Safety and Efficacy of Eltrombopag Plus Pulsed Dexamethasone for Subjects With Idiopathic Thrombocytopenic Purpura

• Register: ClinicalTrials.gov
• Last refreshed on: 11 March 2024
• Main ID: NCT03830749
• Date of registration: 01/02/2019
• Prospective Registration: No
• Primary sponsor: Humanity & Health Medical Group Limited
• Public title: Safety and Efficacy of Eltrombopag Plus Pulsed Dexamethasone for Subjects With Idiopathic Thrombocytopenic Purpura
• Scientific title: A Pilot Study to Evaluate the Safety and Efficacy of Eltrombopag Plus Pulsed Dexamethasone as First Line Therapy for Subjects With Idiopathic Thrombocytopenic Purpura (ITP)
• Date of first enrolment: July 1, 2018
• Target sample size: 53
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/ct2/show/NCT03830749
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 2

Countries of recruitment

Hong Kong

Contacts

• Name: Gregory Cheng, PhD, MD
• Affiliation: Humanity & Health Research Centre

Key inclusion & exclusion criteria

Inclusion Criteria:

1. Subject has signed and dated a written informed consent.

2. Adults (=18 years) diagnosed with ITP according to the American Society for
Hematology/British Committee for Standards in Haematology (ASH/BCSH) Guidelines. In
addition, the peripheral blood smear should support the diagnosis of ITP with no
evidence of other disease causative of thrombocytopenia (e.g., pseudo
thrombocytopenia, myelofibrosis). The physical examination should be normal or at
least not show signs suggestive of any disease likely to be associated with
thrombocytopenia.

3. No prior ITP treatment except platelet transfusions

4. Subject has no intercurrent medical event, including evidence of any thrombosis.

5. Normal prothrombin time (PT/INR) and activated partial thromboplastin time (aPTT), no
history of hypercoagulable state.

6. The following clinical chemistries must be within the normal reference range:

creatinine, ALT, AST, total bilirubin, total albumin and alkaline phosphatase.

7. Subject is practicing an acceptable method of contraception (documented in chart).

8. Subject is able to understand and comply with protocol requirements and instructions.

Exclusion Criteria:

1. Any clinically relevant abnormality, other than ITP, identified on the screening
examination, or any other medical condition or circumstance, which in the opinion of
the investigator makes the subject unsuitable for participation in the study or
suggests another diagnosis.

2. History of active malignancy or on cancer therapy. Patients with a history of
malignancy in complete remission for longer than 5 years are eligible

3. History of arterial or venous thrombosis (stroke, transient ischemic attack,
myocardial infarction, deep vein thrombosis or pulmonary embolism).

4. = two of the following risk factors: Factor V Leiden, hormone replacement therapy,
systemic contraception containing estrogen, smoking, diabetes, hypercholesterolemia,
medications for hypertension or cancer.

5. Pre-existing cardiac disease (including congestive heart failure, and arrhythmia
requiring treatment), or clinically significant findings on resting 12-lead ECG at
screening.

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

7. History of alcohol/drug abuse.

8. Treatment with an investigational drug within 30 days or five half-lives (whichever is
longer) preceding the first dose of study medication



Age minimum: 18 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Immune Thrombocytopenia

Intervention(s)

Drug: Eltrombopag

Primary Outcome(s)

The prolonged response rate [Time Frame: 6 months after completion of therapy]

Secondary Outcome(s)

Early response rate [Time Frame: 6 months after completion of therapy]

Time to relapse [Time Frame: 6 months after completion of therapy]

Health related quality of life [Time Frame: 6 months after completion of therapy]

Secondary ID(s)

HHRC_ITP

Source(s) of Monetary Support

Please refer to primary and secondary sponsors