Evaluation of LUM001 in the Reduction of Pruritus in Alagille Syndrome (ITCH)

Mirum Pharmaceuticals

Status and phase: Completed; Phase 2

Conditions: Alagille Syndrome

Treatments: Drug: Placebo; Drug: LUM001

Study type: Interventional

Funder types: Other; Industry

Identifiers

NCT02057692

LUM001-301

Details and patient eligibility

About

The study is a randomized, double-blind, placebo-controlled study in children with Alagille Syndrome (ALGS). The study will investigate the effects of LUM001, compared to placebo, on pruritus, serum bile acids, liver enzymes, and other biochemical markers in patients with ALGS.

Enrollment: 37 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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37 participants in 2 patient groups, including a placebo group

LUM001

Experimental group

Description:

LUM001 for oral administration

Treatment:

Drug: LUM001

Placebo

Placebo Comparator group

Description:

Placebo administered orally once each day

Treatment:

Drug: Placebo