Completed CINRG Research Studies

How do I find results from completed CINRG research studies?

Below is a brief description of the research studies that CINRG has completed. Related journal publications can be found here. Please feel free to email CINRG at info@cinrgresearch.org and request an article or information on a completed CINRG study.

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Title: Prednisone High Dose vs. Daily in Duchenne Muscular Dystrophy (CNMC0601)

Summary: This study, conducted at 12 CINRG centers, was designed to determine whether a high-dose weekly course (10 mg/kg over two days) of prednisone is safer than and/or as effective as daily dose therapy (0.75 mg/kg/day). A total of 64 participants were enrolled.

Results: To be released concurrently with publication of manuscript.

Manuscript: In Process

Sponsorship: Muscular Dystrophy Association (MDA)

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Title: A Double-Blinded Randomized Placebo Controlled Study of Daily Pentoxifylline as a Rescue Treatment in Duchenne Muscular Dystrophy (CNMC 0705)

Summary: In this study, Pentoxifylline was added as a rescue treatment to patients who were receiving steroids (prednisone, prednisolone or deflazacort) for at least 12 months in a stable dose regardless of weight change. A total of 64 participants were enrolled.

Results: To be released concurrently with publication of manuscript.

Manuscript: In Process

Sponsorship: Foundation to Eradicate Duchenne (FED)

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Title: An Open-Label Pilot Study of Pentoxifylline in Steroid-Naïve Duchenne Muscular Dystrophy (CNMC0302)

Summary: This open label pilot study of oral, immediate release PTX assessed the tolerability and safety of PTX and quantitative muscle strength (QMT) in young DMD boys over twelve months of treatment. We designed the study to identify any potential effects on quantitative muscle strength that could provide us with an effect size to power a future randomized controlled trial.

Results: To be released concurrently with publication of manuscript.

Manuscript: In Process

Sponsorship: Muscular Dystrophy Association (MDA)

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Title: A Multicenter Randomized Placebo-Controlled Double-Blind Study to Assess the Efficacy and Safety of Glutamine and Creatine Monohydrate in Duchenne Muscular Dystrophy (CNMC0599)

Summary: The efficacy and safety of glutamine (0.6gm/kg/day) and creatine (5gm/day) was tested in 50 ambulant boys with Duchenne muscular dystrophy in a 6-month, double blind, placebo-controlled clinical trial. Drug efficacy was tested by measuring muscle strength manually (34 muscle groups) and quantitatively (10 muscle groups). Timed functional tests, functional parameters, and pulmonary function tests were secondary outcome measures.

Results: Although there was no statistically significant effect of either therapy based on manual and quantitative measurements of muscle strength, a disease-modifying effect of creatine in older Duchenne muscular dystrophy and creatine and glutamine in younger Duchenne muscular dystrophy cannot be excluded. Creatine and glutamine were well tolerated.

Manuscript: Annals of Neurology (Ann Neurol 2005; 58: 151-155)

http://dx.doi.org/10.1002/ana.20523

Publication title: CINRG Randomized Controlled Trial of Creatine and Glutamine in Duchenne Muscular Dystrophy

Sponsorship: Muscular Dystrophy Association (MDA)

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Title: An Open-Label Pilot Study of Coenzyme Q10 in Steroid-Treated Duchenne Muscular Dystrophy (CNMC0301)

Summary: Corticosteroid treatment is the standard of care for Duchenne muscular dystrophy, thus clinical trials may require an "add on" design. We report an open label pilot trial of the effects of oral CoQ10 in 16 steroid treated Duchenne muscular dystrophy patients (5-11 years). Target serum levels (2.5 µg/ml) were shown to be subject and administration dependent.

Results: CoQ10 treatment for 6 months resulted in 7.5 ±3.7 % increase in muscle strength (QMT score as primary outcome) and was well tolerated. This result encourages further testing of oral CoQ10 in DMD. The effect size found in this study can be used to power a randomized controlled trial.

Manuscript: In Process

Publication title: CINRG Pilot trial of Coenzyme Q10 in steroid treated Duchenne Muscular Dystrophy

Sponsorship: Muscular Dystrophy Association (MDA)

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Title: An Open-Label Pilot Study of Oxatomide in Steroid-Naive Duchenne Muscular Dystrophy (KUL0401)

Summary: This study was a pilot, open-label two-center therapeutic trial of oxatomide in 14 ambulant, steroid-naive DMD boys aged 5-10 years. Boys received 6 months of therapy after a 6 month medication-free lead-in period and were evaluated at monthly intervals.

Results: Comparison of linear evolutions between 3 months medication-free lead-in periods and 6 months treatment periods showed no significant differences in quantitative (QMT) and manual (MMT) measurements of muscle strength and timed functional tests. A modest mitigation of strength deterioration over time cannot be excluded.

Manuscript: European Journal of Paediatric Neurology

Publication title: CINRG pilot trial of oxatomide in steroid-naive Duchenne muscular dystrophy

http://dx.doi.org/10.1016/j.ejpn.2007.02.009

Sponsorship: Muscular Dystrophy Association (MDA), Association Francaise contre les Myopathies (AFM)