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
|Condition or disease||Intervention/treatment||Phase|
|Alagille Syndrome||Drug: LUM001||Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||19 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|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|
LUM001 administered orally up to twice each day
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).
- 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.
- 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.
- 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.
- 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.
- 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.
- 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.
- 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.
- 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.
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
|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|
|Study Director:||Study Director||Mirum|