An Extension Study of Maralixibat in Patients With Progressive Familial Intrahepatic Cholestasis (PFIC)

Sponsor

Mirum Pharmaceuticals, Inc. (Industry)

Overall Status

Enrolling by invitation

CT.gov ID

NCT04185363

Collaborator

(none)

Enrollment

Locations

Arm

55.8

Anticipated Duration (Months)

3.2

Patients Per Site

0.1

Patients Per Site Per Month

Study Details

Study Description

Brief Summary

The primary objective of this open label extension study is to evaluate the long-term safety and tolerability of maralixibat.

Condition or Disease	Intervention/Treatment	Phase
Progressive Familial Intrahepatic Cholestasis (PFIC)	Drug: Maralixibat	Phase 3

Detailed Description

The study will be conducted at multiple sites in North America, Europe, Asia, and South America.

Study Design

Study Type:

Interventional

Anticipated Enrollment :

90 participants

Allocation:

N/A

Intervention Model:

Single Group Assignment

Masking:

None (Open Label)

Primary Purpose:

Treatment

Official Title:

An Open-label Extension Study to Evaluate the Long-term Safety and Efficacy of Maralixibat in the Treatment of Subjects With Progressive Familial Intrahepatic Cholestasis (PFIC)

Actual Study Start Date :

Jan 8, 2020

Anticipated Primary Completion Date :

Sep 1, 2024

Anticipated Study Completion Date :

Sep 1, 2024

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Maralixibat All subjects will receive Maralixibat oral solution	Drug: Maralixibat All subjects will receive Maralixibat oral solution (up to 600 microgram per kilogram [mcg/kg]) twice daily for up to 104 weeks

Arm

Intervention/Treatment

Experimental: Maralixibat

All subjects will receive Maralixibat oral solution

Drug: Maralixibat

All subjects will receive Maralixibat oral solution (up to 600 microgram per kilogram [mcg/kg]) twice daily for up to 104 weeks

Outcome Measures

Primary Outcome Measures

Incidence of Treatment Emergent Adverse Events (TEAEs) during the study [From baseline through study completion, up to 104 weeks]

Secondary Outcome Measures

Change from maralixibat baseline over the course of the study in the weekly average morning ItchRO(Obs)™ (Itch Reported Outcome) severity score [From baseline through study completion,up to 104 weeks]
Change from maralixibat baseline over the course of the study in the weekly average morning ItchRO(Obs)™ (Itch Reported Outcome) frequency score [From baseline through study completion,up to 104 weeks]
Mean change from baseline over time in serum bile acid (sBA) levels [From baseline through study completion,up to 104 weeks]
Mean change from baseline over time in height and weight z-scores [From baseline through study completion,up to 104 weeks]

Eligibility Criteria

Criteria

Ages Eligible for Study:

1 Year to 18 Years

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

Key Inclusion Criteria:

Provide informed consent and assent (as applicable) per Institutional Review Board/Ethics Committee (IRB/EC)
Completion of study MRX-502

Exclusion Criteria:

Any female who is pregnant or lactating or who is planning to become pregnant
Administration of prohibited medication between the MRX-502 EOT visit and the MRX 503 Baseline Visit (Day 0)
History of non-compliance in study MRX-502, non-adherence to medical regimens, unreliability, mental instability or incompetence that could compromise the validity of informed consent or lead to non-adherence with the study protocol based on Investigator judgment
Experienced an adverse event (AE) or serious adverse event (SAE) related to maralixibat during the MRX-502 study that led to permanent discontinuation of the subject from maralixibat
Any other conditions or laboratory abnormalities that, in the opinion of the Investigator or Sponsor Medical Monitor, may compromise the safety of the subject, or interfere with the subject participating in or completing the study

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Children's Hospital Los Angeles	Los Angeles	California	United States	90027
2	Medstar Georgetown University Hospital	Washington	District of Columbia	United States	20007
3	Advent Health	Orlando	Florida	United States	32803
4	Children's Hospital at Montefiore	Bronx	New York	United States	10461
5	Cincinnati Children's Hospital	Cincinnati	Ohio	United States	45229
6	Children Hospital of Pittsburgh	Pittsburgh	Pennsylvania	United States	15224
7	Medical University of South Carolina	Charleston	South Carolina	United States	29425
8	University of Texas Southwestern Medical Center	Dallas	Texas	United States	75390
9	University of Texas, Health Science Center San Antonio	San Antonio	Texas	United States	78229
10	Seattle Children's Hospital	Seattle	Washington	United States	98105
11	Hospital Italiano de Buenos Aires	Buenos Aires		Argentina
12	Medizinische Universität Wien, Universitätsklinik für Kinder- und Jugendheilkunde	Wien		Austria
13	Cliniques Universitaires Saint-Luc	Brussels		Belgium
14	Sociedade Beneficente de Senhoras Hospital Sírio-Libanês	São Paulo		Brazil
15	University of Alberta - Women and Children's Health Research Institute	Edmonton	Alberta	Canada
16	Fundacion Cardioinfantil	Bogotá		Colombia
17	Groupement Hospitalier Est Hopital, Femme Mère Enfant de Lyon	Lyon		France
18	CHU de Toulouse - Hôpital des Enfants	Toulouse		France	31059
19	Medizinische Hochschule	Hanover		Germany
20	Azienda Ospedaliera Papa Giovanni XXIII - Unita di Pediatria	Bergamo		Italy	24127
21	Ospedale Pediatrico bambino Gesu'	Roma		Italy
22	Hotel Dieu de France, Alfred Naccache	Beirut		Lebanon
23	Consultario de Joshue David Covarrubias Esquer	Zapopan		Mexico
24	Instytut Pomnik Centrum, Zdrowia Dziecka	Warsaw		Poland
25	KK Women's and Children's Hospital	Singapore		Singapore
26	Koc University Hospital	Istanbul		Turkey	34010
27	Birmingham Children's Hospital	Birmingham		United Kingdom
28	King's College Hospital NHS Foundation Trust	London		United Kingdom