Trial · NCT03905330
A Study to Evaluate the Efficacy and Safety of Maralixibat in Subjects With Progressive Familial Intrahepatic Cholestasis (MARCH-PFIC)
NCT03905330, Phase 3, Completed, Mirum Pharmaceuticals, Inc.; results first posted 2023-12-11
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. A Study to Evaluate the Efficacy and Safety of Maralixibat in Subjects With Progressive Familial Intrahepatic Cholestasis (MARCH-PFIC). Trials, record NCT03905330. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03905330
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Study to Evaluate the Efficacy and Safety of Maralixibat in Subjects With Progressive Familial Intrahepatic Cholestasis (MARCH-PFIC)
- NCT number
- NCT03905330
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 93 participants, actual
- Lead sponsor
- Mirum Pharmaceuticals, Inc.
- Conditions
- Progressive Familial Intrahepatic Cholestasis (PFIC)
- Interventions
- Maralixibat, Placebo
- First posted
- 2019-04-05
- Last updated
- 2023-12-11
- Other registries' IDs
- eudract:2019-001211-22
- Results first posted
- 2023-12-11
Source ClinicalTrials.gov
Registered outcomes8
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Mean Change in the Average Morning ItchRO(Obs) Severity Score in the Primary Cohort | 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. |
| Secondary | Mean Change in Total sBA Level in the Primary Cohort. | 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. |
| Secondary | Mean Change in the Average Morning ItchRO(Obs) Severity Score in Participants With PFIC (PFIC1, Nt-PFIC2, PFIC3, PFIC4 and PFIC6) | 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. |
| Secondary | Mean Change in Total sBA Level in Participants With PFIC (PFIC1, PFIC2, PFIC3, PFIC4 and PFIC6) | 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) |
| Secondary | Proportion of ItchRO(Obs) Responders in the Primary Cohort | 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. |
| Secondary | Proportion of sBA Responders in the Primary Cohort | 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. |
| Secondary | Proportion of ItchRO(Obs) Responders PFIC (PFIC1, PFIC2, PFIC3, PFIC4 and PFIC6) | 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. |
| Secondary | Proportion of sBA Responders PFIC (PFIC1, PFIC2, PFIC3, PFIC4 and PFIC6) | 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. |
Source ClinicalTrials.gov
Outcomes8
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Mean Change in the Average Morning ItchRO(Obs) Severity Score in the Primary Cohort | 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. | Primary Cohort, defined as participants with genetic testing results consistent with biallelic disease causing variation in ABCB11 (PFIC2, also referred to as BSEP deficiency), based on standard-of-care genotyping, excluding those PFIC2 participants predicted to have complete absence of BSEP function (PFIC2 participants with heterozygosis, biliary diversion, having low or fluctuating sBA levels). | Score on a scale | LEAST_SQUARES_MEAN |
| SECONDARY | Mean Change in Total sBA Level in the Primary Cohort. | Baseline and average of Weeks 18, 22 and 26 | Primary Cohort, defined as participants with genetic testing results consistent with biallelic disease causing variation in ABCB11 (PFIC2, also referred to as BSEP deficiency), based on standard-of-care genotyping, excluding those PFIC2 participants predicted to have complete absence of BSEP function (PFIC2 participants with heterozygosis, biliary diversion, having low or fluctuating sBA levels). Two maralixibat participants did not have baseline sBA values and were not included in the analysis. | μmol/L | LEAST_SQUARES_MEAN |
| SECONDARY | Mean Change in the Average Morning ItchRO(Obs) Severity Score in Participants With PFIC (PFIC1, Nt-PFIC2, PFIC3, PFIC4 and PFIC6) | Between Baseline and Week 15 through Week 26 | Primary cohort plus PFIC1, PFIC3, PFIC4, PFIC6. | score on a scale | LEAST_SQUARES_MEAN |
| SECONDARY | Mean Change in Total sBA Level in Participants With PFIC (PFIC1, PFIC2, PFIC3, PFIC4 and PFIC6) | Between Baseline and average of Weeks 18, 22 and 26 | Primary cohort plus PFIC1, PFIC3, PFIC4 and PFIC6. | μmol/L | LEAST_SQUARES_MEAN |
| SECONDARY | Proportion of ItchRO(Obs) Responders in the Primary Cohort | Week 15 to Week 26 using the average value from the three 4-week periods (Weeks 15-18, 19-22 and 23-26) | Primary Cohort, defined as participants with genetic testing results consistent with biallelic disease causing variation in ABCB11 (PFIC2, also referred to as BSEP deficiency), based on standard-of-care genotyping, excluding those PFIC2 participants predicted to have complete absence of BSEP function (PFIC2 participants with heterozygosis, biliary diversion, having low or fluctuating sBA levels). | Number of responders | NUMBER |
| SECONDARY | Proportion of sBA Responders in the Primary Cohort | Average value from Weeks 18, 22 and 26 | Primary Cohort, defined as participants with genetic testing results consistent with biallelic disease causing variation in ABCB11 (PFIC2, also referred to as BSEP deficiency), based on standard-of-care genotyping, excluding those PFIC2 participants predicted to have complete absence of BSEP function (PFIC2 participants with heterozygosis, biliary diversion, having low or fluctuating sBA levels). | Number of responders | NUMBER |
| SECONDARY | Proportion of ItchRO(Obs) Responders PFIC (PFIC1, PFIC2, PFIC3, PFIC4 and PFIC6) | From Week 15 to Week 26 | Primary cohort plus PFIC1, PFIC3, PFIC4 and PFIC6. | Number of responders | NUMBER |
| SECONDARY | Proportion of sBA Responders PFIC (PFIC1, PFIC2, PFIC3, PFIC4 and PFIC6) | Week 18 to Week 26 | Primary cohort plus PFIC1, PFIC3, PFIC4 and PFIC6. | Number of responders | NUMBER |
Source ClinicalTrials.gov
Results by group16
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Mean Change in the Average Morning ItchRO(Obs) Severity Score in the Primary Cohort | Primary Cohort Maralixibat | -1.718 | 95% Confidence Interval -2.272 to -1.163 | Score on a scale | |
| Mean Change in the Average Morning ItchRO(Obs) Severity Score in the Primary Cohort | Primary Cohort Placebo | -0.628 | 95% Confidence Interval -1.136 to -0.121 | Score on a scale | |
| Mean Change in Total sBA Level in the Primary Cohort. | Primary Cohort Maralixibat | -175.536 | 95% Confidence Interval -256.716 to -94.356 | μmol/L | |
| Mean Change in Total sBA Level in the Primary Cohort. | Primary Cohort Placebo | 11.187 | 95% Confidence Interval -58.073 to 80.446 | μmol/L | |
| Mean Change in the Average Morning ItchRO(Obs) Severity Score in Participants With PFIC (PFIC1, Nt-PFIC2, PFIC3, PFIC4 and PFIC6) | PFIC Cohort Maralixibat | -1.811 | 95% Confidence Interval -2.178 to -1.444 | score on a scale | |
| Mean Change in the Average Morning ItchRO(Obs) Severity Score in Participants With PFIC (PFIC1, Nt-PFIC2, PFIC3, PFIC4 and PFIC6) | PFIC Cohort Placebo | -0.610 | 95% Confidence Interval -1.000 to -0.221 | score on a scale | |
| Mean Change in Total sBA Level in Participants With PFIC (PFIC1, PFIC2, PFIC3, PFIC4 and PFIC6) | PFIC Cohort Maralixibat | -157.489 | 95% Confidence Interval -200.276 to -114.703 | μmol/L | |
| Mean Change in Total sBA Level in Participants With PFIC (PFIC1, PFIC2, PFIC3, PFIC4 and PFIC6) | PFIC Cohort Placebo | 2.913 | 95% Confidence Interval -42.320 to 48.146 | μmol/L | |
| Proportion of ItchRO(Obs) Responders in the Primary Cohort | Primary Cohort Maralixibat | 8 | Number of responders | ||
| Proportion of ItchRO(Obs) Responders in the Primary Cohort | Primary Cohort Placebo | 4 | Number of responders |
1 to 10 of 16Source ClinicalTrials.gov
See all 16 results by groupParticipant flow14
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Maralixibat | 47 | |
| Overall Study | STARTED | Placebo | 46 | |
| Overall Study | Dosed 150 ug/kg BID | Maralixibat | 47 | |
| Overall Study | Dosed 150 ug/kg BID | Placebo | 0 | |
| Overall Study | Dosed 300 ug/kg BID | Maralixibat | 46 | |
| Overall Study | Dosed 300 ug/kg BID | Placebo | 0 | |
| Overall Study | Dosed 450 ug/kg BID | Maralixibat | 45 | |
| Overall Study | Dosed 450 ug/kg BID | Placebo | 0 | |
| Overall Study | Dosed 600 ug/kg BID | Maralixibat | 45 | |
| Overall Study | Dosed 600 ug/kg BID | Placebo | 0 |
1 to 10 of 14Source ClinicalTrials.gov
See all 14 participant flowAdverse events64
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Blood bilirubin increased | Investigations | Maralixibat | 7 | 47 | 7 |
| other | Blood bilirubin increased | Investigations | Placebo | 4 | 46 | 4 |
| other | Alanine aminotransferase increased | Investigations | Maralixibat | 6 | 47 | 7 |
| other | Alanine aminotransferase increased | Investigations | Placebo | 3 | 46 | 3 |
| other | Vitamin E decreased | Investigations | Maralixibat | 4 | 47 | 4 |
| other | Vitamin E decreased | Investigations | Placebo | 3 | 46 | 3 |
| other | Vitamin D decreased | Investigations | Maralixibat | 4 | 47 | 4 |
| other | Vitamin D decreased | Investigations | Placebo | 2 | 46 | 2 |
| other | International normalised ratio increased | Investigations | Maralixibat | 1 | 47 | 1 |
| other | International normalised ratio increased | Investigations | Placebo | 4 | 46 | 5 |
1 to 10 of 64Source ClinicalTrials.gov
See all 64 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Maralixibat | 0 | 47 |
| Total, all-cause mortality | Placebo | 0 | 46 |
| Total, other adverse events | Maralixibat | 47 | 47 |
| Total, other adverse events | Placebo | 43 | 46 |
| Total, serious adverse events | Maralixibat | 5 | 47 |
| Total, serious adverse events | Placebo | 3 | 46 |
Source ClinicalTrials.gov
Papers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 38723644 | DERIVED |
Source PubMed
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons2
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
| Maralixibat vs Placebo: deaths from any cause | Trial comparisons |
| Maralixibat vs Placebo: serious adverse events | Trial comparisons |
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT03905330
GET https://conductscience.org/api/trials/v1/NCT03905330/events?page=2