EUCTR2013-005373-43-FR进行中（未招募）1 期
ong-Term, Open-Label Study with a Double-Blind, Placebo Controlled, Randomized Drug Withdrawal Period of LUM001, an Apical Sodium-Dependent Bile Acid Transporter Inhibitor (ASBTi), in Patients with Alagille Syndrome - ICONIC
umena Pharmaceuticals Inc0 个研究点目标入组 30 人开始时间: 2015年7月10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
- 30
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
- 性别
- All
入选标准
- •To participate in this study subjects must meet all of the following criteria:
- •1.Male or female between the ages of 12 months and 18 years inclusive.
- •2.Diagnosis of ALGS .
- •3.Evidence of cholestasis (one or more of the following):
- •a.Total serum bile acid > 3x ULN for age.
- •b.Conjugated bilirubin > 1 mg/dL.
- •c.Fat soluble vitamin deficiency otherwise unexplainable.
- •d.GGT > 3x ULN for age.
- •e.Intractable pruritus explainable only by liver disease.
- •4.Females of childbearing potential must have a negative serum pregnancy test during Screening.
- •5.Sexually active females must be prepared to use an effective method of contraception during the trial, such as:
- •a.Hormonal (e.g., contraceptive pill, patch, intramuscular implant or injection).
- •b.Barrier method, i.e., (a) condom (male or female) or (b) diaphragm, with spermicide.
- •c.Intrauterine device (IUD).
- •6.Subject is expected to have a consistent caregiver(s) for the duration of the study.
- •7.Informed consent and assent (per IRB/IEC) as appropriate.
- •8.Access to phone for scheduled calls from study site.
- •9.Caregivers (and age appropriate subjects) must be willing and able to use an eDiary device during the study.
- •10.Caregivers (and age appropriate subjects) must digitally accept the licensing agreement in the eDiary software.
- •11.Caregivers (and age appropriate subjects) must complete at least 10 eDiary reports (morning or evening) during each of two consecutive weeks of the screening period (maximum possible reports = 14 per week).
- •12.Average daily score >2 on the Itch Reported Outcome (ItchRO™) questionnaire (maximum possible daily score of 4) for two consecutive weeks in the screening period, prior to dosing. A daily score is the higher of the scores for the morning and evening ItchRO. The average daily score is the sum of all daily scores divided by the number of days the ItchRO was completed.
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 30
- •F.1.2 Adults (18-64 years) no
- •F.1.2.1 Number of subjects for this age range
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
排除标准
- •Subjects will be excluded from the study if they meet any of the following criteria:
- •1.Chronic diarrhea requiring ongoing intravenous fluid or nutritional intervention.
- •2.Surgical interruption of the enterohepatic circulation.
- •3.Previous liver transplant.
- •4.Decompensated cirrhosis [ALT >15 x ULN, INR >1.5 (unresponsive to vitamin K therapy), albumin < 3.0 gm/dL, history or presence of clinically significant ascites, variceal hemorrhage, and/or encephalopathy].
- •5.History or presence of other concomitant liver disease.
- •6.History or presence of any other disease or condition known to interfere with the absorption, distribution, metabolism or excretion of drugs, including bile salt metabolism in the intestine (e.g., inflammatory bowel disease).
- •7.Known diagnosis of human immunodeficiency virus (HIV) infection.
- •8.Cancers except for in situ carcinoma, or cancers treated at least 5 years prior to Screening with no evidence of recurrence.
- •9.Recent medical history, or current status that suggests that the subject may be unable to complete the study.
- •10.Any female who is pregnant or lactating or who is planning to become pregnant during the study period.
- •11.Known history of alcohol or substance abuse.
- •12.Administration of bile acid or lipid binding resins within 28 days prior to screening and throughout the trial.
- •13.Receipt of investigational drug, biologic, or medical device within 28 days prior to Screening, or 5 half-lives of the study agent, whichever is longer.
- •14.History of non-adherence to medical regimens, unreliability, mental instability or incompetence that could compromise the validity of informed consent or lead to non-adherence with the study protocol based upon Investigator judgment.
- •15.Any other conditions or abnormalities which, in the opinion of the Investigator or Medical Monitor, may compromise the safety of the subject, or interfere with the subject participating in or completing the study.
研究者
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