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).
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
30
Study participants receive rozanolixizumab by subcutaneous infusion at pre-specified time points.
Study participants receive placebo by subcutaneous infusion at pre-specified time points.
Tp0006 50412
St. Petersburg, Florida, United States
Tp0006 50243
Boston, Massachusetts, United States
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.
Time frame: During the last 12 weeks (Week 13 to Week 25)
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.
Time frame: 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 frame: 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.
Time frame: 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.
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Tp0006 40189
Plovdiv, Bulgaria
Tp0006 40008
Sofia, Bulgaria
Tp0006 20201
Bengbu, China
Tp0006 20189
Changchun, China
Tp0006 20188
Changsha, China
Tp0006 20186
Changzhou, China
Tp0006 20179
Fuzhou, China
Tp0006 20187
Hangzhou, China
...and 19 more locations
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: From Baseline to end of Safety Follow-Up Period (up to Week 31)