EUCTR2020-000974-22-IT进行中（未招募）1 期
Randomized, Double-blind, Placebo-Controlled Phase 2 Study to Evaluate the Efficacy and Safety of Maralixibat in the Treatment of Subjects with Biliary Atresia after Hepatoportoenterostomy - EMBARK: Evaluation of Maralixibat in Biliary Atresia Response post Kasai
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 72
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
- •1.Informed consent (by the legally authorized representative) per the Institutional Review Board/Ethics Committee (IRB/EC)
- •2.Male or female participants with a body weight =2500 g who are =21 days old and <90 days old at the time of HPE or Kasai procedure
- •3.Post-conception age =36 weeks at birth
- •4.HPE or Kasai Procedure within 3 weeks prior to randomization
- •5.=31 days old at start of study treatment
- •6.Clinical diagnosis of BA at laparotomy (with subsequent confirmation on histology of the biliary remnant)
- •7.Caregiver willingness to comply with all study visits and requirements, including ability to read and understand the questionnaires and, if applicable, capable of diluting study medication per investigator training and written instructions
- •8. Caregiver access to email or phone for remote participant contacts
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range:
- •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
- •1.Chronic diarrhea requiring ongoing IV fluid or nutritional intervention at screening, during the screening period, or during the 30 days prior to screening
- •2.History of surgical disruption of the enterohepatic circulation other than HPE
- •3.HPE performed by laparoscopy
- •4.Not tolerating full enteral feeds at screening or during the screening period
- •5.Evidence of another pathology involving the intrahepatic bile ducts (e.g., paucity, sclerosing cholangitis)
- •6.Diagnosis of polysplenia syndrome, including evidence of BA splenic malformation syndrome, or major alformation of another organ system
- •7.Diagnosis of cystic BA (based on clinician judgment, including but not limited to ultrasonography and holangiography results)
- •8.Decompensated cirrhosis (international normalized ratio [INR] >1.5 after correction of possible vitamin K deficiency, history or presence of clinically significant ascites, known varices, variceal hemorrhage, and/or encephalopathy)
- •9.Previous or imminent need for liver transplantation
- •10.Known hypersensitivity to maralixibat or any of its excipients
- •11.Previous use of an ileal bile acid transporter inhibitor, an ASBT inhibitor, N-acetyl cysteine, or immunoglobulins
- •12.Receipt of investigational drug, biologic, or medical device within 30 days or 5 half-lives (whichever is longer) prior to screening
- •13.Known caregiver history of unreliability, mental instability, or cognitive impairment that, in the opinion of the investigator or sponsor medical monitor, could compromise the validity of informed consent, compromise the safety of the participant, or lead to nonadherence with the study protocol or inability to conduct the study procedures
- •14.Presence of other significant liver disease or any other conditions or abnormalities which, in the opinion of the investigator or sponsor medical monitor, may compromise the safety of the participant or interfere with the participant participating in or completing the study
- •15.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), per investigator discretion
研究者
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