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. Learn more about MARCH and to see if your child is eligible.

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

For questions about the trial please contact:

medinfo@mirumpharma.com (U.S & International [non-Europe])
medinfoEU@mirumpharma.com (Europe)

Your child must meet all other study criteria to take part in the MARCH study. See the full list of eligibility criteria.

Trial Locations

We have open sites in North America, Europe, Middle East, Asia and Latin America.

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

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

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– Currently Recruiting –
Michigan Medicine
(University of Michigan)
Dr. James Lopez
Ann Arbor, MI

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

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– Currently Recruiting –
University of Texas
Health Science Center
Dr. Naveen Mittal
San Antonio, TX

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– Currently Recruiting –
Children’s Hospital
Dr. Chuan-Hao Lin
Los Angeles, CA

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– Currently Recruiting –
Columbia University
Medical Center
Dr. Jennifer Vittorio
New York, NY

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

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– Currently Recruiting –
USCF Medical Center
Dr. Philip Rosenthal
San Fransisco, CA

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

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– Currently Recruiting –
Hospital for Sick Children
Dr. Binita Kamath
Toronto, CA

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– Currently Recruiting –
Hospital Italiano
de Buenos Aires
Dr. Daniel D’Agostino
Buenos Aires, Argentina

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– Currently Recruiting –
Sociedad Beneficiente de Senhoras
Dr. Gilda Porta
Sao Paulo, Brazil

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– Currently Recruiting –
Consultorio de Joshué David Covarrubias Esquer
Dr. Joshue Covarrubias Esquer
Zapopan, Mexico

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– Currently Recruiting –
Fundación Cardioinfantil
Dr. Felipe Ordonez
Bogota, Colombia

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– Currently Recruiting –
University of Alberta
Edmonton Clinic Health Academy
Dr. Susan Gilmour
Edmonton, Canada

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– Currently Recruiting –
VUMC
Dr. Lynette Gillis
Nashville, Tennessee

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– Currently Recruiting –
Cleveland Clinic
Dr. Vera Hupertz
Cleveland, Ohio

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– Not Recruiting Yet –
Children’s Hospital of Eastern Ontario
Dr. Carolina Jiminez
Ontario, Canada

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– Currently Recruiting –
Advent Health
Dr. Regino Gonzalez-Peralta
Orlando, Florida

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– Currently Recruiting –
Yale University School of Medicine
Dr. Udeme Ekong
Washington, D.C.

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– Not Recruiting Yet –
Saint Louis University
Dr. Jain Ajay
Saint Louis, Missouri

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– Currently Recruiting –
Montefiore Medical Center
Dr. Nadia Ovchinsky
New York, New York

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– Currently Recruiting –
UCL Cliniques st Luc
Dr. Etienne Sokal
Brussels, Belgium

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– Currently Recruiting –
Hôpital Bicêtre
Dr. Emmanuel Gonzales
Le Kremlin-Bicêtre, France

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– Currently Recruiting –
Medizinische Hochschule Hannover
Dr. Ulrich Baumann
Hannover, Germany

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– Currently Recruiting –
Instytut Pomnik Centrum Zdrowia Dziecka
Dr. Piotr Czubkowski
Warsaw, Poland

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– Currently Recruiting –
Hospital Papa Giovanni
Dr. Lorenzo D’Antiga
Bergamo, Italy

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– Currently Recruiting –
Medizinische Universität Wien
Dr. Wolf-Dietrich Huber
Vienna, Austria

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– Currently Recruiting –
Koc University Hospital
Dr. Cigdem Arikan
Istanbul, Turkey

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– Currently Recruiting –
Semmelweis Egyetem
Dr. Antal Dezsofi Tivadar
Budapest, Hungary

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– Currently Recruiting –
King’s College Hospital NHS Foundation Trust
Dr. Richard Thompson
London, UK

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– Currently Recruiting –
Birmingham Children’s Hospital
Dr. Deirdre Kelly
Birmingham, UK

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– Currently Recruiting –
CHU de Marseille – Hôpital de la Timone
Dr. Bertrand Roquelaure
Marseille, France

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– Currently Recruiting –
Groupement Hospitalier Est Hopital
Femme Mère Enfant de Lyon
Dr. Noémie Laverdure
Lyon, France

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– Currently Recruiting –
Hotel Dieu de France
Dr. Adib Moukarzel 
Beirut, Lebanon

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– Currently Recruiting –
Ospedale Pediatrico Bambino Gesu’
Dr. Manila Candusso
Rome, Italy

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– Currently Recruiting –
CHU de Toulouse – Hopital des Enfants

Dr. Nowenn Laborde
Toulouse, France

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– Currently Recruiting –
KK Women’s and Children’s Hospital
Dr. Fang Kuan Chiou
Singapore

FAQs

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.

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.

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.

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.

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.

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.

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.