Immunomodulation With Romiplostim in Young Adults With ITP

NCT ID: NCT02760251

Last Updated: 2020-05-07

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE4
• Total Enrollment: 15 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2016-04-30
• Study Completion Date: 2020-03-31

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

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Romiplostim

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.

Group Type: EXPERIMENTAL

romiplostim

Intervention Type: DRUG

Interventions

romiplostim

Intervention Type: DRUG

Other Intervention Names

Nplate

Eligibility Criteria

Inclusion Criteria

• 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

Exclusion Criteria

• 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
• Drugs with a known impact on the immune system or on platelet function must be recorded and an exclusion of the study should be discussed with the study center
• 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
• Participation in another study with investigational drug within the 30 days preceding and during the present study
• Previous enrolment into the current study
• Previous treatment with romiplostim or eltrombopag
• Hypersensitivity to the active substance or to any of the excipients or to E. coli derived proteins
• Enrolment of the investigator, his/her family members, employees and other dependent persons

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 45 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

University Children's Hospital Basel

OTHER

Sponsor Role: collaborator

University Hospital, Basel, Switzerland

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Thomas Kühne, Prof.Dr.med

Role: PRINCIPAL_INVESTIGATOR

UKBB

Locations

Liestal Cantonal Hospital

Liestal, Basel-Landschaft, Switzerland

Lucerne Cantonal Hospital

Lucerne, Canton of Lucerne, Switzerland

Aarau Cantonal Hospital

Aarau, , Switzerland

University Hospital Basel

Basel, , Switzerland

University Hospital Bern

Bern, , Switzerland

Countries

Switzerland

References

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.

Reference Type: DERIVED

PMID: 37735543 (View on PubMed)

Other Identifiers

20149180

Identifier Type: -

Identifier Source: org_study_id