Immunomodulation With Romiplostim in Young Adults With ITP

NCT02760251 | Completed Phase 4

• 1 other identifier: 20149180
• Study Type: interventional
• Target: 15
• Locations: 1 country
• Sites: 5

Brief Summary

The study aims to investigate immunomodulatory effects of thrombopoietin-receptor Agonist (TPO-RA) in patients with primary ITP, who failed first-line therapy or who became intolerant to it. It is hypothesized that the early phase of this autoimmune disease may exhibit a stronger immunomodulatory potential in response to a stimulus, such as romiplostim. Such a process may subsequently be capable to induce regulatory mechanisms or tolerance. Romiplostim (a thrombopoietin-receptor agonist, TPO-RA) will be administered subcutaneously once weekly over 22 weeks with a starting dose of 1mcg/kg body weight. The dose will be adjusted based on platelet counts as described in the summary of Product Characteristics (SmPC).

Conditions

Immune Thrombocytopenia

Keywords

immunomodulation

Outcome Measures

Primary Outcomes (1)

• Change in Interleukin (IL)-4 concentrations (pg/ml) from baseline to week 22

  The primary aim of the study is to demonstrate an immunomodulatory effect of the study drug. Investigators expect a shift in the Th1/Th2 balance towards Th2. The primary outcome is to compare the pre- and post-treatment IL-4 concentrations (pg/ml) of all included patients (Th2 profile). Assessment of change in pre- and post-treatment IL-4 concentrations (pg/ml).

  baseline and 22 weeks

Secondary Outcomes (16)

• Change in immunomodulation as assessed by immune cell characteristics between baseline and week 22

  baseline and 22 weeks

• Change in immunomodulation as assessed by immune cell characteristics between baseline and week 10

  baseline and 10 weeks

• Change in immunomodulation as assessed by messenger ribonucleic acid (mRNA) of cytokines between baseline and week 22

  baseline and 22 weeks

• Change in immunomodulation as assessed by mRNA of immune cells between baseline and week 22

  baseline and 22 weeks

• Change in immunomodulation as assessed by mRNA of cytokines between baseline and week 10

  baseline and 10 weeks

Study Arms (1)

Romiplostim

EXPERIMENTAL

Romiplostim (a thrombopoietin-receptor agonist, TPO-RA) will be administered subcutaneously once weekly over 22 weeks with a starting dose of 1mcg/kg body weight. The dose will be adjusted based on platelet counts as described in the summary of Product Characteristics (SmPC). Followup examination at week 52.

Drug: romiplostim

Interventions

romiplostim DRUG

Also known as: Nplate

Romiplostim

Eligibility Criteria

• Age: 18 Years - 45 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64)

You may qualify if:

• Informed consent as documented by signature (see informed consent form)
• Primary ITP according to the definition of Rodeghiero et al. (52) and a platelet count of \<30x109/l
• Age range: 18-45 years
• Previously treated patients, with failure or intolerance to first-line therapy, or relapse after first-line therapy, i.e. corticosteroids, intravenous immunoglobulin (IVIG), or anti-D immunoglobulins

You may not qualify if:

• Adults older than 45 and children younger than 18 years
• Platelet count higher than 30x109/l at time of screening
• Suspicion of secondary ITP
• Positive family history for ITP
• Presence or history of autoimmune disease as judged by the investigator
• Hepatosplenomegaly
• Presence or history of relevant hepatic disease as judged by the investigator
• Presence or history of thromboembolic disease as judged by the investigator
• 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 (see 7.1)
• Any vaccination 2 weeks prior start of the study
• Known or suspected non-compliance, drug or alcohol abuse
• Inability to follow the procedures of the study, e.g. due to language problems, psychological disorders, dementia of the study subject

Sponsors & Collaborators

• University Hospital, Basel, Switzerland: lead
• University Children's Hospital Basel: collaborator

Study Sites (5)

Liestal Cantonal Hospital

Liestal, Basel-Landschaft, Switzerland

Location

Lucerne Cantonal Hospital

Lucerne, Canton of Lucerne, Switzerland

Location

Aarau Cantonal Hospital

Aarau, Switzerland

Location

University Hospital Basel

Basel, 4000, Switzerland

Location

University Hospital Bern

Bern, Switzerland

Location

Related Publications (1)

• Schifferli A, Rufer A, Rovo A, Nimmerjahn F, Cantoni N, Holbro A, Favre G, Dirks J, Wieland A, Faeth H, Pereira R, Kuhne T. Immunomodulation with romiplostim as a second-line strategy in primary immune thrombocytopenia: The iROM study. Br J Haematol. 2023 Oct;203(1):119-130. doi: 10.1111/bjh.19074.

  PMID: 37735543 DERIVED

MeSH Terms

Conditions

Purpura, Thrombocytopenic, Idiopathic

Interventions

romiplostim

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

Study Officials

• Thomas Kühne, Prof.Dr.med

  UKBB

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 15, 2016
• First Posted: May 3, 2016
• Study Start: April 1, 2016
• Primary Completion: July 1, 2019
• Study Completion: March 1, 2020
• Last Updated: May 7, 2020

Record last verified: 2020-05

Data Sharing

• IPD Sharing: Will not share

Locations

CH (5)