Evaluation of LUM001 in the Reduction of Pruritus in Alagille Syndrome

Alagille Syndrome Clinical Trial

— ITCH  

Official title:

The Evaluation of the Intestinal Bile Acid Transport (IBAT) Inhibitor LUM001 in the Reduction of Pruritus in Alagille Syndrome, a Cholestatic Liver Disease

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02057692
• Other study ID #: LUM001-301
• Status: Completed
• Phase: Phase 2
• Start date: November 24, 2014
• Est. completion date: November 16, 2016

Study information

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

Clinical Trial Summary

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.

Other known NCT identifiers

• NCT02055768

Recruitment information / eligibility

• Status: Completed
• Enrollment: 37
• Est. completion date: November 16, 2016
• Est. primary completion date: November 16, 2016
• 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

Related Conditions & MeSH terms

• Alagille Syndrome
• Pruritus
• Syndrome

Intervention

Drug:
• LUM001
LUM001 administered orally
• Placebo
Placebo administered orally

Locations

Country	Name	City	State
Canada	The Hospital for Sick Children	Toronto	Ontario
United States	Children's Healthcare of Atlanta	Atlanta	Georgia
United States	Children's Hospital Colorado	Aurora	Colorado
United States	Ann & Robert H. Lurie Children's Hospital of Chicago	Chicago	Illinois
United States	Cincinnati Children's Hospital Medical Center	Cincinnati	Ohio
United States	Baylor College of Medicine/Texas Children's Hospital	Houston	Texas
United States	Riley Hospital for Children	Indianapolis	Indiana
United States	Children's Hospital Los Angeles	Los Angeles	California
United States	The Children's Hospital of Philadelphia	Philadelphia	Pennsylvania
United States	Children's Hospital of Pittsburgh of UPMC	Pittsburgh	Pennsylvania
United States	University of Utah	Salt Lake City	Utah
United States	University of California at San Francisco	San Francisco	California
United States	Seattle Children's Hospital	Seattle	Washington

Sponsors (2)

Lead Sponsor	Collaborator
Mirum Pharmaceuticals, Inc.	Childhood Liver Disease Research and Education Network

Countries where clinical trial is conducted

United States,  Canada, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Other	Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Treatment-emergent Serious Adverse Events (TESAEs)	An AE was any unfavorable and unintended sign (including a clinically significant abnormal laboratory finding, for example), symptom, or disease temporally associated with the study or use of investigational drug product, whether or not the AE was considered related to the investigational drug product. A serious adverse event (SAE) was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged in-patient hospitalization; life -threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly.TEAEs were defined as AEs/SAEs that started or worsened after the study drug treatment.	From the start of study drug administration up to Week 17
Primary	Change From Baseline to Endpoint (Week 13/Early Termination) in Pruritus	Pruritus was assessed using Itch report outcome measure (ItchRO[Obs]), administered as an electronic diary (eDiary) which was completed by the participants twice daily (morning and evening). ItchRO(Obs) score ranged from 0 to 4, with the higher score indicating increasing itch severity. The highest score between the morning and evening ItchRO(Obs) reports represented the daily score: a measure of the worst itching over the previous 24-hour period.	Baseline, Week 13/Early Termination
Secondary	Change From Baseline to Endpoint (Week 13/Early Termination) in Fasting Serum Bile Acid (sBA) Level	Fasting sBA level was measured by using a liquid chromatography mass spectrometry method.	Baseline, Week 13/Early Termination
Secondary	Change From Baseline to Endpoint (Week 13/Early Termination) in Liver Enzyme Levels	Liver enzyme levels of alanine aminotransferase, alkaline phosphatase, aspartate aminotransferase and gamma glutamyl transferase were reported here.	Baseline, Week 13/Early Termination
Secondary	Change From Baseline to Endpoint (Week 13/Early Termination) in Total and Direct Bilirubin Concentrations	Liver enzyme levels of total bilirubin and direct bilirubin were reported here.	Baseline, Week 13/Early Termination