A Study to Assess Efficacy and Safety of Eltrombopag in Combination With a Short Course of Dexamethasone in Patients With Newly Diagnosed ITP

  • STATUS
    Recruiting
  • End date
    Dec 31, 2023
  • participants needed
    106
  • sponsor
    Novartis Pharmaceuticals
Updated on 19 August 2021
Investigator
Novartis Pharmaceuticals
Primary Contact
Novartis Investigative Site (7.4 mi away) Contact
+20 other location
platelet count
dexamethasone
eltrombopag

Summary

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.

Description

This is a Phase II, multicenter, 1:1 randomized, open-label study to compare the efficacy and safety of eltrombopag in combination with a short course of high-dose dexamethasone to 1-3 cycles of high-dose dexamethasone monotherapy, as first-line treatment in adult patients with newly diagnosed ITP.

Adult patients with newly diagnosed ITP who have platelet counts < 30 109/L and require treatment will be screened, and if eligible, will be randomized to either Arm A (eltrombopag in combination with a short course of dexamethasone) or Arm B (1-3 cycles of dexamethasone monotherapy).

The study will be conducted in the following periods:

Screening Period: Patients will be screened for 14 days based on the inclusion and exclusion criteria

Treatment Period: Arm A: Patients will be treated for 26 weeks during the treatment period. Patients who reach platelet counts 30 109/L and maintain counts 30 109/L during the tapering phase will be eligible for treatment discontinuation. Duration of tapering before treatment discontinuation at Week 26 will be 6 weeks. Arm B: Patients will be treated up to 12 weeks during the treatment period. Patients who reach platelet counts 30 109/L and maintain counts 30 109/L after 1-3 cycles of dexamethasone treatment will be eligible for treatment discontinuation. Patients with platelet counts < 30 109/L after 3 cycles of dexamethasone treatment will be offered a course of eltrombopag treatment within the study and will discontinue from study at week 52.

Observation period: After completion of treatment period, all patients will be observed for sustained response off treatment until week 52. Only patients with sustained response at week 52 will be followed for another 26 weeks. Patients who relapse between Week 52 and Week 78 will discontinue the study.

Details
Condition IDIOPATHIC THROMBOCYTOPENIC PURPURA, Autoimmune disease, Immune Thrombocytopenia, Idiopathic Thrombocytopenic Purpura (ITP), Thrombocytopenic, autoimmune thrombocytopenia
Treatment Dexamethasone, Eltrombopag
Clinical Study IdentifierNCT04346654
SponsorNovartis Pharmaceuticals
Last Modified on19 August 2021

Eligibility

Yes No Not Sure

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 < 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-nave patients will be included based on their platelet counts performed at screening

Exclusion Criteria

Previous history of treatment for ITP, except any ITP-directed therapy for a maximum of 3 days within 7 days before randomization
Patients with diagnosis of secondary thrombocytopenia
Patients who have life threatening bleeding complications per physicians discretion
Patients with a history of thromboembolic events or known risk factors for thromboembolism
Serum creatinine > 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 immun 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 19. Women of child-bearing potential and males unwilling to use adequate contraception during the study
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