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
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Condition or disease | Intervention/treatment | Phase |
---|---|---|
Duchenne Muscular Dystrophy | Drug: Prednisone | Phase 3 |
Study Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 64 participants |
Allocation: | Randomized |
Intervention Model: | Parallel Assignment |
Masking: | Triple (Participant, Care Provider, Investigator) |
Primary Purpose: | Treatment |
Official Title: | A Randomized Study of Daily vs. High-dose Weekly Prednisone Therapy in Duchenne Muscular Dystrophy |
Study Start Date : | January 2004 |
Actual Primary Completion Date : | December 2007 |
Actual Study Completion Date : | February 2008 |

Arm | Intervention/treatment |
---|---|
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. |
- 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 ]
- 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 ]

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Ages Eligible for Study: | 4 Years to 10 Years (Child) |
Sexes Eligible for Study: | Male |
Accepts Healthy Volunteers: | No |
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

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00110669
United States, District of Columbia | |
Children's National Medical Center | |
Washington, District of Columbia, United States, 20010 |
Study Chair: | Diana Escolar, MD | Children's National Research Institute |
Responsible Party: | Study Chair, CINRG |
ClinicalTrials.gov Identifier: | NCT00110669 |
Other Study ID Numbers: |
CNMC0601 |
First Posted: | May 13, 2005 Key Record Dates |
Last Update Posted: | October 27, 2011 |
Last Verified: | October 2011 |
Muscular dystrophy, Duchenne and Beckers Beckers Muscular dystrophy |
Muscular Dystrophies Muscular Dystrophy, Duchenne Muscular Disorders, Atrophic Muscular Diseases Musculoskeletal Diseases Neuromuscular Diseases Nervous System Diseases Genetic Diseases, Inborn Genetic Diseases, X-Linked |
Prednisone Anti-Inflammatory Agents Glucocorticoids Hormones Hormones, Hormone Substitutes, and Hormone Antagonists Physiological Effects of Drugs Antineoplastic Agents, Hormonal Antineoplastic Agents |