Maralixibat_RISE Study

• Research type

  Research Study

• Full title

  Open-Label, Phase 2 Study to Evaluate the Safety and Tolerability of Maralixibat in the Treatment of Infants with Cholestatic Liver Diseases Including Progressive Familial Intrahepatic Cholestasis and Alagille Syndrome

• IRAS ID

  291001

• Contact name

  Lara Longpre

• Contact email

  llongpre@mirumpharma.com

• Sponsor organisation

  Mirum Pharmaceutical Inc.

• Eudract number

  2020-004628-40

• Duration of Study in the UK

  1 years, 9 months, 25 days

• Research summary

  The MRX-801, is a multicentre, open-label study of maralixibat in infants <12 months of age diagnosed with cholestatic liver disease as Alagille Syndrome (ALGS) or Progressive Familial Intrahepatic Cholestasis (PFIC). The aim of this study is to assess the safety and tolerability of maralixibat in infants <12 months of age with cholestatic liver disease due to ALGS and PFIC.
  PFIC is a rare autosomal recessive liver disorder characterised by intrahepatic cholestasis due to canalicular bile transport defects associated with early mortality, morbidity, and devastating consequences on patients’ quality of life. Alagille syndrome (ALGS) is an autosomal dominant multisystem genetic disorder with variable penetration. In patients with ALGS, impairment of the egress of bile acids from the liver leads to cholestasis, hepatocellular injury and damage, and progressive liver disease that may ultimately lead to the need for liver transplantation. Itch (pruritus) is the archetypal symptom of cholestasis, occurring at all stages of cholestatic liver disease, with or without jaundice. There is currently no approved treatment for PFIC or ALGS and available medical approaches have limited efficacy.

  By virtue of its ability to inhibit bile acid absorption, thereby increasing fBA excretion and lowering sBAs, maralixibat is currently under development for the treatment of cholestatic liver disease in infants.

• REC name

  London - Hampstead Research Ethics Committee

• REC reference

  20/LO/1308

• Date of REC Opinion

  21 Jan 2021

• REC opinion

  Further Information Favourable Opinion