Alagille Syndrome Clinical Trial
Official title:
A Maralixibat Expanded Access Program for Patients With Cholestatic Pruritus Associated With Alagille Syndrome
NCT number | NCT04530994 |
Other study ID # | MRX-EAP |
Secondary ID | |
Status | Approved for marketing |
Phase | |
First received | |
Last updated |
Verified date | October 2021 |
Source | Mirum Pharmaceuticals, Inc. |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Expanded Access |
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.
Status | Approved for marketing |
Enrollment | 0 |
Est. completion date | |
Est. primary completion date | |
Accepts healthy volunteers | |
Gender | All |
Age group | 12 Months and older |
Eligibility | 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 |
Country | Name | City | State |
---|---|---|---|
United States | Children's Healthcare of Atlanta/Emory University | Atlanta | Georgia |
United States | University of Virginia | Charlottesville | Virginia |
United States | Ann & Robert H. Lurie Children's Hospital | Chicago | Illinois |
United States | Baylor College of Medicine | Houston | Texas |
United States | University of California, Los Angeles | Los Angeles | California |
United States | Medical College of Wisconsin | Milwaukee | Wisconsin |
United States | University of Minnesota | Minneapolis | Minnesota |
United States | Ochsner Clinic Foundation | New Orleans | Louisiana |
United States | The Children's Hospital at Montefiore | New York | New York |
United States | University of Oklahoma | Oklahoma City | Oklahoma |
United States | Advent Health | Orlando | Florida |
United States | Stanford University | Redwood City | California |
United States | University of California San Francisco | San Francisco | California |
United States | Georgetown University | Washington | District of Columbia |
Lead Sponsor | Collaborator |
---|---|
Mirum Pharmaceuticals, Inc. | Clinigen, Inc. |
United States,
Status | Clinical Trial | Phase | |
---|---|---|---|
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