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

This study is ongoing, but not recruiting participants.
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: August 31, 2016
Last verified: August 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: November 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

Locations
United States, California
Children's Hospital Los Angeles
Los Angeles, California, United States, 90027
University of California at San Francisco
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, 19104
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
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: August 31, 2016
Health Authority: United States: Food and Drug Administration
Canada: Health Canada

Additional relevant MeSH terms:
Syndrome
Pruritus
Alagille Syndrome
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 September 23, 2016