A Study to Assess the Ability of Eltrombopag to Induce Sustained Remission in Subjects With ITP

• Status: Active, not recruiting
• Trial end date: 2022-10-26
• Target enrollment: 0
• Participant gender: All

Summary

This study will assess the ability of eltrombopag to induce sustained treatment-free remission in ITP subjects who relapsed or failed to respond to an initial treatment with steroids.

There is limited, mainly retrospective evidence that earlier use of eltrombopag after ITP diagnosis, will allow a larger proportion of subjects to achieve sustained remission after tapering off drug. Clinically there is a need for a less toxic regimen that will provide responses and sustained remission with a shorter treatment interval. This trial is designed to assess this.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Novartis Pharmaceuticals

Criteria

Inclusion Criteria:

1. Signed informed consent must be obtained prior to participation in the study

2. Subjects ≥ 18 years old

3. Subjects with a confirmed diagnosis of primary ITP, who are not responsive or in relapse after a first line of steroid therapy ± intravenous immunoglobulin (IVIG) (used as a rescue therapy)

4. Platelet count < 30×109/L and assessed as needing treatment (per physician's discretion

Exclusion Criteria:

1. ITP subjects previously treated with any ITP second-line therapies, thrombopoietin receptor (TPO-R) agonists for ITP, except steroids / IVIG

2. Subjects who relapsed more than one year after the end of first-line full course of steroid therapy

3. Subjects with a diagnosis of secondary thrombocytopenia

4. Subjects who have life threatening bleeding complications per investigator discretion

5. Subjects who had a deep vein thrombosis or arterial thrombosis in the 6 months preceding enrollment

6. Serum creatinine ≥ 1.5 mg/dL

7. Total bilirubin > 1.5 × upper limit of normal (ULN)

8. Aspartate transaminase (AST) > 3.0 × ULN

9. Alanine transaminase (ALT) > 3.0 × ULN

10. Subjects who are human immune deficiency virus (HIV), hepatitis C virus (HCV), hepatitis B surface antigen (HBsAg) positive

11. Subjects with hepatic impairment (Child-Pugh score > 5)

12. Subjects who have active malignancy

13. Subjects with any serious and/or unstable pre-existing medical, psychiatric disorder or other conditions that could interfere with subject's safety, obtaining informed consent or compliance with the study procedures per investigator discretion

14. History or current diagnosis of cardiac disease indicating significant risk of safety for subjects participating in the study

15. Subjects with known active or uncontrolled infections not responding to appropriate therapy

16. Subjects with evidence of current alcohol/drug abuse

17. Women of child-bearing potential and sexually active males unwilling to use adequate contraception during the study

18. Female subjects who are nursing or pregnant (positive serum or urine B-human chorionic gonadotrophin (B-hCG) pregnancy test) at screening or pre-dose on Day 1

Other protocol-defined inclusion/exclusion criteria may apply.