Investigators with the Center for Gene Therapy at Nationwide Children's are currently conducting numerous clinical research studies in Neuromuscular Disorders including Muscular Dystrophy. Each study is described in detail below.
Interested in learning more information or enrolling in a study? You may also contact the study coordinator by calling (614) 722-2203.
This study will investigate the best corticosteroid regime in boys with Duchenne Muscular Dystrophy.[read more...]
The Newcastle University and the University of Rochester, in collaboration with the United States National Institutes of Health, and the National Institute of Neurological Disorders and Stroke, are conducting a phase III study with corticosteroids in boys with Duchenne Muscular Dystrophy (FOR DMD study).
Corticosteroids are currently the only medicine that has been shown to increase muscle strength in boys with DMD. Doctors have tried different ways of prescribing corticosteroids in order to decrease undesirable side effects. Currently, different doctors in different countries prescribe the drugs in different ways, and some do not prescribe corticosteroids at all.
The FOR DMD study aims to compare three ways of giving corticosteroids to boys with DMD to determine which increases muscle strength the most, and which causes the fewest side effects.
Using the results of this study, we aim to provide patients and families with clearer information about the best way to take these drugs.
This study will look at three ways of taking corticosteroids by the mouth:
All three dosages are commonly used in boys with DMD and have shown to be beneficial.
In this study there is no placebo group, which means that all participants will receive active drugs (Prednisone or Deflazacort). However, neither the boys nor the clinicians will know which treatment or regime the boy is taking.
The study will recruit 300 boys around Europe, United States and Canada.
In North America, 16 centers will take part in the study:
|Alberta Children's Hospital||Penn State Children's Hospital|
|Children's Memorial Hospital, Chicago||SUNY Downstate Med Center|
|Health Sciences Centre Winnipeg||University of California, Davis|
|Kansas University Medical Center||University of California Los Angeles|
|Kennedy Krieger Institute||University of New Mexico|
|London Health Sciences Center||University of Rochester Medical Center|
|Nemours Children’s Hospital, Orlando||University of Texas Southwestern Medical Center|
|Nationwide Children’s Hospital||Vanderbilt Children's Hospital|
Patients who do not attend one of these hospitals for their routine follow-up can also participate, but will have to travel to their closest participating site to receive the study drug and for the check-ups.
Participants will receive study medication for a minimum of three years and a maximum of five years (depending on how early the boy was recruited into the study) and participation involves visits to the study hospital every three months for the first 6 months and every six months thereafter. At these visits we will be repeating many of the tests your child usually has in clinic for his routine DMD follow up.
How do you know if you are eligible to take part?
In order to take part in the study boys need to fulfil a number of criteria. These can only be checked when you come into the clinic. However, at this stage if your child may be eligible if he
If you feel that your child might be able to participate in this trial, please feel free to discuss it with your doctor locally. Alternatively, if you would like further information, please contact the University of Rochester Medical Center: Kim Hart | Phone: 1 (585) 275-3767.[hide]
This is a prospective natural history study of the progression of physical impairment, activity limitation and quality of life in Duchenne Muscular Dystrophy[read more...]
What is this study about?
This study is to:
There is no medication being tested in this study
What do we do in this study?
During a study visit, patients are asked to:
Who can participate?
This study is a Phase 3, multicenter, randomized, double-blind, placebo-controlled study to determine the efficacy and safety of ataluren 10, 10, 20 mg/kg in patients with nonsense mutation dystrophinopathy. Patients will be randomized in a 1:1 ratio to ataluren 10-, 10-, 20-mg/kg dose level or placebo. It is planned that 220 patients will be enrolled and patients will undergo 48 weeks of blinded treatment prior to the final analysis. Study assessments will be performed at clinic visits every 8 weeks. It is anticipated that an open-label extension study will be available to patients (who successfully complete the double-blind study) in countries where ataluren is not commercially available.[read more...]
Ages Eligible for Study: 7 years to 16 years
Genders Eligible for Study: Male
If you are interested in this study, please contact the Research Coordinator, Susan Gailey, CCRC, phone: (614) 355-2897.[hide]
Mucopolysaccharidosis (MPS) type III, also known as Sanfilippo syndrome, is a group of four genetic diseases. We are conducting a natural history study in MPS III. We will enroll a total of 30 subjects in this study; 15 subjects with MPSIIIA and 15 subjects with MPSIIIB.[read more...]
Kevin Flanigan, MD
Mucopolysaccharidosis (MPS) type III, also known as Sanfilippo syndrome, is a group of four genetic diseases. These diseases result in a buildup of specific sugars in the brain and spinal cord. These diseases cause a decrease in mental function and the ability to move. They eventually result in death.
We are looking for patients for a study of the natural history of MPS type III, with the following goals:
We will enroll 30 subjects total in this study. We will enroll 15 subjects with MPSIIIA and 15 subjects with MPSIIIB. All subjects must meet the following criteria:
For more information regarding this study, please contact the Study Coordinator Krista Kunkler at 614-722-2238, or by email at Krista.Kunkler@nationwidechildrens.org.[hide]
Nationwide Children’s Hospital is currently recruiting participants to determine the validity of several outcome measures that may be used for future clinical studies.[read more...]
Your participation will help design the tests to be performed to measure patient improvement in future clinical trials. We will also add your information to our list of patients who you would like to be contacted about upcoming clinical trials for their specific diagnosis.
Participants must be able to walk for 6 minutes and have a documented diagnosis of:
Sporadic Inclusion Body Myositis (sIBM) or
Becker Muscular Dystrophy (BMD)
Participants will be asked to perform timed functional tests such as standing up from a chair, walking for 6 minutes, and having muscle strength tested.
Interested individuals should contact:
or call (614) 722-6881
This NIH-funded project is directed at correlating mutations in the dystrophin gene with the severity and progression of disease in patients with Duchenne and Becker dystrophies.[read more...]
Study requirements include providing a blood sample for dystrophin gene mutation analysis, and visiting one of the participating centers for a yearly examination. Mutation testing is performed at no cost to the patient by collaborators at the University of Utah Genome Center, and patients receive a copy of their genetic testing results.
Participating centers include:
Additional information is available at www.dystrophin.org
|Jerry Mendell, MD|
|In 2004, Jerry Mendell, MD, was awarded the S. Mouchly Small Scientific Achievement Award from the Muscular Dystrophy Association, recognizing researchers who make significant contributions to neuromuscular disease research.|