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A Maralixibat Expanded Access Program for Patients With Cholestatic Pruritus Associated With Alagille Syndrome (ALGS)

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ClinicalTrials.gov Identifier: NCT04530994
Expanded Access Status : Available
First Posted : August 28, 2020
Last Update Posted : September 2, 2021
Sponsor:
Collaborator:
Clinigen, Inc.
Information provided by (Responsible Party):
Mirum Pharmaceuticals, Inc.

Brief Summary:
An open-label, single-arm, multi-centre program providing treatment access to ALGS patients with cholestatic pruritus in the US. Access can be requested through Clinigen Healthcare Ltd via email MirumALGS@clinigengroup.com or telephone +1-877-768-4303.

Condition or disease Intervention/treatment
Alagille Syndrome Drug: maralixibat

Detailed Description:
The objective of this EAP is to provide access to maralixibat for eligible ALGS patients with cholestatic pruritus. Participants will be treated with 400 µg/kg/day with safety and tolerability evaluated on an ongoing basis.

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Study Type : Expanded Access
Expanded Access Type : Treatment IND/Protocol
  See clinical trials of the intervention/treatment in this expanded access record.
Official Title: A Maralixibat Expanded Access Program for Patients With Cholestatic Pruritus Associated With Alagille Syndrome



Intervention Details:
  • Drug: maralixibat
    Maralixibat is an Apical Sodium-dependent Bile acid Transporter (ASBT) inhibitor

Information from the National Library of Medicine

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Ages Eligible for Study:   12 Months and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Criteria

Inclusion Criteria:

  1. Informed consent and assent provided, as applicable, per Institutional Review Board (IRB) or Ethics Committee (EC)
  2. Clinical and/or genetic diagnosis of ALGS
  3. Male or female participants aged >12 months and ≥5 kg with clinically significant cholestatic pruritus associated with ALGS
  4. Willingness to adhere to an acceptable method of contraception until treatment discontinuation by females of childbearing potential who are sexually active or who become sexually active during the program
  5. No other therapeutic options or access to any ongoing ALGS clinical trials

Exclusion Criteria:

  1. Diagnosis with a cholestatic liver disease other than ALGS
  2. Female who is pregnant or breastfeeding
  3. Clinically significant cardiovascular, hepatic, gastro-intestinal, pulmonary, neurologic, infectious or renal disease or other medical condition that, in the opinion of the physician or Medical Monitor, would preclude participation in the program
  4. Past medical history of compensated or decompensated cirrhosis
  5. Presence of any other disease or condition known to interfere with the absorption, distribution, metabolism, or excretion of maralixibat or its mode of action

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): NCT04530994


Contacts
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Contact: Clinigen Healthcare Ltd +1-877-768-4303 MirumALGS@clinigengroup.com
Contact: Mirum Medical Information medinfo@mirumpharma.com

Locations
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United States, California
University of California, Los Angeles Available
Los Angeles, California, United States, 90095
Contact       lwozniak@mednet.ucla.edu   
Principal Investigator: Laura Wozniak, MD         
Stanford University Available
Redwood City, California, United States, 94063
Contact       nebel@stanford.edu   
Principal Investigator: Noelle Ebel, MD         
University of California San Francisco Available
San Francisco, California, United States, 94158
Contact       joanna.curry@ucsf.edu   
Principal Investigator: Rosenthal, MD         
United States, District of Columbia
Georgetown University Available
Washington, District of Columbia, United States, 20007
Principal Investigator: Ekong, MD         
United States, Florida
Advent Health Available
Orlando, Florida, United States, 32804
Contact       Ariana.mora@AdventHealth.com   
Principal Investigator: Gonzalez-Peralta, MD         
United States, Georgia
Children's Healthcare of Atlanta/Emory University Available
Atlanta, Georgia, United States, 30329
Contact       Angela.Stallworth@choa.org   
Principal Investigator: Soler Rodriguez, MD         
United States, Louisiana
Ochsner Clinic Foundation Available
New Orleans, Louisiana, United States, 70121
Contact       shawan.stewart@ochsner.org   
Principal Investigator: Ryan Himes, MD         
United States, Minnesota
University of Minnesota Available
Minneapolis, Minnesota, United States, 55455
Contact       bhatt376@umn.edu   
Principal Investigator: Heli Bhatt, MD         
United States, New York
The Children's Hospital at Montefiore Available
New York, New York, United States, 10467
Contact       trannguy@montefiore.org   
Principal Investigator: Nadia Ovchinsky, MD         
United States, Oklahoma
University of Oklahoma Available
Oklahoma City, Oklahoma, United States, 73104
Contact       sirish-palle@ouhsc.edu   
Principal Investigator: Sirish Palle, MD         
United States, Texas
Baylor College of Medicine Available
Houston, Texas, United States, 77030
Contact       Reza.Amerinia@bcm.edu   
Principal Investigator: Harpavat, MD         
United States, Virginia
University of Virginia Available
Charlottesville, Virginia, United States, 22903
Contact       SLO2N@hscmail.mcc.virginia.edu   
Principal Investigator: Michael Mendoza, MD         
United States, Wisconsin
Medical College of Wisconsin Available
Milwaukee, Wisconsin, United States, 53226
Contact       bvitola@mcw.edu   
Principal Investigator: Vitola, MD         
Sponsors and Collaborators
Mirum Pharmaceuticals, Inc.
Clinigen, Inc.
Additional Information:
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Responsible Party: Mirum Pharmaceuticals, Inc.
ClinicalTrials.gov Identifier: NCT04530994    
Other Study ID Numbers: MRX-EAP
First Posted: August 28, 2020    Key Record Dates
Last Update Posted: September 2, 2021
Last Verified: September 2021
Keywords provided by Mirum Pharmaceuticals, Inc.:
Liver Disease
ALGS
Maralixibat
Bile Duct Diseases
Digestive System Diseases
Biliary Tract Diseases
Cholestasis
Pruritis
Additional relevant MeSH terms:
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Alagille Syndrome
Pruritus
Syndrome
Disease
Pathologic Processes
Skin Diseases
Skin Manifestations
Cholestasis, Intrahepatic
Cholestasis
Bile Duct Diseases
Biliary Tract Diseases
Digestive System Diseases
Liver Diseases
Heart Defects, Congenital
Cardiovascular Abnormalities
Cardiovascular Diseases
Abnormalities, Multiple
Congenital Abnormalities
Genetic Diseases, Inborn