High-dose Prednisone in Duchenne Muscular Dystrophy

NCT00110669 | Completed Phase 3 DMC

• 1 other identifier: CNMC0601
• Study Type: interventional
• Target: 64
• Locations: 1 country
• Sites: 1

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.

Conditions

Duchenne Muscular Dystrophy

Keywords

Muscular dystrophy, Duchenne and Beckers; Beckers Muscular dystrophy

Outcome Measures

Primary Outcomes (2)

• Quantitative muscle strength will be measured using the CINRG Quantitative Measurement System (CQMS)

  February 2008

• Primary strength endpoints will be quantitative myometry (QMT) scores of the upper and lower extremities, consisting of paired flexor/extensor groups.

  February 2008

Secondary Outcomes (2)

• 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.

  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.

  February 2008

Study Arms (2)

High Dose Prednisone

ACTIVE COMPARATOR

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

Daily Prednisone

ACTIVE COMPARATOR

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

Interventions

Prednisone DRUG

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.

Daily Prednisone; High Dose Prednisone

Eligibility Criteria

• Age: 4 Years - 10 Years
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Cooperative International Neuromuscular Research Group: lead

Study Sites (1)

Children's National Medical Center

Washington D.C., District of Columbia, 20010, United States

Location

Related Links

• CINRG public website

MeSH Terms

Conditions

Muscular Dystrophy, Duchenne; Muscular Dystrophies

Interventions

Prednisone

Condition Hierarchy (Ancestors)

Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Pregnadienediols; Pregnadienes; Pregnanes; Steroids; Fused-Ring Compounds; Polycyclic Compounds

Study Officials

• Diana Escolar, MD

  Children's National Research Institute

  STUDY CHAIR

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: NETWORK

Study Record Dates

• First Submitted: May 12, 2005
• First Posted: May 13, 2005
• Study Start: January 1, 2004
• Primary Completion: December 1, 2007
• Study Completion: February 1, 2008
• Last Updated: October 27, 2011

Record last verified: 2011-10

Locations

US (1)