Immunomodulation With Eltrombopag in ITP

NCT04812483 | Completed Phase 2

• 1 other identifier: 2021-00044; ks19Schifferli
• Study Type: interventional
• Target: 2
• Locations: 1 country
• Sites: 6

Brief Summary

The study aims to investigate immunomodulatory effects of eltrombopag combined with dexamethasone in young and midlife adult patients with newly diagnosed primary Immune thrombocytopenia (ITP).

Conditions

Primary Immune Thrombocytopenia (ITP)

Keywords

eltrombopag; thrombopoietin receptor agonist (TPO-RA); ITP in young and midlife adults

Outcome Measures

Primary Outcomes (1)

• Change in percentual T-regulatory cells (Tregs)

  Assessment of the percentage of Tregs (Tregs/CD4) in the study arm compared to the standard arm. The Tregs will be defined as CD4+CD25+ CD127+ in the fluorescence-activated cell sorting (FACS).

  before (Tregs/CD4), at week 3 and at the end of the treatment (week 20)

Secondary Outcomes (3)

• Change in Th1/Th2 balance

  at baseline and weeks 3, 20 and 30

• Clinical response to eltrombopag therapy

  trial duration (baseline to week 30)

• Platelet response to eltrombopag

  at baseline and weeks 6, 20 and 30

Other Outcomes (2)

• Number of Adverse Events

  trial duration (baseline to week 30)

• Severe bleeding

  trial duration (baseline to week 30)

Study Arms (2)

Standard Arm

OTHER

HD-DXM will be administered orally (40 mg) from day 1-4, followed by Arm 1: No planed further treatment. = standard therapy (without eltrombopag)

Drug: standard therapy (without eltrombopag): HD-DXM

Study Arm

EXPERIMENTAL

HD-DXM will be administered orally (40 mg) from day 1-4, followed by Arm 2: The subjects in the experimental arm will be treated with eltrombopag: Eltrombopag (Revolade®), 50 mg PO, from day 5-140. Tapering over 1 week (week 21) from day 141-148 with 50 mg every second day. Eltrombopag will be administered on a starting dose of 50mg. After the end of treatment a clinical and laboratory observation follow-up period until week 30 follows.

Drug: Eltrombopag (Revolade®); Drug: standard therapy (without eltrombopag): HD-DXM

Interventions

Eltrombopag (Revolade®) DRUG

Eltrombopag is a thrombopoietin receptor agonist (TPO-RA) indicated in patients with ITP refractory to first-line drugs or lasting more than 6 months. Administration of Eltrombopag (Revolade®), 50 mg PO, from day 5-140. Tapering over 1 week from day 141-148 with 50 mg every second day.

Study Arm

standard therapy (without eltrombopag): HD-DXM DRUG

standard therapy (without eltrombopag): HD-DXM administered orally (40 mg) from day 1-4

Standard Arm; Study Arm

Eligibility Criteria

• Age: 18 Years - 55 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64)

You may qualify if:

• Informed consent as documented by signature
• Newly diagnosed primary ITP according to the definition of Rodeghiero et al. and a risk of platelet count of \<30x109/l or risk of severe bleeding
• First-line therapy maximum for 1 week prior to enrolment

You may not qualify if:

• Patients previously treated for ITP more than 7 days prior to enrolment (e.g. Steroid, intravenous immunoglobulin (IVIG), platelet infusion)
• Patients treated with second-line drugs prior to enrolment
• Life-threatening bleeding (and inability to sign informed consent)
• Secondary ITP
• Positive family history for ITP
• Presence or history of autoimmune disease as judged by the investigator
• Hepatosplenomegaly in the clinical examination
• Relevant hepatic disease as judged by the investigator
• Presence or history of thromboembolic disease
• Patients with splenectomy
• Women who are pregnant or breast feeding
• Intention to become pregnant during the course of the study
• Lack of safe double contraception
• Any vaccination 2 weeks prior start of the study
• Immunsuppressive and antiplatelet drugs

Sponsors & Collaborators

• University Children's Hospital Basel: lead
• Stiftung zur Förderung medizinischer und biologischer Forschung: collaborator
• Novartis Pharmaceuticals: collaborator
• University of Erlangen-Nürnberg, Department of Biology: collaborator

Study Sites (6)

Aarau Cantonal Hospital, Division of Hematology

Aarau, Switzerland

Location

University Children's Hospital Basel (UKBB)

Basel, 4031, Switzerland

Location

University Hospital Basel, Division of Hematology

Basel, 4031, Switzerland

Location

University Hospital Bern, Division of Hematology

Bern, Switzerland

Location

Liestal Cantonal Hospital, Division of Hematology

Liestal, Switzerland

Location

Lucerne Cantonal Hospital, Division of Hematology

Lucerne, Switzerland

Location

MeSH Terms

Conditions

Purpura, Thrombocytopenic, Idiopathic

Interventions

eltrombopag; Standard of Care

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)

Quality Indicators, Health Care; Quality of Health Care; Health Services Administration; Health Care Quality, Access, and Evaluation

Study Officials

• Alexandra Schifferli, Dr. med.

  University Children's Hospital Basel, UKBB

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: NONE
• Masking Details: The laboratory team will be kept blinded regarding information about response or non-response of patients.
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Model Details: Randomized study evaluating the immunomodulatory effects of eltrombopag versus standard high-dose (HD) DXM therapy (1:1) in newly diagnosed ITP.

Study Record Dates

• First Submitted: March 1, 2021
• First Posted: March 23, 2021
• Study Start: September 1, 2022
• Primary Completion: April 11, 2024
• Study Completion: April 11, 2024
• Last Updated: October 15, 2024

Record last verified: 2024-10

Locations

CH (6)