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Alagille Syndrome

0
Pipeline Programs
5
Companies
14
Clinical Trials
3 recruiting
0
Approved Products

Pipeline by Development Stage

Preclinical
Phase 1
Phase 1/2
Phase 2
Phase 2/3
Phase 3
On Market
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0
0
0
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Early DiscoveryClinical DevelopmentMarket

Competitive Landscape

5 companies ranked by most advanced pipeline stage

Mirum Pharmaceuticals
Mirum PharmaceuticalsFOSTER CITY, CA
9 programs
LivmarliN/A1 trial
Validation of the Itch Reported Outcome (ItchRO) Diaries in Pediatric Cholestatic Liver DiseaseN/A1 trial
maralixibatN/A1 trial
LUM001PHASE_21 trial
LUM001PHASE_21 trial
+4 more programs
Active Trials
NCT06193928Recruiting70Est. Sep 2030
NCT02131623Completed23Est. Mar 2015
NCT04530994Approved For Marketing
+6 more trials
Ipsen
IpsenChina - Tianjin
2 programs
OdevixibatPHASE_32 trials
OdevixibatPHASE_31 trial
Active Trials
NCT07497724Not Yet Recruiting200Est. Sep 2026
NCT04674761Completed52Est. Sep 2022
NCT05035030Recruiting70Est. Dec 2026
Alliance Pharmaceuticals
1 program
Assessment for bleeding disorder with tailored post-operative careN/A1 trial
Active Trials
NCT05846854Unknown40Est. Nov 2024
Genome & Company
Genome & CompanyKorea - Suwon
1 program
Positional Cloning of the Gene(s) Responsible for Alagille SyndromeN/A1 trial
Active Trials
NCT00001642Completed225Est. Mar 2000
Human BioSciences
Human BioSciencesWV - Martinsburg
1 program
Positional Cloning of the Gene(s) Responsible for Alagille SyndromeN/A

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
2027
2028
2029
2030
Mirum PharmaceuticalsLivmarli Oral Product
IpsenOdevixibat
IpsenOdevixibat
Mirum PharmaceuticalsLUM001
Mirum PharmaceuticalsLUM001
Mirum PharmaceuticalsLUM001
Mirum PharmaceuticalsLUM001
Mirum PharmaceuticalsLUM001
IpsenOdevixibat
Mirum PharmaceuticalsLivmarli
Alliance PharmaceuticalsAssessment for bleeding disorder with tailored post-operative care
Mirum PharmaceuticalsValidation of the Itch Reported Outcome (ItchRO) Diaries in Pediatric Cholestatic Liver Disease
Genome & CompanyPositional Cloning of the Gene(s) Responsible for Alagille Syndrome

Clinical Trials (14)

Total enrollment: 921 patients across 14 trials

NCT07290257Mirum PharmaceuticalsLivmarli Oral Product

Long-Term Low-Intervention SafEty and Clinical Outcomes Clinical Study of LivmArli® in Patients With Alagille Syndrome in the European Union (LEAP-EU)

Start: Sep 2025Est. completion: Dec 2030100 patients
Phase 4Recruiting
NCT05035030IpsenOdevixibat

Long-term Safety and Efficacy of Odevixibat in Patients With Alagille Syndrome

Start: Sep 2021Est. completion: Dec 202670 patients
Phase 3Recruiting
NCT04674761IpsenOdevixibat

Efficacy and Safety of Odevixibat in Patients With Alagille Syndrome

Start: Mar 2021Est. completion: Sep 202252 patients
Phase 3Completed
NCT02117713Mirum PharmaceuticalsLUM001

An Extension Study to Evaluate the Long-Term Safety and Durability of Effect of LUM001 in the Treatment of Cholestatic Liver Disease in Pediatric Subjects With Alagille Syndrome

Start: Mar 2015Est. completion: Jun 202034 patients
Phase 2Completed
NCT02057692Mirum PharmaceuticalsLUM001

Evaluation of LUM001 in the Reduction of Pruritus in Alagille Syndrome

Start: Nov 2014Est. completion: Nov 201637 patients
Phase 2Completed
NCT02160782Mirum PharmaceuticalsLUM001

Safety and Efficacy Study of LUM001 (Maralixibat) With a Drug Withdrawal Period in Participants With Alagille Syndrome (ALGS)

Start: Oct 2014Est. completion: May 202031 patients
Phase 2Completed
NCT02047318Mirum PharmaceuticalsLUM001

An Extension Study to Evaluate the Long-Term Safety and Durability of Effect of LUM001 in the Treatment of Cholestatic Liver Disease in Subjects With Alagille Syndrome (ALGS)

Start: Dec 2013Est. completion: Jun 202019 patients
Phase 2Completed
NCT01903460Mirum PharmaceuticalsLUM001

Safety and Efficacy Study of LUM001 in the Treatment of Cholestatic Liver Disease in Patients With Alagille Syndrome

Start: Aug 2013Est. completion: Mar 201520 patients
Phase 2Completed
NCT04530994Mirum Pharmaceuticalsmaralixibat

A Maralixibat Expanded Access Program for Patients With Cholestatic Pruritus Associated With Alagille Syndrome (ALGS)

N/AApproved For Marketing
NCT07497724IpsenOdevixibat

Retrospective Observational Study of Odevixibat Outcomes in Patients With PFIC Versus an External Control Cohort (OvEC-PFIC)

Start: Apr 2026Est. completion: Sep 2026200 patients
N/ANot Yet Recruiting
NCT06193928Mirum PharmaceuticalsLivmarli

Long-Term SafEty and Clinical Outcomes of LivmArli in Patients in the United States (LEAP-US)

Start: Sep 2023Est. completion: Sep 203070 patients
N/ARecruiting
NCT05846854Alliance PharmaceuticalsAssessment for bleeding disorder with tailored post-operative care

Decreasing Hemorrhage Risk in Children With Alagille Syndrome

Start: Apr 2023Est. completion: Nov 202440 patients
N/AUnknown
NCT02131623Mirum PharmaceuticalsValidation of the Itch Reported Outcome (ItchRO) Diaries in Pediatric Cholestatic Liver Disease

Validation of the Itch Reported Outcome (ItchRO) Diaries in Pediatric Cholestatic Liver Disease

Start: Jul 2014Est. completion: Mar 201523 patients
N/ACompleted
NCT00001642Genome & CompanyPositional Cloning of the Gene(s) Responsible for Alagille Syndrome

Positional Cloning of the Gene(s) Responsible for Alagille Syndrome

Start: May 1997Est. completion: Mar 2000225 patients
N/ACompleted

Phase Legend

Preclinical— Lab & animal studies
Phase 1— Safety & dosing
Phase 2— Efficacy testing
Phase 3— Large-scale trials
On Market— Approved & available

Key Insights

3 actively recruiting trials targeting 921 patients
5 companies competing in this space

The information on this page is for informational purposes only and should not be used as a substitute for professional medical advice. Drug information is sourced from FDA, DailyMed, and other government databases. Adverse event data from FAERS does not establish causation. Always consult a healthcare professional for medical decisions.