Clinical Trial Results:
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)
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Summary
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EudraCT number |
2019-000883-40 |
Trial protocol |
HU CZ BG PL DE GB IT RO |
Global end of trial date |
21 Dec 2022
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Results information
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Results version number |
v2(current) |
This version publication date |
06 Mar 2024
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First version publication date |
22 Dec 2023
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Other versions |
v1 |
Version creation reason |
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Trial Information
Subject Disposition
Baseline Characteristics
End Points
Adverse Events
More Information
Subject Disposition
Baseline Characteristics
End Points
Adverse Events
More Information
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Trial identification
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Sponsor protocol code |
TP0004
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Additional study identifiers
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ISRCTN number |
- | ||
US NCT number |
NCT04596995 | ||
WHO universal trial number (UTN) |
- | ||
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Sponsors
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Sponsor organisation name |
UCB Biopharma SRL
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Sponsor organisation address |
Allée de la Recherche 60, Brussels, Belgium, 1070
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Public contact |
Clin Trial Reg & Results Disclosure, UCB BIOSCIENCES GmbH, clinicaltrials@ucb.com
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Scientific contact |
Clin Trial Reg & Results Disclosure, UCB BIOSCIENCES GmbH, clinicaltrials@ucb.com
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Paediatric regulatory details
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Is trial part of an agreed paediatric investigation plan (PIP) |
No
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Does article 45 of REGULATION (EC) No 1901/2006 apply to this trial? |
No
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Does article 46 of REGULATION (EC) No 1901/2006 apply to this trial? |
No
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Results analysis stage
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Analysis stage |
Final
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Date of interim/final analysis |
25 Apr 2023
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Is this the analysis of the primary completion data? |
Yes
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Primary completion date |
21 Dec 2022
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Global end of trial reached? |
Yes
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Global end of trial date |
21 Dec 2022
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Was the trial ended prematurely? |
Yes
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General information about the trial
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Main objective of the trial |
Assess the long-term safety and tolerability of repeated treatment with rozanolixizumab
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Protection of trial subjects |
During the conduct of the study all participants were closely monitored.
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Background therapy |
Not applicable | ||
Evidence for comparator |
Not applicable | ||
Actual start date of recruitment |
06 Jan 2021
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Long term follow-up planned |
No
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Independent data monitoring committee (IDMC) involvement? |
Yes
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Population of trial subjects
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Number of subjects enrolled per country |
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Country: Number of subjects enrolled |
China: 5
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Country: Number of subjects enrolled |
Georgia: 2
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Country: Number of subjects enrolled |
Germany: 1
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Country: Number of subjects enrolled |
Hungary: 2
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Country: Number of subjects enrolled |
Italy: 1
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Country: Number of subjects enrolled |
Japan: 1
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Country: Number of subjects enrolled |
Moldova, Republic of: 1
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Country: Number of subjects enrolled |
Poland: 11
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Country: Number of subjects enrolled |
Russian Federation: 2
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Country: Number of subjects enrolled |
Spain: 3
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Country: Number of subjects enrolled |
Taiwan: 2
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Country: Number of subjects enrolled |
Ukraine: 10
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Country: Number of subjects enrolled |
United Kingdom: 1
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Country: Number of subjects enrolled |
United States: 1
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Worldwide total number of subjects |
43
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EEA total number of subjects |
18
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Number of subjects enrolled per age group |
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In utero |
0
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Preterm newborn - gestational age < 37 wk |
0
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Newborns (0-27 days) |
0
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Infants and toddlers (28 days-23 months) |
0
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Children (2-11 years) |
0
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Adolescents (12-17 years) |
0
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Adults (18-64 years) |
41
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From 65 to 84 years |
2
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85 years and over |
0
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Recruitment
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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. | ||||||||||||||||||
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Pre-assignment
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Screening details |
Participant Flow refers to the Enrolled Set. | ||||||||||||||||||
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Period 1
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Period 1 title |
Overall Study (overall period)
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Is this the baseline period? |
Yes | ||||||||||||||||||
Allocation method |
Not applicable
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Blinding used |
Not blinded | ||||||||||||||||||
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Arms
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Arm title
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Rozanolixizumab | ||||||||||||||||||
Arm description |
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. | ||||||||||||||||||
Arm type |
Experimental | ||||||||||||||||||
Investigational medicinal product name |
Rozanolixizumab
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Investigational medicinal product code |
UCB7665
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Other name |
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Pharmaceutical forms |
Solution for infusion
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Routes of administration |
Subcutaneous use
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Dosage and administration details |
Participants received rozanolixizumab as a weight-tiered fix dose by subcutaneous infusion during the Treatment Period.
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Baseline characteristics reporting groups
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Reporting group title |
Rozanolixizumab
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Reporting group description |
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. | ||||||||||||||||||||||||||||||||||||||||||
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End points reporting groups
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Reporting group title |
Rozanolixizumab
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Reporting group description |
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. | ||
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End point title |
Percentage of Participants With treatment-emergent adverse events (TEAEs) [1] | ||||||||
End point 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. 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).
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End point type |
Primary
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End point timeframe |
From Baseline to end of Safety Follow-Up Period (up to Week 60)
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| Notes [1] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point. Justification: No formal statistical hypothesis testing was planned for this study. Results were summarized as descriptive statistics only. |
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| No statistical analyses for this end point | |||||||||
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End point title |
Percentage of Participants With TEAEs leading to permanent withdrawal of rozanolixizumab (ie, study discontinuation) [2] | ||||||||
End point 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. 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).
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End point type |
Primary
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End point timeframe |
From Baseline to end of Safety Follow-Up Period (up to Week 60)
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| Notes [2] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point. Justification: No formal statistical hypothesis testing was planned for this study. Results were summarized as descriptive statistics only. |
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| No statistical analyses for this end point | |||||||||
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End point title |
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 | ||||||||
End point description |
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).
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End point type |
Secondary
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End point timeframe |
Over the 52-week Treatment Period (starting at Week 4)
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| No statistical analyses for this end point | |||||||||
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End point title |
Change from Baseline in Immune Thrombocytopenia-Patient Assessment Questionnaire (ITP-PAQ) to Week 53 or 55 Symptoms domain score | ||||||||||||
End point description |
ITP-PAQ version 1 is a 44 item disease-specific Health-Related QOL questionnaire developed for use in adults with chronic ITP.It includes 10 scales as physical health:Symptoms (6 items), Bother (3 items), Fatigue (4 items), Activity (2 items);emotional health: Fear (5 items) & Psychological (5 items); quality of life (QOL): Work QOL (4 items), Social QOL (4 items), Women’s Reproductive QOL (6 items) & Overall QOL (5 items).Each item is rated on a Likert-type scale having 4 to 7 responses.All item scores are transformed to a 0 to 100 continuum & are weighted equally to derive individual scale scores & total score(0-100) is calculated as:Sum of item scores within scale/raw sum range*100.Higher scores=better health status.Analysis set was Safety Set.Number of participants analyzed=participants evaluable for this endpoint & Number analyzed=participants evaluable at specified time points.Weeks 53 and 55 was used for participants who finished study on weekly & biweekly dosing respectively.
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End point type |
Secondary
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End point timeframe |
Weeks 53 or 55, compared to Baseline
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| No statistical analyses for this end point | |||||||||||||
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Adverse events information
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Timeframe for reporting adverse events |
From Baseline to end of Safety Follow-Up Period (up to Week 60)
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Adverse event reporting additional description |
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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Assessment type |
Non-systematic | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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Dictionary used for adverse event reporting
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Dictionary name |
MedDRA | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Dictionary version |
24.0
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Reporting groups
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Reporting group title |
Rozanolixizumab
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Reporting group description |
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. | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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| Frequency threshold for reporting non-serious adverse events: 5% | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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Substantial protocol amendments (globally) |
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| Were there any global substantial amendments to the protocol? Yes | |||
Date |
Amendment |
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11 May 2021 |
The primary reason for this substantial protocol amendment (11 May 2021) was to incorporate changes in the objectives, endpoints, the statistical analysis section, as well as a specific study objective and endpoint on post-vaccination biomarkers in study participants who had received a coronavirus disease 2019 (COVID-19) vaccine. Other changes included incorporating local
protocol amendments (0.1-Japan and 0.2-France only) into 1 global protocol. Additional updates were incorporated to provide further clarity on the protocol or to correct errors. |
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03 Dec 2021 |
The primary reason for this substantial protocol amendment (03 Dec 2021) was the recommendation of the external IDMC to modify the dosing regimen of the study. Ongoing study participants receiving the biweekly dosing regimen were required to be switched to the weekly dosing regimen according to Protocol Amendment 2. Note that Protocol Amendment 2 was not submitted to any regulatory authorities prior to issuance of Protocol Amendment 3. Therefore, the implementation
of switching all ongoing study participants from biweekly to weekly dosing actually occurred following the approval of Protocol Amendment 3 at the respective study site. With Protocol Amendment 2, the exploratory arm intended to evaluate wider dosing intervals and reference to maintenance dosing was removed. Additional updates were incorporated to provide further clarity on the protocol or to correct errors. |
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Interruptions (globally) |
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| Were there any global interruptions to the trial? No | |||
Limitations and caveats |
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| Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data. | |||
| None reported | |||
