Phase 2
N=19
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)
Alagille Syndrome
Bottom Line
View on ClinicalTrials.gov: NCT02047318 ↗Enrolled (actual)
19
Serious AEs
16.1%
Results posted
Nov 2021
Primary outcome: Primary: Change From MRX Baseline to Week 48 in Fasting sBA Levels — 261.96; 128.32 μmol/L — p=0.0012
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- LUM001 (Maralixibat) (Drug)
- Age
- Pediatric, Adult · 0+ yrs
- Sex
- All
- Sponsor
- Mirum Pharmaceuticals, Inc.
- Primary completion
- Jun 2020
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Change From MRX Baseline to Week 48 in Fasting sBA Levels |
261.96; 128.32 | 0.0012 sig |
| SECONDARY Change From MRX Baseline Over Time in Fasting sBA Levels |
261.96; 118.32 | 0.032 sig |
| SECONDARY Change From MRX Baseline to Week 48 in Pruritus |
2.435; 1.307 | < 0.0001 sig |
| SECONDARY Change From MRX Baseline Over Time in Pruritus |
2.435; 0.952 | 0.0307 sig |
| SECONDARY Change From MRX Baseline to Week 48 in Clinician Xanthoma Severity Score |
0.4; 0.2 | — |
| SECONDARY Change From MRX Baseline Over Time in Clinician Xanthoma Severity Score |
0.4; 0.2 | — |
| SECONDARY Secondary: Change From MRX Baseline to Week 48 in Alkaline Phosphatase |
601.5; 596.2 | 0.8863 |
| SECONDARY Change From MRX Baseline Over Time in Alkaline Phosphatase |
601.5; 430.5 | 0.22 |
| SECONDARY Change From MRX Baseline to Week 48 in Alanine Aminotransferase |
130.7; 174.5 | 0.0307 sig |
| SECONDARY Change From MRX Baseline Over Time in Alanine Aminotransferase |
130.7; 175.3 | 0.4934 |
| SECONDARY Change From MRX Baseline to Week 48 in Aspartate Aminotransferase |
127.6; 142.4 | 0.1571 |
| SECONDARY Change From MRX Baseline Over Time in Aspartate Aminotransferase |
127.6; 145.0 | 0.7815 |
| SECONDARY Change From MRX Baseline to Week 48 in Gamma Glutamyltransferase |
476.9; 440.5 | 0.9513 |
| SECONDARY Change From MRX Baseline Over Time in Gamma Glutamyltransferase |
476.9; 377.5 | 0.7133 |
| SECONDARY Change From MRX Baseline to Week 48 in Total and Direct Bilirubin |
4.47; 4.25; 3.80; 3.21 | 0.7839 |
| SECONDARY Change From MRX Baseline Over Time in Total and Direct Bilirubin |
4.47; 5.05; 3.80; 3.53 | 0.8218 |
Summary
The purpose of this extension study is to determine the long-term safety and tolerability of an investigational treatment (LUM001 also known as Maralixibat) in children with ALGS who have completed participation in a core LUM001 treatment protocol. Efficacy will be assessed by evaluating the effect of LUM001 on pruritus, biochemical markers of pruritus, as well as biochemical markers of cholestasis and liver disease.
Eligibility Criteria
Participation for an individual patient is expected to be approximately 72 weeks.
Patients who complete 72 weeks of treatment may be eligible to receive treatment for up to 52 weeks during the follow-up treatment period and patients who completed the 124 weeks of treatment may be eligible to enter the additional long-term follow-up period.
Data sourced from ClinicalTrials.gov (NCT02047318). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.