Phase 2
Completed N=105
A Study to Assess the Ability of Eltrombopag to Induce Sustained Response Off Treatment in Subjects With ITP
Immune Thrombocytopenic Purpura (ITP)
Source: ClinicalTrials.gov NCT03524612 ↗
Enrolled (actual)
105
Serious AEs
20.0%
Results posted
Dec 2023
Primary outcomePrimary: Percentage of Participants With Sustained Response Off Treatment (SRoT) by 12 Months — 30.5 Percentage of participants — p=<0.0001
Summary
The purpose of this trial was to assess the ability of eltrombopag to induce sustained treatment-free remission in immune thrombocytopenia purpura (ITP) subjects who relapsed or failed to respond to an initial treatment with steroids.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Percentage of Participants With Sustained Response Off Treatment (SRoT) by 12 Months |
30.5 | <0.0001 sig |
| SECONDARY Median Duration of Sustained Response Off Treatment (SRoT) After Treatment Discontinuation for Participants With Sustained Response Off Treatment |
33.3 | — |
| SECONDARY Estimated Median Duration of Sustained Response Off Treatment (SRoT) for Participants With Sustained Response Off Treatment at Month 12 and Who Enter 12 Months Follow-up Period |
— | — |
| SECONDARY Estimated Median Duration of Sustained Response Off Treatment (SRot) for All Patients |
— | — |
| SECONDARY Percentage of Participants With Sustained Response Off Treatment Until Month 24 |
21; 18; 14; 15 | — |
| SECONDARY Percentage of Participants With Early Response Within First Month |
76.2 | — |
| SECONDARY Percentage of Participants With Recovery Response in Case of Loss of Response During or After Tapering of Eltrombopag Until Month 24 |
11.4; 6.7 | — |
| SECONDARY Relative Change From Baseline in Platelet Count Over Time |
1384.29; 1313.10; 1396.68; 1464.95; 1622.19; 1664.90 | — |
| SECONDARY Percentage of Participants Who Maintain Platelet Counts Level Within 12 Months and Every 3 Months Until Month 24 |
49.5; 38.1; 28.6; 28.6; 20.0; 17.1 | — |
| SECONDARY Change From Baseline in Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-Fatigue) Questionaire |
5.42; 6.15; 6.89; 7.90; 7.12; 8.87 | — |
| SECONDARY Change From Baseline in Functional Assessment of Cancer Therapy- Thrombocytopenia (FACT-Th6) Questionnaire |
3.80; 4.35; 5.60; 5.62; 5.44; 7.04 | — |
| SECONDARY Change From Baseline in Short Form 36 Health Survey (SF-36v2) Questionnaire: Bodily Pain (BP) Score |
7.55; 2.75; 3.82; 7.53; 1.96; 13.61 | — |
| SECONDARY Change From Baseline in Short Form 36 Health Survey (SF-36v2) Questionnaire: General Health (GH) Score |
4.12; 5.39; 7.40; 7.62; 11.17; 14.78 | — |
| SECONDARY Change From Baseline in Short Form 36 Health Survey (SF-36v2) Questionnaire: Mental Health (MH) Score |
6.74; 6.97; 9.78; 10.26; 7.08; 15.22 | — |
| SECONDARY Change From Baseline in Short Form 36 Health Survey (SF-36v2) Questionnaire: Physical Functioning (PF) Score |
8.48; 10.90; 12.67; 14.31; 11.87; 19.35 | — |
| SECONDARY Change From Baseline in Short Form 36 Health Survey (SF-36v2) Questionnaire: Role Emotional (RE) Score |
4.42; 6.15; 8.89; 10.20; 4.86; 13.04 | — |
| SECONDARY Change From Baseline in Short Form 36 Health Survey (SF-36v2) Questionnaire: Role Physical (RP) Score |
13.26; 9.43; 17.36; 19.94; 15.89; 22.55 | — |
| SECONDARY Change From Baseline in Short Form 36 Health Survey (SF-36v2) Questionnaire: Social Functioning (SF) Score |
6.06; 7.17; 10.00; 16.38; 14.58; 26.63 | — |
| SECONDARY Change From Baseline in Short Form 36 Health Survey (SF-36v2) Questionnaire: Vitality (VT) Score |
7.48; 6.45; 12.78; 14.33; 8.33; 12.23 | — |
| SECONDARY Change From Baseline in Short Form 36 Health Survey (SF-36v2) Questionnaire: Physical Component Summary (PCS) Score |
3.46; 2.72; 3.92; 4.85; 4.37; 6.77 | — |
| SECONDARY Change From Baseline in Short Form 36 Health Survey (SF-36v2) Questionnaire: Mental Component Summary (MCS) Score |
2.34; 2.85; 4.52; 5.33; 3.28; 7.12 | — |
| SECONDARY Percentage of Participants With Worst Post-baseline Value in Functional Assessment of Cancer Therapy-G (GP5) |
51; 15; 10; 2; 5; 22 | — |
| SECONDARY Overall Change of Treatment Satisfaction Using Treatment Satisfaction Questionnaire (TSQM-9) |
17.78; 17.69; 9.96; 18.82; 17.99; 14.14 | — |
Eligibility Criteria
Inclusion Criteria
- Signed informed consent must be obtained prior to participation in the study
- Patients ≥ 18 years old
- Patients with a confirmed diagnosis of primary ITP, who are not responsive or in relapse after a first line of steroid therapy ± intravenous immunoglobulin (IVIG) (used as a rescue therapy)
- Platelet count 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), hepatitis B surface antigen (HBsAg) positive
- Patients with hepatic impairment (Child-Pugh score > 5)
- Patients who have active malignancy
- Patients with 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 per investigator discretion
- History or current diagnosis of cardiac disease indicating significant risk of safety for Patients participating in the study
- Patients with known active or uncontrolled infections not responding to appropriate therapy
- Patients with evidence of current alcohol/drug abuse
- Women of child-bearing potential and sexually active males unwilling to use adequate contraception during the study
- Female Patients 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
Other protocol-defined inclusion/exclusion criteria may apply.
Data sourced from ClinicalTrials.gov (NCT03524612). 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.