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Clinical Trials

If you are a patient or a doctor interested in finding out about active and upcoming clinical trials on HIBM, please contact us. We will include your details in our database and send you pertinent information.

Sialic Acid (Phase 1) Trial

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

IVIG Trial

During 2006, the first clinical therapeutic trial on HIBM was conducted at the National Institutes of Health (NIH). IVIG may have “mild benefits” for some patients, and the decision to obtain IVIG therapy will depend on numerous factors which should be discussed with your doctor. Your doctor may need the published results to help you decide.

You may download the full text of the article at BMC Neurology. The study was made possible by patients referred from HIBM Research Group ( HRG), an ARM funded laboratory.

N-Acetylmannosamine (ManNAc) Trial

Research on a mouse model of IBM has offered significant hope for HIBM. See the news release. The result of scientific study regarding N-Acetylmannosamine (ManNAc), a sugar-like molecule, can be downloaded at original scientific article published on June 1, 2007 issue of JCI. The study used a mouse model produced by HIBM Research Group (HRG), an ARM funded laboratory.

ManNAc is not an approved therapy. Upon completion of required safety and toxicology studies according to FDA guidelines, we are hopeful clinical trial will begin in the near future. This project has been selected by NIH TRND program as one of five rare disease projects to pursue. We are hopeful that clinical trial using ManNAc as a promising treatment for HIBM will begin in the next 1-2 years.

Gene Therapy Trial

Several groups worldwide are working hard to develop gene therapy for HIBM. Initial trials will be conducted on very few patients to evaluate both safety and effectiveness. There is currently a single patient trial ongoing under the direction of Dr. Nemunaitis. The initial results of intramuscular injection has been published. This initial work is very encouraging, and has paved the way for continued development of effective gene therapy for HIBM.

Stem Cell Trial

ARM sponsored investigator, Dr. Jacques Tremblay, has been working on developing stem cell based therapy for HIBM for the past several years. Initial trials will be conducted on very few patients. There is currently no projected start date.