This Study Will Investigate the Efficacy and Safety of A4250 in Children With PFIC Types 1 or 2
Summary
Double blind, randomized, placebo controlled, Phase 3 study to investigate the efficacy and safety of low doses and high doses of A4250 compared to placebo in children with progressive familial intrahepatic cholestasis (PFIC) types 1 and 2.
Timeline
- Start
- 2018-05-16
- Primary completion
- 2020-07-27
- Completion
- 2020-07-28
Outcome
Met primary endpoint
registry analysis (superiority test); Placebo vs A4250 Low Dose; p = 0.0015; percentage difference 0.435 (95% CI 0.2195 to 0.6551); Cochran-Mantel-Haenszel NCT03566238 ↗
registry analysis (superiority test); Placebo vs A4250 High Dose; p = 0.0174; percentage difference 0.211 (95% CI 0.021 to 0.4557); Cochran-Mantel-Haenszel NCT03566238 ↗
registry analysis (superiority test); Placebo vs A4250 Low Dose; p = 0.0019; Least Square (LS) mean difference 28.23 (95% CI 9.83 to 46.64); ANCOVA NCT03566238 ↗
registry analysis (superiority test); Placebo vs A4250 High Dose; p = 0.0163; LS mean difference 21.71 (95% CI 1.87 to 41.54); ANCOVA NCT03566238 ↗
release “The PEDFIC trial (n=62) met its first primary endpoint” ipsen.com ↗
Publications
- 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 Nov;39(11):5105-5125. doi: 10.1007/s12325-022-02262-7. Epub 2022 Sep 6.
- Thompson RJ, Arnell H, Artan R, Baumann U, Calvo PL, Czubkowski P, Dalgic B, D'Antiga L, Durmaz O, Fischler B, Gonzales E, Grammatikopoulos T, Gupte G, Hardikar W, Houwen RHJ, Kamath BM, Karpen SJ, Kjems L, Lacaille F, Lachaux A, Lainka E, Mack CL, Mattsson JP, McKiernan P, Ozen H, Rajwal SR, Roquelaure B, Shagrani M, Shteyer E, Soufi N, Sturm E, Tessier ME, Verkade HJ, Horn P. Odevixibat treatment in progressive familial intrahepatic cholestasis: a randomised, placebo-controlled, phase 3 trial. Lancet Gastroenterol Hepatol. 2022 Sep;7(9):830-842. doi: 10.1016/S2468-1253(22)00093-0. Epub 2022 Jul 1.
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | Odevixibat | Small molecule | 40 ug/kg | Oral |
| Subject | Odevixibat | Small molecule | 120 ug/kg | Oral |