CompletedPhase 2

Safety and Efficacy Study of LUM001 in the Treatment of Cholestatic Liver Disease in Patients With Alagille Syndrome

United Kingdom20 participantsStarted 2013-08

Plain-language summary

The study is a randomized, double-blind, placebo-controlled study to evaluate the safety and tolerability of LUM001. Efficacy will be assessed by evaluating the effect of LUM001 versus placebo on the biochemical markers and pruritus associated with Alagille Syndrome.

Who can participate

Age range12 Months – 18 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

✓. Diagnosis of Alagille Syndrome
✓. Evidence of cholestasis
✓. Moderate to severe pruritus
✓. Ability to understand and willingness to sign informed consent/assent prior to initiation of any study procedures

Exclusion criteria

✕. Surgical disruption of the enterohepatic circulation
✕. Liver transplant
✕. History or presence of other concomitant liver disease
✕. Females who are pregnant or lactating
✕. Known HIV infection

What they're measuring

Change From Baseline to Week 13 (End of Treatment) in Fasting Serum Bile Acid Level

Timeframe: Baseline to 13 weeks or end of treatment

Trial details

NCT IDNCT01903460

SponsorMirum Pharmaceuticals, Inc.

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2015-02

Results submitted2015-11-02

View on ClinicalTrials.gov More Alagille Syndrome trials