Gene Therapy Clinical Trials
Investigators with the Center for Gene Therapy currently are conducting numerous clinical research studies, described in detail below. More information about enrolling in these studies is available by contacting the study coordinator or by calling (614) 722-2203.
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
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
Follistatin Gene Transfer Trial for sIBM
Description
Gene therapy trial for sIB
Study Doctors
Coordinator
(614) 355-2695
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
Safety and Tolerability of AVI-4658 (PMO) for Exon Skipping of Exon 51 in Duchenne Muscular Dystrophy
Description
Gene modification trial for Duchenne muscular dystrophy (DMD)
Coordinator
(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
