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)

NCT02047318 | Completed Phase 2 Results DMC FDA Drug

• 2 other identifiers: LUM001-3032013-003832-54
• Study Type: interventional
• Target: 19
• Locations: 1 country
• Sites: 3

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.

Conditions

Alagille Syndrome

Outcome Measures

Primary Outcomes (1)

• Change From MRX Baseline to Week 48 in Fasting sBA Levels

  The primary endpoint of this study was the mean change from MRX baseline to Week 48 in fasting sBA level.

  MRX baseline to Week 48

Secondary Outcomes (15)

• Change From MRX Baseline Over Time in Fasting sBA Levels

  MRX baseline to End of Treatment (maximum exposure was 336 weeks)

• Change From MRX Baseline to Week 48 in Pruritus

  MRX baseline to Week 48

• Change From MRX Baseline Over Time in Pruritus

  MRX baseline to End of Treatment (maximum exposure was 336 weeks)

• Change From MRX Baseline to Week 48 in Clinician Xanthoma Severity Score

  MRX baseline to Week 48

• Change From MRX Baseline Over Time in Clinician Xanthoma Severity Score

  MRX baseline to End of Treatment (maximum exposure was 336 weeks)

Study Arms (1)

LUM001 (Maralixibat)

EXPERIMENTAL

LUM001 also known as Maralixibat (MRX) administered orally up to twice each day

Drug: LUM001 (Maralixibat)

Interventions

LUM001 (Maralixibat) DRUG

Dosing of LUM001 also known as Maralixibat (MRX) with the objective of achieving optimal control of pruritus at a dose level that is tolerated by the participant and up to a maximum daily dose of 560 micrograms per kilogram (mcg/kg).

LUM001 (Maralixibat)

Eligibility Criteria

• Age: 12 Months - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

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.

Contact the study team to discuss eligibility requirements. They can help determine if this study is right for you.

Sponsors & Collaborators

• Mirum Pharmaceuticals, Inc.: lead

Study Sites (3)

Birmingham Children's Hospital

Birmingham, West Midlands, B4 6NH, United Kingdom

Location

Leeds Teaching Hospital NHS Trust

Leeds, West Yorkshire, LS1 3EX, United Kingdom

Location

Kings College Hospital

London, SE5 9RS, United Kingdom

Location

MeSH Terms

Conditions

Alagille Syndrome

Interventions

maralixibat

Condition Hierarchy (Ancestors)

Cholestasis, Intrahepatic; Cholestasis; Bile Duct Diseases; Biliary Tract Diseases; Digestive System Diseases; Liver Diseases; Heart Defects, Congenital; Cardiovascular Abnormalities; Cardiovascular Diseases; Abnormalities, Multiple; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Genetic Diseases, Inborn

Limitations and Caveats

Limitations of the trial such as small numbers of subjects analyzed or technical problems leading to unreliable data.

Results Point of Contact

• Title: Mirum Clinical Trials
• Organization: Mirum Pharmaceuticals

medinfo@mirumpharma.com

650-667-4085

Study Officials

• Study Director

  Mirum

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 23, 2014
• First Posted: January 28, 2014
• Study Start: December 20, 2013
• Primary Completion: June 17, 2020
• Study Completion: June 17, 2020
• Last Updated: November 19, 2021
• Results First Posted: November 19, 2021

Record last verified: 2021-11

Locations

GB (3)