A Study to Assess the Efficacy, Safety, and Tolerability of Rozanolixizumab in Adult Study Participants With Persistent or Chronic Primary Immune Thrombocytopenia (ITP)

Study Overview
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Brief Summary
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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).

Official Title
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A Phase 3 Multicenter, Double-Blind, Randomized, Placebo-Controlled Study to Evaluate the Efficacy, Safety, and Tolerability of Rozanolixizumab in Adult Study Participants With Persistent or Chronic Primary Immune Thrombocytopenia (ITP)

Conditions
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Primary Immune Thrombocytopenia

Intervention / Treatment
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* Drug: Rozanolixizumab
* Other: Placebo


Participation Criteria
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Eligibility Criteria
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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: 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 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, of 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) etc. 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

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Rozanolixizumab<br>Study participants randomized to this arm will receive fixed-unit doses of rozanolixizumab across body weight tiers at pre-specified time points during the Treatment Period. Doses will be adjusted based on platelet count values or medical needs. | Drug: Rozanolixizumab<br>* Study participants receive rozanolixizumab by subcutaneous infusion at pre-specified time points.<br>* Other names: UCB7665;|
| Placebo Comparator: Placebo<br>Study participants randomized to this arm receive placebo at pre-specified time points during the Treatment Period. | Other: Placebo<br>* Study participants receive placebo by subcutaneous infusion at pre-specified time points.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Percentage of Participants With Durable Clinically Meaningful Platelet Response of ≥50×10^9/L, for at Least 8 Out of 12 Weeks During the Last 12 Weeks | Percentage of Participants With Durable Clinically Meaningful Platelet Response of ≥50×10^9/L, for at least 8 out of 12 weeks during the last 12 weeks were reported. | During the last 12 weeks (Week 13 to Week 25) | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Cumulative Number of Weeks With Clinically Meaningful Platelet Response of ≥50×10^9/L Over the 24-week Treatment Period | Total number of weeks with platelet counts ≥50×10^9/L over the 24-week Treatment Period of the study (Week 1 to Week 25) were reported. | Week 1 up to Week 25 | 
| Time to First Clinically Meaningful Platelet Response (CMPR) of ≥50×10^9/L: Time From Starting Treatment to Achievement of First Response of ≥50×10^9/L | Time from starting treatment to achievement of first Clinically Meaningful Platelet Response of ≥50×10^9/L was defined as date of first clinically meaningful response - date of first treatment + 1. Median was calculated based upon the Kaplan-Meier estimate. | Time from starting treatment to achievement of first response of ≥50×10^9/L (up to Week 25) | 
| Percentage of Participants With Clinically Meaningful Platelet Response of ≥50×10^9/L by Day 8 | Clinically meaningful platelet response was defined as platelet count of ≥50×10^9/L. | Baseline to Day 8 | 
| Percentage of Participants With Response Defined as Platelet Count ≥30*10^9/L and at Least Doubling of Baseline, at Least 2 Separate Occasions at Two Adjacent Nominal Visits at Least 7 Days Apart, and Absence of Bleeding | Response was defined as platelet count ≥30*10^9/L and at least doubling of baseline, at least 2 separate occasions at two adjacent nominal visits at least 7 days apart, and absence of bleeding. | From Baseline during Treatment Period (up to Week 25) | 
| Time to First Rescue Therapy | Time to first rescue therapy was defined as date of first rescue therapy use - date of first treatment + 1. Median was calculated based upon the Kaplan-Meier estimate. | From Baseline to first rescue therapy (up to Week 25) | 
| Change From Baseline to Week 25 in Primary Immune Thrombocytopenia Patient Assessment Questionnaire (ITP-PAQ) Symptoms Score | The ITP-PAQ Version 1 is a 44 item disease-specific Health-Related Quality of Life questionnaire developed for use in adults with chronic ITP. It includes 10 scales, Four of the scales measure physical health: Symptoms (6 items), Bother (3 items), Fatigue (4 items), and Activity (2 items). Two of the scales measure emotional health: Fear (5 items) and Psychological (5 items) Health. The remaining four scales measure other aspects of quality of life (QOL): Work QOL (4 items), Social QOL (4 items), Women's Reproductive QOL (6 items) and Overall QOL (5 items). Each item is rated on a Likert-type scale containing 4 to 7 responses. All item scores are transformed to a 0 to 100 continuum and are weighted equally to derive individual scale scores and the total score (0-100) is calculated as per the formula: Sum of item scores within the scale/raw sum range*100. Higher scores indicate better health status. | From Baseline during Treatment Period (up to Week 25) | 
| Percentage of Participants With Treatment-emergent Adverse Events (TEAEs) | An adverse event (AE) is any untoward medical occurrence in a patient or clinical study participant, temporally associated with the use of investigational medicinal product (IMP), whether or not considered related to the IMP. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease (new or exacerbated) temporally associated with the use of IMP. TEAEs are defined as AEs starting after the time of first IMP administration up to and including 8 weeks (56 days) after the final dose. | From Baseline to end of Safety Follow-Up Period (up to Week 31) | 
| Percentage of Participants With TEAEs Leading to Withdrawal of Investigational Medicinal Product (ie, Study Discontinuation) | An AE is any untoward medical occurrence in a patient or clinical study participant, temporally associated with the use of IMP, whether or not considered related to the IMP. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease (new or exacerbated) temporally associated with the use of IMP. TEAEs are defined as AEs starting after the time of first IMP administration up to and including 8 weeks (56 days) after the final dose. | From Baseline to end of Safety Follow-Up Period (up to Week 31) | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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ITP, UCB7665, Rozanolixizumab, Primary Immune Thrombocytopenia