drugset / Trial / NCT03939637
Eltrombopag vs Standard Front Line Management for Newly Diagnosed Immune Thrombocytopenia (ITP) in Children
Phase 3
Completed
122 enrolled
Baylor College of Medicine
Boston Children's Hospital · collabUniversity of California, San Francisco · collab
RandomizedParallel-groupOpen-labelTreatment
Summary
This is an investigator initiated, multicenter, open label, randomized phase 3 study for subjects with newly diagnosed ITP from ages 1 to less than 18 years old.
Timeline
- Start
- 2019-05-02
- Primary completion
- 2024-04-17
- Completion
- 2025-02-26
Publications
- Shimano KA, Grimes AB, Kaicker S, Shah SJ, Gunn E, Bhat RV, Kochhar M, Rothman JA, Rose MJ, Briones M, Nakano TA, Lebensburger JD, Lambert MP, Fritch Lilla SA, Jesudas R, Lee-Miller CA, Thompson AA, Rifkin-Zenenberg S, Majumdar S, Crary SE, Hege K, Ford JB, Bies JJ, Fort J, Wynn TT, Hsieh L, Ruiz ME, Dinu B, Wong JMW, Kao PC, Kim TO, Arnold SD, Bennett CM, Despotovic JM, Klaassen RJ, Neufeld EJ, Neunert CE, London WB, Grace RF. Eltrombopag for Newly Diagnosed Pediatric Immune Thrombocytopenia Requiring Treatment: The PINES Randomized Clinical Trial. JAMA. 2025 Nov 25;334(20):1816-1826. doi: 10.1001/jama.2025.18168.
- Shimano KA, Grace RF, Despotovic JM, Neufeld EJ, Klaassen RJ, Bennett CM, Ma C, London WB, Neunert C. Phase 3 randomised trial of eltrombopag versus standard first-line pharmacological management for newly diagnosed immune thrombocytopaenia (ITP) in children: study protocol. BMJ Open. 2021 Aug 27;11(8):e044885. doi: 10.1136/bmjopen-2020-044885.
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | Eltrombopag | Small molecule | 25 mg | Oral |
| Subject | Eltrombopag | Small molecule | 50 mg | Oral |
| Comparator | Immune Globulin Intravenous (Human) | Protein / enzyme biologic | 1 g/kg | Intravenous |
| Comparator | Prednisolone | Other / unclassified | 4 mg/kg | — |
| Comparator | Prednisone | Other / unclassified | 4 mg/kg | — |
| Comparator | Rho(D) Immune Globulin | Unknown | 75 ug/kg | — |