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)
This trial is active, not recruiting.
|Collaborator||Lumena Pharmaceuticals, Inc.|
|Start date||December 2013|
|End date||August 2017|
|Trial size||19 participants|
|Trial identifier||NCT02047318, 2013-003832-54, LUM001-303|
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.
|United States||No locations recruiting|
|Other countries||No locations recruiting|
|Birmingham, United Kingdom||Birmingham Children's Hospital||no longer recruiting|
|Leeds, United Kingdom||Leeds Teaching Hospital||no longer recruiting|
|London, United Kingdom||Kings College Hospital||no longer recruiting|
|Intervention model||single group assignment|
Safety and tolerability
time frame: 72 weeks
time frame: 72 weeks
All participants from 12 months up to 18 years old.
- 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.
- 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.
|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|
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