Alagille Syndrome | Evaluation of LUM001 in the Reduction of Pruritus in Alagille Syndrome

Alagille Syndrome research study

What is the primary objective of this study?

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.

Who is eligible to participate?

Inclusion Criteria: 1. Diagnosis of Alagille Syndrome 2. Evidence of cholestasis 3. Moderate to severe pruritus 4. Ability to understand and willingness to sign informed consent/assent prior to initiation of any study procedures Exclusion Criteria: 1. Surgical disruption of the enterohepatic circulation 2. Liver transplant 3. History or presence of other concomitant liver disease 4. Females who are pregnant or lactating 5. Known HIV infection

Which medical condition, disease, disorder, syndrome, illness, or injury is researched?

Alagille Syndrome

Study Interventions

Interventions can include giving participants drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available or noninvasive approaches such as surveys, education, and interviews.

Drug:LUM001LUM001 administered orally

Drug:PlaceboPlacebo administered orally

Study Arms

Research studies and clinical trials typically have two or more research arms. An arm is a group of people who receive the same treatment in the study.

LUM001LUM001 for oral administration

PlaceboPlacebo administered orally once each day

Study Status


Start Date: November 24, 2014

Completed Date: November 16, 2016

Phase: Phase 2

Type: Interventional


Primary Outcome: Change From Baseline to Endpoint (Week 13/Early Termination) in Pruritus

Secondary Outcome: Change From Baseline to Endpoint (Week 13/Early Termination) in Fasting Serum Bile Acid (sBA) Level

Study sponsors, principal investigator, and references

Principal Investigator: Shire Study Physician

Lead Sponsor: Shire

Collaborator: Childhood Liver Disease Research and Education Network

More information:

Discuss Pruritus