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 currently recruiting participants. (see Contacts and Locations)
Verified June 2015 by Shire
Information provided by (Responsible Party):
Shire Identifier:
First received: February 5, 2014
Last updated: June 10, 2015
Last verified: June 2015

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


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

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 identifier: NCT02057718

Contact: Sara Barbat, BSN, RN 858-461-0694

United States, California
Children's Hospital Los Angeles Recruiting
Los Angeles, California, United States, 90027
Contact: Cat Goodhue    323-361-4566   
Principal Investigator: Nanda Kerkar, M.D.         
United States, Ohio
Cincinnati Children's Hospital Medical Center Recruiting
Cincinnati, Ohio, United States, 45229
Contact: Susan McMahan    513-636-6215   
Principal Investigator: Alexander Miethke, MD         
United States, Pennsylvania
The Children's Hospital of Philadelphia Recruiting
Philadelphia, Pennsylvania, United States, 19104
Contact: Lindsay Brown    267-426-0970   
Principal Investigator: Kathleen Loomes, M.D.         
Children's Hospital of Pittsburgh of UPMC Recruiting
Pittsburgh, Pennsylvania, United States, 15224
Contact: Kathryn Bukauskas    412-478-8294   
Principal Investigator: Robert Squires, MD         
Hopital Femme Mere Enfant De Lyon Recruiting
Bron, France, 69677
Contact: Ilhem Maine    +33 (0) 4 27855044   
Principal Investigator: Christine Rivet, MD         
The Children's Memorial Health Institute Recruiting
Warsaw, Poland, 04-730
Contact: Elzbieta Cymerman    +48 504-014-027   
Principal Investigator: Irena Jankowska, MD         
United Kingdom
Birmingham Children's Hospital Recruiting
Steelhouse Lane, Birmingham West Midlands, United Kingdom, B4 6NH
Contact: Carla Lloyd    +44 121 333 8257   
Contact: Deirdre Kelly    +44 0 121 333 9999    deirdre.kelly@bch.nhs   
Principal Investigator: Deirdre Kelly         
Leeds Teaching Hospital NHS Trust Recruiting
Great George Street, Leeds, United Kingdom, LS1 3EX
Contact: Nicola Balatoni    0113 3922 185   
Contact: Patricia McClean    +44 0 113 392 5652   
Principal Investigator: Patricia McClean         
Kings College Hospital Recruiting
London, United Kingdom, SE5 9RS
Contact: Palaniswamy Karthikeyan    +44 0 207 346 4230   
Contact: Harpreet Grewal   
Principal Investigator: Richard J Thompson         
Sponsors and Collaborators
  More Information

No publications provided

Responsible Party: Shire Identifier: NCT02057718     History of Changes
Other Study ID Numbers: LUM001-501, 2013-003833-14
Study First Received: February 5, 2014
Last Updated: June 10, 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, Intrahepatic
Liver Diseases
Bile Duct Diseases
Biliary Tract Diseases
Digestive System Diseases processed this record on July 28, 2015