Primary Immune Thrombocytopenia
Conditions
Keywords
ITP, UCB7665, Rozanolixizumab, Primary immune thrombocytopenia
Brief summary
The purpose of this study is to assess the long-term safety, tolerability and clinical efficacy of treatment with rozanolixizumab.
Interventions
DRUGRozanolixizumab
Study participants receive rozanolixizumab by subcutaneous infusion during the Treatment Period.
Sponsors
UCB Biopharma SRL
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
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)
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Percentage of Participants With Treatment-emergent Adverse Events (TEAEs) | 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) | 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. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| 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 | Over the 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. |
| Change From Baseline in Immune Thrombocytopenia-Patient Assessment Questionnaire (ITP-PAQ) to Week 53 or 55 Symptoms Domain Score | Week 53 or 55, compared to Baseline | 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. |
Countries
China, Georgia, Germany, Hungary, Italy, Japan, Moldova, Poland, Russia, Spain, Taiwan, Ukraine, United Kingdom, United States
Participant flow
Recruitment details
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.
Pre-assignment details
Participant Flow refers to the Enrolled Set.
Participants by arm
| Arm | Count |
|---|---|
| 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. | 43 |
| Total | 43 |
Withdrawals & dropouts
| Period | Reason | FG000 |
|---|---|---|
| Overall Study | Enrolled in managed access program | 3 |
| Overall Study | Lack of Efficacy | 8 |
| Overall Study | Lost to Follow-up | 3 |
| Overall Study | Sponsor decision | 3 |
| Overall Study | Withdrawal by Subject | 12 |
Baseline characteristics
| Characteristic | Rozanolixizumab |
|---|---|
| Age, Continuous | 42.7 years STANDARD_DEVIATION 13.7 |
| Age, Customized 18 - <65 years | 41 Participants |
| Age, Customized >=65 - <85 years | 2 Participants |
| Age, Customized >=85 years | 0 Participants |
| Race/Ethnicity, Customized Asian | 8 Participants |
| Race/Ethnicity, Customized Hispanic or Latino | 2 Participants |
| Race/Ethnicity, Customized Missing | 1 Participants |
| Race/Ethnicity, Customized Not Hispanic or Latino | 41 Participants |
| Race/Ethnicity, Customized Other or Mixed | 1 Participants |
| Race/Ethnicity, Customized White | 33 Participants |
| Sex: Female, Male Female | 28 Participants |
| Sex: Female, Male Male | 15 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 0 / 43 |
| other Total, other adverse events | 31 / 43 |
| serious Total, serious adverse events | 9 / 43 |
Outcome results
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.
Time frame: From Baseline to end of Safety Follow-Up Period (up to Week 60)
Population: Safety Set included all study participants who received at least 1 dose of IMP (partial or full).
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Rozanolixizumab | Percentage of Participants With TEAEs Leading to Permanent Withdrawal of Rozanolixizumab (ie, Study Discontinuation) | 0 percentage of Participants |
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.
Time frame: From Baseline to end of Safety Follow-Up Period (up to Week 60)
Population: Safety Set included all study participants who received at least 1 dose of IMP (partial or full).
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Rozanolixizumab | Percentage of Participants With Treatment-emergent Adverse Events (TEAEs) | 90.7 percentage of Participants |
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.
Time frame: Week 53 or 55, compared to Baseline
Population: 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.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Rozanolixizumab | Change From Baseline in Immune Thrombocytopenia-Patient Assessment Questionnaire (ITP-PAQ) to Week 53 or 55 Symptoms Domain Score | Week 53 | 2.98 score on a scale | Standard Deviation 11.21 |
| Rozanolixizumab | Change From Baseline in Immune Thrombocytopenia-Patient Assessment Questionnaire (ITP-PAQ) to Week 53 or 55 Symptoms Domain Score | Week 55 | 4.17 score on a scale | Standard Deviation 12.5 |
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.
Time frame: Over the 52-week Treatment Period (starting at Week 4)
Population: Safety Set included all study participants who received at least 1 dose of IMP (partial or full).
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Rozanolixizumab | 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 | 16.3 percentage of participants |