A Study to Assess the Ability of Eltrombopag to Induce Sustained Response Off Treatment in Subjects With ITP

NCT03524612 | Completed Phase 2 Results FDA Drug

• 2 other identifiers: CETB115J24112018-000452-18
• Study Type: interventional
• Target: 105
• Locations: 15 countries
• Sites: 32

Brief 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.

Conditions

Immune Thrombocytopenic Purpura (ITP)

Keywords

Thrombocytopenic; Immune; Platelets; Bleeding; ITP; eltrombopag; ETB115; induce sustained response off treatment; refractory or relapsed; first-line steroids

Outcome Measures

Primary Outcomes (1)

• Percentage of Participants With Sustained Response Off Treatment (SRoT) by 12 Months

  Sustained response off treatment (SRoT) was defined as: reaching platelet count \>= 100×10\^9/L (complete response \[CR\]) and then maintaining platelet counts around 100×10\^9/L for 2 months (no counts below 70×10\^9/L), AND then tapering off the drug until treatment discontinuation while maintaining platelet count \>= 30×10\^9/L in the absence of bleeding (no bleeding AEs) or use of any rescue therapy until month 12.

  Month 12

Secondary Outcomes (22)

• Median Duration of Sustained Response Off Treatment (SRoT) After Treatment Discontinuation for Participants With Sustained Response Off Treatment

  From last dose of eltrombopag to month 12

• 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

  From last dose of eltrombopag to relapse, assessed up to month 24

• Estimated Median Duration of Sustained Response Off Treatment (SRot) for All Patients

  From last dose of eltrombopag to month 24

• Percentage of Participants With Sustained Response Off Treatment Until Month 24

  Month 15, 18, 21 and 24

• Percentage of Participants With Early Response Within First Month

  By 1 month

Study Arms (1)

eltrombopag

OTHER

Participants were treated with eltrombopag to induce sustained response off treatment to reach a target platelet count of \>=100×10\^9/L (CR), after 1st line steroids had failed.

Drug: eltrombopag

Interventions

eltrombopag DRUG

12.5, 25, 50 and 75 mg tablets for oral use once daily

Also known as: ETB115

eltrombopag

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
• 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 \< 30×10\^9/L and assessed as needing treatment (per physician's discretion

You may not qualify if:

• ITP patients previously treated with any ITP second-line therapies, thrombopoietin receptor (TPO-R) agonists for ITP, except steroids / IVIG
• Patients who relapsed more than one year after the end of first-line full course of steroid therapy
• Patients with a diagnosis of secondary thrombocytopenia
• Patients who have life threatening bleeding complications per investigator discretion
• Patients who had a deep vein thrombosis or arterial thrombosis in the 6 months preceding enrollment
• Serum creatinine ≥ 1.5 mg/dL
• Total bilirubin \> 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

Sponsors & Collaborators

• Novartis Pharmaceuticals: lead

Study Sites (32)

Hematology Oncology Association of Rockland

Nyack, New York, 10960, United States

Location

Case Western Reserve

Cleveland, Ohio, 44106-5000, United States

Location

Novartis Investigative Site

Linz, 4010, Austria

Location

Novartis Investigative Site

Salvador, Estado de Bahia, 41253-190, Brazil

Location

Novartis Investigative Site

Rio de Janeiro, Rio de Janeiro, 20211-030, Brazil

Location

Novartis Investigative Site

São Paulo, São Paulo, 05319-000, Brazil

Location

Novartis Investigative Site

Temuco, Región de la Araucanía, 4800827, Chile

Location

Novartis Investigative Site

Viña del Mar, Región de Valparaíso, 2540364, Chile

Location

Novartis Investigative Site

Caen, 14033, France

Location

Novartis Investigative Site

Pessac, 33604, France

Location

Novartis Investigative Site

Athens, 115 27, Greece

Location

Novartis Investigative Site

Pátrai, 265 00, Greece

Location

Novartis Investigative Site

Bologna, BO, 40138, Italy

Location

Novartis Investigative Site

Trieste, TS, 34129, Italy

Location

Novartis Investigative Site

Nagoya, Aichi-ken, 466-8650, Japan

Location

Novartis Investigative Site

Guadalajara, Jalisco, 44160, Mexico

Location

Novartis Investigative Site

Mexico City, 14000, Mexico

Location

Novartis Investigative Site

Muscat, 123, Oman

Location

Novartis Investigative Site

Moscow, 125167, Russia

Location

Novartis Investigative Site

Saint Petersburg, 191024, Russia

Location

Novartis Investigative Site

Málaga, Andalusia, 29010, Spain

Location

Novartis Investigative Site

Salamanca, Castille and León, 37007, Spain

Location

Novartis Investigative Site

Barcelona, Catalonia, 08035, Spain

Location

Novartis Investigative Site

Madrid, 28009, Spain

Location

Novartis Investigative Site

Madrid, 28046, Spain

Location

Novartis Investigative Site

Madrid, 28222, Spain

Location

Novartis Investigative Site

Murcia, 30008, Spain

Location

Novartis Investigative Site

Bern, 3010, Switzerland

Location

Novartis Investigative Site

Aydin, 09100, Turkey (Türkiye)

Location

Novartis Investigative Site

Edirne, 22030, Turkey (Türkiye)

Location

Novartis Investigative Site

Kocaeli, 41380, Turkey (Türkiye)

Location

Novartis Investigative Site

London, W12 0HS, United Kingdom

Location

Related Publications (1)

• Cooper N, Ghanima W, Vianelli N, Valcarcel D, Yavasoglu I, Melikyan A, Ruiz EY, Haenig J, Somenzi O, Lee J, Clark J, Zhang Y, Zaja F. Sustained response off-treatment in eltrombopag-treated adult patients with ITP who are refractory or relapsed after first-line steroids: Primary, final, and ad-hoc analyses of the Phase II TAPER trial. Am J Hematol. 2024 Jan;99(1):57-67. doi: 10.1002/ajh.27131. Epub 2023 Nov 28.

  PMID: 38014779 DERIVED

MeSH Terms

Conditions

Purpura, Thrombocytopenic, Idiopathic; Hemorrhage; Recurrence

Interventions

eltrombopag

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; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Skin Manifestations; Signs and Symptoms; Disease Attributes

Results Point of Contact

• Title: Study Director
• Organization: Novartis Pharmaceuticals

Novartis.email@Novartis.com

+ 1 862 778 8300

Study Officials

• Novartis Pharmaceuticals

  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: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 2, 2018
• First Posted: May 15, 2018
• Study Start: November 2, 2018
• Primary Completion: October 22, 2021
• Study Completion: October 3, 2022
• Last Updated: January 7, 2025
• Results First Posted: December 27, 2023

Record last verified: 2025-01

Data Sharing

• IPD Sharing: Will share

Locations

US (2); RU (2); IT (2); CL (2); AU (1); TU (3); FR (2); BR (3); JP (1); ME (2); OM (1); CH (1); GB (1); GR (2); ES (7)