NCT03025698

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

A Phase II, Open-label, Non-controlled, Intra-patient Dose-escalation Study to Characterize the Pharmacokinetics After Oral Administration of Eltrombopag in Pediatric Patients With Refractory, Relapsed or Treatment Naive Severe Aplastic Anemia or Recurrent Aplastic Anemia

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 51 (actual)
• Sponsor: Novartis Pharmaceuticals · Industry
• Sex: All
• Age: 1 Year – 18 Years
• Healthy volunteers: Not accepted

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.

Detailed description

All patients were treated with eltrombopag for the 26-week Treatment Period, followed by a 52-week Follow-Up Period. Patients who had been previously untreated with immunosuppressive therapy were treated according to the standard of care, hATG/cyclosporine, in addition to eltrombopag. Patients with relapsed/refractory SAA or recurrent AA were enrolled into one of two treatment options: hATG/cyclosporine plus eltrombopag or cyclosporine plus eltrombopag, depending on prior treatment with immunosuppressive therapy. Patients could receive eltrombopag beyond 26 weeks if the investigator thought that the patient was still receiving clinical benefit from the drug. After initiating treatment with eltrombopag, patients had their dose assessed and modified as tolerated, until the targeted platelet count or maximum dose was achieved. Pharmacokinetic assessments were performed at time points intended to capture steady state PK of the starting dose and highest dose achieved. There are four separate periods of this study: Screening (signing of written informed consent through Day -1), Treatment (for 26 weeks), Follow-up (additional 52 weeks), and Long-term Follow-up (for additional 3 years). The first 3 periods were considered the Core phase of the study. Study completion (Core) will occur when the last patient completes the 26-week treatment and 52-week Follow-up Period \[at Week 78\].

Conditions

• Aplastic Anemia

Interventions

Type	Name	Description
DRUG	Eltrombopag	Tablet for oral use, once daily or Powder for oral suspension (PfOS), once daily
DRUG	hATG	Horse ATG (ATGAM) (hATG) is not considered an investigational medicinal product (IMP)
DRUG	CsA	Cyclosporine (CsA) will be by supplied as either oral capsules or oral solution, administered twice a day

Timeline

• Start date: 2017-09-30
• Primary completion: 2022-04-22
• Completion: 2025-01-27
• First posted: 2017-01-19
• Last updated: 2025-03-04

Locations

20 sites across 6 countries: United States, Hong Kong, Portugal, Russia, Thailand, United Kingdom

Regulatory

• FDA-regulated drug study