Open Label Study to Evaluate Efficacy and Long Term Safety of LUM001 (Maralixibat) in the Treatme… (NCT02057718) | Clinical Trial Compass
CompletedPhase 2
Open Label Study to Evaluate Efficacy and Long Term Safety of LUM001 (Maralixibat) in the Treatment of Cholestatic Liver Disease in Patients With Progressive Familial Intrahepatic Cholestasis
United States33 participantsStarted 2014-03-01
Plain-language summary
This is an open label study in children with Progressive Familial Intrahepatic Cholestasis (PFIC) designed to evaluate the safety and efficacy of LUM001, also known as Maralixibat (MRX). Efficacy will be assessed by evaluating the effect of LUM001 on pruritus and the biochemical markers of pruritus associated with PFIC.
Who can participate
Age range12 Months – 18 Years
SexALL
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Inclusion criteria
✓. Intrahepatic cholestasis manifest by total serum bile acid \>3x upper limit of normal (ULN) for age and, b or c:
✓. Two documented mutant alleles in ATP8B1, or ABCB11.
✓. Evidence of chronic liver disease, excluding those listed in (see Section 16.3), with one or more of the following criteria:
✓. Duration of biochemical or clinical abnormalities of \>6 months, or
✓. Pathologic evidence of progressive liver disease, or
✓. Sibling of known individual affected by PFIC (predicted to be chronic).
Exclusion criteria
✕. Chronic diarrhea requiring specific intravenous fluid or nutritional intervention for the diarrhea and/or its sequelae.
✕. Surgical disruption of the enterohepatic circulation at the time at screening. Subjects who have undergone reversal of a prior surgical procedure intended to disrupt enterohepatic circulation and who and have a permanently restored flow of bile acids from the liver to the terminal ileum may be eligible for the study upon consultation with the Medical Monitor.
✕. Liver transplant.
✕. Decompensated cirrhosis \[international normalized ratio (INR) \> 1.5, albumin \< 30 g/L, history or presence of clinically significant ascites, variceal hemorrhage, and/or encephalopathy\].
✕. ALT \>15×ULN at screening.
What they're measuring
1
Change From Baseline to Endpoint (Week 13) in Fasting sBA Level
✕. History or presence of other liver disease (see Section 16.3).
✕. 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).