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

This study is not yet open for participant recruitment. (see Contacts and Locations)
Verified July 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:
NCT02057692
First received: February 5, 2014
Last updated: July 14, 2014
Last verified: July 2014
  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 Lumena Pharmaceuticals, Inc.:

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: 24
Study Start Date: August 2014
Estimated Study Completion Date: March 2015
Estimated Primary Completion Date: December 2014 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: LUM001
LUM001 for oral administration
Drug: LUM001
Placebo Comparator: Placebo
Placebo administered orally once each day
Drug: Placebo

  Eligibility

Ages Eligible for Study:   2 Years to 18 Years
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: Pamela Wedel 858-461-0694 info@lumenapharma.com

Locations
United States, California
Children's Hospital Los Angeles Not yet recruiting
Los Angeles, California, United States, 90027
Principal Investigator: Nanda Kerkar, M.D.         
University of California at San Francisco Not yet recruiting
San Francisco, California, United States, 94143
Principal Investigator: Philip Rosenthal, MD         
United States, Colorado
Children's Hospital Colorado Not yet recruiting
Aurora, Colorado, United States, 80045
Contact: Michelle Hite       michelle.hite@childrenscolorado.org   
Principal Investigator: Ronald Sokol, MD         
United States, Georgia
Children's Hospital of Atlanta Not yet recruiting
Atlanta, Georgia, United States, 30322
Principal Investigator: Saul Karpen, MD         
United States, Illinois
Ann & Robert H. Lurie Children's Hospital of Chicago Not yet recruiting
Chicago, Illinois, United States, 60611
Principal Investigator: Peter Whitington, M.D.         
United States, Indiana
Riley Hospital for Children Not yet recruiting
Indianapolis, Indiana, United States, 46202
Contact: Ann Klipsch         
Principal Investigator: Jean Molleston, MD         
United States, Ohio
Cincinnati Children's Hospital Medical Center Not yet recruiting
Cincinnati, Ohio, United States, 45229
Principal Investigator: Alexander Miethke, M.D.         
United States, Pennsylvania
The Children's Hospital of Philadelphia Not yet recruiting
Philadelphia, Pennsylvania, United States, 19104
Contact: Jessi Erlichman       ERLICHMAN@email.chop.edu   
Principal Investigator: Kathy Loomes, MD         
Children's Hospital of Pittsburgh Not yet recruiting
Pittsburgh, Pennsylvania, United States, 15224
Principal Investigator: Benjamin Shneider, M.D.         
United States, Washington
Seattle Children's Hospital Not yet recruiting
Seattle, Washington, United States, 98105
Principal Investigator: Karen Murray, MD         
Canada, Ontario
The Hospital for Sick Children Not yet recruiting
Toronto, Ontario, Canada, M5G 1X8
Contact: Claudia Quammie       claudia.quammie@sickkids.ca   
Principal Investigator: Binita Kamath, MD         
Sponsors and Collaborators
Lumena Pharmaceuticals, Inc.
Childhood Liver Disease Research and Education Network
Investigators
Study Director: Alejandro Dorenbaum, M.D. Lumena Pharmaceuticals, Inc.
  More Information

Additional Information:
No publications provided

Responsible Party: Lumena Pharmaceuticals, Inc.
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 14, 2014
Health Authority: United States: Food and Drug Administration

Additional relevant MeSH terms:
Alagille Syndrome
Pruritus
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 August 19, 2014