Long-Term SafEty and Clinical Outcomes of LivmArli in Patients in the United States (LEAP-US)
Recruiting
Observational Study
Alagille Syndrome
Progressive Familial Intrahepatic Cholestasis
No Placebo Group
Every participant receives an active treatment — no one gets a placebo.
No Study Drug
Researchers observe your health over time — no experimental treatment is given.
At a Glance
- Sex
- Any
- Study type
- Observational
- Participants needed
- 70 (estimated)
- Sponsor
- Mirum Pharmaceuticals, Inc. · Industry
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About This Trial
The objective of this 5-year, prospective, observational cohort study is to evaluate the long-term safety and clinical outcomes of patients with Alagille syndrome (ALGS) or Progressive familial intrahepatic cholestasis (PFIC) treated with Livmarli.
Trial Locations
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Eligibility Criteria
Inclusion Criteria:
* A clinically and/or genetically confirmed ALGS diagnosis or PFIC diagnosis
* Participant prescribed Livmarli
Exclusion Criteria:
* Refusal to provide informed consent/assent (if required by the local IRB)
* Previously or currently on Livmarli through participation in a clini…
Contacts