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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.




Title: Comparative Study of Clinical Endpoint in DMD: HHM vs. CQMS (CNMC0609)

Summary: The study compared two commonly used pediatric strength testing measures: handheld myometry (HHM) and CINRG Quantitative Measurement System (CQMS), with the goal of identifying a sensitive and valid tool for measuring muscle strength in children with DMD. The data obtained from this study will be used to make recommendations for strength measurement endpoints in prospective muscular dystrophy trials and provide more reliable and accurate recommendations in the clinic for strength assessment. Thirty-three participants, between the ages of 5 and 18, with a diagnosis of Duchenne Muscular Dystrophy (DMD) were enrolled in the study. Participants were able to transfer to and from a wheel-chair with assistance and had not had any surgeries or muscle injuries in the previous 8 weeks.

Results: To be released concurrently with publication of manuscript.

Manuscript: In Process

Sponsorship: Muscular Dystrophy Association (MDA) Crystal Ball Fund


Title: Evaluation of Limb Girdle Muscular Dystrophy

Study Details: The purpose of this study was to understand the biochemistry of different types of LGMD and collect information about differences in the pathophysiology (causes) of different muscular dystrophies and the effects of the dystrophy process on your quality of life. The information collected aids in our understanding of the natural progression of the disease and can guide future therapeutic trials. A total of 52 participants were enrolled; 11 participants with each of the following diagnoses were enrolled: Limb girdle muscular dystrophy, type 2a (LGMD2A), Limb girdle muscular dystrophy, type 2a (LGMD2B), Limb girdle muscular dystrophy, type 2a (LGMD2I), Becker muscular dystrophy (BMD), and 11 controls (no neuromuscular disorder)

Results: To be released concurrently with publication of manuscript.

Manuscript: In Process

Sponsorship: Muscular Dystrophy Association (MDA) Crystal Ball Fund and Parsons family, and the Department of Health and Human Services (NIH)


Title: Cardiac Outcome Measures in Children with Muscular Dystrophy (PITT1109)

Study Details: The purpose of this study was to gain a better understanding of cardiac measurements in children with muscular dystrophy. This study included 48 participants aged 8 to 18 years old with Duchenne, Becker, or autosomal recessive limb-girdle (specifically: LGMD 2C-2F and 2I) muscular dystrophies. Participants were seen in one of five Cooperative International Neuromuscular Research Group (CINRG) centers located in the United States. All study assessments were completed in the same day and included reviewing past medical and surgical history, collecting vital signs, collecting cardiac measures through echocardiographic tests.

Results: Forty-eight participants (mean age, 13.3 ± 2.7 years) were enrolled. Shortening fraction had a greater interobserver correlation (intraclass correlation coefficient [ICC] = 0.63) compared with ejection fraction (ICC = 0.49). One reader could measure ejection fraction in only 53% of participants. Myocardial performance index measured by pulse-wave Doppler and Doppler tissue imaging showed similar ICCs (0.55 and 0.54). Speckle-tracking echocardiography showed a high ICC (0.96). Focusing on participants with DMD (n = 33), significantly increased mitral A-wave velocities, lower E/A ratios, and lower Doppler tissue imaging mitral lateral E' velocities were observed compared with age-matched control subjects. Speckle-tracking echocardiography demonstrated subclinical myocardial dysfunction with decreased average circumferential and longitudinal strain in three distinct subgroups: participants with DMD with normal shortening fractions, participants with DMD aged < 13 years, and participants with DMD with myocardial performance index scores < 0.40 compared with control subjects.

Manuscript: Journal of The American Society of Echocardiography (2015 Apr 21)

http://dx.doi.org/10.1016/j.echo.2015.03.003

Publication title: Feasibility and Reproducibility of Echocardiographic Measures in Children with Muscular Dystrophies

Sponsorship: NIH (CTSA program)


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: Equivalence was met for weekend and daily dosing of prednisone for the primary outcomes of quantitative muscle testing (QMT) arm score and QMT leg score. Secondary strength scores for QMT elbow flexors also showed equivalence between the 2 treatment groups. Overall side effect profiles of height and weight, bone density, cataract formation, blood pressure, and behavior, analyzed at 12 months, did not differ between weekend and daily dosing of prednisone.

Manuscript: Neurology (2011, Aug 77:444-452).

http://dx.doi.org/10.1212%2FWNL.0b013e318227b164

Publication title: Randomized, blinded trial of weekend versus daily prednisone in Duchenne muscular dystrophy

Sponsorship: Muscular Dystrophy Association (MDA)


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: A total of 64 boys with DMD with a mean age of 9.9 ± 2.9 years were randomly assigned to PTX or placebo in 11 participating Cooperative International Neuromuscular Research Group centers. There was no significant difference between PTX and the placebo group in total QMT scores (p = 0.14) or in most of the secondary outcomes after a 12-month treatment. The use of PTX was associated with mild to moderate gastrointestinal or hematologic AEs.

Manuscript: Neurology (2012, Mar 78:904-913)

http://dx.doi.org/10.1212/WNL.0b013e31824c46be

Publication title: Pentoxifylline as a rescue treatment for Duchenne muscular dystrophy: A randomized, double blind clinical trial

Sponsorship: Foundation to Eradicate Duchenne (FED)


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: Seventeen boys with DMD, between 4 and 8 years of age, were enrolled at one of five Cooperative International Neuromuscular Research Group (CINRG) centers. Only 9 were able to complete the 12-month PTX treatment phase; the primary reason for discontinuation was adverse events. Intolerable gastrointestinal side effects were experienced by 65% of participants. Two participants had severe leukopenia that resolved with medication withdrawal.

Manuscript: Muscle Nerve (2011 Aug 44(2):170-3)

http://dx.doi.org/10.1002/mus.22127

Publication Title: Liquid formulation of pentoxifylline is a poorly tolerated treatment for Duchenne dystrophy

Sponsorship: Muscular Dystrophy Association (MDA)


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)


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: Muscle and Nerve (2011, 44:174-178)

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

Sponsorship: Muscular Dystrophy Association (MDA)


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)

Last Updated: June 22, 2015

 

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