Immunomodulation With Romiplostim in Young Adults With ITP

• Status: Completed
• Trial end date: 2020-03-01
• Target enrollment: 0
• Participant gender: All

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).

• Phase: Phase 4
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: University Hospital, Basel, Switzerland
• Collaborator: University Children's Hospital Basel

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