Trial record 1 of 1 for:
expanded access maralixibat
A Maralixibat Expanded Access Program for Patients With Cholestatic Pruritus Associated With Alagille Syndrome (ALGS)
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| ClinicalTrials.gov Identifier: NCT04530994 |
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Expanded Access Status :
Available
First Posted : August 28, 2020
Last Update Posted : September 2, 2021
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Sponsor:
Mirum Pharmaceuticals, Inc.
Collaborator:
Clinigen, Inc.
Information provided by (Responsible Party):
Mirum Pharmaceuticals, Inc.
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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 |
| 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:
- Informed consent and assent provided, as applicable, per Institutional Review Board (IRB) or Ethics Committee (EC)
- Clinical and/or genetic diagnosis of ALGS
- Male or female participants aged >12 months and ≥5 kg with clinically significant cholestatic pruritus associated with ALGS
- 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
- No other therapeutic options or access to any ongoing ALGS clinical trials
Exclusion Criteria:
- Diagnosis with a cholestatic liver disease other than ALGS
- Female who is pregnant or breastfeeding
- 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
- Past medical history of compensated or decompensated cirrhosis
- 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
| Contact: Clinigen Healthcare Ltd | +1-877-768-4303 | MirumALGS@clinigengroup.com | |
| Contact: Mirum Medical Information | medinfo@mirumpharma.com |
Locations
| 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:
| 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.:
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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 |