PEDFIC 1 study design
PEDFIC 1 is the FIRST COMPLETED GLOBAL Phase 3 clinical trial in PFIC1,2
Primary Endpoint2:
- Proportion of participant’s positive pruritus assessments over 24 weeks*
Secondary Endpoints Included2:
- Proportion of participants with sBA response at Week 24†
- Change in sleep parameters through Week 24‡
Concomitant ursodeoxycholic acid (UDCA) and other antipruritic medications were permitted provided they were at a stable dose at least 4 weeks before enrollment with no dosage changes planned during the study period.2
89% of participants were receiving UDCA or rifampicin at PEDFIC 1 baseline.3
PEDFIC 2 study design
PEDFIC 2 is an open-label extension study to assess long-term efficacy and safety4
Primary Endpoint4:
- Proportion of participant’s positive pruritus assessments over 72 weeks*
Exploratory Endpoint4:
- Change in sleep parameters through Week 72‡
Secondary Endpoints Included4:
- Change from baseline in sBA at Week 72
Exploratory Endpoint4:
- Change in sleep parameters through Week 72‡
in the PEDFIC 1 and PEDFIC 2 trials,
Pruritus was measured using the PRUCISION™ ObsRO scale2,3,5,6
A validated instrument that defines a ≥1-point reduction as a clinically meaningful change in pruritus score.2,6
Caregivers recorded scratching scores twice daily based on the following questions2,3,5,6:
- AM score: How bad was your child's worst scratching since he/she went to bed last night?
- PM score: How bad was your child's worst scratching since he/she woke up this morning?
BYLVAY is approved for the treatment of pruritus in all PFIC types, including newly detected subtypes7
Limitation of Use: BYLVAY is not recommended in a subgroup of PFIC type 2 patients with specific ABCB11 variants resulting in non-functional or complete absence of the bile salt export pump protein.
All images are actor portrayals.
All images are actor portrayals.
BYLVAY is approved for the treatment of pruritus in all PFIC types, including newly detected subtypes7
Limitation of Use: BYLVAY is not recommended in a subgroup of PFIC type 2 patients with specific ABCB11 variants resulting in non-functional or complete absence of the bile salt export pump protein.
ObsRO=observer-reported outcomes; OLE=open-label extension; PFIC=progressive familial intrahepatic cholestasis; sBA=serum bile acid.
*Defined as a scratching score of ≤1 or at least a 1-point reduction from baseline on the PRUCISION™ ObsRO instrument.2,4
†sBA response was defined as at least a 70% decrease in sBA or a final sBA level of ≤70 μmol/L.2
‡Data reported using the PRUCISION™ ObsRO instrument; caregivers answered up to 9 questions about the effects that itch had on the participant’s sleep and sleep-related parameters daily.3-5
References:
- Data on file. Ipsen US. [NON-US-004498].
- Thompson RJ, Amell H, Artan R, et al. Odevixibat treatment in progressive familial intrahepatic cholestasis: a randomised, placebo-controlled, phase 3 trial. Lancet Gastroenterol Hepatol. 2022;7(9):830-842. doi:10.1016/S2468-1253(22)00093-0
- Data on file. Ipsen US. [NON-US-004379].
- Thompson RJ, Artan R, Baumann U, et al. Interim results from an ongoing, open-label, single-arm trial of odevixibat in progressive familial intrahepatic cholestasis. JHEP Rep. 2023;5(8):100782. doi:10.1016/j.jhepr.2023.100782
- Data on file. Ipsen US. [NON-US-004380].
- Gwaltney C, Ivanescu C, Karlsson L, Warholic N, Kjems L, Horn P. Validation of the PRUCISION instruments in pediatric patients with progressive familial intrahepatic cholestasis. Adv Ther. 2022;39(11):5105-5125. doi:10.1007/s12325-022-02262-7
- BYLVAY. Prescribing Information. Ipsen Biopharmaceuticals, Inc.; 2025.