Evaluation of LUM001 in the Reduction of Pruritus in Alagille Syndrome (ITCH)

This study is currently recruiting participants. (see Contacts and Locations)
Verified July 2016 by Shire
Sponsor:
Collaborator:
Childhood Liver Disease Research and Education Network
Information provided by (Responsible Party):
Shire
ClinicalTrials.gov Identifier:
NCT02057692
First received: February 5, 2014
Last updated: July 20, 2016
Last verified: July 2016
  Purpose
The study is a randomized, double-blind, placebo-controlled study in children with Alagille Syndrome (ALGS). The study will investigate the effects of LUM001, compared to placebo, on pruritus, serum bile acids, liver enzymes, and other biochemical markers in patients with ALGS.

Condition Intervention Phase
Alagille Syndrome
Drug: LUM001
Drug: Placebo
Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, Investigator)
Primary Purpose: Treatment
Official Title: The Evaluation of the Intestinal Bile Acid Transport (IBAT) Inhibitor LUM001 in the Reduction of Pruritus in Alagille Syndrome, a Cholestatic Liver Disease

Resource links provided by NLM:


Further study details as provided by Shire:

Primary Outcome Measures:
  • Efficacy [ Time Frame: 13 weeks ] [ Designated as safety issue: No ]
    Change in pruritus compared to placebo from baseline to Week 13


Secondary Outcome Measures:
  • Efficacy [ Time Frame: 13 weeks ] [ Designated as safety issue: No ]
    Change in fasting serum bile acids and liver enzymes compared to placebo from baseline to Week 13


Other Outcome Measures:
  • Safety and Tolerability [ Time Frame: 13 Weeks ] [ Designated as safety issue: Yes ]
    Adverse events, changes in vital signs, laboratory and other safety parameters from baseline to week 13


Estimated Enrollment: 36
Study Start Date: November 2014
Estimated Study Completion Date: December 2016
Estimated Primary Completion Date: October 2016 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: LUM001
LUM001 for oral administration
Drug: LUM001
LUM001 administered orally
Placebo Comparator: Placebo
Placebo administered orally once each day
Drug: Placebo
Placebo administered orally

  Eligibility

Ages Eligible for Study:   12 Months to 18 Years   (Child, Adult)
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Diagnosis of Alagille Syndrome
  2. Evidence of cholestasis
  3. Moderate to severe pruritus
  4. Ability to understand and willingness to sign informed consent/assent prior to initiation of any study procedures

Exclusion Criteria:

  1. Surgical disruption of the enterohepatic circulation
  2. Liver transplant
  3. History or presence of other concomitant liver disease
  4. Females who are pregnant or lactating
  5. Known HIV infection
  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: NCT02057692

Contacts
Contact: Shire Contact clinicaltransparency@shire.com

Locations
United States, California
Children's Hospital Los Angeles Recruiting
Los Angeles, California, United States, 90027
Contact: Cat Goodhue    323-361-4566    cgoodhue@chla.usc.edu   
University of California at San Francisco Not yet recruiting
San Francisco, California, United States, 94143
United States, Colorado
Children's Hospital Colorado Recruiting
Aurora, Colorado, United States, 80045
Contact: Julia Smith    720-777-6669    julia.smith@childrenscolorado.org   
United States, Georgia
Children's Healthcare of Atlanta Recruiting
Atlanta, Georgia, United States, 30322
Contact: Dana Hankerson-Dyson    404-785-6027    dana.hankerson-dyson@choa.org   
United States, Illinois
Ann & Robert H. Lurie Children's Hospital of Chicago Recruiting
Chicago, Illinois, United States, 60611
Contact: Elizabeth Kaurs       ekaurs@luriechildrens.org   
United States, Indiana
Riley Hospital for Children Recruiting
Indianapolis, Indiana, United States, 46202
Contact: Ann Klipsch    317-944-9605    aeye@iu.edu   
United States, Ohio
Cincinnati Children's Hospital Medical Center Recruiting
Cincinnati, Ohio, United States, 45229
Contact: Andrea Ferris    513-803-0675    andrea.ferris@cchmc.org   
United States, Pennsylvania
The Children's Hospital of Philadelphia Recruiting
Philadelphia, Pennsylvania, United States, 19104
Contact: Lindsay Brown    267-426-7223    BrownL7@email.chop.edu   
Children's Hospital of Pittsburgh of UPMC Recruiting
Pittsburgh, Pennsylvania, United States, 15224
Contact: Kathryn Bukauskas    412-478-8294    kathryn.bukauskas@chp.edu   
United States, Texas
Baylor College of Medicine/Texas Children's Hospital Recruiting
Houston, Texas, United States, 77030
Contact: Loriel Liwanag    832-824-3848    loriel.liwanag@bcm.edu   
United States, Utah
University of Utah Recruiting
Salt Lake City, Utah, United States, 84113
Contact: Ann Rutherford    801-585-9495    ann.rutherford@hsc.utah.edu   
United States, Washington
Seattle Children's Hospital Recruiting
Seattle, Washington, United States, 98105
Contact: Melissa Young    206-987-1037    melissa.young@seattlechildrens.org   
Canada, Ontario
The Hospital for Sick Children Recruiting
Toronto, Ontario, Canada, M5G 1X8
Contact: Claudia Quammie    416-813-7654    claudia.quammie@sickkids.ca   
Sponsors and Collaborators
Shire
Childhood Liver Disease Research and Education Network
Investigators
Study Director: Shire Study Physician Shire
  More Information

Responsible Party: Shire
ClinicalTrials.gov Identifier: NCT02057692     History of Changes
Obsolete Identifiers: NCT02055768
Other Study ID Numbers: LUM001-301 
Study First Received: February 5, 2014
Last Updated: July 20, 2016
Health Authority: United States: Food and Drug Administration
Canada: Health Canada

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

ClinicalTrials.gov processed this record on July 21, 2016