Primary immune thrombocytopenia
Conditions
Sponsors
UCB Japan Co., Ltd.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: -Study participant must be >=18 years of age at the time of the Screening Visit -Study participant has a diagnosis of persistent (>3 months duration) or chronic (>12 months duration) primary immune thrombocytopenia (ITP) at the Screening Visit -Study participant has documented intolerance or insufficient response to one or more appropriate courses of standard of care ITP treatments prior to Screening -Study participants must have prior history of a response to a previous ITP therapy. -If taking allowed immunosuppressive drugs, study participant must be on stable doses during defined time periods prior to Baseline (Day 1) -Study participant has a documented history of low platelet count (35x10^9/L (using local laboratories) -Study participant has a current or history of a peripheral blood smear consistent with ITP -Study participants may be male or female: a. A male participant must agree to use contraception during the Treatment Period and for at least 3 months after the final dose of study treatment and refrain from donating sperm during this period b. A female participant is eligible to participate if she is not pregnant as confirmed by a negative serum pregnancy test and not planning to get pregnant during the participation in the study, not breastfeeding, and at least one of the following conditions applies: Not a woman of childbearing potential (WOCBP) OR A WOCBP who agrees to follow the contraceptive guidance during the Treatment Period and for at least 3 months after the dose of study treatment
Exclusion criteria
Exclusion criteria: -Participant has a history of arterial or venous thromboembolism (eg, stroke, transient ischemic attach, myocardial infarction, deep vein thrombosis or pulmonary embolism) within the 6 months prior to randomization or requires current anticoagulant treatment -Study participant has clinically significant bleeding that warrants immediate platelet adjustment (eg, menorrhagia with significant drop in hemoglobin) -Study participant has a known hypersensitivity to any components of the investigational medicinal product (IMP) or comparative drugs (and/or an investigational device) as stated in this protocol -Study participant has evidence of a secondary cause of immune thrombocytopenia (clear association with other medical conditions, eg untreated H. pylori infection , leukemia, lymphoma, common variable immunodeficiency, systemic lupus erythematosus, autoimmune thyroid disease or is drug induced or participant has a multiple immune cytopenia, eg, Evan's syndrome) -Study participant has a clinically relevant active infection (eg, sepsis, pneumonia, or abscess) in the opinion of the investigator, or had a serious infection (resulting in hospitalization or requiring parenteral antibiotic treatment) within 6 weeks prior to the dose of IMP -Study participant with a known tuberculosis (TB) infection, at high risk of acquiring TB infection, or latent tuberculosis infection (LTBI), or current/history of nontuberculous mycobacterial infection (NTMBI) -Study participant has a history of a major organ transplant or hematopoietic stem cell/marrow transplant -Study participant has experienced intracranial bleed in the last 6 months prior to the Screening Visit -Study participant has a history of coagulopathy disorders other than ITP -Study participant with current or medical history of immunoglobulin A (IgA) deficiency, or a measurement of IgA <50 mg/dL at the Screening Visit -Study participant has undergone a splenectomy in the 2 years prior to the Baseline Visit
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| efficacy Durable Clinically Meaningful Platelet Response of >= 50x10^9/L, for at least 8 out of 12 weeks during the last 12 weeks | — |
Secondary
| Measure | Time frame |
|---|---|
| safety efficacy other 1. Cumulative number of visits with Clinically Meaningful Platelet Response of >=50x10^9/L over the 24-week treatment period 2. Time to first Clinically Meaningful Platelet Response of >=50x10^9/L: time from starting treatment to achievement of first response of>=50x10^9/L 3. Clinically Meaningful Platelet Response of >=50x10^9/L by Day 8 4.Response defined as platelet count >=30x10^9/L and at least a 2-fold increase of the Baseline count confirmed on at least 2 separate occasions at two adjacent nominal visits at least 7 days apart, and absence of bleeding by visit 5. Time to first rescue therapy 6. Change from Baseline to Week 25 in ITP Patient Assessment Questionnaire (ITP-PAQ) Symptoms Score 7. Occurrence of treatment-emergent adverse events (TEAEs) 8. Occurrence of TEAEs leading to withdrawal of investigational medicinal product (IMP) (ie, study discontinuation) | — |
Countries
Asia except Japan, Europe, Japan, North America
Outcome results
None listed