Brief Summary
The primary objective of this open label extension study is to evaluate the long-term safety and tolerability of maralixibat.
Brief Title
An Extension Study of Maralixibat in Patients With Progressive Familial Intrahepatic Cholestasis (PFIC)
Detailed Description
The study will be conducted at multiple sites in North America, Europe, Asia, and South America.
Categories
Completion Date
Completion Date Type
Actual
Conditions
Progressive Familial Intrahepatic Cholestasis (PFIC)
Eligibility Criteria
Key Inclusion Criteria:
1. Provide informed consent and assent (as applicable) per Institutional Review Board/Ethics Committee (IRB/EC)
2. Completion of study MRX-502
Exclusion Criteria:
1. Any female who is pregnant or lactating or who is planning to become pregnant
2. Administration of prohibited medication between the MRX-502 EOT visit and the MRX 503 Baseline Visit (Day 0)
3. History of non-compliance in study MRX-502, non-adherence to medical regimens, unreliability, mental instability or incompetence that could compromise the validity of informed consent or lead to non-adherence with the study protocol based on Investigator judgment
4. Experienced an adverse event (AE) or serious adverse event (SAE) related to maralixibat during the MRX-502 study that led to permanent discontinuation of the subject from maralixibat
5. Any other conditions or laboratory abnormalities that, in the opinion of the Investigator or Sponsor Medical Monitor, may compromise the safety of the subject, or interfere with the subject participating in or completing the study
1. Provide informed consent and assent (as applicable) per Institutional Review Board/Ethics Committee (IRB/EC)
2. Completion of study MRX-502
Exclusion Criteria:
1. Any female who is pregnant or lactating or who is planning to become pregnant
2. Administration of prohibited medication between the MRX-502 EOT visit and the MRX 503 Baseline Visit (Day 0)
3. History of non-compliance in study MRX-502, non-adherence to medical regimens, unreliability, mental instability or incompetence that could compromise the validity of informed consent or lead to non-adherence with the study protocol based on Investigator judgment
4. Experienced an adverse event (AE) or serious adverse event (SAE) related to maralixibat during the MRX-502 study that led to permanent discontinuation of the subject from maralixibat
5. Any other conditions or laboratory abnormalities that, in the opinion of the Investigator or Sponsor Medical Monitor, may compromise the safety of the subject, or interfere with the subject participating in or completing the study
Inclusion Criteria
Inclusion Criteria:
1. Provide informed consent and assent (as applicable) per Institutional Review Board/Ethics Committee (IRB/EC)
2. Completion of study MRX-502
1. Provide informed consent and assent (as applicable) per Institutional Review Board/Ethics Committee (IRB/EC)
2. Completion of study MRX-502
Gender
All
Gender Based
false
Keywords
Cholestasis
Maralixibat
Mutation
PFIC
PFIC2
Bile Duct Diseases
Liver Diseases
Biliary Tract Diseases
Digestive System Diseases
Pediatric
Healthy Volunteers
No
Last Update Post Date
Last Update Post Date Type
Actual
Last Update Submit Date
Maximum Age
18 Years
Minimum Age
1 Year
NCT Id
NCT04185363
Org Class
Industry
Org Full Name
Mirum Pharmaceuticals, Inc.
Org Study Id
MRX-503
Overall Status
Completed
Phases
Phase 3
Primary Completion Date
Primary Completion Date Type
Actual
Official Title
An Open-label Extension Study to Evaluate the Long-term Safety and Efficacy of Maralixibat in the Treatment of Subjects With Progressive Familial Intrahepatic Cholestasis (PFIC)
Primary Outcomes
Outcome Measure
Incidence of Treatment Emergent Adverse Events (TEAEs) during the study
Outcome Time Frame
From baseline through time of interim analysis, up to 120 weeks
Secondary Outcomes
Outcome Time Frame
From baseline through study completion, up to approximately 4 years
Outcome Measure
Change from maralixibat baseline over the course of the study in the weekly average morning ItchRO(Obs)™ (Itch Reported Outcome) severity score
Outcome Time Frame
From baseline through study completion, up to approximately 4 years
Outcome Measure
Maintenance of treatment effect based on the average morning ItchRO(Obs)™ severity scores over the time
Outcome Time Frame
From baseline through study completion, up to approximately 4 years
Outcome Measure
Mean change from baseline over time in serum bile acid (sBA) levels
Outcome Time Frame
From baseline through study completion, up to approximately 4 years
Outcome Measure
Change from maralixibat baseline over the course of the study in the weekly average morning ItchRO(Obs)™ (Itch Reported Outcome) frequency score
Outcome Time Frame
From baseline through study completion, up to approximately 4 years
Outcome Measure
Mean change from baseline over time in height and weight z-scores
See Also Links
Start Date
Start Date Type
Actual
Status Verified Date
First Post Date
First Post Date Type
Actual
First Submit Date
First Submit QC Date
Std Ages
Child
Adult
Maximum Age Number (converted to Years and rounded down)
18
Minimum Age Number (converted to Years and rounded down)
1
Investigators
Investigator Type
Principal Investigator
Investigator Name
Nadia Ovchinsky
Investigator Email
NOVCHINS@montefiore.org
Investigator Phone
Categories Mesh Debug
Digestive System --- CHOLESTASIS
Liver --- CHOLESTASIS
Digestive System --- BILE DUCT DISEASES
Liver --- BILE DUCT DISEASES
Digestive System --- LIVER DISEASES
Liver --- LIVER DISEASES
Digestive System --- BILIARY TRACT DISEASES
Liver --- BILIARY TRACT DISEASES
Gastrointestinal (GI) Cancers --- DIGESTIVE SYSTEM DISEASES
Digestive System --- DIGESTIVE SYSTEM DISEASES
Liver --- DIGESTIVE SYSTEM DISEASES
MeSH Terms
CHOLESTASIS, PROGRESSIVE FAMILIAL INTRAHEPATIC 1
CHOLESTASIS
BILE DUCT DISEASES
LIVER DISEASES
BILIARY TRACT DISEASES
DIGESTIVE SYSTEM DISEASES
MARALIXIBAT