Clinical trial NCT04596995: A Study to Investigate the Long-term Safety, Tolerability, and Efficacy of Rozanolixizumab in Study Participants With Persistent or Chronic Primary Immune Thrombocytopenia (ITP)
Official title: An Open-Label Extension Study to Investigate the Long-Term Safety, Tolerability, and Efficacy of Rozanolixizumab in Study Participants With Persistent or Chronic Primary Immune Thrombocytopenia (ITP)
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE3
Conditions: Primary Immune Thrombocytopenia
Keywords: ITP
UCB7665
Rozanolixizumab
Primary immune thrombocytopenia
Lead sponsor: UCB Pharma
Sponsor class: INDUSTRY
First posted: 2020-10-22
Enrollment: 43
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: The purpose of this study is to assess the long-term safety, tolerability and clinical efficacy of treatment with rozanolixizumab.


Eligibility criteria: Inclusion 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:

  1. 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
  2. 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

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 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)

Primary outcomes:
measure: Percentage of Participants With Treatment-emergent Adverse Events (TEAEs)
timeFrame: From Baseline to end of Safety Follow-Up Period (up to Week 60)
measure: Percentage of Participants With TEAEs Leading to Permanent Withdrawal of Rozanolixizumab (ie, Study Discontinuation)
timeFrame: From Baseline to end of Safety Follow-Up Period (up to Week 60)
Secondary outcomes:
measure: 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
timeFrame: Over the 52-week Treatment Period (starting at Week 4)
measure: Change From Baseline in Immune Thrombocytopenia-Patient Assessment Questionnaire (ITP-PAQ) to Week 53 or 55 Symptoms Domain Score
timeFrame: Week 53 or 55, compared to Baseline
Other outcomes:
Locations:
city: Boston
country: United States
facility: Tp0004 50243
state: Massachusetts
city: Fuzhou
country: China
facility: Tp0004 20179
city: Jinan
country: China
facility: Tp0004 20185
city: Wuxi
country: China
facility: Tp0004 20194
city: Tbilisi
country: Georgia
facility: Tp0004 20050
city: Berlin
country: Germany
facility: Tp0004 40369
city: Győr
country: Hungary
facility: Tp0004 40202
city: Nyíregyháza
country: Hungary
facility: Tp0004 40178
city: Firenze
country: Italy
facility: Tp0004 40208
city: Iruma-gun
country: Japan
facility: Tp0004 20039
city: Chisinau
country: Moldova, Republic of
facility: Tp0004 20051
city: Gdansk
country: Poland
facility: Tp0004 40218
city: Skorzewo
country: Poland
facility: Tp0004 40222
city: Slupsk
country: Poland
facility: Tp0004 40219
city: Warszawa
country: Poland
facility: Tp0004 40223
city: Moscow
country: Russian Federation
facility: Tp0004 20052
city: Saint Petersburg
country: Russian Federation
facility: Tp0004 20053
city: Madrid
country: Spain
facility: Tp0004 40268
city: Taipei City
country: Taiwan
facility: Tp0004 20095
city: Taipei
country: Taiwan
facility: Tp0004 20099
city: Cherkasy
country: Ukraine
facility: Tp0004 20061
city: Dnipropetrovsk
country: Ukraine
facility: Tp0004 20060
city: Ivano-frankivsk
country: Ukraine
facility: Tp0004 20062
city: Kyiv
country: Ukraine
facility: Tp0004 20063
city: Kyiv
country: Ukraine
facility: Tp0004 20064
city: Zaporizhzhia
country: Ukraine
facility: Tp0004 20100
city: Plymouth
country: United Kingdom
facility: Tp0004 40234
Study officials:
affiliation: 001 844 599 2273
name: UCB Cares
role: STUDY_DIRECTOR
References: