Avatrombopag for the Treatment of Thrombocytopenia in Pediatric Subjects With Immune Thrombocytopenia for ≥6 Months
Summary
A Phase 3b Study to Evaluate the Efficacy and Safety of Avatrombopag for the Treatment of Thrombocytopenia in Pediatric Subjects with Immune Thrombocytopenia for ≥6 Months
Timeline
- Start
- 2021-03-05
- Primary completion
- 2023-11-08
- Completion
- 2025-10-28
Outcome
Met primary endpoint
registry analysis (superiority test); Experimental: Avatrombopag vs Placebo Comparator:Placebo; p = 0.0077; Fisher Exact NCT04516967 ↗
registry analysis (superiority test); Experimental: Avatrombopag vs Placebo Comparator:Placebo; p <0.0001; Fisher Exact NCT04516967 ↗
paper 15 (28%) patients in the avatrombopag group met the primary endpoint of durable platelet response versus no (0%) patients in the placebo group PMID 40418942 ↗
release “The primary endpoint was met, confirming the efficacy and safety of avatrombopag (Doptelet®) in treating children and adolescents with ITP” sobi.com ↗
Publications
- Results Leblebisatan, G. G., Aydinok, Y., Äcenal, Ş., Grainger, J., Grimes, A., Lambert, M., Zhang, J., Jamieson, B., Vredenburg, M., & Kolodny, S. (2025). PB0364: Evaluation of efficacy and safety of avatrombopag in children with ITP based on disease duration. RPTH, 9(Suppl 2).
- Results Leblebisatan, G. G., Aydinok, Y., Ünal Cangül, Ş., Grainger, J., Grimes, A., Lambert, M., Zhang, J., Jamieson, B., Vredenburg, M., & Kolodny, S. (2025). PB0348: Consistent response to avatrombopag for pediatric ITP across various baseline characteristics. RPTH, 9(Suppl 2).
- Results Grace, R. F., Leblebisatan, G., Aydinok, Y., Ünal Cangül, Ş., Grainger, J., Zhang, J., Alkhori Franzén, L., & Skuban, N. (2025). PS2234: Efficacy and safety of avatrombopag for the treatment of pediatric immune thrombocytopenia in the open-label extension of a Phase 3, randomized, double-blind, placebo-controlled trial. HemaSphere, 9(Suppl 1), 4067.
- Results Scott Kolodny, Rachael Grace, Göksel Leblebisatan, Yesim Aydinok, Şule Ünal, John Grainger, Amanda Grimes, Michelle Lambert, Jessica Zhang, Brian Jamieson, Michael Vredenburg. Baseline Correlates With Durability of Avatrombopag Response in Pediatric Immune Thrombocytopenia. Pediatr Blood Cancer. 2025;72(Suppl S94):e31765. Abstract [# 304].
- Results Scott Kolodny, Rachael Grace, Göksel Leblebisatan, YesimAydinok, Şule Ünal, John Grainger, Amanda Grimes, Michele Lambert, Jessica Zhang, Brian Jamieson, Michael Vredenburg. Clinically Meaningful Response to Avatrombopag for Treatment of Pediatric Immune Thrombocytopenia. Pediatr Blood Cancer. 2025;72(Suppl S06):e31765. Abstract [# 2011].
- Results Grace RF, Leblebisatan G, Aydinok Y, Unal S, Grainger JD, Zhang J, Smallwood L, de Leon E, Jamieson BD; AVA-PED-301 Study Group. Avatrombopag for the treatment of children and adolescents with immune thrombocytopenia (AVA-PED-301): a multicentre, randomised, double-blind, placebo-controlled, phase 3b study. Lancet Haematol. 2025 Jul;12(7):e494-e504. doi: 10.1016/S2352-3026(25)00107-3. Epub 2025 May 23.
- Results Grace RF, Leblebisatan G, Aydinok Y, Unal S, Grainger JD, Zhang J, Smallwood L, de Leon E, Jamieson BD, DiRaimo J. Avatrombopag for treating children and adolescents with immune thrombocytopenia: a plain language summary. Expert Rev Hematol. 2026 Feb;19(2):107-116. doi: 10.1080/17474086.2025.2604524. Epub 2025 Dec 18.
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | Avatrombopag | Small molecule | 10 mg | Oral |
| Subject | Avatrombopag | Small molecule | 20 mg | Oral |