A Study to Evaluate the Efficacy, Safety, and Tolerability of Rozanolixizumab in Adult Study Participants With Persistent or Chronic Primary Immune Thrombocytopenia (ITP)
Condition: Primary Immune Thrombocytopenia
MeSH terms: Thrombocytopenia; Purpura, Thrombocytopenic, Idiopathic
Intervention: Rozanolixizumab; Placebo
Intervention description: Study participants receive rozanolixizumab by subcutaneous infusion at pre-specified time points.; Study participants receive placebo by subcutaneous infusion at pre-specified time points.
Intervention MeSH terms: Rozanolixizumab
Phase: Phase 3
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Masking description: Investigators are blinded to the treatment code, they will see the platelet values.
Enrollment: 33.0
Sex: All
Minimum age: 18 Years
Healthy volunteers: No
Keywords: ITP; UCB7665; Rozanolixizumab; Primary immune thrombocytopenia

Summary:

      The purpose of this study is to demonstrate the clinical efficacy of rozanolixizumab in
      maintenance treatment and assess safety and tolerability of rozanolixizumab in adult study
      participants with primary immune thrombocytopenia (ITP).
    

Eligibility 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 a documented intolerance or insufficient response to two or more
             appropriate 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 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 (<30×10^9/L) prior to
             Screening

          -  Study participant has a platelet count measurement at Screening and at Baseline (Day
             1) with an average of the two <30×10^9/L and no single count may be >35×10^9/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:

               1. 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

               2. 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:

          -  Participant has a history of arterial or venous thromboembolism (eg, stroke, transient
             ischemic attack, 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 study
             medication or any other anti-neonatal Fc receptor (FcRn) medications

          -  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), 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 first dose of investigational medicinal product (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