A Study to Investigate the Long-term Safety, Tolerability, and Efficacy of Rozanolixizumab in Study Participants With Persistent or Chronic Primary Immune Thrombocytopenia (ITP)
- Registration Number
- NCT04596995
- Lead Sponsor
- UCB Biopharma SRL
- Brief Summary
The purpose of this study is to assess the long-term safety, tolerability and clinical efficacy of treatment with rozanolixizumab.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- TERMINATED
- Sex
- All
- Target Recruitment
- 43
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Study participant completed TP0003 [NCT04200456] or TP0006 [NCT04224688] until Visit 27 (Week 25) and, in the opinion of the investigator, has been compliant with the TP0003 or TP0006 study assessments
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The study participant is considered reliable and capable of adhering to the protocol, visit schedule, or medication intake according to the judgment of the investigator
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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 urine 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 final dose of study treatment
- Study participant has any ongoing investigational medicinal product (IMP)-related serious adverse event (SAE) or ongoing severe IMP-related treatment-emergent adverse event (TEAE) experienced during TP0003 or TP0006
- Study participant has, at last available assessment of TP0003 or TP0006, 3.0x upper limit of normal (ULN) of any of the following: alanine aminotransferase (ALT), aspartate aminotransferase (AST), or alkaline phosphatase (ALP)
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description Rozanolixizumab Treatment Arm Rozanolixizumab All study participants 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.
- Primary Outcome Measures
Name Time Method Percentage of Participants With Treatment-emergent Adverse Events (TEAEs) From Baseline to end of Safety Follow-Up Period (up to Week 60) 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.
Percentage of Participants With TEAEs Leading to Permanent Withdrawal of Rozanolixizumab (ie, Study Discontinuation) From Baseline to end of Safety Follow-Up Period (up to Week 60) 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.
- Secondary Outcome Measures
Name Time Method Change From Baseline in Immune Thrombocytopenia-Patient Assessment Questionnaire (ITP-PAQ) to Week 53 or 55 Symptoms Domain Score Week 53 or 55, compared to Baseline 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.
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 Over the 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.
Trial Locations
- Locations (27)
Tp0004 50243
🇺🇸Boston, Massachusetts, United States
Tp0004 20185
🇨🇳Jinan, China
Tp0004 40202
🇭🇺Győr, Hungary
Tp0004 20039
🇯🇵Iruma-gun, Japan
Tp0004 40268
🇪🇸Madrid, Spain
Tp0004 20051
🇲🇩Chisinau, Moldova, Republic of
Tp0004 40219
🇵🇱Slupsk, Poland
Tp0004 40223
🇵🇱Warszawa, Poland
Tp0004 20062
🇺🇦Ivano-frankivsk, Ukraine
Tp0004 20100
🇺🇦Zaporizhzhia, Ukraine
Tp0004 40234
🇬🇧Plymouth, United Kingdom
Tp0004 20064
🇺🇦Kyiv, Ukraine
Tp0004 20179
🇨🇳Fuzhou, China
Tp0004 20194
🇨🇳Wuxi, China
Tp0004 20050
🇬🇪Tbilisi, Georgia
Tp0004 40369
🇩🇪Berlin, Germany
Tp0004 40218
🇵🇱Gdansk, Poland
Tp0004 40208
🇮🇹Firenze, Italy
Tp0004 40178
🇭🇺Nyíregyháza, Hungary
Tp0004 20053
🇷🇺Saint Petersburg, Russian Federation
Tp0004 20052
🇷🇺Moscow, Russian Federation
Tp0004 20095
🇨🇳Taipei City, Taiwan
Tp0004 20061
🇺🇦Cherkasy, Ukraine
Tp0004 20099
🇨🇳Taipei, Taiwan
Tp0004 20060
🇺🇦Dnipropetrovsk, Ukraine
Tp0004 20063
🇺🇦Kyiv, Ukraine
Tp0004 40222
🇵🇱Skorzewo, Poland