Phase 2 N=20 Randomized Triple-blind Treatment

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

Alagille Syndrome

Bottom Line

View on ClinicalTrials.gov: NCT01903460 ↗

Enrolled (actual)

Serious AEs

0.0%

Results posted

Dec 2015

Primary outcome: Primary: Change From Baseline to Week 13 (End of Treatment) in Fasting Serum Bile Acid Level — -82.864; -49.388; -66.126; -42.157 umol/L — p=0.5740

Study Design & Population

Study type: Interventional
Phase: Phase 2
Interventions: LUM001 (Drug); Placebo (Drug)
Age: Pediatric, Adult · 0+ yrs
Sex: All
Sponsor: Mirum Pharmaceuticals, Inc.
Primary completion: Feb 2015

Outcome Measures

Outcome	Result	p-value
PRIMARY Change From Baseline to Week 13 (End of Treatment) in Fasting Serum Bile Acid Level	-82.864; -49.388; -66.126; -42.157	0.5740
SECONDARY Change From Baseline to Week 13 (End of Treatment) in Liver Enzymes	59.3; 10.5; 34.9; 2.7; 37.2; -2.7	0.0783
SECONDARY Change From Baseline to Week 13 (End of Treatment) in Pruritus as Measured by The Patient And Observer Itch Reported Outcome (ItchRO) Average Daily Scores	-1.159; -0.608; -0.883; -0.811; -0.802; -0.419	0.6907

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.

Eligibility Criteria

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

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT01903460). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.