An Extension Study to Evaluate the Long-Term Safety and Durability of Effect of LUM001 in the Treatment of Cholestatic Liver Disease in Subjects With Alagille Syndrome (ALGS) (IMAGINE)

Mirum Pharmaceuticals

Status and phase: Completed; Phase 2

Conditions: Alagille Syndrome

Treatments: Drug: LUM001 (Maralixibat)

Study type: Interventional

Funder types: Industry

Identifiers

NCT02047318

LUM001-303

2013-003832-54 (EudraCT Number)

Details and patient eligibility

About

The purpose of this extension study is to determine the long-term safety and tolerability of an investigational treatment (LUM001 also known as Maralixibat) in children with ALGS who have completed participation in a core LUM001 treatment protocol. Efficacy will be assessed by evaluating the effect of LUM001 on pruritus, biochemical markers of pruritus, as well as biochemical markers of cholestasis and liver disease.

Enrollment: 19 patients

Sex: All

Ages: 12 months to 18 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

Participation for an individual patient is expected to be approximately 72 weeks.

Patients who complete 72 weeks of treatment may be eligible to receive treatment for up to 52 weeks during the follow-up treatment period and patients who completed the 124 weeks of treatment may be eligible to enter the additional long-term follow-up period.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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19 participants in 1 patient group

LUM001 (Maralixibat)

Experimental group

Description:

LUM001 also known as Maralixibat (MRX) administered orally up to twice each day

Treatment:

Drug: LUM001 (Maralixibat)