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Alagille syndrome (ALGS) for Alagille Syndrome (LEAP Trial)
N/A
Recruiting
Research Sponsored by Mirum Pharmaceuticals, Inc.
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Timeline
Screening 3 weeks
Treatment Varies
Follow Up long-term clinical outcomes (sbd, lt, portal hypertension, all-cause mortality) up to 180 days after discontinuation of livmarli will be recorded.
Awards & highlights
LEAP Trial Summary
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).
Eligible Conditions
- Alagille Syndrome
LEAP Trial Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ long-term clinical outcomes (sbd, lt, portal hypertension, all-cause mortality) up to 180 days after discontinuation of livmarli will be recorded.
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~long-term clinical outcomes (sbd, lt, portal hypertension, all-cause mortality) up to 180 days after discontinuation of livmarli will be recorded.
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
Assessment of Height
Assessment of Weight
Incidence of Clinical Events Potentially Related to Fat-Soluble Vitamin Deficiencies
+2 moreSide effects data
From 2022 Phase 3 trial • 93 Patients • NCT0390533057%
Diarrhoea
36%
Pyrexia
21%
Abdominal pain
17%
Rhinorrhoea
15%
Blood bilirubin increased
15%
Cough
13%
Influenza
13%
Alanine aminotransferase increased
11%
Nasopharyngitis
11%
Pruritus
9%
Vitamin E decreased
9%
Constipation
9%
Vitamin D deficiency
9%
Vitamin D decreased
6%
Gastroenteritis
6%
Coronavirus infection
6%
Upper respiratory tract infection
6%
Vomiting
6%
Vitamin E deficiency
4%
Urinary tract infection
4%
Abdominal pain upper
2%
International normalised ratio increased
2%
Cholestasis
2%
Idiopathic pneumonia syndrome
100%
80%
60%
40%
20%
0%
Study treatment Arm
Maralixibat
Placebo
LEAP Trial Design
1Treatment groups
Experimental Treatment
Group I: Alagille syndrome (ALGS)Experimental Treatment1 Intervention
A clinically and/or genetically confirmed ALGS diagnosis
Participant prescribed Livmarli
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Maralixibat
2015
Completed Phase 3
~260
Find a Location
Who is running the clinical trial?
Mirum Pharmaceuticals, Inc.Lead Sponsor
29 Previous Clinical Trials
1,596 Total Patients Enrolled
8 Trials studying Alagille Syndrome
176 Patients Enrolled for Alagille Syndrome
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