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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 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.
This trial's timeline: 3 weeks for screening, Varies for treatment, and long-term clinical outcomes (sbd, lt, portal hypertension, all-cause mortality) up to 180 days after discontinuation of livmarli will be recorded. for reporting.

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 more

Side effects data

From 2022 Phase 3 trial • 93 Patients • NCT03905330
57%
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%
Vitamin D decreased
9%
Constipation
9%
Vitamin D deficiency
6%
Vitamin E deficiency
6%
Gastroenteritis
6%
Coronavirus infection
6%
Upper respiratory tract infection
6%
Vomiting
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

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
Recent research and studies
clinicaltrials.govStudy
Long-Term Safety and Clinical Outcomes of Livmarli in Patients With Alagille Syndrome (LEAP)
2023. "Long-Term Safety and Clinical Outcomes of Livmarli in Patients With Alagille Syndrome (LEAP)". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT06193928.
All > Kansas City > Colorado Springs
~33 spots leftby Sep 2028
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