Open Actively Recruiting

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

About

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 [email protected] or telephone +1-877-768-4303.

Study Type
Expanded Access

Eligibility

Gender
All
Minimum Age
12 Months
Maximum Age
N/A

Inclusion Criteria:

  • Informed consent and assent provided, as applicable, per Institutional Review Board (IRB) or Ethics Committee (EC)
  • Clinical and/or genetic diagnosis of ALGS
  • Male or female participants aged >12 months and ≥5 kg with clinically significant cholestatic pruritus associated with ALGS
  • 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
  • No other therapeutic options or access to any ongoing ALGS clinical trials

Exclusion Criteria:

  • Diagnosis with a cholestatic liver disease other than ALGS
  • Female who is pregnant or breastfeeding
  • 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
  • Past medical history of compensated or decompensated cirrhosis
  • Presence of any other disease or condition known to interfere with the absorption, distribution, metabolism, or excretion of maralixibat or its mode of action

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Study Stats
Protocol No.
21-000255
Category
Pediatrics
Contact
Laura Wozniak
Location
  • UCLA Westwood
For Providers
NCT No.
NCT04530994
For detailed technical eligibility, visit ClinicalTrials.gov.