First human test of gene therapy for devastating muscle disease
NCT ID NCT03362502
Summary
This early-stage study tested the safety of a single dose of an experimental gene therapy, called PF-06939926, in boys with Duchenne muscular dystrophy (DMD). The main goal was to see if the treatment was safe and tolerable, while also checking if it helped produce the missing muscle protein, dystrophin. The study was terminated and included both boys who could walk and those who could not.
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Contacts and locations
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Locations
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Biospecimen Repository & Processing Core - BPRC
Durham, North Carolina, 27710, United States
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CCTS Clinical Research Center
Salt Lake City, Utah, 84108, United States
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Duke Cardiovascular Magnetic Resonance Center
Durham, North Carolina, 27710, United States
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Duke Children's Hospital & Health Center
Durham, North Carolina, 27710, United States
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Duke Neurology
Durham, North Carolina, 27705, United States
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Duke University Hospital Investigational Drug Services (IDS) Pharmacy
Durham, North Carolina, 27710, United States
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Duke University Medical Center, Lenox Baker Children's Hospital
Durham, North Carolina, 27705, United States
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MRI Research Center
Los Angeles, California, 90095, United States
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Primary Children's Hospital
Salt Lake City, Utah, 84113, United States
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Reed Neurological Research Center
Los Angeles, California, 90095, United States
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Ronald Reagan UCLA Medical Center (Investigational Drug Section)
Los Angeles, California, 90095, United States
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Ronald Reagan UCLA Medical Center - Interventional Radiology
Los Angeles, California, 90095, United States
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Ronald Reagan UCLA Medical Center Drug Information Center
Los Angeles, California, 90095, United States
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UCLA (David Geffen School of Medicine)
Los Angeles, California, 90095, United States
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UCLA Children's Heart Center
Los Angeles, California, 90095, United States
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UCLA Mattel Children's Hospital
Los Angeles, California, 90095, United States
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UCLA Medical Center
Los Angeles, California, 90095, United States
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UCLA Outpatient Surgery Center
Los Angeles, California, 90095, United States
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University of Utah Clinical Neurosciences Center
Salt Lake City, Utah, 84132, United States
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University of Utah Hospital
Salt Lake City, Utah, 84112, United States
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University of Utah Hospital & Clinics Investigational Drug Services
Salt Lake City, Utah, 84112, United States
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University of Utah Imaging and Neurosciences Center
Salt Lake City, Utah, 84108, United States
Conditions
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