Phase 2 Study of Triheptanoin (UX007) for the Treatment of Glucose Transporter Type 1 Deficiency … (NCT01993186) | Clinical Trial Compass
CompletedPhase 2
Phase 2 Study of Triheptanoin (UX007) for the Treatment of Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS)
United States36 participantsStarted 2014-02-28
Plain-language summary
The primary objectives of the study are to evaluate the efficacy of UX007 compared to placebo as measured by the reduction from randomization to Week 8 in frequency of seizures and to evaluate the safety of UX007 via adverse event (AE) rates, laboratory values, and electrocardiogram (ECG).
Who can participate
Age range1 Year – 100 Years
SexALL
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Inclusion criteria
✓. Diagnosis of Glut1 DS confirmed by SLC2A1 mutation
✓. Males and females at least 1 of age at the time of informed consent
✓. 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
✓. 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 electroencephalogram (EEG)
✓. Continuing to have seizures despite a prior or current use of at least 1 antiepileptic drug (AED)
✓. 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
✓. Not on, or not fully compliant with a prescribed diet plan (e.g. KD)
✓. Plasma level of beta-hydroxybutyrate (BHB) ≤ 1 mmol/L (non-fasting) at Screening
Exclusion criteria
✕. Serum alanine aminotransferase (ALT) or aspartate aminotransferase (AST) levels exceeding 3 times the upper limit of normal at Screening
✕. Any known hypersensitivity to triheptanoin or safflower oil that, in the judgment of the investigator, places the subject at increased risk for adverse effects
What they're measuring
1
Percent Reduction From Baseline to Week 8 in Frequency of Total Seizures (Normalized to a 4-Week Rate)
Timeframe: Baseline, Week 8
2
Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Serious TEAEs and Discontinuations Due to TEAEs During the Placebo-Controlled Period
Timeframe: Weeks 0 to 8
3
Number of Participants With TEAEs, Serious TEAEs and Discontinuations Due to TEAEs During the Extension Period
✕. Prior use of triheptanoin within 30 days prior to Screening
✕. History of, or current suicidal ideation, behavior and/or attempts
✕. Pregnant and/or breastfeeding an infant at Screening
✕. Participants unwilling or unable to discontinue use of a prohibited medication or other substance that may confound study objectives
✕. 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
✕. Has a condition of such severity and acuity, in the opinion of the investigator, that it warrants immediate surgical intervention or other treatment