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

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ClinicalTrials.gov Identifier: NCT02057692
Recruitment Status : Completed
First Posted : February 7, 2014
Results First Posted : January 23, 2018
Last Update Posted : January 23, 2018
Sponsor:
Collaborator:
Childhood Liver Disease Research and Education Network
Information provided by (Responsible Party):
Shire

Brief Summary:
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 or disease Intervention/treatment Phase
Alagille Syndrome Drug: LUM001 Drug: Placebo Phase 2

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 37 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Triple (Participant, Care Provider, 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
Actual Study Start Date : November 24, 2014
Actual Primary Completion Date : November 16, 2016
Actual Study Completion Date : November 16, 2016

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Itching

Arm Intervention/treatment
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




Primary Outcome Measures :
  1. Change From Baseline to Endpoint (Week 13/Early Termination) in Pruritus [ Time Frame: Baseline, Week 13/Early Termination ]
    Pruritus was assessed using Itch report outcome measure (ItchRO[Obs]) measure, administered as an electronic diary (eDiary) which was completed by the participants twice daily (morning and evening). ItchRO(Obs) score ranged from 0 to 4, with the higher score indicating increasing itch severity. The highest score between the morning and evening ItchRO(Obs) reports represented the daily score: a measure of the worst itching over the previous 24-hour period.


Secondary Outcome Measures :
  1. Change From Baseline to Endpoint (Week 13/Early Termination) in Fasting Serum Bile Acid (sBA) Level [ Time Frame: Baseline, Week 13/Early Termination ]
    Fasting sBA level was measured by using a liquid chromatography mass spectrometry method. Total serum bile acid concentrations were calculated as sum of individual bile acid concentration.

  2. Change From Baseline to Endpoint (Week 13/Early Termination) in Liver Enzyme Levels [ Time Frame: Baseline, Week 13/Early Termination ]
    Liver enzyme levels of alanine aminotransferase, alkaline phosphatase, aspartate aminotransferase and gamma glutamyl transferase were reported here.

  3. Change From Baseline to Endpoint (Week 13/Early Termination) in Total and Direct Bilirubin Concentrations [ Time Frame: Baseline, Week 13/Early Termination ]
    Liver enzyme levels of total bilirubin and direct bilirubin were reported here.


Other Outcome Measures:
  1. Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Treatment-emergent Serious Adverse Events (TESAEs) [ Time Frame: Baseline up to Week 17 (follow up) ]
    An AE was any unfavorable and unintended sign (including a clinically significant abnormal laboratory finding, for example), symptom, or disease temporally associated with the study or use of investigational drug product, whether or not the AE was considered related to the investigational drug product. A serious adverse event (SAE) was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged in-patient hospitalization; life -threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly.TEAEs were defined as AEs/SAEs that started or worsened after the study drug treatment.



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Ages Eligible for Study:   12 Months to 18 Years   (Child, Adult)
Sexes Eligible for Study:   All
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

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): 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

Responsible Party: Shire
ClinicalTrials.gov Identifier: NCT02057692     History of Changes
Obsolete Identifiers: NCT02055768
Other Study ID Numbers: LUM001-301
First Posted: February 7, 2014    Key Record Dates
Results First Posted: January 23, 2018
Last Update Posted: January 23, 2018
Last Verified: January 2018

Additional relevant MeSH terms:
Syndrome
Alagille 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