Active Trials | Completed Trials | Postponed Trials | Discontinued Trials
To search by disease name, select the disease from the list below: Acid Maltase Deficiency (Pompe's Disease) Amyotrophic Lateral Sclerosis Becker Muscular Dystrophy Carnitine Deficiency Carnitine Palmityl Transferase Deficiency Central Core Disease Charcot-Marie-Tooth Disease Congenital Muscular Dystrophy Congenital Myasthenic Syndrome Congenital Myotonic Muscular Dystrophy Debrancher Enzyme Deficiency Dejerine-Sottas Disease Dermatomyositis Distal Muscular Dystrophy Duchenne Muscular Dystrophy Emery-Dreifuss Muscular Dystrophy Facioscapulohumeral Muscular Dystrophy Friedreich's Ataxia General Hyperthyroid Myopathy Hypothyroid Myopathy Inclusion-Body Myositis Juvenile Dermatomyositis Lactate Dehydrogenase Deficiency Lambert-Eaton Syndrome Leigh's Syndrome Limb-Girdle Muscular Dystrophy Mitochondrial Myopathy - CPEO Mitochondrial Myopathy - KSS Mitochondrial Myopathy - MELAS Mitochondrial Myopathy - MERRF Mitochondrial Myopathy - MILS Mitochondrial Myopathy - MNGIE Mitochondrial Myopathy - NARP Mitochondrial Myopathy - PEO Mitochondrial Myopathy General Myasthenia Gravis Myoadenylate Deaminase Deficiency Myotonia Congenita Myotonic Muscular Dystrophy Myotubular Myopathy Nemaline Myopathy Non-MDA Diseases Oculopharyngeal Muscular Dystrophy Paramyotonia Congenita Pearson Syndrome Periodic Paralysis Phosphofructokinase Deficiency Phosphoglycerate Kinase Deficiency Phosphoglycerate Mutase Deficiency Phosphorylase Deficiency Polymyositis Spinal Bulbar Muscular Atrophy Spinal Muscular Atrophy Type 1 Spinal Muscular Atrophy Type 2 Spinal Muscular Atrophy Type 3 Or Search by **: Trial Title Trial Location/City Trial Institution
Clinical trials are experiments conducted using human participants that seek to determine the value of a potential treatment, such as a drug, dietary supplement or exercise program. Most trials compare a treated group of participants with a "control," or comparison, group, which receives an inert substance ("placebo") or a sham treatment.
Many trials are divided into phases, the first of which is usually quite small and almost always designed only to assess the safety of the new treatment and how well it's tolerated. Phases 2 and 3 of a trial are larger and address questions of effectiveness and dose.
The term "study" implies either a trial or a more general research question under investigation, such as how many people with a certain set of symptoms have a particular genetic change or how parents cope with raising a child with a disability.
To learn more about the purposes and risks of clinical trials, please go to www.clinicaltrials.gov. This site, sponsored by the National Institutes of Health, contains general information and listings of specific trials.
For specific information about children and clinical trials, see www.ChildrenAndClinicalStudies.nhlbi.nih.gov.
See also "Being a Co-Adventurer: Clinical trials Involve Risks, Rights, Responsibilities" in the May-June 2008 issue of Quest, MDA's bimonthly magazine.
The full names of the diseases in MDA's program and more information about each disease can be found at http://www.mda.org/disease/.