A Study to Investigate the Long-term Safety, Tolerability, and Efficacy of Rozanolixizumab in Study Participants With Persistent or Chronic Primary Immune Thrombocytopenia (ITP)

Primary Immune Thrombocytopenia Clinical Trial

— myOpportunITy3  

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)

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT04596995
• Other study ID #: TP0004
• Secondary ID: 2019-000883-40
• Status: Terminated
• Phase: Phase 3
• Start date: January 6, 2021
• Est. completion date: December 21, 2022

Study information

• Verified date: January 2024
• Source: UCB Pharma
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 43
• Est. completion date: December 21, 2022
• Est. primary completion date: December 21, 2022
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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)

Related Conditions & MeSH terms

• Primary Immune Thrombocytopenia
• Purpura, Thrombocytopenic, Idiopathic
• Thrombocytopenia

Intervention

Drug:
• Rozanolixizumab
Study participants receive rozanolixizumab by subcutaneous infusion during the Treatment Period.

Locations

Country	Name	City	State
China	Tp0004 20179	Fuzhou
China	Tp0004 20185	Jinan
China	Tp0004 20194	Wuxi
Georgia	Tp0004 20050	Tbilisi
Germany	Tp0004 40369	Berlin
Hungary	Tp0004 40202	Gyor
Hungary	Tp0004 40178	Nyíregyháza
Italy	Tp0004 40208	Firenze
Japan	Tp0004 20039	Iruma-gun
Moldova, Republic of	Tp0004 20051	Chisinau
Poland	Tp0004 40218	Gdansk
Poland	Tp0004 40222	Skorzewo
Poland	Tp0004 40219	Slupsk
Poland	Tp0004 40223	Warszawa
Russian Federation	Tp0004 20052	Moscow
Russian Federation	Tp0004 20053	Saint Petersburg
Spain	Tp0004 40268	Madrid
Taiwan	Tp0004 20099	Taipei
Taiwan	Tp0004 20095	Taipei City
Ukraine	Tp0004 20061	Cherkasy
Ukraine	Tp0004 20060	Dnipropetrovsk
Ukraine	Tp0004 20062	Ivano-frankivsk
Ukraine	Tp0004 20063	Kyiv
Ukraine	Tp0004 20064	Kyiv
Ukraine	Tp0004 20100	Zaporizhzhia
United Kingdom	Tp0004 40234	Plymouth
United States	Tp0004 50243	Boston	Massachusetts

Sponsors (1)

Lead Sponsor	Collaborator
UCB Biopharma SRL

Countries where clinical trial is conducted

United States,  China,  Georgia,  Germany,  Hungary,  Italy,  Japan,  Moldova, Republic of,  Poland,  Russian Federation,  Spain,  Taiwan,  Ukraine,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Percentage of Participants With Treatment-emergent Adverse Events (TEAEs)	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.	From Baseline to end of Safety Follow-Up Period (up to Week 60)
Primary	Percentage of Participants With TEAEs Leading to Permanent Withdrawal of Rozanolixizumab (ie, Study Discontinuation)	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.	From Baseline to end of Safety Follow-Up Period (up to Week 60)
Secondary	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	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.	Over the 52-week Treatment Period (starting at Week 4)
Secondary	Change From Baseline in Immune Thrombocytopenia-Patient Assessment Questionnaire (ITP-PAQ) to Week 53 or 55 Symptoms Domain Score	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.	Week 53 or 55, compared to Baseline