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Completed

NCT Number: NCT02057692

Evaluation of LUM001 in the Reduction of Pruritus in Alagille Syndrome

The 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.

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Key information

Conditions

Alagille Syndrome Abnormalities, Multiple Bile Duct Diseases Biliary Tract Diseases Cardiovascular Abnormalities Cardiovascular Diseases Cholestasis Cholestasis, Intrahepatic Congenital Abnormalities Congenital, Hereditary, and Neonatal Diseases and Abnormalities Digestive System Diseases Genetic Diseases, Inborn Heart Defects, Congenital Liver Diseases

Age range

12 month–18 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

The Hospital for Sick Children, Toronto, Ontario, Canada

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

LUM001

Drug

LUM001 administered orally

Placebo

Drug

Placebo administered orally

Primary outcomes

  1. Change From Baseline to Endpoint (Week 13/Early Termination) in Pruritus

    Time frame: Baseline, Week 13/Early Termination

    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.

Secondary outcomes

  1. Change From Baseline to Endpoint (Week 13/Early Termination) in Fasting Serum Bile Acid (sBA) Level

    Time frame: Baseline, Week 13/Early Termination

    Fasting sBA level was measured by using a liquid chromatography mass spectrometry method.

  2. Change From Baseline to Endpoint (Week 13/Early Termination) in Liver Enzyme Levels

    Time frame: Baseline, Week 13/Early Termination

    Liver enzyme levels of alanine aminotransferase, alkaline phosphatase, aspartate aminotransferase and gamma glutamyl transferase were reported here.

  3. Change From Baseline to Endpoint (Week 13/Early Termination) in Total and Direct Bilirubin Concentrations

    Time frame: Baseline, Week 13/Early Termination

    Liver enzyme levels of total bilirubin and direct bilirubin were reported here.

Other outcomes

  1. Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Treatment-emergent Serious Adverse Events (TESAEs)

    Time frame: From the start of study drug administration up to Week 17

    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.

Sponsors and collaborators

Lead sponsor

Mirum Pharmaceuticals, Inc.

Industry

Collaborators

  • Childhood Liver Disease Research and Education Network

Registry information

Official study title

The Evaluation of the Intestinal Bile Acid Transport (IBAT) Inhibitor LUM001 in the Reduction of Pruritus in Alagille Syndrome, a Cholestatic Liver Disease

Acronym: ITCH

Important dates

Study start
2014
Primary completion
2016
Study completion
2016
First posted
Feb 7, 2014
Registry last updated
Mar 26, 2019

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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