EUCTR2015-000389-69-ES进行中（未招募）1 期
An Open-label Extension Study to Assess the Long-term 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 40 人开始时间: 2017年3月27日最近更新:
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 发起方
- 入组人数
- 40
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 as indicated by cerebrospinal fluid glucose concentration, erythrocyte 3-O-methyl-D-glucose uptake assay, or molecular genetic testing obtained from medical records.
- •2)Males and females, aged at least 1 year at the time of informed consent
- •3)Completion of UX007G-CL201 study (NCT01993186). Glut1 DS patients who received UX007/triheptanoin treatment as part of other clinical studies, ISTs, or expanded access/compassionate use treatment programs may be eligible at the discretion of the sponsor.
- •4)Provide written informed consent or verbal assent (if possible) with written informed consent by a legally authorized representative after the nature of the study has been explained, and prior to any research-related procedures
- •5)Must, in the opinion of the investigator, be willing and able to complete all aspects of the study, and comply with accurate completion of the seizure diary
- •6)Females of childbearing potential must have a negative urine pregnancy test at Baseline and be willing to have additional pregnancy tests during the study. Females considered not of childbearing potential include those who have not experienced menarche, are post-menopausal (defined as having no menses for at least 12 months without an alternative medical cause), or are permanently sterile due to total hysterectomy, bilateral salpingectomy, or bilateral oophorectomy.
- •7)Participants of child-bearing potential or fertile males with partners of child-bearing potential who are sexually active must consent to use a highly-effective method of contraception as determined by the investigator from the period following the signing of the informed consent through 30 days after last dose of study drug.
- •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 0
排除标准
- •1)Any known hypersensitivity to triheptanoin that, in the judgment of the investigator, places the subject at increased risk for adverse effects
- •2)History of, or current suicidal ideation, behavior and/or attempts
- •3)Pregnant and/or breastfeeding an infant
- •4)Unwilling or unable to discontinue use of a prohibited medication (i.e. barbiturates, pancreatic lipase inhibitors) or other substance that may confound study objectives. Use of up to 3 concomitant AEDs is allowed, provided dose has been stable at least 14 days prior to Baseline.
- •5)Use of any investigational product, drug or supplement (other than UX007) within 30 days prior to Baseline, or at any time during the study
- •6)Has a condition of such severity and acuity, in the opinion of the investigator, that it warrants immediate surgical intervention or other treatment
- •7)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 introduce additional safety concerns (e.g. other concurrent neurological or psychiatric disorders)
研究者
相似试验
进行中（未招募）
1 期
A trial to assess the long term safety and efficacy of UX007 in subjects who have already participated in a UX007 study.Glucose Transporter Type 1 deficiency syndromeMedDRA version: 20.0 Level: LLT Classification code 10061032 Term: Carbohydrate transport disorder System Organ Class: 100000004850EUCTR2015-000389-69-DKltragenyx Pharmaceutical Inc40
招募中
1 期
A rollover study for subjects that complete KAR-012 trial, to assess Long-term Safety and Tolerability of Adjunctive KarXT in Subjects with Inadequately Controlled Symptoms of SchizophreniaSchizophreniaCTIS2024-510773-20-00Karuna Therapeutics Inc.264
进行中（未招募）
1 期
A trial to assess the long term safety and efficacy of UX007 in subjects who have already participated in a UX007 study.Glucose Transporter Type 1 deficiency syndromeMedDRA version: 20.0 Level: LLT Classification code 10061032 Term: Carbohydrate transport disorder System Organ Class: 100000004850EUCTR2015-000389-69-GBltragenyx Pharmaceutical Inc40
进行中（未招募）
不适用
Rheumatoid arthritisMedDRA version: 13.1Level: PTClassification code 10039073Term: Rheumatoid arthritisSystem Organ Class: 10028395 - Musculoskeletal and connective tissue disordersRheumatoid arthritisEUCTR2009-015636-15-HUWyeth Pharmaceuticals Inc. Acting through its division Wyeth Research, a Pfizer Company260
进行中（未招募）
1 期
A study to assess the long-term safety and efficacy of dupilumab administered in patients 6 months to <18 year of age with atopic dermatitis (eczema)MedDRA version: 21.1Level: LLTClassification code 10003639Term: Atopic dermatitisSystem Organ Class: 100000004858Atopic dermatitisEUCTR2015-001396-40-PLRegeneron Pharmaceuticals, Inc.812