Clinical Study for Progressive Familial
Intrahepatic Cholestasis (PFIC)

Mirum Pharmaceuticals is striving to bring transformative medicines to patients
who have rare cholestatic liver conditions. In keeping with this mission, we have
initiated a Phase 3 study called MARCH to evaluate the safety and efficacy of
maralixibat in pediatric patients with progressive familialintrahepatic cholestasis (PFIC).
Eligible participants in this clinical study may help provide new insights into PFIC
and could lead to the first approved drug therapy.

Clinical Study for Progressive Familial Intrahepatic Cholestasis (PFIC)

Mirum Pharmaceuticals is striving to bring transformative medicines to patients who have rare cholestatic liver conditions. In keeping with this mission, we have initiated a Phase 3 study called MARCH to evaluate the safety and efficacy of maralixibat in pediatric patients with progressive familial intrahepatic cholestasis (PFIC). Eligible participants in this clinical study may help provide new insights into PFIC and could lead to the first approved drug therapy.

About

What is Progressive Familial Intrahepatic Cholestasis (PFIC)?

PFIC is a rare genetic condition that causes progressive liver disease, often leading to liver failure and requiring a liver transplant. In patients with PFIC, liver cells are less able to eliminate bile, leading to a buildup of bile which causes itching so severe and constant that it becomes a major life disruption for the patients and their families. Itching typically begins in infancy along with other symptoms including jaundice, failure to grow at the expected rate (failure to thrive), and increasingly severe liver disease. Current standard of care involves an invasive and disfiguring surgery (external biliary diversion) or liver transplantation. There is currently no approved drug therapy to treat PFIC or pruritus associated with PFIC.

The MARCH Study

Mirum has recently initiated a Phase 3 study called MARCH (MARalixibat in CHolestasis) to evaluate the safety and efficacy of a drug called maralixibat as a potential novel treatment for pediatric patients with PFIC. The study is enrolling 30 to 90 participants ages 1 to 17 years old to assess the change in itch severity and frequency, as well as reduction of bile acids in the blood. To learn more about MARCH and to see if your child is eligible, please click here.

Maralixibat

Maralixibat is an orally-administered liquid. Maralixibat is an investigational medicine being evaluated as a treatment for children with rare cholestatic liver diseases, including PFIC.

Maralixibat works by blocking an important bile acid transport protein on the surface of the small bowel, thereby reducing systemic levels of bile acids.

Encouraged by promising Phase 2 clinical data, Mirum Pharmaceuticals is now advancing maralixibat into Phase 3 clinical studies. To date, more than 119 children and 69 adults have received maralixibat as a treatment for their cholestatic liver diseases, many of them for more than 4 years. Maralixibat has also been tested in > 1400 subjects in other studies.

Results from the Phase 2 INDIGO study supported maralixibat’s Breakthrough Therapy Designation for the treatment of PFIC2 from the US FDA. Breakthrough Therapy designation is a process designed to expedite the development and review of drugs by the FDA that are intended to treat a serious condition and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over available therapy.

Who Can Take Part?

Your child may be eligible for the MARCH study if they:

  • Have been diagnosed with PFIC, carry the gene that causes the disease and are 1-17 years old
  • Have elevated bile acids in the blood and are moderately to severely itchy
  • Do not have presence of any other disease/condition known to interfere with the drug; are not experiencing severe diarrhea; and do not have a previous or planned liver transplant

Your child must meet all other study criteria to take part in the MARCH study. The full list of eligibility criteria can be found here.

Trial Locations

We are planning on opening sites in North America, Europe, Middle East, Asia and Latin America.

Even if you don’t live close to a trial site, that doesn’t mean you can’t participate.
Please contact pficstudy@clin-edge.com to see how we might be able to connect you to a trial site.

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1

– Currently Recruiting –
Children’s Hospital
Dr. Robert Squires
Pittsburgh, PA

2

– Currently Recruiting –
Children’s Hospital
Dr. Simon Horslen
Seattle, WA

3

– Currently Recruiting –
Michigan Medicine
(University of Michigan)
Dr. Frank DiPaola
Ann Arbor, MI

4

– Currently Recruiting –
Children’s Hospital
Dr. Alex Miethke
Cincinnati, OH

5

– Not Recruiting Yet –
University of Texas
Health Science Center
Dr. Naveen Mittal
San Antonio, TX

6

– Not Recruiting Yet –
Children’s Hospital
Dr. Daniel Thomas
Los Angeles, CA

7

– Not Recruiting Yet –
Columbia University
Medical Center
Dr. Jennifer Vittorio
New York, NY

8

– Currently Recruiting –
Children’s Hospital
Dr. Kathleen Loomes
Philadelphia, PA

9

– Not Recruiting Yet –
USCF Medical Center
Dr. Philip Rosenthal
San Fransisco, CA

10

– Currently Recruiting –
University of Texas
Southwestern Medical Center
Dr. Amal Aqul
Dallas, TX

11

– Not Recruiting Yet –
Hospital for Sick Children
Dr. Binita Kamath
Toronto, CA

12

– Not Recruiting Yet –
Hospital Italiano
de Buenos Aires
Dr. Daniel D’Agostino
Buenos Aires, Argentina

13

– Not Recruiting Yet –
UCL Cliniques st Luc
Dr. Etienne Sokal
Brussels, Belgium

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– Not Recruiting Yet –
Hôpital Bicêtre
Dr. Emmanuel Gonzales
Le Kremlin-Bicêtre, France

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– Not Recruiting Yet –
Medizinische Hochschule Hannover
Dr. Ulrich Baumann
Hannover, Germany

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– Not Recruiting Yet –
Instytut Pomnik Centrum Zdrowia Dziecka
Dr. Piotr Czubkowski
Warsaw, Poland

17

– Not Recruiting Yet –
Hospital Papa Giovanni
Dr. Lorenzo D’Antiga
Bergamo, Italy

18

– Not Recruiting Yet –
Charité Universitätsmedizin
Dr. Philip Bufler
Berlin, Germany

19

– Not Recruiting Yet –
Universitätsklinik Tübingen
Dr. Ekkehard Sturm
Tübingen, Germany

20

– Not Recruiting Yet –
Medizinische Universität Wien
Dr. Wolf-Dietrich Huber
Vienna, Austria

21

– Not Recruiting Yet –
Memorial Atasehir Hospital
Dr. Cigdem Arikan
Istanbul, Turkey

22

– Not Recruiting Yet –
Semmelweis Egyetem
Dr. Antal Dezsofi Tivadar
Budapest, Hungary

23

– Not Recruiting Yet –
Ankara University Faculty
Dr. Ceyda Tuna Kirsaclioglu
Ankara, Turkey

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– Not Recruiting Yet –
King’s College Hospital NHS Foundation Trust
Dr. Richard Thompson
London, UK

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– Not Recruiting Yet –
Hospital Municipal 
Dr. Gilda Porta
Sao Paulo, Brazil

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– Not Recruiting Yet –
Unidad de Hepatología Infantil
Dr. Joshue Covarrubias Esquer
Zapopan, Mexico

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– Not Recruiting Yet –
Birmingham Children’s Hospital
Dr. Deirdre Kelly
Birmingham, UK

28

– Not Recruiting Yet –
Dokuz Eylul Universitesi
Dr. Yesim Ozturk
Izmir, Turkey

29

– Not Recruiting Yet –
KK Women’s and Children’s Hospital
Dr. Fang Kuan Chiou
Bukit, Singapore

30

– Not Recruiting Yet –
CHU de Marseille – Hôpital de la Timone
Dr. Bertrand Roquelaure
Marseille, France

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– Not Recruiting Yet –
Gastroenterologia Pediatrica
Dr. Felipe Ordonez
Bogota, Colombia

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– Not Recruiting Yet –
Groupement Hospitalier Est Hopital
Femme Mère Enfant de Lyon
Dr. Noémie Laverdure
Lyon, France

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– Not Recruiting Yet –
Hotel Dieu de France
Dr. Adib Moukarzel 
Beirut, Lebanon

34

– Not Recruiting Yet –
University of Alberta
Edmonton Clinic Health Academy
Dr. Susan Gilmour
Edmonton, Canada

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– Not Recruiting Yet –
Ospedale Pediatrico bambino Gesu’
Dr. Manila Candusso
Rome, Italy

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– Not Recruiting Yet –
VUMC
Dr. Lynette Gillis
Nashville, Tennessee

FAQs

What are clinical trials?

It can take up to 10 years to develop a new medication. During this process plenty of information to learn if and how a new medication works need to be gathered before doctors are allowed to prescribe it. This information is collected in clinical research studies (also known as clinical trials) that test investigational medications (also known as study drugs) in humans. Each study follows a plan (or “protocol”) that has been reviewed and approved by various groups of people, including health authorities, like the Food and Drug Administration (FDA) in the United States, as well as scientists, doctors, lawyers, and members of the public.

Why do people participate in clinical trials?

Clinical trial participants can play a more active role in their health care, gain access to new research treatments before they are widely available and help others by contributing to medical research.

What are the different phases of clinical trials?

There are four phases of clinical trials. The trials at each phase have a different purpose and help answer different questions.

  • Phase I – test a new drug or treatment in a small group of people for the first time in order to evaluate the treatment’s safety, how much can be given safely, and to identify possible side effects from the treatment.
  • Phase II – typically test the treatment in a larger group of people in order to see if the treatment is effective and to further evaluate the safety of the treatment. Patients are usually given either active medication or placebo without knowing which one they are taking.
  • Phase III – typically test the treatment in a large group of people to confirm that the treatment is effective, monitor side effects, compare the treatment to other commonly used treatments, and collect additional information which should help allow safe use of the treatment. Again, patients are usually given either active medication or placebo without knowing which one they are taking.
  • Phase IV – are usually studies that are conducted once a drug is on the market and establish additional information about the treatment’s risks, benefits, and best use of the treatment.

The number of people included in each stage of a trial increases from one stage to the next, with the total number in a given trial depends on the type of disease the trial is addressing. For example, a clinical trial for a rare disease like PFIC would enroll far fewer patients than a clinical trial for a common condition that impacts millions of people, such as heart disease or diabetes.

What is an investigational medication?

An investigational medication is a drug that is not approved for marketing by the health authorities. It is generally only available for use in clinical research studies to determine its safety and effectiveness.

How often does PFIC occur?

Approximately 3,000 people in the United States and 5,000 people in Europe are affected by PFIC. Approximately 60% of PFIC patients are estimated to be PFIC2.

Are there other available therapeutics for PFIC?

There is currently no therapy approved for PFIC and available medical approaches have limited efficacy. Under current standard of care, patients often require a liver transplant and are at higher risk of many serious health issues.

Who is conducting the MARCH clinical study?

Mirum Pharmaceuticals Inc. is a clinical-stage therapeutics company developing a novel approach for treating liver diseases, with an immediate focus on rare pediatric conditions. The company’s lead product candidate, maralixibat, is an investigational orally-administered drug with an established safety profile and efficacy data in several indications, including Alagille syndrome (ALGS) and progressive familial intrahepatic cholestasis (PFIC). Mirum is dedicated to bringing innovative medicines to patients as quickly and efficiently as possible. For more information, visit www.mirumpharma.com.