An Extension Study to Evaluate the Long-Term Safety and Durability of Effect of LUM001 in the Tre… (NCT02117713) | Clinical Trial Compass
CompletedPhase 2
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
United States34 participantsStarted 2015-03-16
Plain-language summary
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.
Who can participate
Age range1 Year – 18 Years
SexALL
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Inclusion criteria
✓. Male or female, 12 months to 18 years of age.
✓. Competent to provide informed consent and assent (per institutional review board/Ethics Committee \[IRB/EC\]), as appropriate.
✓. Completed participation in the LUM001-301 protocol.
✓. Females of childbearing potential must have a negative urine pregnancy test \[beta human chorionic gonadotropin (beta-hCG)\] at the Baseline Visit.
✓. Sexually active females must be prepared to use an effective method of contraception during the trial.
✓. Hormonal (for example, contraceptive pill, patch, intramuscular implant or injection); or
✓. Barrier method, for example, (a) condom with spermicide, or (b) diaphragm, with spermicide; or
✓. Intrauterine device (IUD).
Exclusion criteria
✕. 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 participant from the core study.
✕. Any conditions or abnormalities (including laboratory abnormalities) which in the opinion of the Investigator, Medical Monitor or ChiLDReN Protocol Chair, may compromise the safety of the participant, or interfere with the participant participating in or completing the study.
✕. History or known presence of gallstones or kidney stones.
What they're measuring
1
Change From MRX Baseline to Week 48 in Fasting Serum Bile Acid (sBA)
✕. History of non-adherence during the participant's participation in the LUM001-301 protocol. Non-adherence is defined by dosing compliance (dosing compliance is calculated by \[the total number of doses that were actually taken by the participant\] divided by \[the total number of doses that should have been taken by the participant\] multiplied by 100) of less than 80% in the LUM001-301 protocol.
✕. Unlikely to comply with the study protocol, or unsuitable for any other reason, as judged by the investigator.
✕. All above exclusion criteria will apply upon re-entry into the long-term, optional follow-up treatment period 2.