Evaluation of Maralixibat in Biliary Atresia Response Post-Kasai (EMBARK)

Mirum Pharmaceuticals

Status and phase: Completed; Phase 2

Conditions: Biliary Atresia

Treatments: Other: Placebo; Drug: Maralixibat

Study type: Interventional

Funder types: Industry

Identifiers

NCT04524390

MRX-701

Details and patient eligibility

About

A study to evaluate the efficacy and safety of maralixibat in infants with Biliary Atresia (BA) after Hepatoportoenterostomy (HPE, also known as the Kasai procedure).

Full description

This is a double-blind randomized, placebo-controlled study in subjects with Biliary Atresia with a primary endpoint at Week 26 followed by long-term open-label period during which all subjects will receive maralixibat to Week 104.

Enrollment: 75 patients

Sex: All

Ages: 21 to 111 days old

Volunteers: No Healthy Volunteers

Inclusion criteria

1. Male or female subjects with body weight ≥2500 g, who are ≥21 days old and <90 days old at the time of HPE (Kasai)
2. HPE or Kasai Procedure within 3 weeks prior to randomization
3. Clinical diagnosis of biliary atresia

Exclusion criteria

1. Subjects with intractable chronic diarrhea at randomization
2. Subjects not tolerating enteral feeds at randomization
3. History of ileal resection
4. Diagnosis of biliary atresia splenic malformation syndrome or cystic biliary atresia
5. Evidence of another non-biliary atresia pathology involving the intrahepatic bile duct (e.g., paucity, sclerosing cholangitis)
6. Evidence of liver failure (e.g. significant ascites)

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Triple Blind

---

75 participants in 3 patient groups, including a placebo group

Double Blind - Maralixibat

Experimental group

Description:

The double-blind period comprised of 4-8 weeks of dose escalation followed by 18 - 22 weeks of stable dosing treatment, after which participants were transferred to the open-label arm.

Treatment:

Drug: Maralixibat

Double Blind - Placebo

Placebo Comparator group

Description:

The double-blind period comprised of 4-8 weeks of dose escalation followed by 18 - 22 weeks of stable dosing treatment, after which participants were transferred to the open-label arm.

Treatment:

Other: Placebo

Open Label - Maralixibat

Experimental group

Description:

The Open-Label period comprised of 4-8 weeks of dose escalation followed by 70 - 74 weeks of stable dosing treatment. During the OLE, all participants, regardless of treatment assignment in the double-blind period, received maralixibat.

Treatment:

Drug: Maralixibat