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Home › Conditions › Alagille Syndrome

Alagille Syndrome

0 published articles · Updated continuously

Clinical Trial Landscape

Clinical Trials for Alagille Syndrome

7 trials tracked for Alagille Syndrome: 1 in phase 3 or 4 and 1 with published results. The most-cited published study has 47 citations.

7Trials tracked
1Phase 3 & 4
0Recruiting
1With published results
Phase distribution
Phase 3 1 Phase 2 6
  1. Phase 3 Efficacy and Safety of Odevixibat in Patients With Alagille Syndrome Completed · 47 cited
  2. Phase 2 Evaluation of LUM001 in the Reduction of Pruritus in Alagille Syndrome Completed
  3. Phase 2 A Study to Evaluate the Safety and Tolerability of Maralixibat in Infant Participants With Cholestatic Liver Diseases Including Progressive Familial Intrahepatic Cholestasis (PFIC) and Alagille Syndrome (ALGS). Completed
  4. Phase 2 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 Completed
  5. Phase 2 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) Completed
  6. Phase 2 Safety and Efficacy Study of LUM001 (Maralixibat) With a Drug Withdrawal Period in Participants With Alagille Syndrome (ALGS) Completed
Show 1 more trials
  1. Phase 2 Safety and Efficacy Study of LUM001 in the Treatment of Cholestatic Liver Disease in Patients With Alagille Syndrome Completed

Showing the 7 most-cited and recently-updated of 7 trials. Browse the full registry →

Trial data sourced from ClinicalTrials.gov. Counts describe the research landscape and are not a treatment recommendation. Informational only — not medical advice.

What the trials found Updated — new results For clinicians

Alagille Syndrome: what the trials found

Odevixibat demonstrated significant reductions in serum bile acid levels (p=0.0006) and significant improvements in scratching scores (p=0.0012) in a Phase 3 trial 2.

Maralixibat (LUM001) has shown significant reductions in fasting serum bile acid (sBA) levels across multiple Phase 2 trials [3, 4]. Specifically, significant reductions were noted at week 18 4 and at week 48 3. However, other Phase 2 trials for LUM001 did not reach statistical significance for sBA reduction at week 13 7 or at weeks 48 and 216 5.

Maralixibat (LUM001) consistently demonstrated significant improvements in pruritus across several Phase 2 trials, including reductions measured by ItchRO 4, patient/observer reports 3, and other pruritus scales [5, 6]. One study evaluating LUM001 did not find a statistically significant change in pruritus scores 7.

Data regarding liver enzymes following LUM001 treatment did not reach statistical significance 7.

Recent results — preliminary, needs further review

  • Maralixibat was evaluated in a Phase 2 trial (N=27), though specific outcome data were not provided in the summary 1.

For the clinician treating this condition

  • Odevixibat is associated with significant reductions in serum bile acid levels and improved scratching scores 2.
  • Maralixibat (LUM001) shows consistent efficacy in reducing pruritus across multiple trials [3, 4, 5, 6].
  • While Maralixibat (LUM001) shows significant reductions in serum bile acid levels in some trials, results are not consistent across all Phase 2 studies [3, 4, 5, 7].

AI synthesis of 6 cited trials, updated Sep 14, 2026. Informational only — not medical advice; trial data sourced from ClinicalTrials.gov. How we use AI.

HCP Mode — summaries include clinical detail, trial data, and statistical outcomes.
Patient Mode — summaries use plain language, avoiding clinical jargon.
No published articles yet for this condition. Check back soon.