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

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ClinicalTrials.gov Identifier: NCT02117713
Recruitment Status : Active, not recruiting
First Posted : April 21, 2014
Last Update Posted : April 20, 2017
Sponsor:
Collaborators:
Information provided by (Responsible Party):

April 16, 2014
April 21, 2014
April 20, 2017
March 16, 2015
October 19, 2018   (Final data collection date for primary outcome measure)
Safety and tolerability [ Time Frame: 96 weeks ]
Evaluate teh long-term safety and tolerability of LUM001 in pediatric subjects with ALGS. This will include Adverse events, changes in vital signs, laboratory and other safety parameters from baseline to week 96
Safety and tolerability [ Time Frame: 48 weeks ]
Adverse events, changes in vital signs, laboratory and other safety parameters from baseline to week 48
Complete list of historical versions of study NCT02117713 on ClinicalTrials.gov Archive Site
  • Efficacy 1 [ Time Frame: 96 weeks ]
    Evaluate the long-term effect of LUM001 on serum bile acid levels associated with ALGS from baseline to week 96
  • Efficacy 2 [ Time Frame: 96 weeks ]
    Evaluate the long-term effect of LUM001 on pruritus associated with ALGS from baseline to Week 96
  • Efficacy 3 [ Time Frame: 96 weeks ]
    Explore the long-term effect of LUM001 on other biochemical markers of cholestasis and liver disease
  • Efficacy 4 [ Time Frame: 96 weeks ]
    Evaluate the long-term effect of LUM001 on xanthomas associated with ALGS
  • Dosing [ Time Frame: 96 weeks ]
    Explore expanded dosing range to identify doses necessary to achieve the optimal benefit-to-risk ration for this patient population
Efficacy [ Time Frame: 48 weeks ]
Changes in serum bile acids, pruritus, and other biochemical markers of cholestasis and liver disease from baseline to week 48
Not Provided
Not Provided
 
An Extension Study to Evaluate the Long-Term Safety and Durability of Effect of LUM001 in the Treatment of Cholestatic Liver Disease in Pediatric Subjects With Alagille Syndrome
A Multicenter 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
This is a multicentre, extension study of LUM001 in children diagnosed with Alagille Syndrome who have completed participation in a core LUM001 treatment protocol. The primary objective is to evaluate long-term safety and tolerability of LUM001. Efficacy will be assessed by evaluating the effect of LUM001 on the biochemical markers and pruritus associated with Alagille Syndrome.
Not Provided
Interventional
Phase 2
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Alagille Syndrome
Drug: LUM001
Administered orally daily
Experimental: LUM001
LUM001 administered orally once each day
Intervention: Drug: LUM001
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Active, not recruiting
36
October 19, 2018
October 19, 2018   (Final data collection date for primary outcome measure)

Inclusion Criteria:

  1. Male or female, 12 months to 18 years of age.
  2. Completed participation in the LUM001-301 protocol.
  3. Females of childbearing potential must have a negative urine pregnancy test at the Baseline Visit.
  4. Sexually active females must be prepared to use an effective method of contraception during the trial.
  5. Caregivers (and age appropriate subjects) must have access to phone for scheduled calls from study site.
  6. Caregivers (and age appropriate subjects) must be willing and able to complete a daily electronic diary.

Exclusion Criteria:

  1. Experienced an adverse event or serious adverse event (SAE) related to the study drug during the LUM001-301 protocol that led to the discontinuation of the subject from the core study.
  2. Any conditions or abnormalities which may compromise the safety of the subject, or interfere with the subject participating in or completing the study.
  3. History or presence of gallstones or kidney stones.
  4. History of non-adherence during the subject's participation in the LUM001-301 protocol. Non-adherence is defined by dosing compliance1 of less than 80% in the LUM001-301 protocol.
Sexes Eligible for Study: All
1 Year to 18 Years   (Child, Adult)
No
Contact information is only displayed when the study is recruiting subjects
Canada,   United States
 
 
NCT02117713
LUM001-305
No
Not Provided
Not Provided
Shire
Shire
  • Lumena Pharmaceuticals, Inc.
  • Childhood Liver Disease Research and Education Network
Study Director: Shire Physician Shire
Shire
April 2017

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP