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)
NCT ID: NCT02047318
Last Updated: 2021-11-19
Study Results
Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.
View full resultsBasic Information
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COMPLETED
PHASE2
19 participants
INTERVENTIONAL
2013-12-20
2020-06-17
Brief Summary
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Detailed Description
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Conditions
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Study Design
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NA
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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LUM001 (Maralixibat)
LUM001 also known as Maralixibat (MRX) administered orally up to twice each day
LUM001 (Maralixibat)
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).
Interventions
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LUM001 (Maralixibat)
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).
Eligibility Criteria
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Inclusion Criteria
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.
12 Months
18 Years
ALL
No
Sponsors
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Mirum Pharmaceuticals, Inc.
INDUSTRY
Responsible Party
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Principal Investigators
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Study Director
Role: STUDY_DIRECTOR
Mirum
Locations
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Birmingham Children's Hospital
Birmingham, West Midlands, United Kingdom
Leeds Teaching Hospital NHS Trust
Leeds, West Yorkshire, United Kingdom
Kings College Hospital
London, , United Kingdom
Countries
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Provided Documents
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Document Type: Study Protocol
Document Type: Statistical Analysis Plan
Other Identifiers
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2013-003832-54
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
LUM001-303
Identifier Type: -
Identifier Source: org_study_id