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NCT Number: NCT01903460

Safety and Efficacy Study of LUM001 in the Treatment of Cholestatic Liver Disease in Patients With Alagille Syndrome

The study is a randomized, double-blind, placebo-controlled study to evaluate the safety and tolerability of LUM001. Efficacy will be assessed by evaluating the effect of LUM001 versus placebo on the biochemical markers and pruritus associated with Alagille Syndrome.

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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

Birmingham Children's Hospital, Birmingham, West Midlands, United Kingdom

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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

Placebo

Drug

Primary outcomes

  1. Change From Baseline to Week 13 (End of Treatment) in Fasting Serum Bile Acid Level

    Time frame: Baseline to 13 weeks or end of treatment

    Participants were required to fast for at least 4 hours; only water was permitted prior to collection. A negative change from baseline indicates that the level of bile acid decreased.

Secondary outcomes

  1. Change From Baseline to Week 13 (End of Treatment) in Liver Enzymes

    Time frame: Baseline to 13 weeks or end of treatment

    Analysis of liver enzymes included alanine aminotransferase (ALT), aspartate aminotransferase (AST), and alkaline phosphatase (ALP). A negative change from baseline indicates that the level of that enzyme decreased.

  2. Change From Baseline to Week 13 (End of Treatment) in Pruritus as Measured by The Patient And Observer Itch Reported Outcome (ItchRO) Average Daily Scores

    Time frame: Baseline to 13 weeks or end of treatment

    The ItchRO was administered as a twice daily electronic diary (eDiary). Children ≥9 years of age completed the patient ItchRO; those between the ages of 5 and 8 completed the patient ItchRO with the assistance of their caregiver. There was no patient report for subjects under the age of 5. ItchRO scores range from 0 to 4, with the higher score indicating increasing itch severity. ItchRO average daily scores were calculated as the sum of daily scores (ie, the maximum of morning and evening scores) divided by the number of days. The average daily score was calculated by using the 7 days pre-treatment for baseline, and the last 7 days of treatment for Week 13. A negative change from Baseline indicates that itch severity decreased.

Sponsors and collaborators

Lead sponsor

Mirum Pharmaceuticals, Inc.

Industry

Registry information

Official study title

A Randomized, Double-blind, Placebo-controlled Study to Evaluate the Safety and Efficacy of LUM001, an Apical Sodium-dependent Bile Acid Transporter Inhibitor (ASBTi), in the Treatment of Cholestatic Liver Disease in Paediatric Patients With Alagille Syndrome

Acronym: IMAGO

Important dates

Study start
2013
Primary completion
2015
Study completion
2015
First posted
Jul 19, 2013
Registry last updated
Mar 28, 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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