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

Status:	Recruiting
Conditions:	Hematology, Hematology, Hematology
Therapuetic Areas:	Hematology
Healthy:	No
Age Range:	18 - Any
Updated:	3/29/2019
Start Date:	November 2, 2018
End Date:	November 16, 2020
Contact:	Novartis Pharmaceuticals
Email:	novartis.email@novartis.com
Phone:	1-888-669-6682

A Phase II, Open-label, Prospective, Single-arm, Study to Assess Ability of Eltrombopag to Induce Sustained Remission in Subjects With ITP Who Are Refractory or Relapsed After First-line Steroids

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.

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.

We found this trial at

sites

Winship Cancer Institute at Emory University

1365 Clifton Rd NE
Atlanta, Georgia 30322

(404) 778-1900