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
This trial is active, not recruiting.
|Start date||September 2014|
|End date||September 2015|
|Trial size||30 participants|
|Trial identifier||NCT02160782, 2013-005373-43, LUM001-304|
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.
|United States||No locations recruiting|
|Other countries||No locations recruiting|
|Westmead, Australia||Children's Hospital Westmead||no longer recruiting|
|Parkville, Australia||The Royal Children's Hospital Melbourne||no longer recruiting|
|Brussels, Belgium||Cliniques Universitaires Saint-Luc||no longer recruiting|
|Bron, France||Hopital Femme Mere Enfant De Lyon||no longer recruiting|
|Paris, France||Hopital Necker-Enfants Malades||no longer recruiting|
|Paris, France||Hopital Kremlin Bicetre||no longer recruiting|
|Warsaw, Poland||The Children's Memorial Health Institute||no longer recruiting|
|Madrid, Spain||Hospital Universitario La Paz- Hospital Materno Infantil||no longer recruiting|
|London, United Kingdom||Paediatric Liver Center, Kings College Hospital||no longer recruiting|
|Endpoint classification||safety/efficacy study|
|Intervention model||crossover assignment|
|Masking||double blind (subject, caregiver, investigator)|
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
Evaluate effect of LUM001 on biochemical markers of cholestasis in children with ALGS
time frame: 48 weeks
Evaluate effect of LUM001 on pruritus in children with ALGS as measured by the average daily Itch Reported Outcome (ItchRO)
time frame: 48 weeks
Male or female participants from 12 months up to 18 years old.
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.
|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|
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