Primary Immune Thrombocytopenia MedDRA version: 20.0 Level: PT Classification code 10043554 Term: Thrombocytopenia System Organ Class: 10005329 - Blood and lymphatic system disorders
Conditions
Sponsors
UCB Biopharma SRL
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: -Study participant completed TP0003 [NCT04200456] or TP0006 [NCT04224688] until Visit 28 (Week 25) and, in the opinion of the investigator and sponsor, 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 -If taking allowed concomitant medications, study participant must have been on stable doses - 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 or not planning to get pregnant during the 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 138 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 72
Exclusion criteria
Exclusion criteria: -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 had any relevant resolved IMP-related SAE or severe IMP-related TEAE experienced during TP0003 or TP0006 that was not discussed and approved as acceptable for enrollment into open-label extension (OLE) study by Medical Monitor or designee -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)
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: Assess the long-term safety and tolerability of repeated treatment with rozanolixizumab (Maintenance Treatment Arm only) ;Secondary Objective: -Assess the long-term clinical efficacy of repeated treatment with rozanolixizumab (Maintenance Treatment Arm only) -Assess effect of rozanolixizumab on health-related quality of life (HRQoL) -Assess the pharmacodynamic (PD) effect of rozanolixizumab (Maintenance Treatment Arm only) -Assess the effect of rozanolixizumab on patient-reported outcomes (PROs) -Assess the reduction in use of steroids in study participants receiving rozanolixizumab ;Primary end point(s): 1. Occurrence of treatment-emergent adverse events (TEAEs) 2. Occurrence of TEAEs leading to withdrawal of rozanolixizumab ;Timepoint(s) of evaluation of this end point: 1. and 2. From Baseline to end of Safety Follow-Up Period (up to Week 61) | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): 1. Stable Clinically Meaningful Response without rescue therapy at =70% of the visits starting after the second dose of rozanolixizumab until Week 54 2. Change from Baseline in European Quality of Life-5 Dimension 5 Levels Assessment (EQ5D-5L) 3. Change from Baseline in Short-Form 36-Item (SF-36) Survey 4. Change from Baseline in ITP-Patient Assessment Questionnaire (ITP-PAQ) 5. Response defined as change from Baseline at or above the defined threshold for ITP Patient Assessment Questionnaire (ITP-PAQ) Symptoms Score 6. Minimum value in total serum immunoglobulin G (IgG) concentration over time 7. Maximum decrease from Baseline in total serum IgG concentration over time 8. Change from Baseline in serum IgG subclasses concentration over time 9. Change from Baseline in serum Ig concentrations (IgA, IgE, IgM) over time 10. Change from Baseline in Physical Fatigue Instrument 11. Change from Baseline in Patient Global Impression of Severity (PGI-S) 12. Change from Baseline in Patient Global Impression of Change (PGI-C) 13. Area under the curve (AUC) of the oral steroid dose over time;Timepoint(s) of evaluation of this end point: 1. After second dose of rozanolixizumab until Week 54 (up to Week 54) 2.-7.; 12.; 13. From Baseline during the Treatment Period (up to Week 55) 8.-11. From Baseline during the Treatment Period (up to Week 53) | — |
Countries
Austria, Belgium, Bulgaria, Canada, China, Czech Republic, France, Georgia, Germany, Hong Kong, Hungary, Italy, Japan, Moldova, Republic of, Netherlands, Poland, Romania, Russian Federation, Spain, Taiwan, Ukraine, United Kingdom, United States
Contacts
Public ContactClin Trial Reg & Results Disclosure
UCB Biosciences GmbH
Outcome results
None listed