Immunomodulation With Romiplostim in Young Adults With ITP (iROM)

University Hospital Basel

Status and phase

Completed

Phase 4

Conditions

Immune Thrombocytopenia

Treatments

Drug: romiplostim

Study type

Interventional

Funder types

Other

Identifiers

NCT02760251

20149180

Details and patient eligibility

About

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).

Enrollment

15 patients

Sex

All

Ages

18 to 45 years old

Volunteers

No Healthy Volunteers

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