A Study to Assess the Efficacy, Safety, and Tolerability of Rozanolixizumab in Adult Study Participants With Persistent or Chronic Primary Immune Thrombocytopenia (ITP) (myOpportunITy2)

• ClinicalTrials.gov Identifier: NCT04224688
• Recruitment Status: Terminated
• First Posted: January 13, 2020
• Results First Posted: August 8, 2023
• Last Update Posted: August 8, 2023
• Sponsor: UCB Biopharma SRL
• Information provided by (Responsible Party): UCB Pharma ( UCB Biopharma SRL )

• Study Type: Interventional
• Study Design: Allocation: Randomized; Intervention Model: Parallel Assignment; Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor); Primary Purpose: Treatment
• Condition: Primary Immune Thrombocytopenia
• Interventions: Drug: Rozanolixizumab; Other: Placebo
• Enrollment: 30

Participant Flow

Recruitment Details	The study started to enroll study participants in June 2020 and terminated in May 2022.
Pre-assignment Details	Participant Flow refers to the Randomized Set.

Arm/Group Title	Placebo	Rozanolixizumab
Arm/Group Description	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.	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.
Period Title: Overall Study
Started	10	20
Completed	8	13
Not Completed	2	7
Reason Not Completed
Withdrawal by Subject	1	3
Lack of Efficacy	1	1
Adverse event, not fatal	0	2
COVID-19	0	1

Baseline Characteristics

Arm/Group Title		Placebo	Rozanolixizumab	Total
Arm/Group Description		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.	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 of all reporting groups
Overall Number of Baseline Participants		10	20	30
Baseline Analysis Population Description		Baseline Characteristics refer to Randomized Set which consisted of all enrolled study participants who were randomized.
Age, Categorical; Measure Type: Count of Participants; Unit of measure: Participants
	Number Analyzed	10 participants	20 participants	30 participants
	<=18 years	0 0.0%	1 5.0%	1 3.3%
	Between 18 and 65 years	10 100.0%	17 85.0%	27 90.0%
	>=65 years	0 0.0%	2 10.0%	2 6.7%
Age, Continuous; Mean (Standard Deviation); Unit of measure: Years
	Number Analyzed	10 participants	20 participants	30 participants
		41.9 (14.5)	42.3 (15.7)	42.2 (15.0)
Sex: Female, Male; Measure Type: Count of Participants; Unit of measure: Participants
	Number Analyzed	10 participants	20 participants	30 participants
	Female	6 60.0%	13 65.0%	19 63.3%
	Male	4 40.0%	7 35.0%	11 36.7%
Race/Ethnicity, Customized; Measure Type: Count of Participants; Unit of measure: Participants
	Number Analyzed	10 participants	20 participants	30 participants
	Asian	3 30.0%	6 30.0%	9 30.0%
	White	7 70.0%	13 65.0%	20 66.7%
	Missing	0 0.0%	1 5.0%	1 3.3%
Race/Ethnicity, Customized; Measure Type: Count of Participants; Unit of measure: Participants
	Number Analyzed	10 participants	20 participants	30 participants
	Hispanic or Latino	0 0.0%	2 10.0%	2 6.7%
	Not Hispanic or Latino	10 100.0%	18 90.0%	28 93.3%
	Missing	0 0.0%	0 0.0%	0 0.0%
Platelet count; Mean (Standard Deviation); Unit of measure: cells*10^9/L
	Number Analyzed	10 participants	20 participants	30 participants
		14.10 (7.77)	14.65 (8.18)	14.46 (7.91)

Outcome Measures

1. Primary Outcome

Title	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
Description	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.
Time Frame	During the last 12 weeks (Week 13 to Week 25)

Outcome Measure Data

Analysis Population Description

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

Arm/Group Title	Placebo	Rozanolixizumab
Arm/Group Description:	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.	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 Number of Participants Analyzed	10	20
Measure Type: Number; Unit of Measure: Percentage of participants
	0	5

2. Secondary Outcome

Title	Cumulative Number of Weeks With Clinically Meaningful Platelet Response of ≥50×10^9/L Over the 24-week Treatment Period
Description	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.
Time Frame	Week 1 up to Week 25

Outcome Measure Data

Analysis Population Description

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

Arm/Group Title	Placebo	Rozanolixizumab
Arm/Group Description:	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.	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 Number of Participants Analyzed	10	20
Median (Full Range); Unit of Measure: Weeks
	0.0; (0 to 10)	1.0; (0 to 18)

3. Secondary Outcome

Title	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
Description	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.
Time Frame	Time from starting treatment to achievement of first response of ≥50×10^9/L (up to Week 25)

Outcome Measure Data

Analysis Population Description

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

Arm/Group Title	Placebo	Rozanolixizumab
Arm/Group Description:	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.	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 Number of Participants Analyzed	10	20
Median (95% Confidence Interval); Unit of Measure: Days
	NA [1]; (4.0 to NA)	8.0 [2]; (5.0 to NA)

[1]

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.

[2]

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.

4. Secondary Outcome

Title	Percentage of Participants With Clinically Meaningful Platelet Response of ≥50×10^9/L by Day 8
Description	Clinically meaningful platelet response was defined as platelet count of ≥50×10^9/L.
Time Frame	Baseline to Day 8

Outcome Measure Data

Analysis Population Description

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

Arm/Group Title	Placebo	Rozanolixizumab
Arm/Group Description:	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.	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 Number of Participants Analyzed	10	20
Measure Type: Number; Unit of Measure: Percentage of participants
	10.0	45.0

5. Secondary Outcome

Title	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
Description	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.
Time Frame	From Baseline during Treatment Period (up to Week 25)

Outcome Measure Data

Analysis Population Description

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

Arm/Group Title	Placebo	Rozanolixizumab
Arm/Group Description:	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.	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 Number of Participants Analyzed	10	20
Measure Type: Number; Unit of Measure: Percentage of participants
	10.0	20.0

6. Secondary Outcome

Title	Time to First Rescue Therapy
Description	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.
Time Frame	From Baseline to first rescue therapy (up to Week 25)

Outcome Measure Data

Analysis Population Description

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

Arm/Group Title	Placebo	Rozanolixizumab
Arm/Group Description:	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.	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 Number of Participants Analyzed	10	20
Median (95% Confidence Interval); Unit of Measure: Days
	NA [1]; (4.0 to NA)	NA [2]; (46.0 to NA)

[1]

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.

[2]

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.

7. Secondary Outcome

Title	Change From Baseline to Week 25 in Primary Immune Thrombocytopenia Patient Assessment Questionnaire (ITP-PAQ) Symptoms Score
Description	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	From Baseline during Treatment Period (up to Week 25)

Outcome Measure Data

Analysis Population Description

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.

Arm/Group Title	Placebo	Rozanolixizumab
Arm/Group Description:	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.	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 Number of Participants Analyzed	8	12
Mean (Standard Deviation); Unit of Measure: units on a scale
	-0.5 (17.0)	4.2 (12.9)

8. Secondary Outcome

Title	Percentage of Participants With Treatment-emergent Adverse Events (TEAEs)
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.
Time Frame	From Baseline to end of Safety Follow-Up Period (up to Week 31)

Outcome Measure Data

Analysis Population Description

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

Arm/Group Title	Placebo	Rozanolixizumab
Arm/Group Description:	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.	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 Number of Participants Analyzed	10	20
Measure Type: Number; Unit of Measure: Percentage of participants
	60.0	95.0

9. Secondary Outcome

Title	Percentage of Participants With TEAEs Leading to Withdrawal of Investigational Medicinal Product (ie, Study Discontinuation)
Description	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.
Time Frame	From Baseline to end of Safety Follow-Up Period (up to Week 31)

Outcome Measure Data

Analysis Population Description

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

Arm/Group Title	Placebo	Rozanolixizumab
Arm/Group Description:	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.	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 Number of Participants Analyzed	10	20
Measure Type: Number; Unit of Measure: Percentage of participants
	0	10

Adverse Events

Time Frame	From Baseline to end of Safety Follow-Up Period (up to Week 31)
Adverse Event Reporting Description	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.
Arm/Group Title	Placebo		Rozanolixizumab
Arm/Group Description	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.		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.
All-Cause Mortality
	Placebo		Rozanolixizumab
	Affected / at Risk (%)		Affected / at Risk (%)
Total	0/10 (0.00%)		0/20 (0.00%)
Serious Adverse Events
	Placebo		Rozanolixizumab
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	1/10 (10.00%)		5/20 (25.00%)
Blood and lymphatic system disorders
Thrombocytopenia * 1	1/10 (10.00%)	1	0/20 (0.00%)	0
Gastrointestinal disorders
Vomiting * 1	0/10 (0.00%)	0	1/20 (5.00%)	1
Infections and infestations
Pneumonia viral * 1	0/10 (0.00%)	0	1/20 (5.00%)	1
Injury, poisoning and procedural complications
Head injury * 1	0/10 (0.00%)	0	1/20 (5.00%)	1
Limb injury * 1	0/10 (0.00%)	0	1/20 (5.00%)	1
Skin injury * 1	0/10 (0.00%)	0	1/20 (5.00%)	1
Investigations
Platelet count decreased * 1	1/10 (10.00%)	1	1/20 (5.00%)	1
Nervous system disorders
Headache * 1	0/10 (0.00%)	0	1/20 (5.00%)	1
Dizziness * 1	0/10 (0.00%)	0	1/20 (5.00%)	1
Skin and subcutaneous tissue disorders
Urticaria * 1	0/10 (0.00%)	0	1/20 (5.00%)	1
1 Term from vocabulary, MedDRA v24.0; * Indicates events were collected by non-systematic assessment
Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events	5%
	Placebo		Rozanolixizumab
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	6/10 (60.00%)		18/20 (90.00%)
Blood and lymphatic system disorders
Anaemia * 1	0/10 (0.00%)	0	2/20 (10.00%)	2
Hypofibrinogenaemia * 1	1/10 (10.00%)	1	0/20 (0.00%)	0
Eye disorders
Eye pain * 1	1/10 (10.00%)	1	0/20 (0.00%)	0
Gastrointestinal disorders
Nausea * 1	0/10 (0.00%)	0	3/20 (15.00%)	3
Diarrhoea * 1	0/10 (0.00%)	0	2/20 (10.00%)	3
Gingival bleeding * 1	0/10 (0.00%)	0	2/20 (10.00%)	3
Abdominal pain * 1	1/10 (10.00%)	1	1/20 (5.00%)	1
Constipation * 1	1/10 (10.00%)	1	1/20 (5.00%)	1
Abdominal distension * 1	1/10 (10.00%)	1	0/20 (0.00%)	0
General disorders
Pyrexia * 1	0/10 (0.00%)	0	6/20 (30.00%)	22
Fatigue * 1	0/10 (0.00%)	0	2/20 (10.00%)	4
Asthenia * 1	1/10 (10.00%)	1	1/20 (5.00%)	4
Infusion site pain * 1	1/10 (10.00%)	1	0/20 (0.00%)	0
Infections and infestations
Upper respiratory tract infection * 1	1/10 (10.00%)	1	2/20 (10.00%)	2
COVID-19 * 1	1/10 (10.00%)	1	1/20 (5.00%)	1
Urinary tract infection * 1	1/10 (10.00%)	1	1/20 (5.00%)	1
Pharyngitis * 1	1/10 (10.00%)	1	0/20 (0.00%)	0
Investigations
Platelet count decreased * 1	1/10 (10.00%)	1	0/20 (0.00%)	0
White blood cell count increased * 1	1/10 (10.00%)	1	0/20 (0.00%)	0
Metabolism and nutrition disorders
Decreased appetite * 1	1/10 (10.00%)	1	0/20 (0.00%)	0
Hyperkalaemia * 1	1/10 (10.00%)	4	0/20 (0.00%)	0
Musculoskeletal and connective tissue disorders
Myalgia * 1	0/10 (0.00%)	0	2/20 (10.00%)	3
Arthralgia * 1	1/10 (10.00%)	1	1/20 (5.00%)	3
Nervous system disorders
Headache * 1	2/10 (20.00%)	2	12/20 (60.00%)	25
Somnolence * 1	1/10 (10.00%)	1	0/20 (0.00%)	0
Psychiatric disorders
Insomnia * 1	1/10 (10.00%)	1	1/20 (5.00%)	1
Renal and urinary disorders
Haematuria * 1	0/10 (0.00%)	0	2/20 (10.00%)	2
Respiratory, thoracic and mediastinal disorders
Epistaxis * 1	0/10 (0.00%)	0	2/20 (10.00%)	2
Oropharyngeal pain * 1	0/10 (0.00%)	0	2/20 (10.00%)	2
Skin and subcutaneous tissue disorders
Urticaria * 1	0/10 (0.00%)	0	2/20 (10.00%)	2
Petechiae * 1	1/10 (10.00%)	2	1/20 (5.00%)	2
Blister * 1	1/10 (10.00%)	1	0/20 (0.00%)	0
Hirsutism * 1	1/10 (10.00%)	1	0/20 (0.00%)	0
Vascular disorders
Haemorrhage * 1	1/10 (10.00%)	1	0/20 (0.00%)	0
1 Term from vocabulary, MedDRA v24.0; * Indicates events were collected by non-systematic assessment

Limitations and Caveats

[Not Specified]

More Information

Certain Agreements

Principal Investigators are NOT employed by the organization sponsoring the study.

There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.

Results Point of Contact

• Name/Title: UCB
• Organization: Cares
• Phone: 001 844 599 2273
• EMail: UCBCares@ucb.com

• Responsible Party: UCB Pharma ( UCB Biopharma SRL )
• ClinicalTrials.gov Identifier: NCT04224688
• Other Study ID Numbers: TP0006; 2019-003451-11 ( EudraCT Number )
• First Submitted: January 8, 2020
• First Posted: January 13, 2020
• Results First Submitted: May 15, 2023
• Results First Posted: August 8, 2023
• Last Update Posted: August 8, 2023