Immunomodulation With Eltrombopag in ITP

• Register: ClinicalTrials.gov
• Last refreshed on: 28 October 2024
• Main ID: NCT04812483
• Date of registration: 01/03/2021
• Prospective Registration: Yes
• Primary sponsor: University Children's Hospital Basel
• Public title: Immunomodulation With Eltrombopag in ITP iROM2
• Scientific title: Immunomodulation in Young and Midlife Adults With Newly Diagnosed Primary Immune Thrombocytopenia (ITP): A Randomized Open Label Trial With High-dose Dexamethasone Versus Eltrombopag and High-dose Dexamethasone
• Date of first enrolment: September 1, 2022
• Target sample size: 2
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/ct2/show/NCT04812483
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 2

Countries of recruitment

Switzerland

Contacts

• Name: Alexandra Schifferli, Dr. med.
• Affiliation: University Children's Hospital Basel, UKBB

Key inclusion & exclusion criteria

Inclusion Criteria:

- 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

- Bleeding severity and quality of life are neither an inclusion nor an exclusion
criterion.

Exclusion Criteria:

- 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

- 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, incompetence to judge

- Participation in another study with investigational drug within the 30 days
preceding and during the present study

- Enrolment of the investigator, his/her family members, employees and other dependent
persons


Age minimum: 18 Years
Age maximum: 55 Years
Gender: All

Health Condition(s) or Problem(s) studied

Primary Immune Thrombocytopenia (ITP)

Intervention(s)

Drug: Eltrombopag (Revolade®)

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

Primary Outcome(s)

Change in percentual T-regulatory cells (Tregs) [Time Frame: before (Tregs/CD4), at week 3 and at the end of the treatment (week 20)]

Secondary Outcome(s)

Platelet response to eltrombopag [Time Frame: at baseline and weeks 6, 20 and 30]

Change in Th1/Th2 balance [Time Frame: at baseline and weeks 3, 20 and 30]

Clinical response to eltrombopag therapy [Time Frame: trial duration (baseline to week 30)]

Secondary ID(s)

2021-00044; ks19Schifferli

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Novartis Pharmaceuticals

Stiftung zur Förderung medizinischer und biologischer Forschung

University of Erlangen-Nürnberg, Department of Biology