Phase 2
Completed N=26
A Study to Assess Efficacy and Safety of Eltrombopag in Combination With a Short Course of Dexamethasone in Patients With Newly Diagnosed ITP
Immune Thrombocytopenia (ITP)
Source: ClinicalTrials.gov NCT04346654 ↗
Enrolled (actual)
26
Serious AEs
40.0%
Results posted
Mar 2025
Primary outcomePrimary: Percentage of Patients With Sustained Response Off Treatment at 52 Weeks — 2; 2 Participants — p=0.5133
Summary
The purpose of this study was 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.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Percentage of Patients With Sustained Response Off Treatment at 52 Weeks |
2; 2 | 0.5133 |
| SECONDARY Percentage of Patients With Overall Response at Week 52 |
2; 2 | 0.5133 |
| SECONDARY Duration of Sustained Response Off Treatment |
49.1; 45.7 | — |
| SECONDARY Overall Response by Week 4 |
10; 11 | — |
| SECONDARY Complete Response by Week 4 |
8; 9 | — |
| SECONDARY Absolute Change in Platelet Count From Pre-treatment/Screening to Baseline and to Various Time Points |
32.0; 23.2; 145.2; 80.7; 192.5; 92.0 | — |
| SECONDARY Relative Change in Platelet Count From Pre-treatment/Screening to Baseline and to Various Time Points |
347.9; 293.4; 5795.5; 2028.2; 11400.9; 2865.4 | — |
| SECONDARY Time to Overall Response (TOR) |
1.1; 1.0 | — |
| SECONDARY Time to Complete Response |
1.1; 2.1 | — |
| SECONDARY Duration of Overall Response (OR) and Complete Response (CR) |
8.3; 3.6; 9.7; 1.3 | — |
| SECONDARY Change From Baseline in Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-Fatigue) Questionnaire |
-4.2; -4.5; 1.0; -2.9; 1.2; -5.1 | — |
| SECONDARY Change From Baseline in Short Form 36 Health Survey (SF-36v2) Questionnaire (Physical (PS), Mental Score (MS) and Alternative Scoring (PS-QM)) |
1.9; -3.3; 2.5; -1.4; 1.5; -2.7 | — |
| SECONDARY Incidence and Severity of Bleeding Events |
10; 12; 6; 2; 2; 1 | — |
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 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 immune 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
- 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
- Women of child-bearing potential and males unwilling to use adequate contraception during the study
Data sourced from ClinicalTrials.gov (NCT04346654). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.