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

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

20 participants

Study Classification

INTERVENTIONAL

Study Start Date

2013-08-31

Study Completion Date

2015-03-31

Brief Summary

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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.

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Detailed Description

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Conditions

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Alagille Syndrome

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

TRIPLE

Participants Caregivers Investigators

Study Groups

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LUM001

LUM001 administered orally once each day

Group Type EXPERIMENTAL

LUM001

Intervention Type DRUG

Placebo

Placebo administered orally once each day

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

Interventions

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LUM001

Intervention Type DRUG

Placebo

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

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

Exclusion Criteria

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

Minimum Eligible Age

12 Months

Maximum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No