Complex immunomodulatory therapy for patients with immune thrombocytopenia (T-MEM)

Phase 1

• Conditions: Immune thrombocytopenia; MedDRA version: 23.0Level: PTClassification code: 10083842Term: Immune thrombocytopenia Class: 100000004851; Therapeutic area: Diseases [C] - Hemic and Lymphatic Diseases [C15]; Therapeutic area: Diseases [C] - Immune System Diseases [C20]

• Registration Number: CTIS2023-505788-35-00
• Lead Sponsor: Fakultni Nemocnice Hradec Kralove

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2023-05-21
• Last Update Posted: 22 July 2024

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 73

Inclusion Criteria

Age = 18 years - 70 years, Ability to understand the nature and course of the study and sign written informed consent to participate in the study., Ability and willingness to complete study visit and commute to study center., Diagnosis of primary immune thrombocytopenia., Platelets count below 80x109/l (after termination or on corticotherapy) and fulfillment of the indication for therapy (bleeding, need for anticoagulation, constitutional symptoms, documented refractoriness when stopping corticoids, etc.)., Previous corticosteroid therapy lasting at least 14 days (pulse or continuous), after which, according to the doctor's decision, it is necessary/appropriate to continue ITP therapy., Women of childbearing potential must have a negative pregnancy test (from blood serum) and use effective methods of contraception before starting treatment., For women of childbearing potential: Willingness to prevent pregnancy and breast-feeding during treatment and for 12 months after the last dose of rituximab, using at least one of the highly reliable methods of contraception. For men: Willingness to prevent paternity during treatment and for the next 90 days after stopping mycophenolate treatment.

Exclusion Criteria

Severe bleeding (grade 2 or more according to CTCAE), which requires a more intensive approach (hospitalization, IVIG)., Active cancer in the last 5 years, 11.Previous therapy: •Previous rituximab therapy in the last 12 months. •IVIG therapy or plasmapheresis in the last 28 days before the 1st study treatment administration (visit M1D1). •TPO-RA therapy, fostamatinib, immunosuppressants (MMF, CSA) in the last 28 days before the 1st study treatment administration (visit M1D1). •Cytostatic therapy in the last 28 days before the 1st study treatment administration (visit M1D1)., Participation in a clinical study involving administration of investigational medicinal product within 3 months (or 5 half-terms, whichever is longer) prior to screening., Laboratory findings more than 1.5 x upper limit - ALT, AST, GMT, ALP, creatine, PT, aPTT., Uncontrolled arterial hypertension., Infectious disease in the last 14 days before starting the study treatment (visit M1D1)., Planned surgical procedure in the next 6 months, or until visit M6., Splenectomy in the last 3 months., The presence of another significant disease limiting therapy (liver disease, GIT disease, psychiatric disease /suicide, depression/, etc.), Contraindications to administered drugs (simultaneous use of contraindicated drugs, HIV, hepatitis, liver disease, etc.), Breastfeeding, pregnancy, trying to get pregnant, Myelodysplastic syndrome (MDS) or other abnormalities in the blood count, except for thrombocytopenia (a sternal puncture may be necessary to exclude MDS)., History of Thrombocytopenic thrombotic purpura (TTP), Hemolytic-uremic syndrome (HUS), antiphospholipid syndrome (AFS) or acquired hemophilia, Immunodeficiency, History of anaphylaxis

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: Evaluation of the effectiveness of treatment of a four-combination of evaluated medicinal products compared to standard practice in patients with immune thrombocytopenia.;Secondary Objective: Evaluation of the safety of the treatment of the four-combination of evaluated medicinal products compared to standard practice in patients with immune thrombocytopenia, Comparison of patients' quality of life before, during and after treatment, Determination of the level of biomarkers (kynurenine, neopterin, tryptophan) and their changes in connection with treatment, Evaluation of possible predictive parameters of response to therapy (CD4/8+ lymphocytes, amount of anti-platelet antibodies).;Primary end point(s): Platelets count at visit M3, M7, M12 and M18,, Number of patients in remission without the need for therapy at visit M7, M12, M18

Secondary Outcome Measures

Name	Time	Method
Secondary end point(s):The number of relapses within 12 months of the end of therapy;Secondary end point(s):The number of patients who complete the entire cycle;Secondary end point(s):The results of the standardized SF-36 quality of life questionnaire before starting, during and after treatment;Secondary end point(s):Biomarker levels (kynurenine, neopterin, tryptophan) and their changes in connection with treatment;Secondary end point(s):Values of CD4/8+ lymphocytes and antiplatelet antibodies as possible predictive parameters of response to therapy