Seeking Participants for hIBM/GNE Clinical Trial

A phase 1 trial of Ultragenyx’ treatment for hereditary inclusion-body myopathy caused by GNE mutations is taking place in New York and California

by Quest Staff on November 30, 2011 - 2:46pm

Update (Feb. 11, 2013): The phase 2 trial is no longer recruiting participants.


Update (March 15, 2012): This phase 1 trial is no longer recruiting participants. A phase 2 trial of sialic acid extended release tablets in people with GNE-related IBM is getting under way. See Phase 2 Study of SA-ER Tablets or enter NCT01517880 in the search box at ClinicalTrials.gov.

Ultragenyx Pharmaceutical in Novato, Calif., is developing an experimental treatment for a form of hereditary inclusion-body myopathy, or hIBM, that’s caused by mutations in the GNE gene.

This gene codes for a protein that’s needed to make sialic acid. The Ultragenyx product is an extended-release form of sialic acid.

The company announced Nov. 30, 2011, that it is seeking more participants for its phase 1 hIBM/GNE myopathy trial. Phase 1 trial sites are located in New York and California. Travel and lodging costs for participating in the trial will be covered. 

Participation in the phase 1 trial does not preclude individuals from participating in later trials.

To learn more about the trial, contact one of these centers:

California
West Coast Clinical Trials
(714) 668-1500, ext. 4009
ptinfo@wcct.com

New York
Clinilabs Inc.
(212) 994-4567
participate@clinilabs.com

 

About Clinical Trials

About Clinical Trials

A clinical trial is a test, in humans, of an experimental treatment. Although it's possible that benefit may be derived from participating in a clinical trial, it's also possible that no benefit, or even harm, may occur.

MDA has no ability to influence who is chosen to participate in a clinical trial.

To learn more, see Understanding Clinical Trials and Being a Co-Adventurer, which is about neuromuscular disease clinical trials. To see a continuously updated database of clinical trials, go to ClinicalTrials.gov.

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