Open Label Study to Evaluate Efficacy and Long Term Safety of LUM001 in the Treatment of Cholestatic Liver Disease in Patients With Progressive Familial Intrahepatic Cholestasis (INDIGO)

This study is ongoing, but not recruiting participants.
Sponsor:
Information provided by (Responsible Party):
Shire
ClinicalTrials.gov Identifier:
NCT02057718
First received: February 5, 2014
Last updated: August 19, 2015
Last verified: August 2015
  Purpose

This is an open label study in children with Progressive Familial Intrahepatic Cholestasis (PFIC) designed to evaluate the safety and efficacy of LUM001. Efficacy will be assessed by evaluating the effect of LUM001 on the biochemical markers and pruritus associated with PFIC.


Condition Intervention Phase
Progressive Familial Intrahepatic Cholestasis (PFIC)
Drug: LUM001
Phase 2

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Open Label Study of the Efficacy and Long Term Safety of LUM001, an Apical Sodium-Dependent Bile Acid Transporter Inhibitor (ASBTi), in the Treatment of Cholestatic Liver Disease in Pediatric Patients With Progressive Familial Intrahepatic Cholestasis

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 fasting serum bile acids from baseline to Week 13


Secondary Outcome Measures:
  • Efficacy [ Time Frame: 13 weeks ] [ Designated as safety issue: No ]
    Change in liver enzymes and pruritus from baseline to Week 13


Other Outcome Measures:
  • Safety and Tolerability [ Time Frame: 72 weeks ] [ Designated as safety issue: Yes ]
    Adverse events, changes in vital signs, laboratory and other safety parameters from baseline to week 48


Estimated Enrollment: 24
Study Start Date: February 2014
Estimated Study Completion Date: November 2016
Estimated Primary Completion Date: October 2016 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: LUM001
LUM001 administered orally once each day
Drug: LUM001

  Eligibility

Ages Eligible for Study:   12 Months to 18 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Diagnosis of Progressive Familial Intrahepatic Cholestasis
  2. Ability to understand and willingness to sign informed consent/assent prior to initiation

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
  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: NCT02057718

Locations
United States, California
Children's Hospital Los Angeles
Los Angeles, California, United States, 90027
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
France
Hopital Femme Mere Enfant De Lyon
Bron, France, 69677
Poland
The Children's Memorial Health Institute
Warsaw, Poland, 04-730
United Kingdom
Birmingham Children's Hospital
Steelhouse Lane, Birmingham West Midlands, United Kingdom, B4 6NH
Leeds Teaching Hospital NHS Trust
Great George Street, Leeds, United Kingdom, LS1 3EX
Kings College Hospital
London, United Kingdom, SE5 9RS
Sponsors and Collaborators
Shire
  More Information

No publications provided

Responsible Party: Shire
ClinicalTrials.gov Identifier: NCT02057718     History of Changes
Other Study ID Numbers: LUM001-501, 2013-003833-14
Study First Received: February 5, 2014
Last Updated: August 19, 2015
Health Authority: United States: Food and Drug Administration
United Kingdom: Medicines and Healthcare Products Regulatory Agency
Canada: Health Canada
France: Agence Nationale de Sécurité du Médicament et des produits de santé
Poland: Office for Registration of Medicinal Products, Medical Devices and Biocidal Products

Additional relevant MeSH terms:
Cholestasis
Cholestasis, Intrahepatic
Liver Diseases
Bile Duct Diseases
Biliary Tract Diseases
Digestive System Diseases

ClinicalTrials.gov processed this record on September 02, 2015