LBCTR2020063422招募中3 期
MRX-502: Randomized Double-blind Placebo-controlled Phase 3 Study to Evaluate the Efficacy and Safety of Maralixibat in the Treatment of Subjects with Progressive Familial Intrahepatic Cholestasis (PFIC) – MARCH-PFIC - N/A
适应症
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 入组人数
- 3
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized controlled trial
- 主要目的
- Treatment
- 盲法
- Blinded (masking used)
入排标准
- 年龄范围
- 1 至 17（—）
- 性别
- All
入选标准
- •Informed consent and assent (as applicable) per Institutional Review Board/Ethics Committee
- •2. Male or female subjects with a body weight = 5 kg, who are =12 months and < 18 years of age at
- •time of consent
- •3. Cholestasis as manifested by total sBA = 3× ULN
- •4. An average AM ItchRO(Obs) score = 1.5 during 4 consecutive weeks of the screening period, leading
- •to the baseline visit (Visit 1)
- •5. Completion of at least 21 valid* morning ItchRO(Obs) entries during 4 consecutive weeks of the
- •screening period, leading to the baseline visit (Visit 1) (*valid = completed and not answered as I
- •don’t know”; maximum allowed invalid reports = 7, no more than 2 invalid reports during the last
- •7 days before randomization)
- •6. Diagnosis of PFIC based on the following:
- •Chronic cholestasis as manifested by persistent (>6 months) pruritus, biochemical abnormalities
- •or pathological evidence of progressive liver disease
- •Primary Cohort:
- •Subjects with genetic testing results consistent with biallelic disease-causing variation in
- •ABCB11 (PFIC2), based on standard of care genotyping
- •Supplemental Cohort:
- •i. Subjects with genetic testing results consistent with biallelic disease-causing variation
- •in ATP8B1 (PFIC1), ABCB4 (PFIC3), or TJP2 (PFIC4), based on standard of care
- •ii. Subjects with PFIC phenotype without a known mutation or with another known
- •mutation not described above
- •iii. Subjects with PFIC after internal or external biliary diversion surgery or for whom
- •internal or external biliary diversion surgery was reversed
- •7. Male and females of non-childbearing potential. Males and non-pregnant, non-lactating females of
- •childbearing potential who are sexually active must agree to use acceptable contraception during the
- •study and 30 days following the last dose of the study medication. Females of childbearing potential
- •must have a negative pregnancy test
- •8. Access to email or phone for scheduled remote visits
- •9. Ability to read and understand the questionnaires (both caregivers and subjects above the age of
- •10. Access to consistent caregiver(s) during the study
- •11. Subject and caregiver willingness to comply with all study visits and requirements
排除标准
- •Predicted complete absence of bile salt excretion pump (BSEP) function based on the type of
- •ABCB11 mutation (PFIC2), as determined by a standard of care genotyping (applies to primary
- •cohort only).
- •History of surgical disruption of the enterohepatic circulation (applies to primary cohort only)
- •3. Chronic diarrhea requiring intravenous fluid or nutritional intervention for the diarrhea and/or its
- •sequelae at screening or during the 6 months prior to screening
- •4. Previous or planned liver transplant
- •5. Decompensated cirrhosis (international normalized ratio [INR] > 1.5, albumin < 30 g/L, history or
- •presence of clinically significant ascites, variceal hemorrhage, and/or encephalopathy)
- •6. ALT or total serum bilirubin (TSB) > 15× ULN at screening
- •7. Presence of other liver disease
- •8. 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
- •9. Liver mass on imaging, including screening ultrasound
- •10. Known diagnosis of human immunodeficiency virus (HIV) infection
- •11. Any prior cancer diagnosis (except for in situ carcinoma) within 5 years of the screening visit
- •12. Any known history of alcohol or substance abuse
- •13. Administration of bile acids or lipid binding resins, or sodium phenylbutyrate during the screening
- •14. Administration of growth hormones at any time before or during the study
- •15. Administration of any investigational drug, biologic, or medical device during the screening period
- •16. Previous use of an ileal bile acid transporter inhibitor (IBATi)
- •17. History of non-adherence to medical regimens, unreliability, medical condition, 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 subject, or lead to
- •nonadherence with the study protocol or inability to conduct the study procedures
- •18. Known hypersensitivity to maralixibat or any of its excipients
研究者
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