Phase 2
Completed N=20
Safety and Efficacy Study of LUM001 in the Treatment of Cholestatic Liver Disease in Patients With Alagille Syndrome
Alagille Syndrome
Source: ClinicalTrials.gov NCT01903460 ↗
Enrolled (actual)
20
Serious AEs
0.0%
Results posted
Dec 2015
Primary outcomePrimary: Change From Baseline to Week 13 (End of Treatment) in Fasting Serum Bile Acid Level — -82.864; -49.388; -66.126; -42.157 umol/L — p=0.5740
Summary
The study is a randomized, double-blind, placebo-controlled study to evaluate the safety and tolerability of LUM001. Efficacy will be assessed by evaluating the effect of LUM001 versus placebo on the biochemical markers and pruritus associated with Alagille Syndrome.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Change From Baseline to Week 13 (End of Treatment) in Fasting Serum Bile Acid Level |
-82.864; -49.388; -66.126; -42.157 | 0.5740 |
| SECONDARY Change From Baseline to Week 13 (End of Treatment) in Liver Enzymes |
59.3; 10.5; 34.9; 2.7; 37.2; -2.7 | 0.0783 |
| SECONDARY Change From Baseline to Week 13 (End of Treatment) in Pruritus as Measured by The Patient And Observer Itch Reported Outcome (ItchRO) Average Daily Scores |
-1.159; -0.608; -0.883; -0.811; -0.802; -0.419 | 0.6907 |
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 (NCT01903460). 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. Informational only — not medical advice.