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Clinical Trials

Date: 2015-11-06

Type of information: update on patient enrollment

phase: 3

Announcement: initiation of development program

Company: Ultragenyx Pharmaceutical (USA - CA)

Product: triheptanoin (UX007)

Action mechanism:

  • triglyceride.Triheptanoin is a purified, pharmaceutical-grade, specially designed synthetic triglyceride compound created via a multi-step chemical process. Triheptanoin is metabolized to and intended to provide patients with heptanoate, which can diffuse across the blood-brain barrier and be converted into glucose. Heptanoate can also be further metabolized to four- and five-carbon ketone bodies in the liver that also cross the blood-brain-barrier and provide an additional energy source to the brain. Heptanoate and five-carbon ketone bodies can also regenerate new glucose in the brain, which is deficient in these patients.
  • Ultragenyx is currently conducting a randomized, placebo-controlled Phase 2 study in the U.S. and Europe to evaluate the potential of triheptanoin to treat Glut1 DS patients who have failed the ketogenic diet and who continue to have breakthrough seizures. An investigator-initiated pilot study of UX007 in six Glut1 DS patients with movement disorder was recently presented at the American Association of Neurology 2015 meeting showing a substantial reduction in the frequency of movement disorders during treatment that rebounded after withdrawal and suggested a reasonable safety profile.

Disease: Glut1 DS - Encephalopathy due to GLUT1 deficiency

Therapeutic area: Rare diseases - CNS diseases - Neurological diseases

Country:

Trial details:

Latest news:

  • • On November 6, 2015, Ultragenyx Pharmaceutical announced an update to its development plan for UX007 in Glut1 DS patients. Following an End-of-Phase 2 meeting with the FDA, the company now plans to initiate a Phase 3 study in Glut1 DS patients with the movement disorder phenotype in mid-2016. The ongoing Phase 2 study in patients with the seizure phenotype will continue to enroll up to 40 patients as the movement disorder study progresses. If positive, the two studies are intended to support an NDA filing for the treatment of Glut1 DS.
  • The Phase 3 movement disorder study is designed to enroll approximately 40 patients. It is intended to be a randomized, double-blind, placebo-controlled, double cross-over study. The primary endpoint will be an assessment of the impact of UX007 on movement disorder events as recorded by a patient diary that will be further refined in discussions with the FDA. The company will continue enrollment of up to 40 patients in the randomized placebo-controlled Phase 2 seizure study. As the pivotal Phase 3 movement disorder study will be ongoing in 2016 and given the value of the seizure study in potentially supporting the filing, we will no longer conduct an interim analysis of the current Phase 2 study in the seizure phenotype. This will allow us to preserve the integrity of the Phase 2 study and maximize its utility from a regulatory perspective.

Is general: Yes