CompletedPhase 2

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)

United Kingdom19 participantsStarted 2013-12-20

Plain-language 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.

Who can participate

Age range12 Months – 18 Years

SexALL

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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.

What they're measuring

Change From MRX Baseline to Week 48 in Fasting sBA Levels

Timeframe: MRX baseline to Week 48

Trial details

NCT IDNCT02047318

SponsorMirum Pharmaceuticals, Inc.

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2020-06-17

Results submitted2021-03-29

View on ClinicalTrials.gov More Alagille Syndrome trials