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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 (IMAGINE)

This study is currently recruiting participants. (see Contacts and Locations)
Verified April 2014 by Lumena Pharmaceuticals, Inc.
Sponsor:
Collaborator:
Childhood Liver Disease Research and Education Network
Information provided by (Responsible Party):
Lumena Pharmaceuticals, Inc.
ClinicalTrials.gov Identifier:
NCT02047318
First received: January 23, 2014
Last updated: April 16, 2014
Last verified: April 2014
  Purpose

This is a multicentre, extension study of LUM001 in children diagnosed with Alagille Syndrome who have completed participation in a core LUM001 treatment protocol. The primary objective is to evaluate long-term safety and tolerability of LUM001. Efficacy will be assessed by evaluating the effect of LUM001 on the biochemical markers and pruritus associated with Alagille Syndrome.


Condition Intervention Phase
Alagille Syndrome
Drug: LUM001
Phase 2

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: 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

Resource links provided by NLM:


Further study details as provided by Lumena Pharmaceuticals, Inc.:

Primary Outcome Measures:
  • Safety and tolerability [ Time Frame: 48 weeks ] [ Designated as safety issue: Yes ]
    Adverse events, changes in vital signs, laboratory and other safety parameters from baseline to week 48


Secondary Outcome Measures:
  • Efficacy [ Time Frame: 48 weeks ] [ Designated as safety issue: No ]
    Changes in serum bile acids, pruritus, and other biochemical markers of cholestasis and liver disease from baseline to week 48


Estimated Enrollment: 18
Study Start Date: December 2013
Estimated Study Completion Date: December 2015
Estimated Primary Completion Date: June 2015 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: LUM001
LUM001 administered orally once each day
Drug: LUM001

  Eligibility

Ages Eligible for Study:   12 Months to 18 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Completed a core treatment protocol of LUM001 in the treatment of cholestatic liver disease in pediatric subjects with Alagille Syndrome
  2. Ability to understand and willingness to sign informed consent/assent prior to initiation of any study procedures.

Exclusion Criteria:

  1. Experienced an adverse event or serious adverse event related to the study drug during a core LUM001 treatment protocol that led to the discontinuation of the subject from the core LUM001 treatment study
  2. History of non-adherence during the subject's participation in the core LUM001 treatment protocol.
  Contacts and Locations
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, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT02047318

Contacts
Contact: Sara Barbat 858-461-0694 info@lumenapharma.com

Locations
United Kingdom
Birmingham Children's Hospital Recruiting
Birmingham, West Midlands, United Kingdom, B4 6NH
Contact: Carla Lloyd       carla.lloyd@bch.nhs.uk   
Contact: Fazeelat Sharif       fazeelat.sharif@bch.nhs.uk   
Principal Investigator: Deidre Kelly, FRCPI, FRCP,FRCPCH         
Leeds Teaching Hospital Recruiting
Leeds, West Yorkshire, United Kingdom, LS9 7TF
Contact: Nicola Balatoni       nicola.balatoni@leedsth.nhs.uk   
Principal Investigator: Patricia McClean, MD,FRCP,FRCPCH         
Kings College Hospital Recruiting
London, United Kingdom, SE5 9RS
Contact: Palaniswamy Karthikeyan       pkarthikeyan@nhs.net   
Contact: Denise Holley       denise.holley@nhs.net   
Principal Investigator: Alastair Baker, MBChB,FFARCS,MRCP,FRCPCH         
Sponsors and Collaborators
Lumena Pharmaceuticals, Inc.
Childhood Liver Disease Research and Education Network
Investigators
Study Director: Alejandro Dorenbaum, MD Lumena Pharmaceuticals, Inc.
  More Information

Additional Information:
No publications provided

Responsible Party: Lumena Pharmaceuticals, Inc.
ClinicalTrials.gov Identifier: NCT02047318     History of Changes
Other Study ID Numbers: LUM001-303
Study First Received: January 23, 2014
Last Updated: April 16, 2014
Health Authority: United States: Food and Drug Administration
United Kingdom: Medicines and Healthcare Products Regulatory Agency

Additional relevant MeSH terms:
Alagille Syndrome
Cholestasis
Liver Diseases
Syndrome
Abnormalities, Multiple
Bile Duct Diseases
Biliary Tract Diseases
Cardiovascular Abnormalities
Cardiovascular Diseases
Cholestasis, Intrahepatic
Congenital Abnormalities
Digestive System Diseases
Disease
Genetic Diseases, Inborn
Heart Defects, Congenital
Pathologic Processes

ClinicalTrials.gov processed this record on November 25, 2014