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

Mirum Pharmaceuticals

Status and phase: Completed; Phase 2

Conditions: Alagille Syndrome

Treatments: Drug: LUM001; Drug: Placebo

Study type: Interventional

Funder types: Industry

Identifiers

NCT01903460

2012-005346-38 (EudraCT Number)

LUM001-302

SHP625-302 (Other Identifier)

Details and patient eligibility

About

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.

Enrollment: 20 patients

Sex: All

Ages: 12 months to 18 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Triple Blind

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20 participants in 2 patient groups, including a placebo group

LUM001

Experimental group

Description:

LUM001 administered orally once each day

Treatment:

Drug: LUM001

Placebo

Placebo Comparator group

Description:

Placebo administered orally once each day

Treatment:

Drug: Placebo