FindTrial
Sign In

A Maralixibat Expanded Access Program for Patients With Cholestatic Pruritus Associated With Alagille Syndrome (ALGS)

A Maralixibat Expanded Access Program for Patients With Cholestatic Pruritus Associated With Alagille Syndrome

Status
APPROVED_FOR_MARKETING
Phases
Unknown
Study type
Expanded Access
Source
ClinicalTrials.gov
Registry ID
NCT04530994
Enrollment
Unknown
Registered
2020-08-28
Start date
Unknown
Completion date
Unknown
Last updated
2021-10-20

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Alagille Syndrome

Keywords

Liver Disease, ALGS, Maralixibat, Bile Duct Diseases, Digestive System Diseases, Biliary Tract Diseases, Cholestasis, Pruritis

Brief summary

An open-label, single-arm, multi-centre program providing treatment access to ALGS patients with cholestatic pruritus in the US. Access can be requested through Clinigen Healthcare Ltd via email MirumALGS@clinigengroup.com or telephone +1-877-768-4303.

Detailed description

The objective of this EAP is to provide access to maralixibat for eligible ALGS patients with cholestatic pruritus. Participants will be treated with 400 µg/kg/day with safety and tolerability evaluated on an ongoing basis.

Interventions

DRUGmaralixibat

Maralixibat is an Apical Sodium-dependent Bile acid Transporter (ASBT) inhibitor

Sponsors

Clinigen, Inc.
CollaboratorINDUSTRY
Mirum Pharmaceuticals, Inc.
Lead SponsorINDUSTRY

Eligibility

Sex/Gender
ALL
Age
12 Months to No maximum

Inclusion criteria

1. Informed consent and assent provided, as applicable, per Institutional Review Board (IRB) or Ethics Committee (EC) 2. Clinical and/or genetic diagnosis of ALGS 3. Male or female participants aged \>12 months and ≥5 kg with clinically significant cholestatic pruritus associated with ALGS 4. Willingness to adhere to an acceptable method of contraception until treatment discontinuation by females of childbearing potential who are sexually active or who become sexually active during the program 5. No other therapeutic options or access to any ongoing ALGS clinical trials

Exclusion criteria

1. Diagnosis with a cholestatic liver disease other than ALGS 2. Female who is pregnant or breastfeeding 3. Clinically significant cardiovascular, hepatic, gastro-intestinal, pulmonary, neurologic, infectious or renal disease or other medical condition that, in the opinion of the physician or Medical Monitor, would preclude participation in the program 4. Past medical history of compensated or decompensated cirrhosis 5. Presence of any other disease or condition known to interfere with the absorption, distribution, metabolism, or excretion of maralixibat or its mode of action

Countries

United States

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026