus related topics: Muscular Dystrophy Steroids

Drug Information available for: Prednisone

Genetic and Rare Diseases Information Center resources: Becker Muscular Dystrophy Duchenne Muscular Dystrophy Muscular Dystrophy

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

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

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

ClinicalTrials.gov processed this record on February 27, 2015