Long-Term Low-Intervention SafEty and Clinical Outcomes Clinical Study of LivmArli® in Patients With Alagille Syndrome or Progressive Familial Intrahepatic Cholestasis in the European Union (LEAP-EU)
Recruiting
Phase 4
Interventional Study
Alagille Syndrome
Progressive Familial Intrahepatic Cholestasis
No Placebo Group
Every participant receives an active treatment — no one gets a placebo.
Prior Safety Data
This treatment has already been tested in at least one earlier human trial.
At a Glance
- Age
- 2 Months and older
- Sex
- Any
- Trial phase
- Phase 4
- Study type
- Interventional
- Purpose
- Other
- Participants needed
- 230 (estimated)
- Sponsor
- Mirum Pharmaceuticals, Inc. · Industry
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About This Trial
In patients with Alagille syndrome (ALGS), the key objectives are to evaluate the tolerability, long-term safety (including possible liver toxicity) and long-term efficacy of Livmarli treatment.
In patients with Progressive Familial Intrahepatic Cholestasis (PFIC), the key objectives are to evaluate tolerability, long-term safety (including possible liver toxicity and potential impact of chronic …
Trial Locations
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Eligibility Criteria
Inclusion Criteria:
* Understand and execute an Informed consent and assent (as applicable)
* For participants with ALGS ≥2 months of age at Day 1
* For participants with PFIC, ≥3 months of age at Day 1
* A clinically and/or genetically confirmed ALGS diagnosis with pruritus secondary to chronic ch…
Contacts