Other Clinical Trial - Crossover Study to Assess the Efficacy & Safety of

Crossover Study to Assess the Efficacy and Safety of UX007 in the Treatment of Movement Disorders Associated With Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS)

Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS) Clinical Trial

Official title:
A Phase 3, Randomized, Double-blind, Placebo-controlled, Crossover Study to Assess the Efficacy and Safety of UX007 in the Treatment of Movement Disorders Associated With Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS)

Clinical Trial Description

n/a

Study Design

Related Conditions & MeSH terms

Carbohydrate Metabolism, Inborn Errors
Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS)
Movement Disorders
Syndrome

See also
	Status	Clinical Trial	Phase
	Completed	`NCT01993186` - Phase 2 Study of Triheptanoin (UX007) for the Treatment of Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS)	Phase 2
	Available	`NCT03773770` - Expanded Access to Triheptanoin

See also

Status

Clinical Trial

Phase

Completed

NCT01993186 - Phase 2 Study of Triheptanoin (UX007) for the Treatment of Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS)

Phase 2

Available

NCT03773770 - Expanded Access to Triheptanoin

Interventional studies are often prospective and are specifically tailored to evaluate direct impacts of treatment or preventive measures on disease.

Observational studies are often retrospective and are used to assess potential causation in exposure-outcome relationships and therefore influence preventive methods.

Expanded access is a means by which manufacturers make investigational new drugs available, under certain circumstances, to treat a patient(s) with a serious disease or condition who cannot participate in a controlled clinical trial.

Clinical trials are conducted in a series of steps, called phases - each phase is designed to answer a separate research question.

Phase 1: Researchers test a new drug or treatment in a small group of people for the first time to evaluate its safety, determine a safe dosage range, and identify side effects.
Phase 2: The drug or treatment is given to a larger group of people to see if it is effective and to further evaluate its safety.
Phase 3: The drug or treatment is given to large groups of people to confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the drug or treatment to be used safely.
Phase 4: Studies are done after the drug or treatment has been marketed to gather information on the drug's effect in various populations and any side effects associated with long-term use.

NCT number	NCT02960217
Study type	Interventional
Source	Ultragenyx Pharmaceutical Inc
Contact
Status	Terminated
Phase	Phase 3
Start date	April 19, 2017
Completion date	October 9, 2019

Crossover Study to Assess the Efficacy and Safety of UX007 in the Treatment of Movement Disorders Associated With Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS)

Clinical Trial Summary

Clinical Trial Description

Study Design

Related Conditions & MeSH terms