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
6. Cognitive impairment of the subject or caregiver that would, in the opinion of the investigator, preclude appropriate understanding of study information and compliance with study procedures
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
6. Cognitive impairment of the subject or caregiver that would, in the opinion of the investigator, preclude appropriate understanding of study information and compliance with study procedures
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 Description
The Score on the ItchRo scale is a score on a 5-point scale from 0 (no itch) to 4 (very severe itch)
Outcome Measure
Mean Change From Baseline Over Time in the Average Morning ItchRO(Obs) Severity Score
Outcome Time Frame
From Baseline to Weeks 75-78
Secondary Outcomes
Outcome Description
Maintenance of treatment effect is defined as the proportion of participants in the MRX-MRX treatment group (MRX-502 and MRX-503 data for subjects on MRX in the MRX-502 study) who obtain an ItchRO (Obs) response from Week 15 to Week 26 in Study MRX-503 in the primary cohort and PFIC cohort.
Outcome Time Frame
Week 15 - 26
Outcome Measure
Maintenance of ItchRO(Obs) Response (Weeks 15 - 26)
Outcome Description
Proportion of ItchRO responders over time at each study visit using the 4-week study period prior to the visit as per ItchRO(Obs) responder definition.
Outcome Time Frame
Baseline to EOT
Outcome Measure
Proportion of ItchRO(Obs) Responders Over Time
Outcome Description
The Score on the ItchRo scale is a score on a 5-point scale from 0 (no itch) to 4 (very severe itch)
Outcome Time Frame
From Baseline to Weeks 75-78
Outcome Measure
Mean Change From Baseline Over Time in the Average Morning ItchRO(Obs) Frequency Score
Outcome Description
Mean change from baseline over time in total serum bile acid (sBA) levels
Outcome Time Frame
Baseline to week 70
Outcome Measure
Mean Change From Baseline Over Time in Total Serum Bile Acid (sBA) Levels
Outcome Description
Proportion of subjects who experience an sBA control over time from Week 18 to Week 26
Outcome Time Frame
From Week 18 to Week 26
Outcome Measure
Proportion of Subjects Who Experience an sBA Control Over Time From Week 18 to Week 26
Outcome Description
Height-for-age Z-score measures a participant's height relative to a reference population of children of the same age and sex. Z-scores were derived using World Health Organization growth charts for participants less than 24 months of age and Centers for Disease Control and Prevention growth charts for participants 24 months of age or older. A Z-score of 0 represents the population mean for age and sex. Positive Z-scores indicate height above the reference population mean, and negative Z-scores indicate height below the reference population mean. Higher Z-scores generally indicate greater linear growth relative to the reference population. Change from baseline was calculated as post-baseline height-for-age Z-score minus baseline height-for-age Z-score.
Outcome Time Frame
From baseline to Week 70
Outcome Measure
Change From Baseline in Height Z-score
Outcome Description
Weight-for-age Z-score measures a participant's weight relative to a reference population of children of the same age and sex. Z-scores were derived using World Health Organization growth charts for participants less than 24 months of age and Centers for Disease Control and Prevention growth charts for participants 24 months of age or older. A Z-score of 0 represents the population mean for age and sex. Positive Z-scores indicate weight above the reference population mean, and negative Z-scores indicate weight below the reference population mean. Higher Z-scores generally indicate greater body weight relative to the reference population. Change from baseline was calculated as post-baseline weight-for-age Z-score minus baseline weight-for-age Z-score.
Outcome Time Frame
From Baseline to Week 70
Outcome Measure
Change From Baseline in Weight Z-score
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
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