CINRG - Study Needs Volunteers
| 05 August 2009
Originally Posted: 03 June 2009
The Cooperative International Neuromuscular Research Group (CINRG) at the Children's Hospital in Washington, DC is actively recruiting patients for an important clinical research study. If you are 18 years or older with Becker or Limb-Girdle Muscular Dystrophy this study might be of special interest to you.
The purpose of this study is 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 will help us understand the natural progression of the disease and can guide future therapeutic trials.
The evaluation will take approximately six hours, and will involve the following tests:
Strength and functional muscle testing by a physical therapist,
Pulmonary function testing by a physical therapist,
Physical evaluation by a physician,
Complete cardiac evaluation with electrocardiogram (ECG or EKG) and echocardiogram (Echo) by a cardiologist,
Two blood draws, one before we start testing and one after you have finished all evaluations. Both will be performed by a trained nurse
You will be asked to complete questionnaires about quality of life, activity limitations, and your understanding about the etiology (cause of your muscle disorder), genetics, and inheritance of your particular form of LGMD.
In order to be eligible for this study, you must be over the age of 18 years and able to travel to Children’s National Medical Center. You must be diagnosed with one of the following forms of muscular dystrophy, and be willing to provide muscle biopsy or genetic testing confirming the diagnosis:
Becker muscular dystrophy
Limb-girdle muscular dystrophy, type 2A or calpain-3 deficiency
Limb-girdle muscular dystrophy, type 2B, Myoshi myopathy, or dysferlin deficiency
Limb-girdle muscular dystrophy, type 2I or FKRP (fukutin-related protein) deficiency
Enclosed you will find a copy of the consent that will be discussed and that your kids be asked to sign before any testing or form completion.
We greatly appreciate your consideration of this study. Your participation will make this very important project possible.
Please do not hesitate to contact me if you have additional questions.
Sincerely,
Paola
Paola Canelos, MS
Evaluation of Limb-girdle Muscular Dystrophy
Project Manager
202-476-2427
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