A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Assess the Safety and Efficacy of UX007 in Subjects with Glucose Transporter Type 1 Deficiency Syndrome

ltragenyx Pharmaceutical Inc0 sites36 target enrollmentNovember 10, 2014

ConditionsGlucose Transporter Type 1 deficiency syndrome MedDRA version: 17.1 Level: HLGT Classification code 10039911 Term: Seizures (incl subtypes) System Organ Class: 10029205 - Nervous system disorders Therapeutic area: Diseases [C] - Nervous System Diseases [C10]

Overview

Phase: Phase 1
Intervention: Not specified
Conditions: Glucose Transporter Type 1 deficiency syndrome
Sponsor: ltragenyx Pharmaceutical Inc
Enrollment: 36
Status: Active, not recruiting
Last Updated: 7 years ago

Overview Investigators Eligibility Criteria Outcomes Similar Trials

Overview

Brief Summary

No summary available.

Registry

who.int

Start Date

November 10, 2014

End Date

September 20, 2017

Last Updated

7 years ago

Study Type

Interventional clinical trial of medicinal product

Investigators

Sponsor

ltragenyx Pharmaceutical Inc

Eligibility Criteria

Inclusion Criteria

•1\) Diagnosis of Glut1 DS confirmed by SLC2A1 mutation
•2\) Males and females, aged 1\-35 years (inclusive) at the time of informed consent
•3\) Average of at least 2 observable seizures (generalized or partial\-onset \[simple partial motor, complex partial, absence, or secondarily generalized] seizures) in 4 weeks over the last 24 weeks, by subject or caregiver report
•4\) At least 2 observable seizures (generalized or partial\-onset \[simple partial motor, complex partial, or secondarily generalized] seizures) in 4 weeks during the Baseline Period, with no 3\-week seizure\-free period during the Baseline Period OR absence seizures documented on Screening EEG
•5\) Continuing to have seizures despite a prior or current use of at least 1 AED
•6\) Allowed to be on up to 3 concomitant AEDs that must have been stable in dose at least 2 weeks prior to the beginning of screening and anticipated to remain stable in dose through the end of the 8\-week, placebo\-controlled Treatment Period
•7\) Not on, or not fully compliant with a prescribed diet plan (e.g. ketogenic diet) comprised of at least 60% total daily caloric intake from fat during previous 14 days (confirmed by 3\-day diet diary at Screening), or at any time during the course of the trial
•8\) Plasma level of beta\-hydroxybutyrate (BHB) \= 1 mmol/L (non\-fasting) at Screening
•9\) Provide written or verbal assent (if possible) and written informed consent by a legally authorized representative after the nature of the study has been explained, and prior to any research\-related procedures
•10\) Must, in the opinion of the investigator, be willing and able to complete all aspects of the study, comply with accurate completion of the seizures diary, and likely to complete the 8 week, placebo\-controlled, Treatment Period

Exclusion Criteria

•1\) Serum alanine aminotransferase (ALT) or aspartate aminotransferase (AST) levels exceeding 2X the upper limit of normal at Screening
•2\) Any known hypersensitivity to triheptanoin or safflower oil that, in the judgment of the investigator, places the subject at increased risk for adverse effects
•3\) History of, or current suicidal ideation, behavior and/or attempts
•4\) Pregnant and/or breastfeeding an infant at Screening
•5\) Participants unwilling or unable to discontinue use of a prohibited medication or other substance that may confound study objectives
•6\) Use of any investigational product (drug or supplement, including medium chain triglyceride \[MCT] oil) within 30 days prior to Screening, or at any time during the study
•7\) Has a condition of such severity and acuity, in the opinion of the investigator, that it warrants immediate surgical intervention or other treatment
•8\) Has a concurrent disease or condition, or laboratory abnormality that, in the view of the investigator, places the subject at high risk of poor treatment compliance or of not completing the study, or would interfere with study participation or introduces additional safety concerns (e.g., diabetes mellitus, other concurrent neurological or psychiatric disorders)