First human test of gene therapy for devastating muscle disease
NCT ID NCT03362502
Summary
This was an early safety study by Pfizer for a new gene therapy, PF-06939926, for Duchenne muscular dystrophy (DMD). It tested a single intravenous dose in a small group of boys, both walking and non-walking, to see if it was safe and tolerable. The study also looked for early signs that the therapy might help produce the missing muscle protein, dystrophin.
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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
Explore the condition pages connected to this study.