Trial Outcomes & Findings for A Study to Assess the Efficacy, Safety, and Tolerability of Rozanolixizumab in Adult Study Participants With Persistent or Chronic Primary Immune Thrombocytopenia (ITP) (NCT NCT04224688)

NCT ID: NCT04224688

Last Updated: 2023-08-08

Results Overview

Percentage of Participants With Durable Clinically Meaningful Platelet Response of ≥50×10\^9/L, for at least 8 out of 12 weeks during the last 12 weeks were reported.

• Recruitment status: TERMINATED
• Study phase: PHASE3
• Target enrollment: 30 participants
• Primary outcome timeframe: During the last 12 weeks (Week 13 to Week 25)
• Results posted on: 2023-08-08

Participant Flow

The study started to enroll study participants in June 2020 and terminated in May 2022.

Participant Flow refers to the Randomized Set.

Participant milestones

Measure	Placebo Participants received a fixed-unit starting dose of placebo subcutaneous (sc) infusion matched to rozanolixizumab Dose A on Day 1. Following the initial dose, participants received a fixed-unit dose of placebo sc infusion matched to rozanolixizumab Dose B every 2 weeks until Week 23. Participants were followed up to a maximum of Week 31.	Rozanolixizumab Participants received a fixed-unit starting dose of rozanolixizumab sc infusion equivalent to Dose A on Day 1. Following the initial dose, participants received a fixed-unit dose of rozanolixizumab sc infusion equivalent to Dose B every 2 weeks until Week 23. After protocol amendment 3, the starting dose was removed and the frequency of administration of the Dose B was changed to weekly. Participants were followed up to a maximum of Week 31.
Overall Study STARTED	10	20
Overall Study COMPLETED	8	13
Overall Study NOT COMPLETED	2	7

Reasons for withdrawal

Measure	Placebo Participants received a fixed-unit starting dose of placebo subcutaneous (sc) infusion matched to rozanolixizumab Dose A on Day 1. Following the initial dose, participants received a fixed-unit dose of placebo sc infusion matched to rozanolixizumab Dose B every 2 weeks until Week 23. Participants were followed up to a maximum of Week 31.	Rozanolixizumab Participants received a fixed-unit starting dose of rozanolixizumab sc infusion equivalent to Dose A on Day 1. Following the initial dose, participants received a fixed-unit dose of rozanolixizumab sc infusion equivalent to Dose B every 2 weeks until Week 23. After protocol amendment 3, the starting dose was removed and the frequency of administration of the Dose B was changed to weekly. Participants were followed up to a maximum of Week 31.
Overall Study Withdrawal by Subject	1	3
Overall Study Lack of Efficacy	1	1
Overall Study Adverse event, not fatal	0	2
Overall Study COVID-19	0	1

Baseline Characteristics

A Study to Assess the Efficacy, Safety, and Tolerability of Rozanolixizumab in Adult Study Participants With Persistent or Chronic Primary Immune Thrombocytopenia (ITP)

Baseline characteristics by cohort

Measure	Placebo n=10 Participants Participants received a fixed-unit starting dose of placebo sc infusion matched to rozanolixizumab Dose A on Day 1. Following the initial dose, participants received a fixed-unit dose of placebo sc infusion matched to rozanolixizumab Dose B every 2 weeks until Week 23. Participants were followed up to a maximum of Week 31.	Rozanolixizumab n=20 Participants Participants received a fixed-unit starting dose of rozanolixizumab sc infusion equivalent to Dose A on Day 1. Following the initial dose, participants received a fixed-unit dose of rozanolixizumab sc infusion equivalent to Dose B every 2 weeks until Week 23. After protocol amendment 3, the starting dose was removed and the frequency of administration of the Dose B was changed to weekly. Participants were followed up to a maximum of Week 31.	Total n=30 Participants Total of all reporting groups
Age, Categorical <=18 years	0 Participants n=5 Participants	1 Participants n=7 Participants	1 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	10 Participants n=5 Participants	17 Participants n=7 Participants	27 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants	2 Participants n=7 Participants	2 Participants n=5 Participants
Age, Continuous	41.9 years STANDARD_DEVIATION 14.5 • n=5 Participants	42.3 years STANDARD_DEVIATION 15.7 • n=7 Participants	42.2 years STANDARD_DEVIATION 15.0 • n=5 Participants
Sex: Female, Male Female	6 Participants n=5 Participants	13 Participants n=7 Participants	19 Participants n=5 Participants
Sex: Female, Male Male	4 Participants n=5 Participants	7 Participants n=7 Participants	11 Participants n=5 Participants
Race/Ethnicity, Customized Asian	3 Participants n=5 Participants	6 Participants n=7 Participants	9 Participants n=5 Participants
Race/Ethnicity, Customized White	7 Participants n=5 Participants	13 Participants n=7 Participants	20 Participants n=5 Participants
Race/Ethnicity, Customized Missing	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race/Ethnicity, Customized Hispanic or Latino	0 Participants n=5 Participants	2 Participants n=7 Participants	2 Participants n=5 Participants
Race/Ethnicity, Customized Not Hispanic or Latino	10 Participants n=5 Participants	18 Participants n=7 Participants	28 Participants n=5 Participants
Platelet count	14.10 cells*10^9/L STANDARD_DEVIATION 7.77 • n=5 Participants	14.65 cells*10^9/L STANDARD_DEVIATION 8.18 • n=7 Participants	14.46 cells*10^9/L STANDARD_DEVIATION 7.91 • n=5 Participants

PRIMARY outcome

Timeframe: During the last 12 weeks (Week 13 to Week 25)

Population: Randomized Set consisted of all enrolled study participants who were randomized. No formal analysis was carried out as the program was terminated.

Percentage of Participants With Durable Clinically Meaningful Platelet Response of ≥50×10^9/L, for at least 8 out of 12 weeks during the last 12 weeks were reported.

Outcome measures

Measure	Placebo n=10 Participants Participants received a fixed-unit starting dose of placebo sc infusion matched to rozanolixizumab Dose A on Day 1. Following the initial dose, participants received a fixed-unit dose of placebo sc infusion matched to rozanolixizumab Dose B every 2 weeks until Week 23. Participants were followed up to a maximum of Week 31.	Rozanolixizumab n=20 Participants Participants received a fixed-unit starting dose of rozanolixizumab sc infusion equivalent to Dose A on Day 1. Following the initial dose, participants received a fixed-unit dose of rozanolixizumab sc infusion equivalent to Dose B every 2 weeks until Week 23. After protocol amendment 3, the starting dose was removed and the frequency of administration of the Dose B was changed to weekly. Participants were followed up to a maximum of Week 31.
Percentage of Participants With Durable Clinically Meaningful Platelet Response of ≥50×10^9/L, for at Least 8 Out of 12 Weeks During the Last 12 Weeks	0 Percentage of participants	5.0 Percentage of participants

SECONDARY outcome

Timeframe: Week 1 up to Week 25

Population: Randomized Set consisted of all enrolled study participants who were randomized.

Total number of weeks with platelet counts ≥50×10^9/L over the 24-week Treatment Period of the study (Week 1 to Week 25) were reported.

Outcome measures

Measure	Placebo n=10 Participants Participants received a fixed-unit starting dose of placebo sc infusion matched to rozanolixizumab Dose A on Day 1. Following the initial dose, participants received a fixed-unit dose of placebo sc infusion matched to rozanolixizumab Dose B every 2 weeks until Week 23. Participants were followed up to a maximum of Week 31.	Rozanolixizumab n=20 Participants Participants received a fixed-unit starting dose of rozanolixizumab sc infusion equivalent to Dose A on Day 1. Following the initial dose, participants received a fixed-unit dose of rozanolixizumab sc infusion equivalent to Dose B every 2 weeks until Week 23. After protocol amendment 3, the starting dose was removed and the frequency of administration of the Dose B was changed to weekly. Participants were followed up to a maximum of Week 31.
Cumulative Number of Weeks With Clinically Meaningful Platelet Response of ≥50×10^9/L Over the 24-week Treatment Period	0.0 Weeks Interval 0.0 to 10.0	1.0 Weeks Interval 0.0 to 18.0

SECONDARY outcome

Timeframe: Time from starting treatment to achievement of first response of ≥50×10^9/L (up to Week 25)

Population: Randomized Set consisted of all enrolled study participants who were randomized.

Time from starting treatment to achievement of first Clinically Meaningful Platelet Response of ≥50×10^9/L was defined as date of first clinically meaningful response - date of first treatment + 1. Median was calculated based upon the Kaplan-Meier estimate.

Outcome measures

Measure	Placebo n=10 Participants Participants received a fixed-unit starting dose of placebo sc infusion matched to rozanolixizumab Dose A on Day 1. Following the initial dose, participants received a fixed-unit dose of placebo sc infusion matched to rozanolixizumab Dose B every 2 weeks until Week 23. Participants were followed up to a maximum of Week 31.	Rozanolixizumab n=20 Participants Participants received a fixed-unit starting dose of rozanolixizumab sc infusion equivalent to Dose A on Day 1. Following the initial dose, participants received a fixed-unit dose of rozanolixizumab sc infusion equivalent to Dose B every 2 weeks until Week 23. After protocol amendment 3, the starting dose was removed and the frequency of administration of the Dose B was changed to weekly. Participants were followed up to a maximum of Week 31.
Time to First Clinically Meaningful Platelet Response (CMPR) of ≥50×10^9/L: Time From Starting Treatment to Achievement of First Response of ≥50×10^9/L	NA Days Interval 4.0 to NA for median signifies that the probability of participants achieving a CMPR did not reach 0.5 so the Kaplan-Meier median could not be estimated. NA signifies that upper confidence limit for placebo is not provided for the 95% CI of the median time to first CMPR as there is no time at which the upper bound of the CI for the Kaplan-Meier estimator is less than or equal to 0.5.	8.0 Days Interval 5.0 to NA signifies that upper confidence limit for rozanolixizumab is not provided for the 95% CI of the median time to first CMPR as there is no time at which the upper bound of the CI for the Kaplan-Meier estimator is less than or equal to 0.5.

SECONDARY outcome

Timeframe: Baseline to Day 8

Population: Randomized Set consisted of all enrolled study participants who were randomized.

Clinically meaningful platelet response was defined as platelet count of ≥50×10^9/L.

Outcome measures

Measure	Placebo n=10 Participants Participants received a fixed-unit starting dose of placebo sc infusion matched to rozanolixizumab Dose A on Day 1. Following the initial dose, participants received a fixed-unit dose of placebo sc infusion matched to rozanolixizumab Dose B every 2 weeks until Week 23. Participants were followed up to a maximum of Week 31.	Rozanolixizumab n=20 Participants Participants received a fixed-unit starting dose of rozanolixizumab sc infusion equivalent to Dose A on Day 1. Following the initial dose, participants received a fixed-unit dose of rozanolixizumab sc infusion equivalent to Dose B every 2 weeks until Week 23. After protocol amendment 3, the starting dose was removed and the frequency of administration of the Dose B was changed to weekly. Participants were followed up to a maximum of Week 31.
Percentage of Participants With Clinically Meaningful Platelet Response of ≥50×10^9/L by Day 8	10.0 Percentage of participants	45.0 Percentage of participants

SECONDARY outcome

Timeframe: From Baseline during Treatment Period (up to Week 25)

Population: Randomized Set consisted of all enrolled study participants who were randomized.

Response was defined as platelet count ≥30*10^9/L and at least doubling of baseline, at least 2 separate occasions at two adjacent nominal visits at least 7 days apart, and absence of bleeding.

Outcome measures

Measure	Placebo n=10 Participants Participants received a fixed-unit starting dose of placebo sc infusion matched to rozanolixizumab Dose A on Day 1. Following the initial dose, participants received a fixed-unit dose of placebo sc infusion matched to rozanolixizumab Dose B every 2 weeks until Week 23. Participants were followed up to a maximum of Week 31.	Rozanolixizumab n=20 Participants Participants received a fixed-unit starting dose of rozanolixizumab sc infusion equivalent to Dose A on Day 1. Following the initial dose, participants received a fixed-unit dose of rozanolixizumab sc infusion equivalent to Dose B every 2 weeks until Week 23. After protocol amendment 3, the starting dose was removed and the frequency of administration of the Dose B was changed to weekly. Participants were followed up to a maximum of Week 31.
Percentage of Participants With Response Defined as Platelet Count ≥30*10^9/L and at Least Doubling of Baseline, at Least 2 Separate Occasions at Two Adjacent Nominal Visits at Least 7 Days Apart, and Absence of Bleeding	10.0 Percentage of participants	20.0 Percentage of participants

SECONDARY outcome

Timeframe: From Baseline to first rescue therapy (up to Week 25)

Population: Randomized Set consisted of all enrolled study participants who were randomized.

Time to first rescue therapy was defined as date of first rescue therapy use - date of first treatment + 1. Median was calculated based upon the Kaplan-Meier estimate.

Outcome measures

Measure	Placebo n=10 Participants Participants received a fixed-unit starting dose of placebo sc infusion matched to rozanolixizumab Dose A on Day 1. Following the initial dose, participants received a fixed-unit dose of placebo sc infusion matched to rozanolixizumab Dose B every 2 weeks until Week 23. Participants were followed up to a maximum of Week 31.	Rozanolixizumab n=20 Participants Participants received a fixed-unit starting dose of rozanolixizumab sc infusion equivalent to Dose A on Day 1. Following the initial dose, participants received a fixed-unit dose of rozanolixizumab sc infusion equivalent to Dose B every 2 weeks until Week 23. After protocol amendment 3, the starting dose was removed and the frequency of administration of the Dose B was changed to weekly. Participants were followed up to a maximum of Week 31.
Time to First Rescue Therapy	NA Days Interval 4.0 to NA for median signifies that the probability of participants requiring rescue medication did not reach 0.5 so the Kaplan-Meier median could not be estimated. NA signifies that upper confidence limit for placebo is not provided for the 95% CI of the median time to rescue therapy as there is no time at which the upper bound of the CI for the Kaplan-Meier estimator is less than or equal to 0.5.	NA Days Interval 46.0 to NA for median signifies that the probability of participants requiring rescue medication did not reach 0.5 so the Kaplan-Meier median could not be estimated. NA signifies that upper confidence limit for rozanolixizumab is not provided for the 95% CI of the median time to rescue therapy as there is no time at which the upper bound of the CI for the Kaplan-Meier estimator is less than or equal to 0.5.

SECONDARY outcome

Timeframe: From Baseline during Treatment Period (up to Week 25)

Population: Randomized Set consisted of all enrolled study participants who were randomized. Here, Number of Participants analyzed signifies those participants who were evaluable for this outcome measure.

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.

Outcome measures

Measure	Placebo n=8 Participants Participants received a fixed-unit starting dose of placebo sc infusion matched to rozanolixizumab Dose A on Day 1. Following the initial dose, participants received a fixed-unit dose of placebo sc infusion matched to rozanolixizumab Dose B every 2 weeks until Week 23. Participants were followed up to a maximum of Week 31.	Rozanolixizumab n=12 Participants Participants received a fixed-unit starting dose of rozanolixizumab sc infusion equivalent to Dose A on Day 1. Following the initial dose, participants received a fixed-unit dose of rozanolixizumab sc infusion equivalent to Dose B every 2 weeks until Week 23. After protocol amendment 3, the starting dose was removed and the frequency of administration of the Dose B was changed to weekly. Participants were followed up to a maximum of Week 31.
Change From Baseline to Week 25 in Primary Immune Thrombocytopenia Patient Assessment Questionnaire (ITP-PAQ) Symptoms Score	-0.5 units on a scale Standard Deviation 17.0	4.2 units on a scale Standard Deviation 12.9

SECONDARY outcome

Timeframe: From Baseline to end of Safety Follow-Up Period (up to Week 31)

Population: Safety set included all randomized study participants who received at least one dose of IMP.

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.

Outcome measures

Measure	Placebo n=10 Participants Participants received a fixed-unit starting dose of placebo sc infusion matched to rozanolixizumab Dose A on Day 1. Following the initial dose, participants received a fixed-unit dose of placebo sc infusion matched to rozanolixizumab Dose B every 2 weeks until Week 23. Participants were followed up to a maximum of Week 31.	Rozanolixizumab n=20 Participants Participants received a fixed-unit starting dose of rozanolixizumab sc infusion equivalent to Dose A on Day 1. Following the initial dose, participants received a fixed-unit dose of rozanolixizumab sc infusion equivalent to Dose B every 2 weeks until Week 23. After protocol amendment 3, the starting dose was removed and the frequency of administration of the Dose B was changed to weekly. Participants were followed up to a maximum of Week 31.
Percentage of Participants With Treatment-emergent Adverse Events (TEAEs)	60.0 Percentage of participants	95.0 Percentage of participants

SECONDARY outcome

Timeframe: From Baseline to end of Safety Follow-Up Period (up to Week 31)

Population: Safety set included all randomized study participants who received at least one dose of IMP.

An AE is any untoward medical occurrence in a patient or clinical study participant, temporally associated with the use of 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.

Outcome measures

Measure	Placebo n=10 Participants Participants received a fixed-unit starting dose of placebo sc infusion matched to rozanolixizumab Dose A on Day 1. Following the initial dose, participants received a fixed-unit dose of placebo sc infusion matched to rozanolixizumab Dose B every 2 weeks until Week 23. Participants were followed up to a maximum of Week 31.	Rozanolixizumab n=20 Participants Participants received a fixed-unit starting dose of rozanolixizumab sc infusion equivalent to Dose A on Day 1. Following the initial dose, participants received a fixed-unit dose of rozanolixizumab sc infusion equivalent to Dose B every 2 weeks until Week 23. After protocol amendment 3, the starting dose was removed and the frequency of administration of the Dose B was changed to weekly. Participants were followed up to a maximum of Week 31.
Percentage of Participants With TEAEs Leading to Withdrawal of Investigational Medicinal Product (ie, Study Discontinuation)	0 Percentage of participants	10.0 Percentage of participants

Adverse Events

Placebo

Serious events: 1 serious events

Other events: 6 other events

Deaths: 0 deaths

Rozanolixizumab

Serious events: 5 serious events

Other events: 18 other events

Deaths: 0 deaths

Serious adverse events

Measure	Placebo n=10 participants at risk Participants received a fixed-unit starting dose of placebo sc infusion matched to rozanolixizumab Dose A on Day 1. Following the initial dose, participants received a fixed-unit dose of placebo sc infusion matched to rozanolixizumab Dose B every 2 weeks until Week 23. Participants were followed up to a maximum of Week 31.	Rozanolixizumab n=20 participants at risk Participants received a fixed-unit starting dose of rozanolixizumab sc infusion equivalent to Dose A on Day 1. Following the initial dose, participants received a fixed-unit dose of rozanolixizumab sc infusion equivalent to Dose B every 2 weeks until Week 23. After protocol amendment 3, the starting dose was removed and the frequency of administration of the Dose B was changed to weekly. Participants were followed up to a maximum of Week 31.
Blood and lymphatic system disorders Thrombocytopenia	10.0% 1/10 • Number of events 1 • From Baseline to end of Safety Follow-Up Period (up to Week 31) 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. TEAEs were analyzed for Safety Set.	0.00% 0/20 • From Baseline to end of Safety Follow-Up Period (up to Week 31) 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. TEAEs were analyzed for Safety Set.
Gastrointestinal disorders Vomiting	0.00% 0/10 • From Baseline to end of Safety Follow-Up Period (up to Week 31) 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. TEAEs were analyzed for Safety Set.	5.0% 1/20 • Number of events 1 • From Baseline to end of Safety Follow-Up Period (up to Week 31) 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. TEAEs were analyzed for Safety Set.
Infections and infestations Pneumonia viral	0.00% 0/10 • From Baseline to end of Safety Follow-Up Period (up to Week 31) 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. TEAEs were analyzed for Safety Set.	5.0% 1/20 • Number of events 1 • From Baseline to end of Safety Follow-Up Period (up to Week 31) 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. TEAEs were analyzed for Safety Set.
Injury, poisoning and procedural complications Head injury	0.00% 0/10 • From Baseline to end of Safety Follow-Up Period (up to Week 31) 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. TEAEs were analyzed for Safety Set.	5.0% 1/20 • Number of events 1 • From Baseline to end of Safety Follow-Up Period (up to Week 31) 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. TEAEs were analyzed for Safety Set.
Injury, poisoning and procedural complications Limb injury	0.00% 0/10 • From Baseline to end of Safety Follow-Up Period (up to Week 31) 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. TEAEs were analyzed for Safety Set.	5.0% 1/20 • Number of events 1 • From Baseline to end of Safety Follow-Up Period (up to Week 31) 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. TEAEs were analyzed for Safety Set.
Injury, poisoning and procedural complications Skin injury	0.00% 0/10 • From Baseline to end of Safety Follow-Up Period (up to Week 31) 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. TEAEs were analyzed for Safety Set.	5.0% 1/20 • Number of events 1 • From Baseline to end of Safety Follow-Up Period (up to Week 31) 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. TEAEs were analyzed for Safety Set.
Investigations Platelet count decreased	10.0% 1/10 • Number of events 1 • From Baseline to end of Safety Follow-Up Period (up to Week 31) 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. TEAEs were analyzed for Safety Set.	5.0% 1/20 • Number of events 1 • From Baseline to end of Safety Follow-Up Period (up to Week 31) 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. TEAEs were analyzed for Safety Set.
Nervous system disorders Headache	0.00% 0/10 • From Baseline to end of Safety Follow-Up Period (up to Week 31) 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. TEAEs were analyzed for Safety Set.	5.0% 1/20 • Number of events 1 • From Baseline to end of Safety Follow-Up Period (up to Week 31) 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. TEAEs were analyzed for Safety Set.
Nervous system disorders Dizziness	0.00% 0/10 • From Baseline to end of Safety Follow-Up Period (up to Week 31) 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. TEAEs were analyzed for Safety Set.	5.0% 1/20 • Number of events 1 • From Baseline to end of Safety Follow-Up Period (up to Week 31) 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. TEAEs were analyzed for Safety Set.
Skin and subcutaneous tissue disorders Urticaria	0.00% 0/10 • From Baseline to end of Safety Follow-Up Period (up to Week 31) 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. TEAEs were analyzed for Safety Set.	5.0% 1/20 • Number of events 1 • From Baseline to end of Safety Follow-Up Period (up to Week 31) 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. TEAEs were analyzed for Safety Set.

Other adverse events

Measure	Placebo n=10 participants at risk Participants received a fixed-unit starting dose of placebo sc infusion matched to rozanolixizumab Dose A on Day 1. Following the initial dose, participants received a fixed-unit dose of placebo sc infusion matched to rozanolixizumab Dose B every 2 weeks until Week 23. Participants were followed up to a maximum of Week 31.	Rozanolixizumab n=20 participants at risk Participants received a fixed-unit starting dose of rozanolixizumab sc infusion equivalent to Dose A on Day 1. Following the initial dose, participants received a fixed-unit dose of rozanolixizumab sc infusion equivalent to Dose B every 2 weeks until Week 23. After protocol amendment 3, the starting dose was removed and the frequency of administration of the Dose B was changed to weekly. Participants were followed up to a maximum of Week 31.
Blood and lymphatic system disorders Anaemia	0.00% 0/10 • From Baseline to end of Safety Follow-Up Period (up to Week 31) 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. TEAEs were analyzed for Safety Set.	10.0% 2/20 • Number of events 2 • From Baseline to end of Safety Follow-Up Period (up to Week 31) 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. TEAEs were analyzed for Safety Set.
Blood and lymphatic system disorders Hypofibrinogenaemia	10.0% 1/10 • Number of events 1 • From Baseline to end of Safety Follow-Up Period (up to Week 31) 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. TEAEs were analyzed for Safety Set.	0.00% 0/20 • From Baseline to end of Safety Follow-Up Period (up to Week 31) 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. TEAEs were analyzed for Safety Set.
Eye disorders Eye pain	10.0% 1/10 • Number of events 1 • From Baseline to end of Safety Follow-Up Period (up to Week 31) 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. TEAEs were analyzed for Safety Set.	0.00% 0/20 • From Baseline to end of Safety Follow-Up Period (up to Week 31) 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. TEAEs were analyzed for Safety Set.
Gastrointestinal disorders Nausea	0.00% 0/10 • From Baseline to end of Safety Follow-Up Period (up to Week 31) 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. TEAEs were analyzed for Safety Set.	15.0% 3/20 • Number of events 3 • From Baseline to end of Safety Follow-Up Period (up to Week 31) 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. TEAEs were analyzed for Safety Set.
Gastrointestinal disorders Diarrhoea	0.00% 0/10 • From Baseline to end of Safety Follow-Up Period (up to Week 31) 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. TEAEs were analyzed for Safety Set.	10.0% 2/20 • Number of events 3 • From Baseline to end of Safety Follow-Up Period (up to Week 31) 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. TEAEs were analyzed for Safety Set.
Gastrointestinal disorders Gingival bleeding	0.00% 0/10 • From Baseline to end of Safety Follow-Up Period (up to Week 31) 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. TEAEs were analyzed for Safety Set.	10.0% 2/20 • Number of events 3 • From Baseline to end of Safety Follow-Up Period (up to Week 31) 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. TEAEs were analyzed for Safety Set.
Gastrointestinal disorders Abdominal pain	10.0% 1/10 • Number of events 1 • From Baseline to end of Safety Follow-Up Period (up to Week 31) 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. TEAEs were analyzed for Safety Set.	5.0% 1/20 • Number of events 1 • From Baseline to end of Safety Follow-Up Period (up to Week 31) 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. TEAEs were analyzed for Safety Set.
Gastrointestinal disorders Constipation	10.0% 1/10 • Number of events 1 • From Baseline to end of Safety Follow-Up Period (up to Week 31) 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. TEAEs were analyzed for Safety Set.	5.0% 1/20 • Number of events 1 • From Baseline to end of Safety Follow-Up Period (up to Week 31) 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. TEAEs were analyzed for Safety Set.
Gastrointestinal disorders Abdominal distension	10.0% 1/10 • Number of events 1 • From Baseline to end of Safety Follow-Up Period (up to Week 31) 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. TEAEs were analyzed for Safety Set.	0.00% 0/20 • From Baseline to end of Safety Follow-Up Period (up to Week 31) 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. TEAEs were analyzed for Safety Set.
General disorders Pyrexia	0.00% 0/10 • From Baseline to end of Safety Follow-Up Period (up to Week 31) 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. TEAEs were analyzed for Safety Set.	30.0% 6/20 • Number of events 22 • From Baseline to end of Safety Follow-Up Period (up to Week 31) 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. TEAEs were analyzed for Safety Set.
General disorders Fatigue	0.00% 0/10 • From Baseline to end of Safety Follow-Up Period (up to Week 31) 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. TEAEs were analyzed for Safety Set.	10.0% 2/20 • Number of events 4 • From Baseline to end of Safety Follow-Up Period (up to Week 31) 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. TEAEs were analyzed for Safety Set.
General disorders Asthenia	10.0% 1/10 • Number of events 1 • From Baseline to end of Safety Follow-Up Period (up to Week 31) 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. TEAEs were analyzed for Safety Set.	5.0% 1/20 • Number of events 4 • From Baseline to end of Safety Follow-Up Period (up to Week 31) 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. TEAEs were analyzed for Safety Set.
General disorders Infusion site pain	10.0% 1/10 • Number of events 1 • From Baseline to end of Safety Follow-Up Period (up to Week 31) 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. TEAEs were analyzed for Safety Set.	0.00% 0/20 • From Baseline to end of Safety Follow-Up Period (up to Week 31) 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. TEAEs were analyzed for Safety Set.
Infections and infestations Upper respiratory tract infection	10.0% 1/10 • Number of events 1 • From Baseline to end of Safety Follow-Up Period (up to Week 31) 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. TEAEs were analyzed for Safety Set.	10.0% 2/20 • Number of events 2 • From Baseline to end of Safety Follow-Up Period (up to Week 31) 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. TEAEs were analyzed for Safety Set.
Infections and infestations COVID-19	10.0% 1/10 • Number of events 1 • From Baseline to end of Safety Follow-Up Period (up to Week 31) 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. TEAEs were analyzed for Safety Set.	5.0% 1/20 • Number of events 1 • From Baseline to end of Safety Follow-Up Period (up to Week 31) 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. TEAEs were analyzed for Safety Set.
Infections and infestations Urinary tract infection	10.0% 1/10 • Number of events 1 • From Baseline to end of Safety Follow-Up Period (up to Week 31) 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. TEAEs were analyzed for Safety Set.	5.0% 1/20 • Number of events 1 • From Baseline to end of Safety Follow-Up Period (up to Week 31) 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. TEAEs were analyzed for Safety Set.
Infections and infestations Pharyngitis	10.0% 1/10 • Number of events 1 • From Baseline to end of Safety Follow-Up Period (up to Week 31) 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. TEAEs were analyzed for Safety Set.	0.00% 0/20 • From Baseline to end of Safety Follow-Up Period (up to Week 31) 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. TEAEs were analyzed for Safety Set.
Investigations Platelet count decreased	10.0% 1/10 • Number of events 1 • From Baseline to end of Safety Follow-Up Period (up to Week 31) 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. TEAEs were analyzed for Safety Set.	0.00% 0/20 • From Baseline to end of Safety Follow-Up Period (up to Week 31) 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. TEAEs were analyzed for Safety Set.
Investigations White blood cell count increased	10.0% 1/10 • Number of events 1 • From Baseline to end of Safety Follow-Up Period (up to Week 31) 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. TEAEs were analyzed for Safety Set.	0.00% 0/20 • From Baseline to end of Safety Follow-Up Period (up to Week 31) 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. TEAEs were analyzed for Safety Set.
Metabolism and nutrition disorders Decreased appetite	10.0% 1/10 • Number of events 1 • From Baseline to end of Safety Follow-Up Period (up to Week 31) 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. TEAEs were analyzed for Safety Set.	0.00% 0/20 • From Baseline to end of Safety Follow-Up Period (up to Week 31) 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. TEAEs were analyzed for Safety Set.
Metabolism and nutrition disorders Hyperkalaemia	10.0% 1/10 • Number of events 4 • From Baseline to end of Safety Follow-Up Period (up to Week 31) 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. TEAEs were analyzed for Safety Set.	0.00% 0/20 • From Baseline to end of Safety Follow-Up Period (up to Week 31) 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. TEAEs were analyzed for Safety Set.
Musculoskeletal and connective tissue disorders Myalgia	0.00% 0/10 • From Baseline to end of Safety Follow-Up Period (up to Week 31) 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. TEAEs were analyzed for Safety Set.	10.0% 2/20 • Number of events 3 • From Baseline to end of Safety Follow-Up Period (up to Week 31) 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. TEAEs were analyzed for Safety Set.
Musculoskeletal and connective tissue disorders Arthralgia	10.0% 1/10 • Number of events 1 • From Baseline to end of Safety Follow-Up Period (up to Week 31) 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. TEAEs were analyzed for Safety Set.	5.0% 1/20 • Number of events 3 • From Baseline to end of Safety Follow-Up Period (up to Week 31) 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. TEAEs were analyzed for Safety Set.
Nervous system disorders Headache	20.0% 2/10 • Number of events 2 • From Baseline to end of Safety Follow-Up Period (up to Week 31) 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. TEAEs were analyzed for Safety Set.	60.0% 12/20 • Number of events 25 • From Baseline to end of Safety Follow-Up Period (up to Week 31) 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. TEAEs were analyzed for Safety Set.
Nervous system disorders Somnolence	10.0% 1/10 • Number of events 1 • From Baseline to end of Safety Follow-Up Period (up to Week 31) 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. TEAEs were analyzed for Safety Set.	0.00% 0/20 • From Baseline to end of Safety Follow-Up Period (up to Week 31) 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. TEAEs were analyzed for Safety Set.
Psychiatric disorders Insomnia	10.0% 1/10 • Number of events 1 • From Baseline to end of Safety Follow-Up Period (up to Week 31) 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. TEAEs were analyzed for Safety Set.	5.0% 1/20 • Number of events 1 • From Baseline to end of Safety Follow-Up Period (up to Week 31) 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. TEAEs were analyzed for Safety Set.
Renal and urinary disorders Haematuria	0.00% 0/10 • From Baseline to end of Safety Follow-Up Period (up to Week 31) 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. TEAEs were analyzed for Safety Set.	10.0% 2/20 • Number of events 2 • From Baseline to end of Safety Follow-Up Period (up to Week 31) 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. TEAEs were analyzed for Safety Set.
Respiratory, thoracic and mediastinal disorders Epistaxis	0.00% 0/10 • From Baseline to end of Safety Follow-Up Period (up to Week 31) 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. TEAEs were analyzed for Safety Set.	10.0% 2/20 • Number of events 2 • From Baseline to end of Safety Follow-Up Period (up to Week 31) 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. TEAEs were analyzed for Safety Set.
Respiratory, thoracic and mediastinal disorders Oropharyngeal pain	0.00% 0/10 • From Baseline to end of Safety Follow-Up Period (up to Week 31) 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. TEAEs were analyzed for Safety Set.	10.0% 2/20 • Number of events 2 • From Baseline to end of Safety Follow-Up Period (up to Week 31) 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. TEAEs were analyzed for Safety Set.
Skin and subcutaneous tissue disorders Urticaria	0.00% 0/10 • From Baseline to end of Safety Follow-Up Period (up to Week 31) 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. TEAEs were analyzed for Safety Set.	10.0% 2/20 • Number of events 2 • From Baseline to end of Safety Follow-Up Period (up to Week 31) 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. TEAEs were analyzed for Safety Set.
Skin and subcutaneous tissue disorders Petechiae	10.0% 1/10 • Number of events 2 • From Baseline to end of Safety Follow-Up Period (up to Week 31) 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. TEAEs were analyzed for Safety Set.	5.0% 1/20 • Number of events 2 • From Baseline to end of Safety Follow-Up Period (up to Week 31) 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. TEAEs were analyzed for Safety Set.
Skin and subcutaneous tissue disorders Blister	10.0% 1/10 • Number of events 1 • From Baseline to end of Safety Follow-Up Period (up to Week 31) 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. TEAEs were analyzed for Safety Set.	0.00% 0/20 • From Baseline to end of Safety Follow-Up Period (up to Week 31) 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. TEAEs were analyzed for Safety Set.
Skin and subcutaneous tissue disorders Hirsutism	10.0% 1/10 • Number of events 1 • From Baseline to end of Safety Follow-Up Period (up to Week 31) 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. TEAEs were analyzed for Safety Set.	0.00% 0/20 • From Baseline to end of Safety Follow-Up Period (up to Week 31) 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. TEAEs were analyzed for Safety Set.
Vascular disorders Haemorrhage	10.0% 1/10 • Number of events 1 • From Baseline to end of Safety Follow-Up Period (up to Week 31) 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. TEAEs were analyzed for Safety Set.	0.00% 0/20 • From Baseline to end of Safety Follow-Up Period (up to Week 31) 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. TEAEs were analyzed for Safety Set.

Additional Information

UCB

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Email: UCBCares@ucb.com

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place

Restriction type: GT60