EUCTR2019-001211-22-FR进行中（未招募）1 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. - MARCH-PFIC
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 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
入选标准
- •1.Informed consent and assent (as applicable) per Institutional Review Board/Ethics Committee (IRB/EC)
- •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)
- •6.6.Diagnosis of PFIC based on:
- •Chronic cholestasis as manifested by persistent (>6 months) pruritus, biochemical abnormalities or pathological evidence of progressive liver disease and 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 genotyping
- •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 assent)
- •10.Access to consistent caregiver(s) during the study
- •11.Subject and caregiver willingness to comply with all study visits and requirements
- •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 0
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range 0
排除标准
- •1.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). Subjects can enter the study in the Supplemental Cohort (under inclusion criteria 6.ii or 6.iii)
- •2.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 (Visit 0)
- •12.Any known history of alcohol or substance abuse
- •13.Administration of bile acids or lipid binding resins, or sodium phenylbutyrate during the screening period
- •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
相似试验
进行中（未招募）
1 期
A Placebo-controlled study of Maralixibat in Subjects with Progressive Familial Intrahepatic Cholestasis (MARCH-PFIC).EUCTR2019-001211-22-GBMirum Pharmaceuticals Inc.30
进行中（未招募）
1 期
A Placebo-controlled study of Maralixibat in Subjects with Progressive Familial Intrahepatic Cholestasis (MARCH-PFIC).In patients with progressive familial intrahepatic cholestasis (PFIC),impairment of the egress of bile acids from the liver leads to cholestasis,hepatocellular injury and damage, and progressive liver disease thatmay ultimately lead to the need for liver transplantation. Itch is acommon symptom associated with cholestasis, it can occur at all stages of cholestatic liver disease, with or without jaundice.MedDRA version: 20.0Level: PTClassification code 10076033Term: Progressive familial intrahepatic cholestasisSystem Organ Class: 10010331 - Congenital, familial and genetic disordersEUCTR2019-001211-22-ATMirum Pharmaceuticals Inc.30
进行中（未招募）
1 期
A Placebo-controlled study of Maralixibat in Subjects with Progressive Familial Intrahepatic Cholestasis (MARCH-PFIC).In patients with progressive familial intrahepatic cholestasis (PFIC),impairment of the egress of bile acids from the liver leads to cholestasis,hepatocellular injury and damage, and progressive liver disease thatmay ultimately lead to the need for liver transplantation. Itch is acommon symptom associated with cholestasis, it can occur at all stages of cholestatic liver disease, with or without jaundice.MedDRA version: 20.0Level: PTClassification code 10076033Term: Progressive familial intrahepatic cholestasisSystem Organ Class: 10010331 - Congenital, familial and genetic disordersEUCTR2019-001211-22-DEMirum Pharmaceuticals Inc.30
招募中
3 期
A Placebo-controlled study of Maralixibat in Subjects with Progressive Familial Intrahepatic Cholestasis (MARCH-PFIC)Evaluate the Efficacy and Safety of Maralixibat in the Treatment of Subjects with Progressive Familial Intrahepatic Cholestasis (PFIC)PFICLBCTR2020063422Mirum Pharmaceuticals Inc3
进行中（未招募）
1 期
A Placebo-controlled study of Maralixibat in Subjects with Progressive Familial Intrahepatic Cholestasis (MARCH-PFIC).EUCTR2019-001211-22-PLMirum Pharmaceuticals Inc.30