The purpose of this study is to assess the long-term safety, tolerability and clinical efficacy of treatment with rozanolixizumab.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
43
Study participants receive rozanolixizumab by subcutaneous infusion during the Treatment Period.
Tp0004 50243
Boston, Massachusetts, United States
Tp0004 20179
Fuzhou, China
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 60)
Percentage of Participants With TEAEs Leading to Permanent Withdrawal of Rozanolixizumab (ie, Study Discontinuation)
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 60)
Percentage of Participants With Stable Clinically Meaningful Response Without Rescue Therapy at ≥70% of the Visits Over the Planned 52-week Treatment Period Starting at Week 4
Stable Clinically Meaningful Response was defined as Clinically Meaningful Response (ie, platelet count ≥50×10\^9/L) without rescue therapy at ≥70% of the visits over the planned 52-week Treatment Period starting at Week 4.
Time frame: Over the 52-week Treatment Period (starting at Week 4)
Change From Baseline in Immune Thrombocytopenia-Patient Assessment Questionnaire (ITP-PAQ) to Week 53 or 55 Symptoms Domain Score
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Tp0004 20185
Jinan, China
Tp0004 20194
Wuxi, China
Tp0004 20050
Tbilisi, Georgia
Tp0004 40369
Berlin, Germany
Tp0004 40202
Győr, Hungary
Tp0004 40178
Nyíregyháza, Hungary
Tp0004 40208
Florence, Italy
Tp0004 20039
Iruma-gun, Japan
...and 17 more locations
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: Week 53 or 55, compared to Baseline