Phase 2 N=33 Treatment

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

Progressive Familial Intrahepatic Cholestasis (PFIC)

Bottom Line

View on ClinicalTrials.gov: NCT02057718 ↗

Enrolled (actual)

Serious AEs

45.5%

Results posted

Jul 2021

Primary outcome: Primary: Change From Baseline to Endpoint (Week 13) in Fasting sBA Level — -23; 18; -38 umol/L

Study Design & Population

Study type: Interventional
Phase: Phase 2
Interventions: LUM001 (Maralixibat) (Drug)
Age: Pediatric, Adult · 0+ yrs
Sex: All
Sponsor: Mirum Pharmaceuticals, Inc.
Primary completion: May 2020

Outcome Measures

Outcome	Result	p-value
PRIMARY Change From Baseline to Endpoint (Week 13) in Fasting sBA Level	-23; 18; -38	—
SECONDARY Change From Baseline to Week 13/ET in Pruritus as Measured by ItchRO(Obs)	-0.7; -0.8; -0.7	—
SECONDARY Change From Baseline to Week 13/ET in Pruritus as Measured by ItchRO(Pt)	-0.6; -0.4; -0.7	—
SECONDARY Change From Baseline to Week 13/ET in ALT	-9; -2; -11	—
SECONDARY Change From Baseline to Week 13/ET in Total Bilirubin	-0.2; -0.8; -0.0	—
SECONDARY Change From Baseline to Week 13/ET in Direct Bilirubin	-0.1; -0.3; -0.0	—

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.

Eligibility Criteria

Inclusion Criteria

Male or female subjects between the ages of 12 months and 18 years inclusive.
Diagnosis of PFIC based on:
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).
GGTP 1.5, albumin 15×ULN at screening.
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).
Liver mass on imaging.
Known diagnosis of human immunodeficiency virus (HIV) infection.
Cancers except for in situ carcinoma, or cancers treated at least 5 years prior to screening with no evidence of recurrence.
Any female who is pregnant or lactating or who is planning to become pregnant within 20 weeks of assignment.
Any known history of alcohol or substance abuse.
Administration of bile acid or lipid binding resins within 30 days prior to Baseline / Day 0 and throughout the trial.
Administration of sodium phenylbutyrate within 30 days prior to Baseline / Day 0 and throughout the trial.
Investigational drug, biologic, or medical device within 30 days prior to screening, or 5 half-lives of the study agent, whichever is longer.
History of non-adherence to medical regimens, unreliability, mental instability or incompetence that could compromise the validity of informed consent or lead to non-adherence with the study protocol based on Investigator judgment.
Any other conditions or abnormalities which, in the opinion of the Investigator or Medical monitor, may compromise the safety of the subject, or interfere with the subject participating in or completing the study

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT02057718). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.