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

NCT04346654 | Completed Phase 2 Results

• 2 other identifiers: CETB115JDE012019-002658-21
• Study Type: interventional
• Target: 26
• Locations: 1 country
• Sites: 10

Brief 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.

Conditions

Immune Thrombocytopenia (ITP)

Keywords

ITP; Eltrombopag; Dexamethasone; Sustained response off treatment; TFR; Adult; ETB115; Platelets; Thrombocytopenic; Thrombopoietin receptor Agonist; Newly-diagnosed; Blood bleeding disorder

Outcome Measures

Primary Outcomes (1)

• Percentage of Patients With Sustained Response Off Treatment at 52 Weeks

  Sustained response off treatment at 52 weeks is defined as maintenance of platelet count ≥ 30 × 10\^9/L after treatment discontinuation until Week 52 in the absence of bleeding events ≥ Grade II or use of any rescue medication at all visits until Week 52. Bleeding events ≥ Grade II are assessed by the modified World Health Organization (WHO) Bleeding Scale; Bleeding is graded based on a 1-4 scale (1=minor bleeding to 4=severe bleeding). Characteristics of bleeding events ≥ Grade II include: epistaxis ≥30 minutes, large purpura, joint bleeding, melanotic stool, hematemesis, gross hematuria, abnormal vaginal bleeding, hemoptysis, Visible blood in body cavity fluid, retinal bleeding, bleeding at invasive sites, bleeding requiring transfusion, bleeding associated with moderate or severe hemodynamic instability, fatal bleeding, CNS bleeding.

  Study treatment discontinuation until week 52

Secondary Outcomes (12)

• Percentage of Patients With Overall Response at Week 52

  Study treatment discontinuation until week 52

• Duration of Sustained Response Off Treatment

  from last dose of study treatment until loss of response, approx. 52 weeks

• Overall Response by Week 4

  By Week 4

• Complete Response by Week 4

  By Week 4

• Absolute Change in Platelet Count From Pre-treatment/Screening to Baseline and to Various Time Points

  Pre-treatment/screening, Week 1 (baseline), 2, 4, 13, 27, and 53

Study Arms (2)

Eltrombopag + Dexamethasone

EXPERIMENTAL

Patients were treated with eltrombopag in combination with a standard high-dose dexamethasone (1 cycle: 40 mg once daily (QD) from day 1-4) to induce sustained response off treatment.

Drug: Eltrombopag; Drug: Dexamethasone

Dexamethasone

ACTIVE COMPARATOR

Patients were treated with a standard high-dose dexamethasone (1-3 cycles: 40 mg QD day 1-4 at 4 weeks intervals (or at 14-28 days intervals if needed) to induce sustained response off treatment.

Drug: Dexamethasone

Interventions

Eltrombopag DRUG

Eltrombopag is for oral use and comes in 25, 50 and 75 mg tablets. Prescribed dose is taken once daily.

Also known as: ETB115

Eltrombopag + Dexamethasone

Dexamethasone DRUG

Dexamethasone is for oral use and comes in 8 mg tablets. Prescribed dose is taken once daily.

Dexamethasone; Eltrombopag + Dexamethasone

Eligibility Criteria

• Age: 18 Years - 99 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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)

You may not qualify if:

• 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 physician´s discretion
• Patients with a history of thromboembolic events in the 6 months preceding enrollment 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 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

Sponsors & Collaborators

• Novartis Pharmaceuticals: lead

Study Sites (10)

Novartis Investigative Site

Aschaffenburg, Bavaria, 63739, Germany

Location

Novartis Investigative Site

Aachen, 52074, Germany

Location

Novartis Investigative Site

Chemnitz, 09113, Germany

Location

Novartis Investigative Site

Donauwörth, 86609, Germany

Location

Novartis Investigative Site

Dortmund, 44263, Germany

Location

Novartis Investigative Site

Dresden, 01307, Germany

Location

Novartis Investigative Site

Frankfurt, 60590, Germany

Location

Novartis Investigative Site

Jena, 07740, Germany

Location

Novartis Investigative Site

Kiel, 24116, Germany

Location

Novartis Investigative Site

Kronach, 96317, Germany

Location

Related Links

• A Plain Language Trial Summary is available on www.novctrd.com

MeSH Terms

Conditions

Purpura, Thrombocytopenic, Idiopathic

Interventions

eltrombopag; Dexamethasone

Condition Hierarchy (Ancestors)

Purpura, Thrombocytopenic; Purpura; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Thrombotic Microangiopathies; Thrombocytopenia; Blood Platelet Disorders; Cytopenia; Hemorrhagic Disorders; Autoimmune Diseases; Immune System Diseases; Hemorrhage; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Skin Manifestations; Signs and Symptoms

Intervention Hierarchy (Ancestors)

Pregnadienetriols; Pregnadienes; Pregnanes; Steroids; Fused-Ring Compounds; Polycyclic Compounds; Steroids, Fluorinated

Results Point of Contact

• Title: Clinical Disclosure Office
• Organization: Novartis Pharmaceuticals

novartis.email@novartis.com

862-778-3000

Study Officials

• Novartis Pharmaacueticals

  Novartis Pharmaceuticals

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 12, 2020
• First Posted: April 15, 2020
• Study Start: October 9, 2020
• Primary Completion: September 22, 2023
• Study Completion: September 22, 2023
• Last Updated: May 16, 2025
• Results First Posted: March 17, 2025

Record last verified: 2025-05

Data Sharing

• IPD Sharing: Will share

Locations

DE (10)