NCT04530994Approved For Marketing不适用
A Maralixibat Expanded Access Program for Patients With Cholestatic Pruritus Associated With Alagille Syndrome
适应症
相关药物
试验速览
- 阶段
- 不适用
- 状态
- Approved For Marketing
- 试验地点
- 14
研究概览
简要总结
An open-label, single-arm, multi-centre program providing treatment access to ALGS patients with cholestatic pruritus in the US. Access can be requested through Clinigen Healthcare Ltd via email MirumALGS@clinigengroup.com or telephone +1-877-768-4303.
详细描述
The objective of this EAP is to provide access to maralixibat for eligible ALGS patients with cholestatic pruritus. Participants will be treated with 400 µg/kg/day with safety and tolerability evaluated on an ongoing basis.
研究设计
- 研究类型
- Expanded Access
入排标准
- 年龄范围
- 12 Months 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Informed consent and assent provided, as applicable, per Institutional Review Board (IRB) or Ethics Committee (EC)
- •Clinical and/or genetic diagnosis of ALGS
- •Male or female participants aged >12 months and ≥5 kg with clinically significant cholestatic pruritus associated with ALGS
- •Willingness to adhere to an acceptable method of contraception until treatment discontinuation by females of childbearing potential who are sexually active or who become sexually active during the program
- •No other therapeutic options or access to any ongoing ALGS clinical trials
排除标准
- •Diagnosis with a cholestatic liver disease other than ALGS
- •Female who is pregnant or breastfeeding
- •Clinically significant cardiovascular, hepatic, gastro-intestinal, pulmonary, neurologic, infectious or renal disease or other medical condition that, in the opinion of the physician or Medical Monitor, would preclude participation in the program
- •Past medical history of compensated or decompensated cirrhosis
- •Presence of any other disease or condition known to interfere with the absorption, distribution, metabolism, or excretion of maralixibat or its mode of action
研究者
研究点 (14)
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