Working…
COVID-19 is an emerging, rapidly evolving situation.
Get the latest public health information from CDC: https://www.coronavirus.gov.

Get the latest research information from NIH: https://www.nih.gov/coronavirus.
ClinicalTrials.gov
ClinicalTrials.gov Menu
Trial record 1 of 8 for:    pfic
Previous Study | Return to List | Next Study

An Extension Study of Maralixibat in Patients With Progressive Familial Intrahepatic Cholestasis (PFIC)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04185363
Recruitment Status : Enrolling by invitation
First Posted : December 4, 2019
Last Update Posted : January 13, 2020
Sponsor:
Information provided by (Responsible Party):
Mirum Pharmaceuticals, Inc.

Brief Summary:
The primary objective of this open label extension study is to evaluate the long-term safety and tolerability of maralixibat.

Condition or disease Intervention/treatment Phase
Progressive Familial Intrahepatic Cholestasis (PFIC) Drug: Maralixibat Phase 3

Detailed Description:
The study will be conducted at multiple sites in North America, Europe, Asia, and South America.

Layout table for study information
Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 30 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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)
Actual Study Start Date : January 8, 2020
Estimated Primary Completion Date : November 2022
Estimated Study Completion Date : December 2022


Arm Intervention/treatment
Experimental: Maralixibat
All subjects will receive Maralixibat oral solution
Drug: Maralixibat
All subjects will receive Maralixibat oral solution (up to 600 microgram per kilogram [mcg/kg]) twice daily for up to 104 weeks




Primary Outcome Measures :
  1. Incidence of Treatment Emergent Adverse Events (TEAEs) during the study [ Time Frame: From baseline through study completion, up to 104 weeks ]

Secondary Outcome Measures :
  1. Change from maralixibat baseline over the course of the study in the weekly average morning ItchRO(Obs)™ (Itch Reported Outcome) severity score [ Time Frame: From baseline through study completion,up to 104 weeks ]
  2. Change from maralixibat baseline over the course of the study in the weekly average morning ItchRO(Obs)™ (Itch Reported Outcome) frequency score [ Time Frame: From baseline through study completion,up to 104 weeks ]
  3. Mean change from baseline over time in serum bile acid (sBA) levels [ Time Frame: From baseline through study completion,up to 104 weeks ]
  4. Mean change from baseline over time in height and weight z-scores [ Time Frame: From baseline through study completion,up to 104 weeks ]


Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Layout table for eligibility information
Ages Eligible for Study:   1 Year to 18 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04185363


Locations
Layout table for location information
United States, Pennsylvania
Children Hospital of Pittsburgh
Pittsburgh, Pennsylvania, United States, 15224
Sponsors and Collaborators
Mirum Pharmaceuticals, Inc.

Additional Information:
Layout table for additonal information
Responsible Party: Mirum Pharmaceuticals, Inc.
ClinicalTrials.gov Identifier: NCT04185363    
Other Study ID Numbers: MRX-503
First Posted: December 4, 2019    Key Record Dates
Last Update Posted: January 13, 2020
Last Verified: January 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Layout table for additional information
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Mirum Pharmaceuticals, Inc.:
Cholestasis
Maralixibat
Mutation
PFIC
PFIC2
Bile Duct Diseases
Liver Diseases
Biliary Tract Diseases
Digestive System Diseases
Pediatric
Additional relevant MeSH terms:
Layout table for MeSH terms
Cholestasis
Cholestasis, Intrahepatic
Bile Duct Diseases
Biliary Tract Diseases
Digestive System Diseases
Liver Diseases