A study to investigate the long-term safety, tolerability, and efficacy of Rozanolixizumab in study participants with persistent or chronic primary immune thrombocytopenia (ITP)
- Conditions
- Primary Immune ThrombocytopeniaMedDRA version: 20.0Level: PTClassification code 10043554Term: ThrombocytopeniaSystem Organ Class: 10005329 - Blood and lymphatic system disordersTherapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
- Registration Number
- EUCTR2019-000883-40-DE
- Lead Sponsor
- CB Biopharma SR
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 180
- 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
- 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
- Study participants may be male or female:
a) 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
b) 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
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 120
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 60
-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 clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Main Objective: Assess the long-term safety and tolerability of repeated treatment with rozanolixizumab<br>;Secondary Objective: - Assess the long-term clinical efficacy of treatment with rozanolixizumab<br>- Assess the effect of rozanolixizumab on study participant perceived symptoms<br>- Assess the reduction in use of steroids and other concomitant ITP medications<br>;Primary end point(s): 1. Occurrence of treatment-emergent adverse events (TEAEs)<br>2. Occurrence of TEAEs leading to withdrawal of rozanolixizumab (ie, study discontinuation)<br><br>;Timepoint(s) of evaluation of this end point: 1. and 2. From Baseline to end of Safety Follow-Up Period (up to Week 60)<br>
- Secondary Outcome Measures
Name Time Method Secondary end point(s): 1. Stable Clinically Meaningful Response without rescue therapy at = 70% of the visits over the planned 52-week Treatment Period starting at Week 4<br>2. Change from Baseline to Week 54 including all intermediate timepoints for ITP Patient Assessment Questionnaire (ITP-PAQ) Symptoms domain score<br>3. Area under the curve (AUC) of the oral steroid dose over time<br>4. Change in dose and/or frequency of concomitant ITP medications (excluding corticosteroids) over time;Timepoint(s) of evaluation of this end point: 1. Over the planned 52-week Treatment Period (starting at Week 4)<br>2.; 3.; 4 From Baseline during the Treatment Period (up to Week 53)