Evaluation of Maralixibat in Pruritus Associated With General Cholestatic Liver Disease (EXPAND) (NCT06553768) | Clinical Trial Compass
Active — Not RecruitingPhase 3
Evaluation of Maralixibat in Pruritus Associated With General Cholestatic Liver Disease (EXPAND)
United States90 participantsStarted 2024-10-14
Plain-language summary
The purpose of this study is to determine whether the investigational treatment (maralixibat) is safe and effective in pediatric and adult participants who have cholestatic liver disease with pruritus that has been refractory to other therapies, and who have no other treatment options.
Who can participate
Age range6 Months
SexALL
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Inclusion criteria
✓. Informed consent and assent (as applicable)
✓. Age ≥6 months at time of baseline visit
✓. Diagnosis of a rare cholestatic liver disease with cholestatic pruritus based on the following:
✓. Chronic liver biochemical abnormalities (\>90 days) and/or pathological evidence of progressive liver disease. Total sBA \>2× ULN is required.
✓. Persistent pruritus (\>90 days). An average worst-daily (morning and evening) ItchRO(Obs)/ItchRO(Pt) score ≥1.5 during the 2 consecutive weeks of the screening period leading to the baseline visit. If both instruments are administered, a score ≥1.5 is required only for ItchRO(Obs).
✓. Completion of at least 10 valid daily (morning and evening) ItchRO(Obs)/ItchRO(Pt) entries during 2 consecutive weeks of the screening period, leading to the baseline visit. Each week should have at least 4 valid daily (morning and evening) entries. If both instruments are administered, the completion criterion is required only for ItchRO(Obs).
✓. If taking antipruritics or ursodeoxycholic acid, the participant has to be on a stable dosing regimen (i.e., same dose and frequency in the 30 days prior to the screening visit and will continue this dosing regimen up to Week 40 \[adjustment for body weight is allowed\]).
✓. Non-pregnant, non-lactating females of childbearing potential who are sexually active must agree to use at least an acceptable effective method of contraception during the study. Females of childbearing potential must have a negative pregnancy test result.
Exclusion criteria
✕
What they're measuring
1
Mean change in the ItchRO(Obs) severity score
Timeframe: From baseline to average of week 13 to week 20
✕. Active atopic dermatitis or other non-cholestatic diseases associated with pruritus that are not controlled by standard treatment and that may interfere with the severity assessment of cholestasis-associated pruritus
✕. Decompensated cirrhosis or complications of cirrhosis (e.g., esophageal or gastric variceal bleeding in the last 6 months, high-risk esophageal or gastric varices \[e.g., large, coiled, occupying \>1/3 of the esophageal lumen, red varices or red signs\], ascites, hepatic encephalopathy, hepatorenal syndrome). Patients with compensated cirrhosis with preserved hepatic synthetic function (see Exclusion Criterion #6) and absence of complications are eligible.
✕. Suspected or proven cholangiocarcinoma or hepatocellular carcinoma
✕. Unstable and/or serious medical disease that is likely to impair the ability to participate in all aspects of the study, confound efficacy and/or safety assessments, or result in substantially shortened life expectancy (e.g., any active malignancy including hematological malignancy, end-stage heart failure, active infection, acute and chronic diarrhea). Exceptionally, previous history of malignancy, adequately treated/in remission, that in opinion of investigator and medical monitor does not impact participant safety and participation in the study, may be allowed. The investigator should contact the medical monitor to discuss these cases and seek approval before the screening period.
✕. Laboratory results during the screening visit as follows: