An Open-label, Multicenter Extension Study to Evaluate the Long-term Safety of LUM001, an Apical Sodium-dependent Bile Acid Transporter Inhibitor (ASBTi), in Patients with Alagille Syndrome (ALGS) or Progressive Familial Intrahepatic Cholestasis (PFIC)

Shire Human Genetic Therapies Inc0 个研究点目标入组 999,999 人开始时间: 2015年4月1日最近更新: 2019年12月10日

适应症

试验速览

暂无简介。

•1.Competent to provide informed consent and assent, as age appropriate, (per IRB/EC).
•2.Completed a treatment protocol of LUM001 in the treatment of cholestatic liver disease in patients with ALGS or PFIC, which includes Study LUM001-303, LUM001-304, LUM001 305, or LUM001-501.
•3.Females of childbearing potential must have a negative urine pregnancy test [ß human chorionic gonadotropin (ß-hCG)] at the Week 0 Visit.
•4.Sexually active females must be prepared to use an effective method (= 1% failure rate) of contraception during the trial. Effective methods of contraception are considered to be:
•a.Hormonal (e.g., contraceptive pill, patch, intramuscular implant or injection); or
•b.Barrier method, e.g., (a) condom with spermicide, or (b) diaphragm, with spermicide; or
•c.Intrauterine device (IUD).
•5.Access to phone for scheduled calls from study site.
•Are the trial subjects under 18? yes
•Number of subjects for this age range: 120
•F.1.2 Adults (18-64 years) no
•F.1.2.1 Number of subjects for this age range 5
•F.1.3 Elderly (>=65 years) no
•F.1.3.1 Number of subjects for this age range

•1.Experienced an adverse event or serious adverse event (SAE) related to the study drug during a core LUM001 treatment protocol that led to the discontinuation of the subject from the core LUM001 treatment study.
•2.Any conditions or abnormalities (including laboratory 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.
•3.History of non-adherence during the subject’s participation in the core LUM001 treatment protocol. Non-adherence is defined by dosing compliance of less than 80% in the core LUM001 treatment protocol.
•4.Unlikely to comply with the study protocol, or unsuitable for any other reason, as judged by the investigator.

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