A Study to Evaluate the Efficacy and Safety of Maralixibat in Subjects With Progressive Familial … (NCT03905330) | Clinical Trial Compass
CompletedPhase 3
A Study to Evaluate the Efficacy and Safety of Maralixibat in Subjects With Progressive Familial Intrahepatic Cholestasis (MARCH-PFIC)
United States93 participantsStarted 2019-07-09
Plain-language summary
The purpose of this study is to determine whether the investigational treatment (maralixibat) is safe and effective in pediatric participants with Progressive Familial Intrahepatic Cholestasis (PFIC).
Who can participate
Age range1 Year – 17 Years
SexALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
✓. Informed consent and assent (as applicable) per Institutional Review Board/Ethics Committee (IRB/EC)
✓. Male or female subjects with a body weight ≥5 kg, who are ≥12 months and \<18 years of age at time of baseline
✓. Cholestasis as manifested by total sBA ≥3× ULN (applies to primary cohort only)
✓. An average AM ItchRO(Obs) score ≥1.5 during 4 consecutive weeks of the screening period, leading to the baseline visit (Visit 1)
✓. Completion of at least 21 valid\* morning ItchRO(Obs) entries during 4 consecutive weeks of the screening period, leading to the baseline visit (Visit 1) (\*valid = completed and not answered as "I don't know"; maximum allowed invalid reports = 7, no more than 2 invalid reports during the last 7 days before randomization)
✓. Diagnosis of PFIC based on the following:
✓. Male and females of non-childbearing potential. Males and non-pregnant, non-lactating females of childbearing potential who are sexually active must agree to use acceptable methods of contraception during the study and 30 days following the last dose of the study medication. Females of childbearing potential must have a negative pregnancy test
✓. Access to email or phone for scheduled remote visits
Exclusion criteria
✕. Predicted complete absence of bile salt excretion pump (BSEP) function based on the type of ABCB11 mutation (PFIC2), as determined by a standard of care genotyping (applies to primary cohort only). Subjects can enter the study in the Supplemental Cohort (under inclusion criteria 6.ii or 6.iii).
What they're measuring
1
Mean Change in the Average Morning ItchRO(Obs) Severity Score in the Primary Cohort
Timeframe: MMRM model was used with inputs of the average changes from baseline (BL) to Weeks 1-6, 7-10, 11-14, 15-18, 19-22, 23-26, and other covariates. The average change from BL over Weeks 15-26 is calculated in the model and presented as a single number.
✕. Recurrent intrahepatic cholestasis, indicated by a history of sBA levels \<3x ULN or intermittent pruritus (applies to primary cohort only)
✕. Current or recent history (\<1 year) of atopic dermatitis or other non-cholestatic diseases associated with pruritus.
✕. History of surgical disruption of the enterohepatic circulation (applies to primary cohort only)
✕. Chronic diarrhea requiring intravenous fluid or nutritional intervention for the diarrhea and/or its sequelae at screening or during the 6 months prior to screening
✕. Previous or need for imminent liver transplant
✕. Decompensated cirrhosis (international normalized ratio \[INR\] \>1.5, albumin \<30 g/L, history or presence of clinically significant ascites, variceal hemorrhage, and/or encephalopathy)
✕. ALT or total serum bilirubin (TSB) \>15× ULN at screening