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

An Extension Study to Evaluate the Long-Term Safety and Durability of Effect of LUM001 in the Treatment of Cholestatic Liver Disease in Subjects With Alagille Syndrome (ALGS) (IMAGINE)

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: NCT02047318
Recruitment Status : Completed
First Posted : January 28, 2014
Last Update Posted : June 19, 2020
Sponsor:
Information provided by (Responsible Party):
Mirum Pharmaceuticals, Inc.

Brief Summary:
The purpose of this extension study is to determine the long-term safety and tolerability of an investigational treatment (LUM001) in children with ALGS who have completed participation in a core LUM001 treatment protocol. Efficacy will be assessed by evaluating the effect of LUM001 on pruritus, biochemical markers of pruritus, as well as biochemical markers of cholestasis and liver disease.

Condition or disease Intervention/treatment Phase
Alagille Syndrome Drug: LUM001 Phase 2

Layout table for study information
Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 19 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Multicentre Extension Study to Evaluate the Long-Term Safety and Durability of the Therapeutic Effect of LUM001, an Apical Sodium-Dependent Bile Acid Transporter Inhibitor (ASBTi), in the Treatment of Cholestatic Liver Disease in Pediatric Subjects With Alagille Syndrome
Actual Study Start Date : December 20, 2013
Actual Primary Completion Date : June 17, 2020
Actual Study Completion Date : June 17, 2020

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Liver Diseases

Arm Intervention/treatment
Experimental: LUM001
LUM001 administered orally up to twice each day
Drug: LUM001
Dosing of LUM001 with the objective of achieving optimal control of pruritus at a dose level that is tolerated by the participant and up to a maximum daily dose of 560 micrograms per kilogram (mcg/kg).




Primary Outcome Measures :
  1. Number of Participants with Clinically Significant Changes in Vital Signs Reported as Adverse Events [ Time Frame: Up to 72 Weeks ]
    Clinically significant changes in vital signs (body temperature, blood pressure, respiration and pulse) will be assessed from baseline to week 72.

  2. Number of Participants with Clinically Significant Changes in Laboratory Parameters Reported as Adverse Events [ Time Frame: Up to 72 Weeks ]
    Clinically significant changes in laboratory parameters (clinical chemistry, fasting lipid panel and determination of fat-soluble vitamins, bile acids and other cholestasis biochemical markers) will be assessed from baseline to week 72.


Secondary Outcome Measures :
  1. Evaluate the Long-term Efficacy of LUM001 on Serum Bile Acid Levels [ Time Frame: Up to 72 Weeks ]
    Mean change from baseline in serum bile acids will be assessed.

  2. Long term Effect of LUM001 on Pruritus Measured by ItchRO Patient Reported Pruritus Instrument [ Time Frame: Up to 72 Weeks ]
    The ItchRO will be completed using an electronic diary (eDiary) twice daily (morning and evening). Change from baseline will be assessed.

  3. Long term effect on xanthomas associated with ALGS as measured by clinician xanthoma scale [ Time Frame: Up to 72 Weeks ]
    The degree and severity of xanthomatosis will be evaluated for all participants.

  4. Long term Effect on Biochemical Markers of Cholestasis and Liver Disease [ Time Frame: Up to 72 Weeks ]
    Blood and urine samples will be taken for clinical laboratory testing. Changes in ALT (alanine aminotransferase), GGT (gamma glutamyltransferase), and total bilirubin will be assessed.


Other Outcome Measures:
  1. Assessment of Alpha-fetoprotein (AFP), a Marker of Hepatocellular Carcinoma [ Time Frame: 52 Weeks ]
    Alpha-fetoprotein (AFP), a marker of hepatocellular carcinoma, will be measured every 6 months throughout optional follow-up period.

  2. Assessment of Palatability of LUM-001 [ Time Frame: 52 Weeks ]
    Palatability will be assessed by the study participant and or caregiver. Assessments over time will be evaluated.



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:   12 Months to 18 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Participation for an individual patient is expected to be approximately 72 weeks.

Patients who complete 72 weeks of treatment may be eligible to receive treatment for up to 52 weeks during the follow-up treatment period. Planned participation for each patient enrolled in the optional follow-up treatment study is 128 weeks, inclusive of a 4-week follow-up phone call.


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): NCT02047318


Locations
Layout table for location information
United Kingdom
Birmingham Children's Hospital
Birmingham, West Midlands, United Kingdom, B4 6NH
Leeds Teaching Hospital NHS Trust
Leeds, West Yorkshire, United Kingdom, LS1 3EX
Kings College Hospital
London, United Kingdom, SE5 9RS
Sponsors and Collaborators
Mirum Pharmaceuticals, Inc.
Investigators
Layout table for investigator information
Study Director: Study Director Mirum
Layout table for additonal information
Responsible Party: Mirum Pharmaceuticals, Inc.
ClinicalTrials.gov Identifier: NCT02047318    
Other Study ID Numbers: LUM001-303
2013-003832-54 ( EudraCT Number )
First Posted: January 28, 2014    Key Record Dates
Last Update Posted: June 19, 2020
Last Verified: June 2020

Layout table for additional information
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
Layout table for MeSH terms
Liver Diseases
Alagille Syndrome
Syndrome
Disease
Pathologic Processes
Digestive System Diseases
Cholestasis, Intrahepatic
Cholestasis
Bile Duct Diseases
Biliary Tract Diseases
Heart Defects, Congenital
Cardiovascular Abnormalities
Cardiovascular Diseases
Abnormalities, Multiple
Congenital Abnormalities
Genetic Diseases, Inborn