Title
Evaluation of LUM001 in the Reduction of Pruritus in Alagille Syndrome
The Evaluation of the Intestinal Bile Acid Transport (IBAT) Inhibitor LUM001 in the Reduction of Pruritus in Alagille Syndrome, a Cholestatic Liver Disease
Phase
Phase 2Lead Sponsor
ShireStudy Type
InterventionalStatus
Completed Results PostedIndication/Condition
Alagille SyndromeIntervention/Treatment
maralixibat ...Study Participants
37The study is a randomized, double-blind, placebo-controlled study in children with Alagille Syndrome (ALGS). The study will investigate the effects of LUM001, compared to placebo, on pruritus, serum bile acids, liver enzymes, and other biochemical markers in patients with ALGS.
LUM001 administered orally
Placebo administered orally
Inclusion Criteria: Diagnosis of Alagille Syndrome Evidence of cholestasis Moderate to severe pruritus Ability to understand and willingness to sign informed consent/assent prior to initiation of any study procedures Exclusion Criteria: Surgical disruption of the enterohepatic circulation Liver transplant History or presence of other concomitant liver disease Females who are pregnant or lactating Known HIV infection
| Event Type | Organ System | Event Term | Maralixibat 14 mcg/kg/Day | Maralixibat 70 mcg/kg/Day | Maralixibat 140 mcg/kg/Day | Maralixibat 280 mcg/kg/Day | Placebo |
|---|
Pruritus was assessed using Itch report outcome measure (ItchRO[Obs]), administered as an electronic diary (eDiary) which was completed by the participants twice daily (morning and evening). ItchRO(Obs) score ranged from 0 to 4, with the higher score indicating increasing itch severity. The highest score between the morning and evening ItchRO(Obs) reports represented the daily score: a measure of the worst itching over the previous 24-hour period.
Fasting sBA level was measured by using a liquid chromatography mass spectrometry method.
Liver enzyme levels of alanine aminotransferase, alkaline phosphatase, aspartate aminotransferase and gamma glutamyl transferase were reported here.
Liver enzyme levels of total bilirubin and direct bilirubin were reported here.
An AE was any unfavorable and unintended sign (including a clinically significant abnormal laboratory finding, for example), symptom, or disease temporally associated with the study or use of investigational drug product, whether or not the AE was considered related to the investigational drug product. A serious adverse event (SAE) was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged in-patient hospitalization; life -threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly.TEAEs were defined as AEs/SAEs that started or worsened after the study drug treatment.
