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sialic_trials [2012/05/24 20:15] – created kamsialic_trials [2012/08/27 18:41] – [Ultragenyx - HIBM Sialic Acid Phase II Trial Recruitment] daniel
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 Study Contact; Russ Byrns  \\ Study Contact; Russ Byrns  \\
 Study Coordinator  \\ Study Coordinator  \\
 +310-825-3264 \\
 RByrns@mednet.ucla.edu  \\ RByrns@mednet.ucla.edu  \\
    
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 212-263-8344 \\ 212-263-8344 \\
 Pankaj.patel@nyumc.org \\  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.  \\ 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.  \\
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   * Medication history   * Medication history
   * Office notes from last office visit with current physician   * Office notes from last office visit with current physician
- 
 ====== Natural History Study ====== ====== Natural History Study ======
  
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 \\ \\ \\ \\
  
 +====== 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|}}
 +
 +
 +
 +\\ \\