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Clinical Trial Details — Status: Completed

Administrative data

NCT number NCT01903460
Other study ID # LUM001-302
Secondary ID 2012-005346-38SH
Status Completed
Phase Phase 2
First received
Last updated
Start date August 2013
Est. completion date March 2015

Study information

Verified date March 2019
Source Mirum Pharmaceuticals, Inc.
Contact n/a
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial Summary

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.


Recruitment information / eligibility

Status Completed
Enrollment 20
Est. completion date March 2015
Est. primary completion date February 2015
Accepts healthy volunteers No
Gender All
Age group 12 Months to 18 Years
Eligibility 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

Study Design


Related Conditions & MeSH terms


Intervention

Drug:
LUM001

Placebo


Locations

Country Name City State
United Kingdom Birmingham Children's Hospital Birmingham West Midlands
United Kingdom Leeds Teaching Hospitals Leeds West Yorkshire
United Kingdom Kings College Hospital London

Sponsors (1)

Lead Sponsor Collaborator
Mirum Pharmaceuticals, Inc.

Country where clinical trial is conducted

United Kingdom, 

Outcome

Type Measure Description Time frame Safety issue
Primary Change From Baseline to Week 13 (End of Treatment) in Fasting Serum Bile Acid Level 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. Baseline to 13 weeks or end of treatment
Secondary Change From Baseline to Week 13 (End of Treatment) in Liver Enzymes 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. Baseline to 13 weeks or end of treatment
Secondary 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 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. Baseline to 13 weeks or end of treatment
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