A study to investigate the long-term safety, tolerability, and efficacy of rozanolixizumab in study participants with persistent or chronic primary immune thrombocytopenia (ITP)

JRCT ID: jRCT2031210245

Registered date:16/08/2021

Basic Information

• Recruitment status: Not Recruiting
• Health condition(s) or Problem(s) studied: Primary immune thrombocytopenia
• Date of first enrollment: 31/08/2021
• Target sample size: 10
• Countries of recruitment: Austria,Japan,Belgium,Japan,Bulgaria,Japan,Canada,Japan,China,Japan,Croatia,Japan,Czechia,Japan,Denmark,Japan,France,Japan,Georgia,Japan,Germany,Japan,Greece,Japan,Hong Kong,Japan,Hungary,Japan,Italy,Japan,Korea,Japan,Republic of Moldova,Japan,Poland,Japan,Romania,Japan,Russian Federation,Japan,Serbia,Japan,Spain,Japan,Taiwan,Japan,Turkey,Japan,Ukraine,Japan,United Kingdom,Japan,United States,Japan
• Study type: Interventional

Intervention(s)

Study participants receive rozanolixizumab by subcutaneous infusion during the Treatment Period.
Study participants will receive fixed-unit doses of rozanolixizumab across body weight tiers.

Outcome(s)

Primary Outcome

- Occurrence of treatment-emergent adverse events (TEAEs)
- Occurrence of TEAEs leading to withdrawal of rozanolixizumab (ie, study discontinuation

Secondary Outcome

Stable Clinically Meaningful Response without rescue therapy at equal and over 70% of the visits over the planned 52-week Treatment Period starting at Week 4
- Change from Baseline to Week 53 including all intermediate timepoints
for ITP Patient Assessment Questionnaire (ITP-PAQ) Symptoms domain score
- Area under the curve (AUC) of the oral steroid dose over time
- Change in dose and/or frequency of concomitant ITP medications (excluding corticosteroids) over time

Key inclusion & exclusion criteria

• Age minimum: >= 18age old
• Age maximum: Not applicable
• Gender: Both

Include criteria

-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 or 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

Exclude 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 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)

Related Information

• Primary Sponsor: Sugihara Kazuhiro
• Secondary ID(s): 2019-000883-40,NCT04596995