Clinical trial NCT02047318: An Extension Study to Evaluate the Long-Term Safety and Durability of Effect of LUM001 in the Treatment of Cholestatic Liver Disease in Subjects With Alagille Syndrome (ALGS)
Official title: A Multicentre Extension Study to Evaluate the Long-Term Safety and Durability of the Therapeutic Effect of LUM001 Also Known as Maralixibat (MRX), an Apical Sodium-Dependent Bile Acid Transporter Inhibitor (ASBTi), in the Treatment of Cholestatic Liver Disease in Pediatric Subjects With Alagille Syndrome
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Alagille Syndrome
Lead sponsor: Mirum Pharmaceuticals, Inc.
Sponsor class: INDUSTRY
First posted: 2014-01-28
Enrollment: 19
Sex: ALL
Minimum age (years): 12
Maximum age (years): 18
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: The purpose of this extension study is to determine the long-term safety and tolerability of an investigational treatment (LUM001 also known as Maralixibat) in children with ALGS who have completed participation in a core LUM001 treatment protocol. Efficacy will be assessed by evaluating the effect of LUM001 on pruritus, biochemical markers of pruritus, as well as biochemical markers of cholestasis and liver disease.


Eligibility criteria: Participation for an individual patient is expected to be approximately 72 weeks.

Patients who complete 72 weeks of treatment may be eligible to receive treatment for up to 52 weeks during the follow-up treatment period and patients who completed the 124 weeks of treatment may be eligible to enter the additional long-term follow-up period.

Primary outcomes:
measure: Change From MRX Baseline to Week 48 in Fasting sBA Levels
timeFrame: MRX baseline to Week 48
Secondary outcomes:
measure: Change From MRX Baseline Over Time in Fasting sBA Levels
timeFrame: MRX baseline to End of Treatment (maximum exposure was 336 weeks)
measure: Change From MRX Baseline to Week 48 in Pruritus
timeFrame: MRX baseline to Week 48
measure: Change From MRX Baseline Over Time in Pruritus
timeFrame: MRX baseline to End of Treatment (maximum exposure was 336 weeks)
measure: Change From MRX Baseline to Week 48 in Clinician Xanthoma Severity Score
timeFrame: MRX baseline to Week 48
measure: Change From MRX Baseline Over Time in Clinician Xanthoma Severity Score
timeFrame: MRX baseline to End of Treatment (maximum exposure was 336 weeks)
measure: Secondary: Change From MRX Baseline to Week 48 in Alkaline Phosphatase
timeFrame: MRX baseline to Week 48
measure: Change From MRX Baseline Over Time in Alkaline Phosphatase
timeFrame: MRX baseline to end of treatment (maximum exposure was 336 weeks)
measure: Change From MRX Baseline to Week 48 in Alanine Aminotransferase
timeFrame: MRX baseline to Week 48
measure: Change From MRX Baseline Over Time in Alanine Aminotransferase
timeFrame: MRX baseline to End of Treatment (maximum exposure was 336 weeks)
measure: Change From MRX Baseline to Week 48 in Aspartate Aminotransferase
timeFrame: MRX baseline to Week 48
measure: Change From MRX Baseline Over Time in Aspartate Aminotransferase
timeFrame: MRX baseline to End of treatment (maximum exposure was 336 weeks)
measure: Change From MRX Baseline to Week 48 in Gamma Glutamyltransferase
timeFrame: MRX baseline to Week 48
measure: Change From MRX Baseline Over Time in Gamma Glutamyltransferase
timeFrame: MRX baseline to End of Treatment (maximum exposure was 336 weeks)
measure: Change From MRX Baseline to Week 48 in Total and Direct Bilirubin
timeFrame: MRX baseline to Week 48
measure: Change From MRX Baseline Over Time in Total and Direct Bilirubin
timeFrame: MRX baseline to End of Treatment (maximum exposure was 336 weeks)
Other outcomes:
Locations:
city: Birmingham
country: United Kingdom
facility: Birmingham Children's Hospital
state: West Midlands
city: Leeds
country: United Kingdom
facility: Leeds Teaching Hospital NHS Trust
state: West Yorkshire
city: London
country: United Kingdom
facility: Kings College Hospital
Study officials:
affiliation: Mirum
name: Study Director
role: STUDY_DIRECTOR
References: