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

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:

West Coast Clinical Trials
(714) 668-1500, ext. 4009

New York
Clinilabs Inc.
(212) 994-4567


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

No votes yet
MDA cannot respond to questions asked in the comments field. For help with questions, contact your local MDA office or clinic or email See comment policy