A Phase 3 interventional study of Maralixibat and Placebo in Progressive Familial Intrahepatic Cholestasis (PFIC), sponsored by Mirum Pharmaceuticals, Inc.. Withdrawn. Open to participants aged Up to 18 Years. Per ClinicalTrials.gov, last updated 2019-03-18.
Sponsored by Mirum Pharmaceuticals, Inc. · Phase 3, Interventional, and Treatment
The purpose of this study is to determine if the investigational treatment (maralixibat) is safe and effective in pediatric participants with Progressive Familial Intrahepatic Cholestasis (PFIC).
33 studies on the registry are indexed under Cholestasis, progressive familial intrahepatic 1; 13 are open to participants now.
Browse Cholestasis, progressive familial intrahepatic 1 studies →Mirum Pharmaceuticals, Inc. is the lead sponsor of 40 studies on the registry; 7 are open to participants now.
Of its 12 completed or terminated interventional studies of FDA-regulated products, 11 (92%) have results posted.
Counted across the registry records on this site, refreshed daily.
Key Inclusion Criteria:
Diagnosis of PFIC based on:
a. Primary cohort: i. Participants with 2 documented mutant alleles in ABCB11 (PFIC2); participants without bile salt export pump (BSEP) function (biallelic truncating mutations in ABCB11) will not be enrolled into the primary cohort. b. Exploratory cohort: i. Participants with PFIC1/3/4 or PFIC2 with biallelic truncating mutationsiii.Infants from birth to \<12 months of age with PFIC ii. Participants with PFIC after internal or external (eg, PEBD) biliary diversion surgery with unsatisfactory pruritus control or where biliary diversion was reversed.
Key Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
Blood tests of how long blood takes to clot. The INR is the prothrombin time (PT) put on a common scale; about 1 is usual for someone not taking a blood thinner.
Criteria often make an exception for people on a blood thinner such as warfarin, whose INR is raised on purpose.
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ULN (upper limit of normal)
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
INR, PT and aPTT (clotting tests)
Blood tests of how long blood takes to clot. The INR is the prothrombin time (PT) put on a common scale; about 1 is usual for someone not taking a blood thinner.
Criteria often make an exception for people on a blood thinner such as warfarin, whose INR is raised on purpose.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Clinically significant
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ALT and AST (liver enzymes)
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
HIV
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Participants will be randomized to Maralixibat oral solution (up to 600 microgram per kilogram \[mcg/kg\]) orally twice daily for 26 weeks.
Drug: Maralixibat
Participants will receive placebo matched to maralixibat oral solution twice daily for 26 weeks.
Drug: Placebo
Maralixibat oral solution (up to 600 mcg/kg) orally twice daily for 26 weeks.
Also known as: SHP625
Placebo matching to maralixibat orally twice daily for 26 weeks.
Treatment Response as Measured by the Observer Itch Reported Outcome (ItchRO[Obs])
Compare the percentage of participants on active treatment versus (vs.) placebo who meet response criteria which is defined as improvement in before midday (AM) Observer Itch Reported Outcome (ItchRO\[Obs\]) severity decrease from baseline demonstrated on at least 2 of the last 3 study visits.
Time frame: Baseline up to Week 26
Treatment Response as Measured by the Observer Itch Reported Outcome (ItchRO[Obs]) and Serum Bile Acids (sBA)
Compare the percentage of participants on active treatment versus (vs.) placebo who meet response criteria which is defined as improvement in average before midday (AM) Observer Itch Reported Outcome (ItchRO\[Obs\]) severity decrease from baseline and normalization or reduction from baseline sBA demonstrated on at least 2 of the last 3 study visits.
Time frame: Baseline up to Week 26
Normalization or Reduction From Baseline in Serum Bile Acids (sBA)
Compare the percentage of participants on active treatment vs. placebo with normalization or significant reduction from baseline in sBA.
Time frame: Baseline up to Week 26
Change Over Time in Daily Average Itch Reported Outcome (ItchRO[Obs]) Score
Change over time in daily average ItchRO scores will be reported.
Time frame: Baseline up to Week 26
Change Over Time in Before Midday (AM) Itch Reported Outcome (ItchRO[Obs]) Score
Change over time in AM ItchRO scores will be reported.
Time frame: Baseline up to Week 26
Change Over Time in After Midday (PM) Itch Reported Outcome (ItchRO[Obs]) Score
Change over time in PM ItchRO scores will be reported.
Time frame: Baseline up to Week 26
Disappearance of Pruritus as Measured by Observer Itch Reported Outcome (ItchRO[Obs])
Compare the percentage of participants on active treatment vs. placebo of participants who experience disappearance of pruritus as measured by ItchRO(Obs).
Time frame: Baseline up to Week 26
Improvement in Height
Number of participants on active treatment vs. placebo with a height z-score change from baseline \>0.
Time frame: Baseline up to Week 26
Improvement in Weight
Number of participants on active treatment vs. placebo with a weight z-score change from baseline \>0.
Time frame: Baseline up to Week 26
Change From Baseline in Nutritional Status as Measured by Mid-arm Circumference
Compare the change in nutritional status as measured by mid-arm circumference in participants on active treatment vs. placebo.
Time frame: Baseline, Week 26
Change From Baseline in Nutritional Status as Measured by Triceps Skin Fold
Compare the change in nutritional status as measured by triceps skin fold in participants on active treatment vs. placebo.
Time frame: Baseline, Week 26
Change From Baseline in Clinician Scratch Scale (CSS)
Compare the change in Clinician Scratch Scale score in participants on active treatment vs. placebo.
Time frame: Baseline, Week 26
Change From Baseline in Quality of Life as Measured by Pediatric Quality of Life Inventory (PedsQL)
Compare the change from baseline of PedsQL in participants on active treatment vs. placebo.
Time frame: Baseline, Week 26
Change From Baseline in Quality of Sleep as Measured by Children's Sleep Habits Questionnaire (CSHQ)
Compare the change from baseline of CSHQ in participants on active treatment vs. placebo.
Time frame: Baseline, Week 26
Normalization or Meaningful Reduction From Baseline of Alanine Aminotransferase (ALT)
Number of participants whose ALT normalizes on treatment or has decreased \>=50%.
Time frame: Baseline up to Week 26
Normalization or Meaningful Decrease From Baseline of Total Bilirubin
Number of participants whose total bilirubin normalizes on treatment or has decreased \>=50%.
Time frame: Baseline up to Week 26
Change From Baseline in Biomarkers of Bile Acid Synthesis
Change from baseline in biomarkers of bile acid synthesis (serum 7 alpha-hydroxy-4-cholesten-3-one \[C4\]).
Time frame: Baseline, Week 26
Evaluate the safety of SHP625
Adverse events, changes in vital signs, laboratory, and other safety parameters will be compared between participants on active treatment vs. placebo.
Time frame: Baseline up to Week 26
Plasma Levels of Maralixibat Over Time
Systemic concentrations of maralixibat in plasma will be assessed.
Time frame: Baseline, Week 6, 10, 14, 18, 22 and 26
No study locations are listed for this record.
Plan to share: No
No publications or documents are linked to this record.
This study is withdrawn, as verified in Mar 2019. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: Maralixibat
Cholestasis, progressive familial intrahepatic 1 →