Completed Clinical Trials

CNMC0599: A multicenter randomized placebo-controlled double-blind study to assess the efficacy and safety of glutamine and creatine monohydrate in Duchenne muscular dystrophy

Purpose: 1. To establish a collaborative group of clinical trial centers, with standardized equipment and protocols, able to conduct both drug and gene therapy trials in DMD. 2. To evaluate the therapeutic effect of glutamine and creatine monohydrate on muscle strength in children with DMD. 3. To validate the use of QMT (quantitative muscle strength testing) and gait analysis in children with DMD as reliable tools to quantify muscle strength, monitor disease progression and assess therapeutic response.

Further Information: The effect of glutamine (0.6 g/kg/day) and creatine monohydrate (5 g/day) on muscle strength will be evaluated in a multicenter randomized double-blind placebocontrolled 3-armed study. Ambulant children aged 5 to 10 years with an established DMD diagnosis will be studied. Patients will undergo 2 screening evaluations within 2 weeks. Patients will be randomized into treatment groups on the second screening visit, followed by a 6-month treatment period. During the treatment period, patients will be evaluated at monthly intervals.

*The results of this study have been published in the Annal of Neurology, please goto http://www.ncbi.nlm.nih.gov/entrez/query.fcgi?cmd=Retrieve
&db=pubmed&dopt=Abstract&list_uids=15984021&query_hl=8 for more information.*

Purpose: To evaluate the therapeutic effect of Coenzyme Q10 on muscle strength in children with DMD.

Further Information: The effect of Coenzyme Q10 on muscle strength, at a therapeutic level of 2.5 ug/ml or higher, will be evaluated in an open-label pilot study. Ambulant children aged 5 to 11 years with an established DMD diagnosis, on a stable steroids regimen, will be studied. Subjects will undergo one screening visit to ensure that they are able to participate in strength testing sessions. Successful subjects will be enrolled and placed on oral CoQ10 supplements, after which a dose escalation period will assure blood levels in the desired range. During the treatment period, patients will be evaluated at monthly intervals.

Purpose: To evaluate the therapeutic effect of the mast cell stabilizer oxatomide on muscle strength in children with DMD, through the CINRG network of clinical trial centers with standardized equipment/protocols and web-based data and safety review infrastructure.

Further Information: Oxatomide has been widely used in humans as an anti-histaminic (Tinset®, Janssen-Cilag N.V.). Its mechanism of action is twofold: oxatomide inhibits the release of mediators from mast cells, and it acts as an H1-antihistaminic.