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Ultragenyx Pharmaceutical has announced that recruitment has begun for a Phase II Trial study for HIBM patients. Recruiting will soon begin at 3 locations in the United States.
Here are the following sites:
Washington University School of Medicine
St. Louis, MO
Investigator; Alan Pestronk, MD
Study Contact; Charlie Wulf, BA
Admin/Study Coordinator
314-362-6980
wulfc@neuro.wustl.edu
Ronald Regan UCLA Medical Center
Los Angeles, CA
Investigator; Perry Shieh, MD, PhD
Study Contact; Russ Byrns
Study Coordinator
RByrns@mednet.ucla.edu
NYU Medical Center/Dept. of Neurogenetics New York, NY
Investigator; Edwin Kolodny MD
Study Contact; Pankaj Patel
Research Coordinator
212-263-8344
Pankaj.patel@nyumc.org
To be eligible, patients must be between 18 and 65 years of age, must have a documented diagnosis of GNE myopathy (HIBM), must be able to walk 20 meters independently (may use orthotics and assistive devices), must be able to provide reproducible force in bilateral elbow flexors and knee extensors during hand-held dynamometry testing, and must be willing and able to comply with all study procedures including fine needle muscle biopsies of the upper and lower extremities at Baseline and 24 and 48 weeks.
The details of the Phase 2 study, including full inclusion/exclusion criteria can be found at: ClinicalTrials.Gov HIBM Phase 2 Study.
For more eligibility criteria or information visit: http://clinicaltrials.gov/ct2/show/NCT01517880?term=HIBM&rank=4
Desired medical records by Investigators:
Beyond the Phase 2 Trial, Ultragenyx will be commencing a natural history study in the fall of 2012 to further characterize the rate of progression of the disease and to elucidate functional outcome measures for future therapeutic trials. For information regarding the Natural History Study, please contact;
John Ditton, VP of Commercial Planning
Ultragenyx Pharmaceutical
jditton@ultragenyx.com