A Study to Assess Efficacy and Safety of Eltrombopag in Combination With a Short Course of Dexamethasone in Patients With Newly Diagnosed ITP

A Phase II, Randomized (1:1) Open Label Study to Assess the Efficacy and Safety of Eltrombopag in Combination With Dexamethasone Compared to Dexamethasone, as First-line Treatment in Adult Patients With Newly Diagnosed Immune Thrombocytopenia

ClinicalTrials.gov Identifier: NCT04346654

Novartis Reference Number: CETB115JDE01

Last Update: Dec 20, 2022

All compounds are either investigational or being studied for (a) new use(s). Efficacy and safety have not been established. There is no guarantee that they will become commercially available for the use(s) under investigation.

Study Description

The purpose of this study is to compare the ability of eltrombopag in combination with a short course of high-dose dexamethasone to induce sustained response off treatment in patients with newly-diagnosed ITP versus 1-3 cycles of dexamethasone monotherapy.

The unmet clinical need and the potential for eltrombopag when added to steroids to improve the treatment outcome and the potential to induce sustained response off treatment serve as the basis for clinical investigation of eltrombopag in first-line ITP.

Condition

Immune Thrombocytopenia (ITP)

Phase

Phase 2

Overall status

Recruiting

Start date

Oct 09, 2020

Completion date

Jan 03, 2024

Gender

All

Age(s)

18 Years and older (Adult, Older Adult)

Interventions

Drug

Eltrombopag

Eltrombopag is for oral use and comes in 25, 50 and 75 mg tablets. Prescribed dose is taken once daily.

Drug

Dexamethasone

Dexamethasone is for oral use and comes in 8 mg tablets. Prescribed dose is taken once daily.

Eligibility Criteria

Inclusion Criteria:

Signed informed consent must be obtained prior to participation in the study.
Men and women ≥ 18 years of age
Newly diagnosed with primary ITP (time from diagnosis within 3 months)
Platelet count < 30 × 109/L at screening and a need for treatment (per physician's discretion) Note: If pre-treatment is necessary, platelet count data performed directly before pre-treatment (can be used for study inclusion (screening value). Treatment-naïve patients will be included based on their platelet counts performed at screening

Exclusion Criteria:

Previous history of treatment for ITP, except any ITP-directed therapy for a maximum of 3 days within 7 days before randomization
Patients with diagnosis of secondary thrombocytopenia
Patients who have life threatening bleeding complications per physician´s discretion
Patients with a history of thromboembolic events or known risk factors for thromboembolism
Serum creatinine > 1.5 mg/dL
Total bilirubin (TBIL) > 1.5 × upper limit of normal (ULN)
Aspartate transaminase (AST) > 3.0 × ULN
Alanine transaminase (ALT) > 3.0 × ULN
Patients who are human immun deficiency virus (HIV),hepatitis C virus (HCV) or hepatitis B surface antigen (HBsAg) positive
Patients with hepatic impairment (Child-Pugh score > 5)
Patients with known active or uncontrolled infections not responding to appropriate therapy
History of current diagnosis of cardiac disease or impaired cardiac function denoted
Patients who have active malignancy
Patients with evidence of current alcohol/drug abuse
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

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