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Clinical trial

Long-Term Safety and Clinical Outcomes of Livmarli in Patients With Alagille Syndrome (LEAP)

ID
Name
MRX-310
Description
The goal of this observational study is to evaluate the long-term safety and clinical outcomes of Livmarli prescribed to patients with Alagille Syndrome (ALGS).
Trial arms
Trial start
2023-09-21
Estimated PCD
2028-09-20
Trial end
2028-09-20
Status
Recruiting
Treatment
Maralixibat
The recommended dosage is 380 mcg/kg once daily.
Arms:
Alagille syndrome (ALGS)
Size
50
Primary endpoint
Long-term clinical outcome events
Long-term clinical outcomes (SBD, LT, portal hypertension, all-cause mortality) up to 180 days after discontinuation of Livmarli will be recorded.
Liver Transplant Waitlist Status
LT waitlist status will be collected at enrollment and every 6 months for 5 years.
Assessment of Height
Height z-score (centimeters) will be collected every year for 5 years.
Assessment of Weight
Weight z-score (kilograms) will be collected every year for 5 years.
Incidence of Clinical Events Potentially Related to Fat-Soluble Vitamin Deficiencies
The incidence of events will be assessed and reported every year for 5 years.
Eligibility criteria
Inclusion Criteria: * A clinically and/or genetically confirmed ALGS 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 clinical study or expanded access program * Participants who have previously received an SBD or LT * Any condition or abnormalities that, in the opinion of the investigator, may interfere with the participant participating in or completing the study * Participants who have received an investigational drug within 30 days of the first dose of Livmarli
Protocol
{'studyType': 'OBSERVATIONAL', 'patientRegistry': True, 'targetDuration': '5 Years', 'designInfo': {'observationalModel': 'COHORT', 'timePerspective': 'PROSPECTIVE'}, 'enrollmentInfo': {'count': 50, 'type': 'ESTIMATED'}}
Updated at
2024-03-12

1 organization

1 product

1 indication

Organization
Mirum Pharmaceuticals
Product
Maralixibat
Indication
Alagille syndrome