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

CETB115JDE01

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.

2020-10-09

2023-09-22

2023-09-22

Completed

Early phase I

28

Inclusion Criteria: 1. Signed informed consent must be obtained prior to participation in the study. 2. Men and women ≥ 18 years of age 3. Newly diagnosed with primary ITP (time from diagnosis within 3 months) 4. 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: 1. Previous history of treatment for ITP, except any ITP-directed therapy for a maximum of 3 days within 7 days before randomization 2. Patients with diagnosis of secondary thrombocytopenia 3. Patients who have life threatening bleeding complications per physician´s discretion 4. Patients with a history of thromboembolic events or known risk factors for thromboembolism 5. Serum creatinine \> 1.5 mg/dL 6. Total bilirubin (TBIL) \> 1.5 × upper limit of normal (ULN) 7. Aspartate transaminase (AST) \> 3.0 × ULN 8. Alanine transaminase (ALT) \> 3.0 × ULN 9. Patients who are human immun deficiency virus (HIV),hepatitis C virus (HCV) or hepatitis B surface antigen (HBsAg) positive 10. Patients with hepatic impairment (Child-Pugh score \> 5) 11. Patients with known active or uncontrolled infections not responding to appropriate therapy 12. History of current diagnosis of cardiac disease or impaired cardiac function denoted 13. Patients who have active malignancy 14. Patients with evidence of current alcohol/drug abuse 15. 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

{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE2'], 'designInfo': {'allocation': 'RANDOMIZED', 'interventionModel': 'PARALLEL', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'NONE'}}, 'enrollmentInfo': {'count': 28, 'type': 'ACTUAL'}}

2024-02-01