CompletedPhase 2

Safety and Efficacy Study of LUM001 (Maralixibat) With a Drug Withdrawal Period in Participants With Alagille Syndrome (ALGS)

Australia, Belgium, France31 participantsStarted 2014-10-28

Plain-language summary

This is a long-term, open-label study with a double-blind, placebo-controlled, randomized drug withdrawal period in children with Alagille Syndrome (ALGS) designed to evaluate the safety and efficacy of LUM001 (Also known as maralixibat or MRX).

Who can participate

Age range12 Months – 18 Years

SexALL

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Inclusion criteria

✓. Total serum bile acid \> 3x ULN for age.
✓. Conjugated bilirubin \> 1 mg/dL.
✓. Fat soluble vitamin deficiency otherwise unexplainable.
✓. GGT \> 3x ULN for age.
✓. Intractable pruritus explainable only by liver disease.

What they're measuring

Change From Week 18 to Week 22 in Fasting sBA Levels in Participants Who Had a Reduction in sBA ≥50% From Baseline to Week 12 or Week 18

Timeframe: Week 18 to Week 22

Trial details

NCT IDNCT02160782

SponsorMirum Pharmaceuticals, Inc.

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2020-05-28

Results submitted2021-03-02

View on ClinicalTrials.gov More Alagille Syndrome trials