PEDFIC 1 and PEDFIC 2 study designs
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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 Endpoint:
- Change in sleep parameters through Week 72‡
Secondary Endpoints Included4:
- Change from baseline in sBA at Week 72
Exploratory Endpoint:
- 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
Scratching scores were recorded twice daily by an observer2,3,5,6:
- AM score recorded worst scratching since going to bed last night
- PM score recorded worst scratching since waking up
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.