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

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 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