Back to resultsA Study to Investigate the Long-term Safety, Tolerability, and Efficacy of Rozanolixizumab in Study Participants With Persistent or Chronic Primary Immune Thrombocytopenia (ITP) (myOpportunITy3)
Primary Purpose
Primary Immune Thrombocytopenia
Status
Completed
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
Rozanolixizumab
Sponsored by
About this trial
This is an interventional treatment trial for Primary Immune Thrombocytopenia focused on measuring ITP, UCB7665, Rozanolixizumab, Primary immune thrombocytopenia
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:
- 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
- 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)
Sites / Locations
- Tp0004 50243
- Tp0004 20179
- Tp0004 20185
- Tp0004 20194
- Tp0004 20050
- Tp0004 40369
- Tp0004 40202
- Tp0004 40178
- Tp0004 40208
- Tp0004 20039
- Tp0004 20051
- Tp0004 40218
- Tp0004 40222
- Tp0004 40219
- Tp0004 40223
- Tp0004 20052
- Tp0004 20053
- Tp0004 40268
- Tp0004 20095
- Tp0004 20099
- Tp0004 20061
- Tp0004 20060
- Tp0004 20062
- Tp0004 20063
- Tp0004 20064
- Tp0004 20100
- Tp0004 40234
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Rozanolixizumab Treatment Arm
Arm Description
All study participants will receive fixed-unit doses of rozanolixizumab across body weight tiers at pre-specified time points during the Treatment Period. Doses will be adjusted based on platelet count values or medical needs.
Outcomes
Primary Outcome Measures
Occurrence of 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.
Occurrence of 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.
Secondary Outcome Measures
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, 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
Change from Baseline to Week 53 or Week 55 for ITP Patient Assessment Questionnaire (ITP-PAQ) Symptoms domain score
The ITP-PAQ is a 44-item disease-specific quality of life (QoL) questionnaire developed for use in adults with chronic ITP. It includes 11 scales: Symptoms, Fatigue, Physical Health - Bother, Physical Health - Activity, Emotional Health - Psychological, Emotional Health - Fear, Overall QoL, Social Activity, Women's Reproductive Health - Fertility, Women's Reproductive Health - Menstrual Symptoms, and Work. 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. Higher scores indicate better health status.
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT04596995
Brief Title
A Study to Investigate the Long-term Safety, Tolerability, and Efficacy of Rozanolixizumab in Study Participants With Persistent or Chronic Primary Immune Thrombocytopenia (ITP)
Acronym
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)
Study Type
Interventional
2. Study Status
Record Verification Date
September 2023
Overall Recruitment Status
Completed
Study Start Date
December 30, 2020 (Actual)
Primary Completion Date
December 23, 2022 (Actual)
Study Completion Date
December 23, 2022 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
UCB Biopharma SRL
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Product Manufactured in and Exported from the U.S.
No
Data Monitoring Committee
Yes
5. Study Description
Brief Summary
The purpose of this study is to assess the long-term safety, tolerability and clinical efficacy of treatment with rozanolixizumab.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Primary Immune Thrombocytopenia
Keywords
ITP, UCB7665, Rozanolixizumab, Primary immune thrombocytopenia
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
43 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Rozanolixizumab Treatment Arm
Arm Type
Experimental
Arm Description
All study participants will receive fixed-unit doses of rozanolixizumab across body weight tiers at pre-specified time points during the Treatment Period. Doses will be adjusted based on platelet count values or medical needs.
Intervention Type
Drug
Intervention Name(s)
Rozanolixizumab
Other Intervention Name(s)
UCB7665
Intervention Description
Study participants receive rozanolixizumab by subcutaneous infusion during the Treatment Period.
Primary Outcome Measure Information:
Title
Occurrence of treatment-emergent adverse events (TEAEs)
Description
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.
Time Frame
From Baseline to end of Safety Follow-Up Period (up to Week 60)
Title
Occurrence of TEAEs leading to permanent withdrawal of rozanolixizumab (ie, study discontinuation)
Description
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.
Time Frame
From Baseline to end of Safety Follow-Up Period (up to Week 60)
Secondary Outcome Measure Information:
Title
Stable Clinically Meaningful Response without rescue therapy at ≥70% of the visits over the planned 52-week Treatment Period starting at Week 4
Description
Stable Clinically Meaningful Response, 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
Time Frame
Over the planned 52-week Treatment Period (starting at Week 4)
Title
Change from Baseline to Week 53 or Week 55 for ITP Patient Assessment Questionnaire (ITP-PAQ) Symptoms domain score
Description
The ITP-PAQ is a 44-item disease-specific quality of life (QoL) questionnaire developed for use in adults with chronic ITP. It includes 11 scales: Symptoms, Fatigue, Physical Health - Bother, Physical Health - Activity, Emotional Health - Psychological, Emotional Health - Fear, Overall QoL, Social Activity, Women's Reproductive Health - Fertility, Women's Reproductive Health - Menstrual Symptoms, and Work. 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. Higher scores indicate better health status.
Time Frame
Week 53 or Week 55, compared to Baseline
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
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:
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
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)
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
UCB Cares
Organizational Affiliation
001 844 599 2273
Official's Role
Study Director
Facility Information:
Facility Name
Tp0004 50243
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02114
Country
United States
Facility Name
Tp0004 20179
City
Fuzhou
Country
China
Facility Name
Tp0004 20185
City
Jinan
Country
China
Facility Name
Tp0004 20194
City
Wuxi
Country
China
Facility Name
Tp0004 20050
City
Tbilisi
Country
Georgia
Facility Name
Tp0004 40369
City
Berlin
Country
Germany
Facility Name
Tp0004 40202
City
Győr
Country
Hungary
Facility Name
Tp0004 40178
City
Nyíregyháza
Country
Hungary
Facility Name
Tp0004 40208
City
Firenze
Country
Italy
Facility Name
Tp0004 20039
City
Iruma-gun
Country
Japan
Facility Name
Tp0004 20051
City
Chisinau
Country
Moldova, Republic of
Facility Name
Tp0004 40218
City
Gdansk
Country
Poland
Facility Name
Tp0004 40222
City
Skorzewo
Country
Poland
Facility Name
Tp0004 40219
City
Slupsk
Country
Poland
Facility Name
Tp0004 40223
City
Warszawa
Country
Poland
Facility Name
Tp0004 20052
City
Moscow
Country
Russian Federation
Facility Name
Tp0004 20053
City
Saint Petersburg
Country
Russian Federation
Facility Name
Tp0004 40268
City
Madrid
Country
Spain
Facility Name
Tp0004 20095
City
Taipei City
Country
Taiwan
Facility Name
Tp0004 20099
City
Taipei
Country
Taiwan
Facility Name
Tp0004 20061
City
Cherkasy
Country
Ukraine
Facility Name
Tp0004 20060
City
Dnipropetrovsk
Country
Ukraine
Facility Name
Tp0004 20062
City
Ivano-frankivsk
Country
Ukraine
Facility Name
Tp0004 20063
City
Kyiv
Country
Ukraine
Facility Name
Tp0004 20064
City
Kyiv
Country
Ukraine
Facility Name
Tp0004 20100
City
Zaporizhzhia
Country
Ukraine
Facility Name
Tp0004 40234
City
Plymouth
Country
United Kingdom
12. IPD Sharing Statement
Plan to Share IPD
Yes
IPD Sharing Plan Description
Data from this trial may be requested by qualified researchers six months after product approval in the US and/or Europe, or global development is discontinued, and 18 months after trial completion. Investigators may request access to anonymized individual patient-level data and redacted trial documents which may include: analysis-ready datasets, study protocol, annotated case report form, statistical analysis plan, dataset specifications, and clinical study report. Prior to use of the data, proposals need to be approved by an independent review panel at www.Vivli.org and a signed data sharing agreement will need to be executed. All documents are available in English only, for a prespecified time, typically 12 months, on a password protected portal. This plan may change if the risk of re-identifying trial participants is determined to be too high after the trial is completed; in this case and to protect participants, individual patient-level data would not be made available.
IPD Sharing Time Frame
Data from this trial may be requested by qualified researchers six months after product approval in the US and/or Europe or global development is discontinued, and 18 months after trial completion.
IPD Sharing Access Criteria
Qualified researchers may request access to anonymized IPD and redacted study documents which may include: raw datasets, analysis-ready datasets, study protocol, blank case report form, annotated case report form, statistical analysis plan, dataset specifications, and clinical study report. Prior to use of the data, proposals need to be approved by an independent review panel at www.Vivli.org and a signed data sharing agreement will need to be executed. All documents are available in English only, for a pre-specified time, typically 12 months, on a password protected portal.
IPD Sharing URL
https://www.vivli.org
Learn more about this trial
A Study to Investigate the Long-term Safety, Tolerability, and Efficacy of Rozanolixizumab in Study Participants With Persistent or Chronic Primary Immune Thrombocytopenia (ITP)
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