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| ID | Type | Description | Link |
|---|---|---|---|
| 2019-000883-40 | EudraCT Number |
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Strategic Business Decision; Not a safety decision
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The purpose of this study is to assess the long-term safety, tolerability and clinical efficacy of treatment with rozanolixizumab.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| 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. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Rozanolixizumab | Drug | Study participants receive rozanolixizumab by subcutaneous infusion during the Treatment Period. |
|
| Measure | Description | Time Frame |
|---|---|---|
| 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) |
| 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) |
| Measure | Description | Time Frame |
|---|---|---|
| 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. |
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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:
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:
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| Name | Affiliation | Role |
|---|---|---|
| UCB Cares | 001 844 599 2273 | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Tp0004 50243 | Boston | Massachusetts | 02114 | United States | ||
| Tp0004 20179 |
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.
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.
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.
Participant Flow refers to the Enrolled Set.
The study started to enroll study participants in January 2021 and completed prematurely in December 2022. Study participants from TP0003 (NCT04200456) or TP0006 (NCT04224688) who had completed the 24-week Treatment Period (irrespective of rescue therapy) and met eligibility criteria for TP0004 were enrolled in this study.
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| ID | Title | Description |
|---|---|---|
| FG000 | Rozanolixizumab | In TP0004, participants received a fixed unit dose of rozanolixizumab subcutaneous (sc) infusion at the assigned dose level in the parent studies (TP0003 and TP0006), for one year, starting from Day 1 (which corresponds to Week 25 of the parent studies). The dose of rozanolixizumab could be increased or decreased based upon the platelet count and across body weight tiers. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Apr 4, 2022 | Dec 20, 2023 |
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| 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. | Week 53 or 55, compared to Baseline |
| Fuzhou |
| China |
| Tp0004 20185 | Jinan | China |
| Tp0004 20194 | Wuxi | China |
| Tp0004 20050 | Tbilisi | Georgia |
| Tp0004 40369 | Berlin | Germany |
| Tp0004 40202 | Győr | Hungary |
| Tp0004 40178 | Nyíregyháza | Hungary |
| Tp0004 40208 | Florence | Italy |
| Tp0004 20039 | Iruma-gun | Japan |
| Tp0004 20051 | Chisinau | Moldova |
| Tp0004 40218 | Gdansk | Poland |
| Tp0004 40222 | Skorzewo | Poland |
| Tp0004 40219 | Słupsk | Poland |
| Tp0004 40223 | Warsaw | Poland |
| Tp0004 20052 | Moscow | Russia |
| Tp0004 20053 | Saint Petersburg | Russia |
| Tp0004 40268 | Madrid | Spain |
| Tp0004 20095 | Taipei | Taiwan |
| Tp0004 20099 | Taipei | Taiwan |
| Tp0004 20061 | Cherkasy | Ukraine |
| Tp0004 20060 | Dnipropetrovsk | Ukraine |
| Tp0004 20062 | Ivano-Frankivsk | Ukraine |
| Tp0004 20063 | Kyiv | Ukraine |
| Tp0004 20064 | Kyiv | Ukraine |
| Tp0004 20100 | Zaporizhzhia | Ukraine |
| Tp0004 40234 | Plymouth | United Kingdom |
| COMPLETED |
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| NOT COMPLETED |
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Baseline Characteristics refer to Enrolled Set which consisted of all study participants who signed the informed consent form (ICF).
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| ID | Title | Description |
|---|---|---|
| BG000 | Rozanolixizumab | In TP0004, participants received a fixed unit dose of rozanolixizumab sc infusion at the assigned dose level in the parent studies (TP0003 and TP0006), for one year, starting from Day 1 (which corresponds to Week 25 of the parent studies). The dose of rozanolixizumab could be increased or decreased based upon the platelet count and across body weight tiers. |
| Units | Counts |
|---|---|
| Participants |
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| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | years |
| |||||||||||||||||
| Age, Customized | Count of Participants | Participants |
| ||||||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| ||||||||||||||||||
| Race/Ethnicity, Customized | Count of Participants | Participants |
| ||||||||||||||||||
| Race/Ethnicity, Customized | Count of Participants | Participants |
|
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 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. | Safety Set included all study participants who received at least 1 dose of IMP (partial or full). | Posted | Number | percentage of Participants | 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. | Safety Set included all study participants who received at least 1 dose of IMP (partial or full). | Posted | Number | percentage of Participants | 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. | Safety Set included all study participants who received at least 1 dose of IMP (partial or full). | Posted | Number | percentage of participants | 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. | Safety Set included all study participants who received at least 1 dose of IMP (partial or full). Here, number of participants analyzed signifies those who were evaluable for this outcome measure and number analyzed signifies participants who were evaluable at specified time points. Week 53 was used for study participants who finished the study on weekly dosing, and Week 55 was used for study participants who finished the study on biweekly dosing. | Posted | Mean | Standard Deviation | score on a scale | Week 53 or 55, compared to Baseline |
|
From Baseline to end of Safety Follow-Up Period (up to Week 60)
Treatment-emergent AEs are defined as AEs starting after the time of first investigational medicinal product (IMP) administration up to and including 8 weeks (56 days) after the final dose. TEAEs were analyzed for Safety Set.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Rozanolixizumab | In TP0004, participants received a fixed unit dose of rozanolixizumab sc infusion at the assigned dose level in the parent studies (TP0003 and TP0006), for one year, starting from Day 1 (which corresponds to Week 25 of the parent studies). The dose of rozanolixizumab could be increased or decreased based upon the platelet count and across body weight tiers. | 0 | 43 | 9 | 43 | 31 | 43 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Immune thrombocytopenia | Blood and lymphatic system disorders | MedDRA v24.0 | Non-systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA v24.0 | Non-systematic Assessment |
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| COVID-19 | Infections and infestations | MedDRA v24.0 | Non-systematic Assessment |
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| Joint dislocation | Injury, poisoning and procedural complications | MedDRA v24.0 | Non-systematic Assessment |
| |
| Radius fracture | Injury, poisoning and procedural complications | MedDRA v24.0 | Non-systematic Assessment |
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| Platelet count decreased | Investigations | MedDRA v24.0 | Non-systematic Assessment |
| |
| Leiomyoma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA v24.0 | Non-systematic Assessment |
| |
| Uterine haemorrhage | Reproductive system and breast disorders | MedDRA v24.0 | Non-systematic Assessment |
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| Purpura | Skin and subcutaneous tissue disorders | MedDRA v24.0 | Non-systematic Assessment |
| |
| Skin haemorrhage | Skin and subcutaneous tissue disorders | MedDRA v24.0 | Non-systematic Assessment |
| |
| Haemorrhage | Vascular disorders | MedDRA v24.0 | Non-systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Anaemia | Blood and lymphatic system disorders | MedDRA v24.0 | Non-systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA v24.0 | Non-systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA v24.0 | Non-systematic Assessment |
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| Vomiting | Gastrointestinal disorders | MedDRA v24.0 | Non-systematic Assessment |
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| Fatigue | General disorders | MedDRA v24.0 | Non-systematic Assessment |
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| Pyrexia | General disorders | MedDRA v24.0 | Non-systematic Assessment |
| |
| COVID-19 | Infections and infestations | MedDRA v24.0 | Non-systematic Assessment |
| |
| Pharyngitis | Infections and infestations | MedDRA v24.0 | Non-systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA v24.0 | Non-systematic Assessment |
| |
| Contusion | Injury, poisoning and procedural complications | MedDRA v24.0 | Non-systematic Assessment |
| |
| Body temperature increased | Investigations | MedDRA v24.0 | Non-systematic Assessment |
| |
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA v24.0 | Non-systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA v24.0 | Non-systematic Assessment |
| |
| Petechiae | Skin and subcutaneous tissue disorders | MedDRA v24.0 | Non-systematic Assessment |
| |
| Rash | Skin and subcutaneous tissue disorders | MedDRA v24.0 | Non-systematic Assessment |
|
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| UCB | Cares | 001 844 599 2273 | UCBCares@ucb.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Oct 21, 2022 | Dec 20, 2023 | SAP_001.pdf |
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| ID | Term |
|---|---|
| D016553 | Purpura, Thrombocytopenic, Idiopathic |
| ID | Term |
|---|---|
| D011696 | Purpura, Thrombocytopenic |
| D011693 | Purpura |
| D001778 | Blood Coagulation Disorders |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D057049 | Thrombotic Microangiopathies |
| D013921 | Thrombocytopenia |
| D001791 | Blood Platelet Disorders |
| D000095542 | Cytopenia |
| D006474 | Hemorrhagic Disorders |
| D001327 | Autoimmune Diseases |
| D007154 | Immune System Diseases |
| D006470 | Hemorrhage |
| D010335 | Pathologic Processes |
| D013568 | Pathological Conditions, Signs and Symptoms |
| D012877 | Skin Manifestations |
| D012816 | Signs and Symptoms |
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| ID | Term |
|---|---|
| C000627812 | rozanolixizumab |
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| >=85 years |
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| Other or Mixed |
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| Missing |
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