Primary Immune ThrombocytopeniaPhase 3myOpportunITy3NCT04596995
A Study to Investigate the Long-term Safety, Tolerability, and Efficacy of Rozanolixizumab in Study Participants With Persistent or Chronic Primary Immune Thrombocytopenia (ITP)
A Phase 3 study testing Rozanolixizumab in people with Primary Immune Thrombocytopenia, run by UCB Biopharma SRL at 27 sites in 14 countries.
Trial summary
The purpose of this study is to assess the long-term safety, tolerability and clinical efficacy of treatment with rozanolixizumab. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Primary Immune Thrombocytopenia
- What is being tested?
- Rozanolixizumab
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 43 participants
- Where
- 27 sitesin 14 countries
- Study dates
- 6 Jan 2021to 21 Dec 2022
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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)
Trial locations and contacts
27 sites in 14 countries.
27 sites in 14 countries
Ukraine6 sites
Tp0004 20061
Cherkasy
Tp0004 20060
Dnipropetrovsk
Tp0004 20062
Ivano-Frankivsk
Tp0004 20063
Kyiv
Tp0004 20064
Kyiv
Tp0004 20100
Zaporizhzhya
Poland4 sites
Tp0004 40218
Gdansk
Tp0004 40222
Skorzewo
Tp0004 40219
Słupsk
Tp0004 40223
Warsaw
About this study
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)
The purpose of this study is to assess the long-term safety, tolerability and clinical efficacy of treatment with rozanolixizumab.
- Conditions
- Primary Immune Thrombocytopenia
- Being tested
- Rozanolixizumab
- Keywords
- ITP, UCB7665, Rozanolixizumab, Primary immune thrombocytopenia
- Medical subject headings
- Purpura, Thrombocytopenic, Idiopathic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RozanolixizumabDrug
Also called UCB7665
Study participants receive rozanolixizumab by subcutaneous infusion during the Treatment Period.
| Study group | Role | What they receive |
|---|---|---|
| Rozanolixizumab Treatment Arm | Experimental | 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. Receives: Drug: Rozanolixizumab |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: From Baseline to end of Safety Follow-Up Period (up to Week 60)
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.
Measured: From Baseline to end of Safety Follow-Up Period (up to Week 60)
Secondary outcomes (2)
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.
Measured: Over the 52-week Treatment Period (starting at Week 4)
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.
Measured: Week 53 or 55, compared to Baseline
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Oct 2020
- Study start6 Jan 2021
- Primary completion21 Dec 2022
- Study completion21 Dec 2022
Why it stopped: Strategic Business Decision; Not a safety decision
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Primary Immune ThrombocytopeniaStudies of the same condition, open and under way.
- More trials run by UCB Biopharma SRLThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 43 participants (actual)
Sponsor and investigators
- Lead sponsor
- UCB Biopharma SRL
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- UCB Cares, Study director, 001 844 599 2273
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04596995
- Sponsor's protocol ID
- TP0004
- Other IDs
- 2019-000883-40, 2019
- Acronym
- myOpportunITy3
- Status last verified
- Jan 2024
- First submitted
- 15 Oct 2020
- Last updated
- 5 Feb 2024
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: 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.
Study at a glance
From the registry record, last updated by the sponsor on 5 Feb 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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)
- ClinicalTrials.gov ID
- NCT04596995
- Acronym / sponsor ID
- myOpportunITy3 / TP0004
- Conditions
- Primary Immune Thrombocytopenia
- Keywords
- ITP, UCB7665, Rozanolixizumab, Primary immune thrombocytopenia
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Strategic Business Decision; Not a safety decision
- Enrolment
- 43 participants (actual)
- Lead sponsor
- UCB Biopharma SRLIndustry (pharma, biotech, devices)
- Study start
- 6 Jan 2021 (actual)
- Primary completion
- 21 Dec 2022 (actual)
- Study completion
- 21 Dec 2022 (actual)
- First posted
- 22 Oct 2020 (actual)
- Results
- Posted 5 Feb 2024
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this study is to assess the long-term safety, tolerability and clinical efficacy of treatment with rozanolixizumab.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Rozanolixizumab Treatment Arm | Experimental | Drug: RozanolixizumabAll 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. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- 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:
- 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
Key exclusion criteria (2 listed)
- 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)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| RozanolixizumabAlso: UCB7665Study participants receive rozanolixizumab by subcutaneous infusion during the Treatment Period. | Drug | Rozanolixizumab Treatment Arm |
Primary outcome
Percentage of Participants With Treatment-emergent Adverse Events (TEAEs)
Time frame: From Baseline to end of Safety Follow-Up Period (up to Week 60)
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.
Percentage of Participants With TEAEs Leading to Permanent Withdrawal of Rozanolixizumab (ie, Study Discontinuation)
Time frame: From Baseline to end of Safety Follow-Up Period (up to Week 60)
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.
2 secondary outcomes
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
Time frame: Over the 52-week Treatment Period (starting at Week 4)
Change From Baseline in Immune Thrombocytopenia-Patient Assessment Questionnaire (ITP-PAQ) to Week 53 or 55 Symptoms Domain Score
Time frame: Week 53 or 55, compared to Baseline
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Feb 2024.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: Yes. 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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
UCB Cares study director
001 844 599 2273
27 sites in 14 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Rozanolixizumab in people with Primary Immune Thrombocytopenia, run by UCB Biopharma SRL at 27 sites in 14 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 5 Feb 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 27 sites in Ukraine, Poland, China, Hungary, Russia and 9 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 5 Feb 2024). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Percentage of Participants With Treatment-emergent Adverse Events (TEAEs) (time frame: From Baseline to end of Safety Follow-Up Period (up to Week 60)). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 5 Feb 2024.