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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 Pediatric Subjects With Alagille Syndrome (IMAGINE-II)

This study is ongoing, but not recruiting participants.
Sponsor:
ClinicalTrials.gov Identifier:
NCT02117713
First Posted: April 21, 2014
Last Update Posted: April 20, 2017
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.
Collaborators:
Lumena Pharmaceuticals, Inc.
Childhood Liver Disease Research and Education Network
Information provided by (Responsible Party):
Shire
  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: Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Multicenter 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 Shire:

Primary Outcome Measures:
  • Safety and tolerability [ Time Frame: 96 weeks ]
    Evaluate teh long-term safety and tolerability of LUM001 in pediatric subjects with ALGS. This will include Adverse events, changes in vital signs, laboratory and other safety parameters from baseline to week 96


Secondary Outcome Measures:
  • Efficacy 1 [ Time Frame: 96 weeks ]
    Evaluate the long-term effect of LUM001 on serum bile acid levels associated with ALGS from baseline to week 96

  • Efficacy 2 [ Time Frame: 96 weeks ]
    Evaluate the long-term effect of LUM001 on pruritus associated with ALGS from baseline to Week 96

  • Efficacy 3 [ Time Frame: 96 weeks ]
    Explore the long-term effect of LUM001 on other biochemical markers of cholestasis and liver disease

  • Efficacy 4 [ Time Frame: 96 weeks ]
    Evaluate the long-term effect of LUM001 on xanthomas associated with ALGS

  • Dosing [ Time Frame: 96 weeks ]
    Explore expanded dosing range to identify doses necessary to achieve the optimal benefit-to-risk ration for this patient population


Estimated Enrollment: 36
Actual Study Start Date: March 16, 2015
Estimated Study Completion Date: October 19, 2018
Estimated Primary Completion Date: October 19, 2018 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: LUM001
LUM001 administered orally once each day
Drug: LUM001
Administered orally daily

  Eligibility

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.


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

Inclusion Criteria:

  1. Male or female, 12 months to 18 years of age.
  2. Completed participation in the LUM001-301 protocol.
  3. Females of childbearing potential must have a negative urine pregnancy test at the Baseline Visit.
  4. Sexually active females must be prepared to use an effective method of contraception during the trial.
  5. Caregivers (and age appropriate subjects) must have access to phone for scheduled calls from study site.
  6. Caregivers (and age appropriate subjects) must be willing and able to complete a daily electronic diary.

Exclusion Criteria:

  1. Experienced an adverse event or serious adverse event (SAE) related to the study drug during the LUM001-301 protocol that led to the discontinuation of the subject from the core study.
  2. Any conditions or abnormalities which may compromise the safety of the subject, or interfere with the subject participating in or completing the study.
  3. History or presence of gallstones or kidney stones.
  4. History of non-adherence during the subject's participation in the LUM001-301 protocol. Non-adherence is defined by dosing compliance1 of less than 80% in the LUM001-301 protocol.
  Contacts and Locations
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): NCT02117713


Locations
United States, California
Children's Hospital Los Angeles
Los Angeles, California, United States, 90027
University of California at San Francisco Children's Hospital
San Francisco, California, United States, 94143
United States, Colorado
Children's Hospital Colorado
Aurora, Colorado, United States, 80045
United States, Georgia
Children's Healthcare of Atlanta
Atlanta, Georgia, United States, 30322
United States, Illinois
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States, 60611
United States, Indiana
Riley Hospital for Children
Indianapolis, Indiana, United States, 46202
United States, Ohio
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States, 45229
United States, Pennsylvania
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19147
Children's Hospital of Pittsburgh of UPMC
Pittsburgh, Pennsylvania, United States, 15224
United States, Texas
Baylor College of Medicine/Texas Children's Hospital
Houston, Texas, United States, 77030
United States, Utah
University of Utah
Salt Lake City, Utah, United States, 84113
United States, Washington
Seattle Children's Hospital
Seattle, Washington, United States, 98105
Canada, Ontario
The Hospital for Sick Children
Toronto, Ontario, Canada, M5G 1X8
Sponsors and Collaborators
Shire
Lumena Pharmaceuticals, Inc.
Childhood Liver Disease Research and Education Network
Investigators
Study Director: Shire Physician Shire
  More Information

Responsible Party: Shire
ClinicalTrials.gov Identifier: NCT02117713     History of Changes
Other Study ID Numbers: LUM001-305
First Submitted: April 16, 2014
First Posted: April 21, 2014
Last Update Posted: April 20, 2017
Last Verified: April 2017

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