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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. Identifier: NCT04530994
Expanded Access Status : Available
First Posted : August 28, 2020
Last Update Posted : August 28, 2020
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 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
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

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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

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 identifier (NCT number): NCT04530994

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Contact: Clinigen Healthcare Ltd +1-877-768-4303
Contact: Mirum Medical Information

Sponsors and Collaborators
Mirum Pharmaceuticals, Inc.
Additional Information:
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Responsible Party: Mirum Pharmaceuticals, Inc. Identifier: NCT04530994    
Other Study ID Numbers: MRX-EAP
First Posted: August 28, 2020    Key Record Dates
Last Update Posted: August 28, 2020
Last Verified: August 2020
Keywords provided by Mirum Pharmaceuticals, Inc.:
Liver Disease
Bile Duct Diseases
Digestive System Diseases
Biliary Tract Diseases
Additional relevant MeSH terms:
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Alagille Syndrome
Pathologic Processes
Skin Diseases
Skin Manifestations
Cholestasis, Intrahepatic
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