EUCTR2013-003771-35-GB进行中（未招募）1 期
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 个研究点目标入组 36 人开始时间: 2014年11月10日最近更新:
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 发起方
- 入组人数
- 36
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional clinical trial of medicinal product
入排标准
入选标准
- •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
- •11) Females of childbearing potential must have a negative pregnancy test at Screening, be willing to use an acceptable method of contraception, and have additional pregnancy tests during the study. Females considered not of childbearing potential include those who have not reached menarche, had total hysterectomy, have been in menopause for at least two years, or have had tubal ligation at least one year prior to Screening
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 30
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range 10
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
排除标准
- •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)
研究者
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