Phase 2
N=37
Evaluation of LUM001 in the Reduction of Pruritus in Alagille Syndrome
Alagille Syndrome
Bottom Line
View on ClinicalTrials.gov: NCT02057692 ↗Enrolled (actual)
37
Serious AEs
2.7%
Results posted
Jan 2018
Primary outcome: Primary: Change From Baseline to Endpoint (Week 13/Early Termination) in Pruritus — -1.470; -1.486; -0.620; -0.580 Score on a scale — p=0.0321
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- LUM001 (Drug); Placebo (Drug)
- Age
- Pediatric, Adult · 0+ yrs
- Sex
- All
- Sponsor
- Mirum Pharmaceuticals, Inc.
- Primary completion
- Nov 2016
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Change From Baseline to Endpoint (Week 13/Early Termination) in Pruritus |
-1.470; -1.486; -0.620; -0.580 | 0.0321 sig |
| SECONDARY Change From Baseline to Endpoint (Week 13/Early Termination) in Fasting Serum Bile Acid (sBA) Level |
-117.401; -40.358; -27.437; -10.442 | — |
| SECONDARY Change From Baseline to Endpoint (Week 13/Early Termination) in Liver Enzyme Levels |
14.616; 13.236; 29.539; -11.928; 46.276; -9.570 | — |
| SECONDARY Change From Baseline to Endpoint (Week 13/Early Termination) in Total and Direct Bilirubin Concentrations |
-0.291; -0.353; -0.804; 0.104; -0.333; -0.305 | — |
Summary
The study is a randomized, double-blind, placebo-controlled study in children with Alagille Syndrome (ALGS). The study will investigate the effects of LUM001, compared to placebo, on pruritus, serum bile acids, liver enzymes, and other biochemical markers in patients with ALGS.
Eligibility Criteria
Inclusion Criteria
- Diagnosis of Alagille Syndrome
- Evidence of cholestasis
- Moderate to severe pruritus
- Ability to understand and willingness to sign informed consent/assent prior to initiation of any study procedures
Exclusion Criteria
- Surgical disruption of the enterohepatic circulation
- Liver transplant
- History or presence of other concomitant liver disease
- Females who are pregnant or lactating
- Known HIV infection
Data sourced from ClinicalTrials.gov (NCT02057692). 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.