An Extension Study to Evaluate the Long-Term Safety and Durability of Effect of LUM001 in the Treatment of Cholestatic Liver Disease in Subjects With Alagille Syndrome (ALGS) (IMAGINE)

This study is ongoing, but not recruiting participants.
Sponsor:
Collaborator:
Lumena Pharmaceuticals, Inc.
Information provided by (Responsible Party):
Shire
ClinicalTrials.gov Identifier:
NCT02047318
First received: January 23, 2014
Last updated: July 7, 2016
Last verified: July 2016
  Purpose
The purpose of this extension study is to determine the long-term safety and tolerability of an investigational treatment (LUM001) in children with ALGS who have completed participation in the LUM001-302 study.

Condition Intervention Phase
Alagille Syndrome
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: A Multicentre Extension Study to Evaluate the Long-Term Safety and Durability of the Therapeutic Effect of LUM001, an Apical Sodium-Dependent Bile Acid Transporter Inhibitor (ASBTi), in the Treatment of Cholestatic Liver Disease in Pediatric Subjects With Alagille Syndrome

Resource links provided by NLM:


Further study details as provided by Shire:

Primary 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 72


Secondary Outcome Measures:
  • Efficacy [ Time Frame: 72 weeks ] [ Designated as safety issue: No ]
    Changes in serum bile acids, pruritus, and other biochemical markers of cholestasis and liver disease from baseline to week 72


Other Outcome Measures:
  • Optional Follow-Up [ Time Frame: 52 weeks ] [ Designated as safety issue: Yes ]
    An optional follow-up period for eligible subjects who choose to stay on treatment with LUM001 after Week 72. Subjects' participation in the optional follow-up treatment period will continue until the first of the following occur: i) completion of 52 weeks of additional treatment or ii) in the event that a new study of LUM001 opens to enrollment


Estimated Enrollment: 19
Study Start Date: December 2013
Estimated Study Completion Date: August 2017
Estimated Primary Completion Date: August 2017 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: LUM001
LUM001 administered orally once each day
Drug: LUM001
Dosing of LUM001 with the objective of achieving optimal control of pruritus at a dose level that is tolerated by the subject and up to a maximum daily dose of 280 μg/kg LUM001 or 20 mg total dose.

  Eligibility

Ages Eligible for Study:   12 Months to 18 Years   (Child, Adult)
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Male or female, 12 months to 18 years of age.
  • Competent to provide informed consent and assent (per IRB/EC), as appropriate.
  • Completed participation in study LUM001-302.
  • Females of childbearing potential must have a negative urine pregnancy test.
  • Sexually active females must be prepared to use an effective method of contraception during the trial.
  • Subjects above the age of assent and caregivers and children must be able to read and understand English.
  • Caregivers (and age appropriate subjects) must have access to phone for scheduled calls from study site.
  • Caregivers (and age appropriate subjects) must be willing and able to complete a daily electronic diary (ItchRO) during the first consecutive 12 weeks of the study and then for 4 consecutive weeks following the Week 24 and Week 44 visits.
  • Caregivers (and age appropriate subjects) must digitally accept the licensing agreement in the ItchRO electronic diary software at the outset of the study.

Exclusion Criteria:

  • Experienced an adverse event or serious adverse event (SAE) related to the study drug during the LUM001-302 protocol that led to the discontinuation of the subject from the core study.
  • Any conditions or abnormalities (including laboratory abnormalities) which, in the opinion of the Investigator or the Sponsor Medical Monitor, may compromise the safety of the subject, or interfere with the subject participating in or completing the study.
  • History or presence of gallstones or kidney stones.
  • History of non-adherence during the subject's participation in the LUM001-302 protocol. Non-adherence is defined by dosing compliance of less than 80% in the LUM001-302 protocol.
  • Unlikely to comply with the study protocol, or unsuitable for any other reason, as judged by the investigator.
  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: NCT02047318

Locations
United Kingdom
Birmingham Children's Hospital
Birmingham, West Midlands, United Kingdom, B4 6NH
Leeds Teaching Hospital
Leeds, West Yorkshire, United Kingdom, LS9 7TF
Kings College Hospital
London, United Kingdom, SE5 9RS
Sponsors and Collaborators
Shire
Lumena Pharmaceuticals, Inc.
Investigators
Study Director: Beatriz Caballero, MD
  More Information

Responsible Party: Shire
ClinicalTrials.gov Identifier: NCT02047318     History of Changes
Other Study ID Numbers: LUM001-303  2013-003832-54 
Study First Received: January 23, 2014
Last Updated: July 7, 2016
Health Authority: United States: Food and Drug Administration
United Kingdom: Medicines and Healthcare Products Regulatory Agency

Additional relevant MeSH terms:
Syndrome
Liver Diseases
Alagille Syndrome
Cholestasis
Disease
Pathologic Processes
Digestive System Diseases
Cholestasis, Intrahepatic
Bile Duct Diseases
Biliary Tract Diseases
Heart Defects, Congenital
Cardiovascular Abnormalities
Cardiovascular Diseases
Abnormalities, Multiple
Congenital Abnormalities
Genetic Diseases, Inborn

ClinicalTrials.gov processed this record on July 25, 2016