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?
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
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
Start Date: November 24, 2014
Completed Date: November 16, 2016
Phase: Phase 2
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