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Neurosciences at Nationwide Children's

Neurosciences at Nationwide Children's offers patients and families a comprehensive approach to the care for children and adolescents with disorders of the brain, spine and nervous system; from initial diagnosis and treatment to rehabilitation and long-term follow-up care.

Learn more about Neurosciences

ClinicalTrials.gov

Center for Gene Therapy Clinical Trials

Investigators with the Center for Gene Therapy currently are conducting numerous clinical research studies, described in detail below. For more information about individual studies please fill out the request form at the bottom of this page. 
 

Assessing Safety and Tolerability of GSK2402968 in Non-Ambulant Subjects with Duchenne Muscular Dystrophy

A double-blind, escalating dose, randomized, placebo-controlled study to assess the pharmacokinetics, safety and tolerability of single subcutaneous injections of GSK2402968 in non-ambulant subjects with Duchenne muscular dystrophy

Description  This study is directed to assess the pharmacokinetics, safety and tolerability of drug GSK2402968 in non-ambulant subjects with Duchenne muscular dystrophy.  This drug is designed to induce skipping of exon 51 in the DMD gene.
The study will be conducted at Nationwide Children’s Hospital, in Columbus, OH.

To be considered to be part of the trial, subjects must

  • Be diagnosed with Duchenne muscular dystrophy

  • Have been in a wheelchair full-time for at least one year, but no more than four years.  If a boy is walking, or uses a wheelchair only part-time, he will not be a candidate for this study

  • Have one of the following out-of-frame deletions in the DMD gene:

    exons 13-50

    exons 29-50

    exons 43-50

    exons 45-50

    exons 47-50

    exons 48-50

    exons 49-50

    exon 50

    exon 52

Subjects will undergo a screening that will include a DEXA scan and cardiology examination, along with blood tests.  More than two weeks later, they will receive a subcutaneous injection of the study agent, after which blood samples will be drawn over 24 hours.  They will need to return to Nationwide Children’s Hospital for follow-up visits one week and four weeks after the injection for further blood and urine tests, and examination by a study doctor. 

Travel costs will be reimbursed under guidelines discussed with subjects before enrollment.

Study Doctors


If you are interested in this study, please contact the Study Coordinator

Xiomara Quintero Rosales, MD
(614) 722-6961
 

Phase 1 Clinical Trial of rAAV1.tMCK.human-alpha-sarcoglycan Gene Vector in Limb Girdle Muscular Dystrophy type 2D (alpha-sarcoglycanopathy)

Description  Gene therapy trial for rare form of Limb Girdle muscular dystrophy

Study Doctors

Coordinator
Xiomara Rosales, MD
(614) 722-6961


Newborn Screening for Duchenne Muscular Dystrophy

Learn more about the Newborn Screening for Duchenne Muscular Dystrophy

Description  Newborn screening for Duchenne muscular dystrophy in the state of Ohio; run by the Ohio Department of Health

Study Doctors

Coordinator

Chelsea Rankin
(614) 355-2897


Cardiomyopathy in Patients with Duchenne Muscular Dystrophy

Description  A double-blind randomized clinical trial of lisinopril versus losartan is proposed. Both drugs are known to be effective for the treatment of dilated cardiomyopathy. ACEi have both delayed the onset and progression of left ventricle dysfunction in children with DMD.

Study Doctors

Coordinator
Laurence Viollet, PhD

(614) 355-2695

 

The United Dystrophinopathy Project: Genotype/Phenotype Correlation and Natural History in the Dystrophinopathies

Description  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.
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:

  • Nationwide Children’s Hospital, Columbus, OH
  • Washington University, St. Louis, MO
  • University of Utah, Salt Lake City, UT
  • University of Iowa, Iowa City, IA
  • Children’s Hospital of Philadelphia, Philadelphia, PA
  • University of Minnesota, Minneapolis, MN
  • University of California, Davis, Sacramento, CA

Additional information is available at www.dystrophin.org

Study Doctors

Coordinator
Chelsea Rankin
(614) 355-2897

Center for Gene Therapy Clinical Trials
Complete the form to receive information about the following clinical trials. Descriptions of each clinical trial is listed below the form.
First Name *
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Phase I Clinical Trial for LGMD2D
DMD Newborn Screening
Cardiomyopathy in DMD
Genotype/Phenotype Correlation in Dystrophinopathies
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