A Phase II Dose-escalation Study Characterizing the PK of Eltrombopag in Pediatric Patients With Previously Untreated or Relapsed Severe Aplastic Anemia or Recurrent Aplastic Anemia

Phase 2 Completed 51 enrolled Novartis Pharmaceuticals

Non-randomizedParallel-groupOpen-labelTreatment

Summary

This is a phase II, open label, multi-center, intra-patient dose escalation study to characterize the pharmacokinetics (PK) after oral administration of eltrombopag in combination with immunosuppressive therapy in pediatric patients with previously untreated or relapsed/refractory severe aplastic anemia or recurrent aplastic anemia.

Timeline

Start: 2017-09-30
Primary completion: 2022-04-22
Completion: 2025-01-27

Publications

Shimamura A, Maschan A, Bennett C, Samarasinghe S, Farrar JE, Li CK, Sirachainan N, Pongtanakul B, Komvilaisak P, Zubarovskaya L, Rothman JA, Walkovich K, Nakano TA, Bertuch AA, Ferrao A, Bhat R, Hanna R, Overholt K, Boklan J, Wong TF, Wang Q, Urban P, Strahm B, Wang W, Vlachos A, Williams DA. Eltrombopag in combination with immunosuppressive therapy in pediatric severe aplastic anemia: phase 2 ESCALATE trial. Blood Adv. 2025 Aug 12;9(15):3728-3738. doi: 10.1182/bloodadvances.2024015102.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Eltrombopag	Small molecule	25 mg	Oral
Subject	Eltrombopag	Small molecule	50 mg	Oral
Subject	Eltrombopag	Small molecule	150 mg	Oral
Background	ANTILYMPHOCYTE IMMUNOGLOBULIN (HORSE)	Protein / enzyme biologic	—	—
Background	Cyclosporine	Peptide	—	Oral

A Phase II Dose-escalation Study Characterizing the PK of Eltrombopag in Pediatric Patients With Previously Untreated or Relapsed Severe Aplastic Anemia or Recurrent Aplastic Anemia

Summary

Timeline

Publications

Drugs

Indications