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Trial record 2 of 13 for:    "Alagille syndrome"

A Long-Term, Open-Label Study of LUM001 With a Double-Blind, Placebo Controlled, Randomized Drug Withdrawal Period to Evaluate Safety and Efficacy in Children With Alagille Syndrome (ICONIC)

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ClinicalTrials.gov Identifier: NCT02160782
Recruitment Status : Active, not recruiting
First Posted : June 11, 2014
Last Update Posted : January 23, 2017
Sponsor:
Collaborator:
Lumena Pharmaceuticals, Inc.
Information provided by (Responsible Party):
Shire

Brief Summary:
This is a long-term, open-label study with a double-blind, placebo-controlled, randomized drug withdrawal period in children with ALGS designed to evaluate the safety and efficacy of LUM001. The study is divided into 5 parts: a 6-week open-label, dose escalation period, a 12-week stable dosing period, a 4-week randomized, double-blind, placebo-controlled drug withdrawal period, a 26-week long-term stable dosing period, and an optional 52 week follow-up treatment period for eligible subjects who choose to stay on treatment with LUM001. 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 (Week 100), or (ii) in the event that a new study of LUM001 opens to enrollment.

Condition or disease Intervention/treatment Phase
Alagille Syndrome Drug: LUM001 Drug: Placebo Phase 2

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 30 participants
Allocation: Randomized
Intervention Model: Crossover Assignment
Masking: Triple (Participant, Care Provider, Investigator)
Primary Purpose: Treatment
Official Title: Long-Term, Open-Label Study With a Double-Blind, Placebo-Controlled, Randomized Drug Withdrawal Period of LUM001, an Apical Sodium-Dependent Bile Acid Transporter Inhibitor (ASBTi), in Patients With Alagille Syndrome
Study Start Date : September 2014
Actual Primary Completion Date : September 2015
Estimated Study Completion Date : September 2017

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Experimental: LUM001
LUM001 administered orally once each day
Drug: LUM001
LUM001 administered orally once each day

Placebo Comparator: Placebo
Placebo administered orally once each day during randomized withdrawal period
Drug: Placebo
Placebo administered orally once each day during randomized withdrawal period




Primary Outcome Measures :
  1. Evaluate effect of LUM001 on serum bile acid levels in children with ALGS using an analysis of covariance (ANCOVA) model treatment [ Time Frame: 48 weeks ]
  2. Evaluate effect of LUM001 on biochemical markers of cholestasis in children with ALGS [ Time Frame: 48 weeks ]
    Change from Baseline in Liver enzymes (ALT, ALP) and bilirubin (total and direct) and other biochemical markers of cholestasis [total cholesterol, low-density lipoprotein cholesterol (LDL-C)].

  3. Evaluate effect of LUM001 on pruritus in children with ALGS as measured by the average daily Itch Reported Outcome (ItchRO) [ Time Frame: 48 weeks ]

Other Outcome Measures:
  1. Safety and Tolerability [ Time Frame: 100 weeks ]
    Evaluation of adverse events, changes in vital signs, laboratory and other safety parameters



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

Inclusion Criteria:

  1. Diagnosis of Alagille Syndrome
  2. Evidence of cholestasis
  3. Moderate to severe pruritus
  4. Females of childbearing potential must have a negative serum pregnancy test during screening.
  5. Sexually active females must be prepared to use an effetive method of contraception during the trial.
  6. Subject is expected to have a consistent caregiver(s) for the duration of the study.
  7. Caregivers (and age appropriate subjects) must complete at least 10 eDiary reports (morning or evening) during each of two consecutive weeks of the screening period .

Exclusion Criteria:

  1. Chronic diarrhea requiring ongoing intravenous fluid or nutritional intervention.
  2. Surgical interruption of the enterohepatic circulation.
  3. Previous liver transplant.
  4. Decompensated cirrhosis, history or presence of clinically significant ascites, variceal hemorrhage, and/or encephalopathy.
  5. History or presence of other concomitant liver disease, gallstones, or kidney stones.
  6. Diagnosis of human immunodeficiency virus (HIV).
  7. Cancers, except for in situ carcinoman, or cancers treated at least 5 years prior to screening with no evidence of recurrence.
  8. Any female who is pregnant or lactating, or planning to become pregnant during the study period.

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 ClinicalTrials.gov identifier (NCT number): NCT02160782


Locations
Australia, New South Wales
Children's Hospital Westmead
Westmead, New South Wales, Australia, 2145
Australia, Victoria
The Royal Children's Hospital Melbourne
Parkville, Victoria, Australia, 3052
Belgium
Cliniques Universitaires Saint-Luc
Brussels, Belgium
France
Hopital Femme Mere Enfant De Lyon
Bron, France, 69677
Hopital Necker-Enfants Malades
Paris, France, 75015
Hopital Kremlin Bicetre
Paris, France, 94275
Poland
The Children's Memorial Health Institute
Warsaw, Poland, 04-730
Spain
Hospital Universitario La Paz- Hospital Materno Infantil
Madrid, Spain, 261
United Kingdom
Paediatric Liver Center, Kings College Hospital
London, United Kingdom, SE5 9RS
Sponsors and Collaborators
Shire
Lumena Pharmaceuticals, Inc.
Investigators
Study Director: Shire Physician Shire

Responsible Party: Shire
ClinicalTrials.gov Identifier: NCT02160782     History of Changes
Other Study ID Numbers: LUM001-304
2013-005373-43 ( EudraCT Number )
First Posted: June 11, 2014    Key Record Dates
Last Update Posted: January 23, 2017
Last Verified: January 2017

Additional relevant MeSH terms:
Alagille Syndrome
Syndrome
Substance Withdrawal Syndrome
Disease
Pathologic Processes
Cholestasis, Intrahepatic
Cholestasis
Bile Duct Diseases
Biliary Tract Diseases
Digestive System Diseases
Liver Diseases
Heart Defects, Congenital
Cardiovascular Abnormalities
Cardiovascular Diseases
Abnormalities, Multiple
Congenital Abnormalities
Genetic Diseases, Inborn
Substance-Related Disorders
Chemically-Induced Disorders
Mental Disorders