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Open Label Study to Evaluate Efficacy and Long Term Safety of LUM001 (Maralixibat) in the Treatment of Cholestatic Liver Disease in Patients With Progressive Familial Intrahepatic Cholestasis (INDIGO)

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ClinicalTrials.gov Identifier: NCT02057718
Recruitment Status : Completed
First Posted : February 7, 2014
Results First Posted : July 12, 2021
Last Update Posted : July 12, 2021
Sponsor:
Information provided by (Responsible Party):
Mirum Pharmaceuticals, Inc.

Brief Summary:
This is an open label study in children with Progressive Familial Intrahepatic Cholestasis (PFIC) designed to evaluate the safety and efficacy of LUM001, also known as Maralixibat (MRX). Efficacy will be assessed by evaluating the effect of LUM001 on pruritus and the biochemical markers of pruritus associated with PFIC.

Condition or disease Intervention/treatment Phase
Progressive Familial Intrahepatic Cholestasis (PFIC) Drug: LUM001 (Maralixibat) Phase 2

Detailed Description:
The study is divided into 5 parts: a 4-week dose escalation period, a 4-week stable dosing period, a 5-week stable dosing period, a 59-week long-term exposure period, and an optional follow-up treatment period for eligible participants who continue treatment with LUM001. Participants in the optional follow-up treatment period will continue to receive study drug until they are eligible to enter another LUM001 study or until LUM001 is available commercially, whichever occurs first.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 33 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Open Label Study of the Efficacy and Long Term Safety of LUM001 (Maralixibat), an Apical Sodium-Dependent Bile Acid Transporter Inhibitor (ASBTi), in the Treatment of Cholestatic Liver Disease in Pediatric Patients With Progressive Familial Intrahepatic Cholestasis
Actual Study Start Date : March 1, 2014
Actual Primary Completion Date : May 8, 2020
Actual Study Completion Date : May 8, 2020


Arm Intervention/treatment
Experimental: LUM001 (Maralixibat)
Participants will receive LUM001, also known as Maralixibat (MRX) twice a day (BID).
Drug: LUM001 (Maralixibat)
LUM001 also known as Maralixibat (MRX) oral dose up to twice a day (BID).




Primary Outcome Measures :
  1. Change From Baseline to Endpoint (Week 13) in Fasting sBA Level [ Time Frame: Baseline (Day 0) to Week 13 ]

Secondary Outcome Measures :
  1. Change From Baseline to Week 13/ET in Pruritus as Measured by ItchRO(Obs) [ Time Frame: Baseline (Day 0) to Week 13 ]
    This secondary efficacy endpoint is the change from baseline to Week 13 in pruritus as measured by ItchRO(Obs) weekly average morning score. ItchRO scores range from 0 to 4; the higher score indicates increasing itch severity (0 = none; 4 = very severe).

  2. Change From Baseline to Week 13/ET in Pruritus as Measured by ItchRO(Pt) [ Time Frame: Baseline (Day 0) to Week 13 ]
    This secondary efficacy endpoint is the change from baseline to Week 13 in pruritus as measured by ItchRO(Pt) weekly average morning score. ItchRO scores range from 0 to 4; the higher score indicates increasing itch severity (0 = none; 4 = very severe).

  3. Change From Baseline to Week 13/ET in ALT [ Time Frame: Baseline (Day 0) to Week 13 ]
  4. Change From Baseline to Week 13/ET in Total Bilirubin [ Time Frame: Baseline (Day 0) to Week 13 ]
  5. Change From Baseline to Week 13/ET in Direct Bilirubin [ Time Frame: Baseline (Day 0) to Week 13 ]


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Ages Eligible for Study:   12 Months to 18 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02057718


Locations
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United States, California
Children's Hospital Los Angeles
Los Angeles, California, United States, 90027
United States, Colorado
Children's Hospital Colorado
Aurora, Colorado, United States, 80045
United States, Ohio
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States, 45229
United States, Pennsylvania
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
Children's Hospital of Pittsburgh of UPMC
Pittsburgh, Pennsylvania, United States, 15224
France
Hopital Femme Mere Enfant De Lyon
Bron, France, 69677
Poland
The Children's Memorial Health Institute
Warsaw, Poland, 04-730
United Kingdom
Birmingham Children's Hospital
Birmingham, West Midlands, United Kingdom, B4 6NH
Leeds Teaching Hospital NHS Trust
Leeds, United Kingdom, LS1 3EX
Kings College Hospital
London, United Kingdom, SE5 9RS
Sponsors and Collaborators
Mirum Pharmaceuticals, Inc.
Investigators
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Study Director: Study Director Mirum
  Study Documents (Full-Text)

Documents provided by Mirum Pharmaceuticals, Inc.:
Study Protocol  [PDF] February 8, 2019
Statistical Analysis Plan  [PDF] June 30, 2020

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Responsible Party: Mirum Pharmaceuticals, Inc.
ClinicalTrials.gov Identifier: NCT02057718    
Other Study ID Numbers: LUM001-501
2013-003833-14 ( EudraCT Number )
First Posted: February 7, 2014    Key Record Dates
Results First Posted: July 12, 2021
Last Update Posted: July 12, 2021
Last Verified: June 2021
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Mirum Pharmaceuticals, Inc.:
Bile duct diseases
Intrahepatic Cholestasis
Cholestasis
PFIC
Pruritus
Cholestatic Liver Disease
Additional relevant MeSH terms:
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Liver Diseases
Cholestasis
Cholestasis, Intrahepatic
Digestive System Diseases
Bile Duct Diseases
Biliary Tract Diseases