Adults 1 to 17, any sex, with Progressive Familial Intrahepatic Cholestasis (PFIC). Patients with the condition only — healthy volunteers not accepted.
Results — posted to ClinicalTrials.gov
Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.
Mean Change in the Average Morning ItchRO(Obs) Severity Score in the Primary CohortPrimary· MMRM model was used with inputs of the average changes from baseline (BL) to Weeks 1-6, 7-10, 11-14, 15-18, 19-22, 23-26, and other covariates. The average change from BL over Weeks 15-26 is calculated in the model and presented as a single number.
The primary efficacy endpoint is the mean change in the average morning ItchRO(Obs) severity score between baseline and Weeks 15-26, using 4-week average morning ItchRO(Obs) severity scores (Mixed Model Repeated Measures). The baseline average morning ItchRO(Obs) severity score is defined as the 4-week average morning ItchRO(Obs) severity score prior to the first dose of the study medication.
ItchRo(Obs) Severity Score = Itch Reported Outcome Observer assessment severity score; scale between 0 (not itchy at all) and 4 (extremely itchy); the lower the score the better.
Group
Value
95% CI
Primary Cohort Maralixibat
-1.718
-2.272 – -1.163
Primary Cohort Placebo
-0.628
-1.136 – -0.121
Mean Change in Total sBA Level in the Primary Cohort.Secondary· Baseline and average of Weeks 18, 22 and 26
Mean change in total sBA level between baseline and average of Weeks 18, 22 and 26.
Group
Value
95% CI
Primary Cohort Maralixibat
-175.536
-256.716 – -94.356
Primary Cohort Placebo
11.187
-58.073 – 80.446
Mean Change in the Average Morning ItchRO(Obs) Severity Score in Participants With PFIC (PFIC1, Nt-PFIC2, PFIC3, PFIC4 and PFIC6)Secondary· Between Baseline and Week 15 through Week 26
Mean change in the average morning ItchRO(Obs) severity score between baseline and Week 15 through Week 26 in participants with PFIC (PFIC1, PFIC2, PFIC3, PFIC4 and PFIC6).
ItchRo(Obs) Severity Score = Itch Reported Outcome Observer assessment severity score; scale between 0 (not itchy at all) and 4 (extremely itchy); the lower the score the better.
Group
Value
95% CI
PFIC Cohort Maralixibat
-1.811
-2.178 – -1.444
PFIC Cohort Placebo
-0.610
-1.000 – -0.221
Mean Change in Total sBA Level in Participants With PFIC (PFIC1, PFIC2, PFIC3, PFIC4 and PFIC6)Secondary· Between Baseline and average of Weeks 18, 22 and 26
Mean change in total sBA level between baseline and average of Weeks 18, 22, and 26 in participants with PFIC (PFIC1, PFIC2, PFIC3, PFIC4 and PFIC6)
Group
Value
95% CI
PFIC Cohort Maralixibat
-157.489
-200.276 – -114.703
PFIC Cohort Placebo
2.913
-42.320 – 48.146
Proportion of ItchRO(Obs) Responders in the Primary CohortSecondary· Week 15 to Week 26 using the average value from the three 4-week periods (Weeks 15-18, 19-22 and 23-26)
Proportion of ItchRO(Obs) responders from Week 15 to Week 26 in participants with PFIC2 using the average value from the three 4-week periods (Weeks 15-18, 19-22 and 23-26). The number in the Subject Analysis Set refers to the number of responders.
ItchRO responders are defined as a subject having a 4-week average morning ItchRO(Obs) severity change from baseline of ≤-1.0 or an average severity score of ≤1.0.
Group
Value
95% CI
Primary Cohort Maralixibat
8
Primary Cohort Placebo
4
Proportion of sBA Responders in the Primary CohortSecondary· Average value from Weeks 18, 22 and 26
Proportion of sBA responders from Week 18 to Week 26 in participants with PFIC2, using the average value from Weeks 18, 22 and 26 values. The number in the Subject Analysis Set refers to the number of responders.
sBA responders are defined as a subject having an average sBA level of \<102 umol/L (applies only if baseline sBA level was ≥102 umol/L), OR a ≤-75% average percent change from baseline.
Group
Value
95% CI
Primary Cohort Maralixibat
5
Primary Cohort Placebo
1
Proportion of ItchRO(Obs) Responders PFIC (PFIC1, PFIC2, PFIC3, PFIC4 and PFIC6)Secondary· From Week 15 to Week 26
Proportion of ItchRO(Obs) responders from Week 15 to Week 26 in participants with PFIC (PFIC1, PFIC2, PFIC3, PFIC4 and PFIC6). The number in the Subject Analysis Set refers to the number of responders.
ItchRO responders are defined as a subject having a 4-week average morning ItchRO(Obs) severity change from baseline of ≤-1.0 OR an average severity score of ≤1.0.
Group
Value
95% CI
PFIC Cohort Maralixibat
21
PFIC Cohort Placebo
8
Proportion of sBA Responders PFIC (PFIC1, PFIC2, PFIC3, PFIC4 and PFIC6)Secondary· Week 18 to Week 26
Proportion of sBA responders from Week 18 to Week 26 in participants with PFIC (PFIC1, PFIC2, PFIC3, PFIC4 and PFIC6). The number in the Subject Analysis Set refers to the number of responders.
sBA responders are defined as a subject having an average sBA level of \<102 umol/L (applies only if baseline sBA level was ≥102 umol/L), or a ≤-75% average percent change from baseline.
Group
Value
95% CI
PFIC Cohort Maralixibat
15
PFIC Cohort Placebo
2
Adverse events — posted to ClinicalTrials.gov
Time frame: Baseline to end of treatment (EOT); where EOT can be up to 26 weeks if participant did not early terminate..
Reporting threshold: 5%.
Adverse-event reports describe events observed during the trial — not all are caused by the drug.
The purpose of this study is to determine whether the investigational treatment (maralixibat) is safe and effective in pediatric participants with Progressive Familial Intrahepatic Cholestasis (PFIC).
Publications & conference data
8 peer-reviewed publications reference this trial (live from Europe PMC):
NCT07389031 — Maralixibat for Intrahepatic Cholestasis of Pregnancy
· Phase 2
· not yet recruiting
NCT07293897 — A Database Study of Maralixibat (TAK-625) in Participants With Alagille Syndrome (ALGS) and Progressive Familial Intrahe
· recruiting
NCT04729751 — A Study to Evaluate the Safety and Tolerability of Maralixibat in Infant Participants With Cholestatic Liver Diseases In
· Phase 2
· completed
NCT04524390 — Evaluation of Maralixibat in Biliary Atresia Response Post-Kasai
· Phase 2
· completed
NCT04168385 — MRX-800: A Long-Term Safety Study of Maralixibat in the Treatment of Cholestatic Liver Disease in Subjects Who Previousl
· Phase 2
· completed
Other recruiting trials for Progressive Familial Intrahepatic Cholestasis (PFIC)
Currently open trials in the same condition.
NCT07293897 — A Database Study of Maralixibat (TAK-625) in Participants With Alagille Syndrome (ALGS) and Progressive Familial Intrahe
· recruiting
Other Mirum Pharmaceuticals, Inc. trials
Trials by the same sponsor.
NCT07454837 — Phase 2b/3 Study to Evaluate Switching to Brelovitug for the Treatment of CHD in Participants Receiving Bulevirtide
· Phase 2, PHASE3
· recruiting
NCT07290257 — Long-Term Low-Intervention SafEty and Clinical Outcomes Clinical Study of LivmArli® in Patients With Alagille Syndrome i
· Phase 4
· recruiting
NCT07200908 — A Trial Evaluating Brelovitug (BJT-778) vs Bulevirtide for the Treatment of Chronic Hepatitis Delta Infection (AZURE-2)
· Phase 3
· recruiting
NCT06193928 — Long-Term SafEty and Clinical Outcomes of LivmArli in Patients in the United States (LEAP-US)
· recruiting
NCT04729751 — A Study to Evaluate the Safety and Tolerability of Maralixibat in Infant Participants With Cholestatic Liver Diseases In
· Phase 2
· completed
Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by Mirum Pharmaceuticals, Inc.
Last refreshed: 11 December 2023
Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT03905330.