Trial record 7 of 14 for:    Alagille Syndrome

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)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT02047318
Recruitment Status : Unknown
Verified January 2017 by Shire.
Recruitment status was:  Active, not recruiting
First Posted : January 28, 2014
Last Update Posted : January 23, 2017
Lumena Pharmaceuticals, Inc.
Information provided by (Responsible Party):

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

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 19 participants
Intervention Model: Single Group Assignment
Masking: None (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
Study Start Date : December 2013
Estimated Primary Completion Date : August 2017
Estimated Study Completion Date : August 2017

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Liver Diseases

Arm Intervention/treatment
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.

Primary Outcome Measures :
  1. Safety and tolerability [ Time Frame: 72 weeks ]
    Adverse events, changes in vital signs, laboratory and other safety parameters from baseline to week 72

Secondary Outcome Measures :
  1. Efficacy [ Time Frame: 72 weeks ]
    Changes in serum bile acids, pruritus, and other biochemical markers of cholestasis and liver disease from baseline to week 72

Other Outcome Measures:
  1. Optional Follow-Up [ Time Frame: 52 weeks ]
    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

Information from the National Library of Medicine

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, Learn About Clinical Studies.

Ages Eligible for Study:   12 Months to 18 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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.

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 identifier (NCT number): NCT02047318

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
Lumena Pharmaceuticals, Inc.
Study Director: Beatriz Caballero, MD Shire

Responsible Party: Shire Identifier: NCT02047318     History of Changes
Other Study ID Numbers: LUM001-303
2013-003832-54 ( EudraCT Number )
First Posted: January 28, 2014    Key Record Dates
Last Update Posted: January 23, 2017
Last Verified: January 2017

Additional relevant MeSH terms:
Alagille Syndrome
Liver Diseases
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