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 (XPAG-ITP) - XPAG-ITP

Phase 1

Conditions: immune thrombocytopenia
MedDRA version: 23.0Level: LLTClassification code 10050245Term: Autoimmune thrombocytopeniaSystem Organ Class: 100000004851
MedDRA version: 22.1Level: LLTClassification code 10036735Term: Primary thrombocytopeniaSystem Organ Class: 100000004851
Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]

Registration Number: EUCTR2019-002658-21-DE

Lead Sponsor: ovartis Pharma GmbH

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: ot Recruiting

Sex: All

Target Recruitment: 24

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). Platelet count < 30 × 10^9/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 (prior to signing the study specific informed consent form and start of screening for the study as per local practice or under a local protocol) will be used for study inclusion (screening value to be used for inclusion/exclusion check and for analysis as a covariate). Treatment-naïve patients will be included based on their platelet counts performed at screening.

Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 16
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 8

Exclusion Criteria

1. Previous history of treatment for ITP.
Note: Patients in need of immediate treatment for thrombocytopenia while diagnosis or eligibility are being determined may receive treatment with any ITP-directed therapy for a maximum of 3 days within 7 days before randomization. These therapies must be discontinued before the patient receives the first dose of study treatment
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 in the 6 months preceding enrollment or known risk factors for thromboembolism (e.g. Factor V Leiden, ATIII deficiency, antiphospholipid syndrome) are excluded. Patients with other risk factors which may pose an increased thromboembolic event (TEE) risk (prolonged periods of immobilization, malignancies, contraceptives and hormone replacement therapy, surgery/trauma, obesity and smoking) would be excluded according to the discretion of the investigator.
5. Presence of moderate to severe impaired renal function as indicated by any or all of the following criteria:
• Creatinine clearance < 45 mL/min as calculated using Cockcroft-Gault formula
• 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 immunodeficiency virus (HIV), 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 by any of the following:
• Corrected QTc >450 msec using Fridericia correction (QTcF) on the screening electrocardiogram (ECG)
• History of myocardial infarction and unstable angina within 6 months prior to starting study treatment
• Clinically significant cardiac arrhythmias or other clinically significant cardiovascular disease (e.g., congestive heart failure, uncontrolled hypertension) within the six months prior to starting study treatment
13. Patients who have active malignancy
14. Patients with evidence of current alcohol/drug abuse
15. Any serious and/or unstable pre-existing medical (including any advanced malignancy other than the disease under study), psychiatric disorder, or other conditions that could interfere with subject’s safety, obtaining informed consent or compliance with the study procedures.
16. Patients with a known immediate or delayed hypersensitivity reaction or idiosyncrasy to eltrombopag or drugs chemically related to eltrombopag or excipients that contraindicate their participation
17. Patients with pre-existing medical conditions that are known precautions with corticosteroid use, in whom the potential risks of participating in the study outweigh the potential benefits as determined by the investigator
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, defined as all women physiologically capable of becoming pregnant, unless they are using highly effective methods of contraception while taking study treatment and for 7 days after stopping medication. Highly effective contraception methods include:
• combined (estrogen and progestogen containi

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method

Secondary Outcome Measures

Name	Time	Method