Evaluation of LUM001 in the Reduction of Pruritus in Alagille Syndrome

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT02057692
• Date of registration: 05/02/2014
• Prospective Registration: Yes
• Primary sponsor: Mirum Pharmaceuticals, Inc.
• Public title: Evaluation of LUM001 in the Reduction of Pruritus in Alagille Syndrome ITCH
• Scientific title: The Evaluation of the Intestinal Bile Acid Transport (IBAT) Inhibitor LUM001 in the Reduction of Pruritus in Alagille Syndrome, a Cholestatic Liver Disease
• Date of first enrolment: November 24, 2014
• Target sample size: 37
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT02057692
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Triple (Participant, Care Provider, Investigator).
• Phase: Phase 2

Countries of recruitment

Canada, United States

Contacts

• Name: Study Director
• Affiliation: Mirum

Key inclusion & exclusion criteria

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



Age minimum: 12 Months
Age maximum: 18 Years
Gender: All

Health Condition(s) or Problem(s) studied

Alagille Syndrome

Intervention(s)

Drug: Placebo

Drug: LUM001

Primary Outcome(s)

Change From Baseline to Endpoint (Week 13/Early Termination) in Pruritus [Time Frame: Baseline, Week 13/Early Termination]

Secondary Outcome(s)

Change From Baseline to Endpoint (Week 13/Early Termination) in Fasting Serum Bile Acid (sBA) Level [Time Frame: Baseline, Week 13/Early Termination]

Change From Baseline to Endpoint (Week 13/Early Termination) in Liver Enzyme Levels [Time Frame: Baseline, Week 13/Early Termination]

Change From Baseline to Endpoint (Week 13/Early Termination) in Total and Direct Bilirubin Concentrations [Time Frame: Baseline, Week 13/Early Termination]

Secondary ID(s)

LUM001-301

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Childhood Liver Disease Research and Education Network

Results

• Results available: Yes
• Date Posted: 23/01/2018
• URL: https://clinicaltrials.gov/ct2/show/results/NCT02057692