ASSERT study design
ASSERT is the FIRST and ONLY randomized Phase 3 trial of an IBAT inhibitor in ALGS1,2
Primary Endpoint2:
- Change from baseline in average scratching score to Month 6 (Weeks 21 to 24)
Secondary Endpoints Included2:
- Change from baseline in sBA levels through Week 24
- Proportion of participants achieving a clinically meaningful decrease in pruritus
- Change from baseline 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
98% of participants were receiving antipruritic medications and 89% were receiving UDCA at baseline.4
The only pivotal trial of an IBAT inhibitor to enroll participants with JAG1 (92%) and NOTCH2 (8%) variants.3,5
ASSERT-EXT study design
ASSERT is an open-label extension study to assess long-term efficacy and safety6,7
Primary Endpoint6,7:
- Change from baseline in average scratching score through Week 72
Secondary Endpoints Included6,7:
- Change from baseline in sBA at Week 72
- Change from baseline in sleep parameters through Week 72*
in the assert and assert-ext trials,
Cholestatic pruritus was measured using the PRUCISION™ ObsRO scale2,4,8,9
A validated instrument that defines a ≥1-point reduction as a clinically meaningful change in pruritus score.9
Caregivers recorded scratching scores twice daily based on the following questions4,8,9:
- 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?
ALGS=Alagille syndrome; IBAT=ileal bile acid transporter; ObsO=observer-reported outcome; OLE=open-label extension; sBA=serum bile acid; UDCA=ursodeoxycholic acid.
*Data reported using the PRUCISION™ ObsRO instrument; caregivers answered 9 questions about the effects that itch had on their child’s sleep and sleep-related parameters daily.2,4,8
References:
- Data on file. Ipsen US. [NON-US-004497].
- Ovchinsky N, Aumar M, Baker A, et al. Efficacy and safety of odevixibat in patients with Alagille syndrome (ASSERT): a phase 3, double-blind, randomised, placebo-controlled trial. Lancet Gastroenterol Hepatol. 2024:9(7):632-645. doi:10.1016/S2468-1253(24)00074-8
- BYLVAY. Prescribing Information. Ipsen Biopharmaceuticals, Inc.; 2025.
- Data on file. Ipsen US. [NON-US-004381].
- Data on file. Ipsen US. [NON-US-004502].
- ClinicalTrials.gov. Long-term safety and efficacy of odevixibat in patients with Alagille syndrome. NCT05035030. Updated March 2, 2026. Accessed March 12, 2026. https://clinicaltrials.gov/study/NCT05035030
- Data on file. Ipsen US. [NON-US-004504].
- Data on file. Ipsen US. [NON-US-004230].
- 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