====== Ultragenyx - HIBM Sialic Acid Phase II Trial Recruitment ====== 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 \\ 310-825-3264 \\ 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 \\ Hadassah University Hospital \\ Jerusalem, Israel, 91120 \\ Principal Investigator: Yoseph Caraco, MD \\ Contact: Hannah Drory, BN, MN \\ 972-2-6779398 \\ hannahd@hadassah.org.il 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: * Documentation of the genetic confirmation of HIBM * Medical history * Medication history * Office notes from last office visit with current physician ====== Natural History Study ====== 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\\ \\ \\ ====== Ultragenyx-HIBM Sialic Acid Trial Phase IRecruitment ====== {{:ux001_cl101_phase_1_online_ad_w_irb_changes_2011jul20_2_.jpg?200 |}} **Ultragenyx Pharmaceutical Announces the Start of a Phase 1 Clinical Trial for Hereditary Inclusion Body Myopathy (HIBM) - also known as Nonaka Myopathy or Distal Myopathy with Rimmed Vacuoles (DMRV)** (Novato, CA, July 20, 2011) -- Ultragenyx Pharmaceutical has announced that recruitment has begun for a first clinical study for HIBM patients. To be eligible, patients need to have their HIBM diagnosis confirmed with a genetic test. This first study is a short study to see how the investigational drug circulates in the body. “People with this rare disorder previously had no compelling reason to undergo genetic testing to understand their genetic mutation or to obtain a definitive diagnosis,” said Emil Kakkis, MD, PhD, President and CEO of Ultragenyx. “As clinical trials are initiated patients will need a documented diagnosis of HIBM to be eligible, so we are helping them to get genetic testing.” To obtain testing at no cost, patients should contact their doctor and ask them to order sample collection kits from Ultragenyx for testing. * * * * Contacts: \\ To order test collection kit; John Ditton\\ jditton@ultragenyx.com \\ To learn more about the Clinical Trials; Mari Maurer, RN, BSN\\ mmaurer@ultragenyx.com \\ Phase 1 Trial Site Contacts: \\ Clinilabs, Inc.\\ 212-994-4567\\ 423 W. 55th Street, 4th Floor\\ New York, NY 10019\\ participate@clinilabs.com \\ www.clinilabs.com\\ West Coast Clinical Trials\\ 714-668-1500 ext 4009\\ 3545 Howard Way, Suite 100\\ Costa Mesa, Ca. 92626\\ ptinfo@wcct.com\\ www.wcct.com\\ **Ultragenyx PDF Press Release** \\ {{:ux001_cl101_phase_1_online_ad_w_irb_changes_2011jul20_2_.pdf|}} \\ \\