Trial Outcomes & Findings for A Study to Assess the Efficacy and Safety of Efgartigimod in Adult Patients With Primary Immune Thrombocytopenia (ITP). (NCT NCT04188379)

Last Updated: 2025-03-13

Results Overview

Percentage of participants with chronic ITP with a sustained platelet count response was defined as achieving platelet counts of at least 50 × 10\^9/L for at least 4 of the 6 visits between Week 19 and 24 of the study.

Recruitment status

COMPLETED

Study phase

PHASE3

Target enrollment

131 participants

Primary outcome timeframe

From Week 19 up to Week 24

Results posted on

2025-03-13

Participant Flow

This study was conducted at 71 active sites that enrolled participants in 18 countries. Recruitment started on 09 December 2019.

During the screening period (up to 2 weeks) the participant's eligibility for trial participation was evaluated. A total of 205 participants were screened, of which 74 participants were screen failures. 131 of 205 were enrolled and randomized at a ratio of 2:1 to receive efgartigimod or placebo.

Participant milestones

Participant milestones
Measure	Efgartigimod Participants receiving efgartigimod efgartigimod: The participants entered a 24-week treatment period and were randomized to receive efgartigimod 10mg/kg IV , weekly from visits 1 to 4 and then from visits 5 to 16 either weekly or q2w, adjusted according to their platelet counts. From visits 17 to 24, subjects were fixed on the dosing schedule at visit 16 or at the last visit at which IMP was administered (ie, either weekly or q2w).	Placebo Participants receiving placebo Placebo: The participants entered a 24-week treatment period and were randomized to receive placebo IV, weekly from visits 1 to 4 and then from visits 5 to 16 either weekly or q2w, adjusted according to their platelet counts. From visits 17 to 24, participants were fixed on the dosing schedule at visit 16 or at the last visit at which IMP was administered (ie, either weekly or q2w).
Overall Study STARTED	86	45
Overall Study COMPLETED	64	32
Overall Study NOT COMPLETED	22	13

Reasons for withdrawal

Reasons for withdrawal
Measure	Efgartigimod Participants receiving efgartigimod efgartigimod: The participants entered a 24-week treatment period and were randomized to receive efgartigimod 10mg/kg IV , weekly from visits 1 to 4 and then from visits 5 to 16 either weekly or q2w, adjusted according to their platelet counts. From visits 17 to 24, subjects were fixed on the dosing schedule at visit 16 or at the last visit at which IMP was administered (ie, either weekly or q2w).	Placebo Participants receiving placebo Placebo: The participants entered a 24-week treatment period and were randomized to receive placebo IV, weekly from visits 1 to 4 and then from visits 5 to 16 either weekly or q2w, adjusted according to their platelet counts. From visits 17 to 24, participants were fixed on the dosing schedule at visit 16 or at the last visit at which IMP was administered (ie, either weekly or q2w).
Overall Study Withdrawal by Subject	10	3
Overall Study Physician Decision	0	1
Overall Study Adverse Event	3	1
Overall Study Pregnancy	1	0
Overall Study Lack of Efficacy	8	5
Overall Study Other	0	3

Baseline Characteristics

A Study to Assess the Efficacy and Safety of Efgartigimod in Adult Patients With Primary Immune Thrombocytopenia (ITP).

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Efgartigimod n=86 Participants Participants receiving efgartigimod efgartigimod: The participants entered a 24-week treatment period and were randomised to receive efgartigimod 10mg/kg intravenous (IV). The investigational medicinal product (IMP) infusion (efgartigimod) was administered weekly from visits 1 to 4, either weekly or every other week (q2w) from visits 5 to 16, and fixed on the dosing schedule of visit 16 (or the last visit at which IMP was administered) from visits 17 to 24 (ie, either weekly or q2w).	Placebo n=45 Participants Participants receiving placebo Placebo: The participants entered a 24-week treatment period and were randomized to receive placebo intravenous (IV). The placebo infusion was administered weekly from visits 1 to 4, either weekly or q2w from visits 5 to 16, and fixed on the dosing schedule of visit 16 (or the last visit at which placebo was administered) from visits 17 to 24 (ie, either weekly or q2w).	Total n=131 Participants Total of all reporting groups
Age, Categorical <=18 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	75 Participants n=5 Participants	32 Participants n=7 Participants	107 Participants n=5 Participants
Age, Categorical >=65 years	11 Participants n=5 Participants	13 Participants n=7 Participants	24 Participants n=5 Participants
Age, Continuous	46.9 years STANDARD_DEVIATION 16.55 • n=5 Participants	51.7 years STANDARD_DEVIATION 17.93 • n=7 Participants	48.6 years STANDARD_DEVIATION 17.12 • n=5 Participants
Sex: Female, Male Female	47 Participants n=5 Participants	24 Participants n=7 Participants	71 Participants n=5 Participants
Sex: Female, Male Male	39 Participants n=5 Participants	21 Participants n=7 Participants	60 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Asian	5 Participants n=5 Participants	3 Participants n=7 Participants	8 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) White	80 Participants n=5 Participants	41 Participants n=7 Participants	121 Participants n=5 Participants
Race (NIH/OMB) More than one race	1 Participants n=5 Participants	0 Participants n=7 Participants	1 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	1 Participants n=7 Participants	1 Participants n=5 Participants
Race/Ethnicity, Customized Hispanic or Latino	4 Participants n=5 Participants	1 Participants n=7 Participants	5 Participants n=5 Participants
Race/Ethnicity, Customized Not Hispanic or Latino	77 Participants n=5 Participants	40 Participants n=7 Participants	117 Participants n=5 Participants
Race/Ethnicity, Customized Japanese	5 Participants n=5 Participants	3 Participants n=7 Participants	8 Participants n=5 Participants
Race/Ethnicity, Customized Unknown or Not reported	0 Participants n=5 Participants	1 Participants n=7 Participants	1 Participants n=5 Participants

PRIMARY outcome

Timeframe: From Week 19 up to Week 24

Population: Full Analysis Set-Chronic population: all randomized participants with chronic ITP.

Outcome measures

Outcome measures
Measure	Efgartigimod n=78 Participants Participants receiving efgartigimod Efgartigimod: The participants entered a 24-week treatment period and were randomized to receive efgartigimod 10mg/kg IV, weekly from visits 1 to 4 and then from visits 5 to 16 either weekly or q2w, adjusted according to their platelet counts. From visits 17 to 24, subjects were fixed on the dosing schedule at visit 16 or at the last visit at which IMP was administered (ie, either weekly or q2w).	Placebo n=40 Participants Participants receiving placebo Placebo: The participants entered a 24-week treatment period and were randomized to receive placebo IV, weekly from visits 1 to 4 and then from visits 5 to 16 either weekly or q2w, adjusted according to their platelet counts. From visits 17 to 24, participants were fixed on the dosing schedule at visit 16 or at the last visit at which IMP was administered (ie, either weekly or q2w).
Percentage of Participants With Chronic ITP With a Sustained Platelet Count Response Defined as Achieving Platelet Counts of at Least 50×10^9/L for at Least 4 of the 6 Visits Between Week 19 and 24 of the Trial.	21.8 Percentage of participants	5.0 Percentage of participants

SECONDARY outcome

Timeframe: From Week 1 up to Week 24

Population: Full Analysis Set-Chronic population: all randomized participants with chronic ITP.

Extent of disease control, defined as the cumulative number of weeks over the planned 24-week treatment period with platelet counts of ≥50 × 10\^9/L in the chronic ITP population.

Outcome measures

Outcome measures
Measure	Efgartigimod n=78 Participants Participants receiving efgartigimod Efgartigimod: The participants entered a 24-week treatment period and were randomized to receive efgartigimod 10mg/kg IV, weekly from visits 1 to 4 and then from visits 5 to 16 either weekly or q2w, adjusted according to their platelet counts. From visits 17 to 24, subjects were fixed on the dosing schedule at visit 16 or at the last visit at which IMP was administered (ie, either weekly or q2w).	Placebo n=40 Participants Participants receiving placebo Placebo: The participants entered a 24-week treatment period and were randomized to receive placebo IV, weekly from visits 1 to 4 and then from visits 5 to 16 either weekly or q2w, adjusted according to their platelet counts. From visits 17 to 24, participants were fixed on the dosing schedule at visit 16 or at the last visit at which IMP was administered (ie, either weekly or q2w).
Extent of Disease Control Defined as the Number of Cumulative Weeks Over the Planned 24-week Treatment Period With Platelet Counts of ≥50×10^9/L in the Chronic ITP Population	2.00 Weeks Interval 0.0 to 11.0	0.00 Weeks Interval 0.0 to 1.0

SECONDARY outcome

Timeframe: From Week 19 up to Week 24

Population: Full Analysis Set: all randomized participants.

Percentage of participants in the overall population (chronic and persistent ITP) with a sustained platelet count response, defined as achieving platelet counts of at least 50 × 10\^9/L for at least 4 of the 6 visits between weeks 19 and 24 of the study.

Outcome measures

Outcome measures
Measure	Efgartigimod n=86 Participants Participants receiving efgartigimod Efgartigimod: The participants entered a 24-week treatment period and were randomized to receive efgartigimod 10mg/kg IV, weekly from visits 1 to 4 and then from visits 5 to 16 either weekly or q2w, adjusted according to their platelet counts. From visits 17 to 24, subjects were fixed on the dosing schedule at visit 16 or at the last visit at which IMP was administered (ie, either weekly or q2w).	Placebo n=45 Participants Participants receiving placebo Placebo: The participants entered a 24-week treatment period and were randomized to receive placebo IV, weekly from visits 1 to 4 and then from visits 5 to 16 either weekly or q2w, adjusted according to their platelet counts. From visits 17 to 24, participants were fixed on the dosing schedule at visit 16 or at the last visit at which IMP was administered (ie, either weekly or q2w).
Percentage of Participants With a Sustained Platelet Count Response for at Least 4 of the 6 Visits Between Week 19 and 24 of the Study	25.6 Percentage of participants	6.7 Percentage of participants

SECONDARY outcome

Timeframe: From Week 1 to Week 24

Population: Full Analysis Set: all randomized participants.

Incidence of the World Health Organization (WHO)-classified bleeding events in the overall population. This secondary endpoint used the WHO-classified bleeding scale. Bleeding was the predominant clinical manifestation of ITP and was typically related to platelet count. Accordingly, measuring bleeding was important for monitoring this participant population.

Outcome measures

Outcome measures
Measure	Efgartigimod n=86 Participants Participants receiving efgartigimod Efgartigimod: The participants entered a 24-week treatment period and were randomized to receive efgartigimod 10mg/kg IV, weekly from visits 1 to 4 and then from visits 5 to 16 either weekly or q2w, adjusted according to their platelet counts. From visits 17 to 24, subjects were fixed on the dosing schedule at visit 16 or at the last visit at which IMP was administered (ie, either weekly or q2w).	Placebo n=45 Participants Participants receiving placebo Placebo: The participants entered a 24-week treatment period and were randomized to receive placebo IV, weekly from visits 1 to 4 and then from visits 5 to 16 either weekly or q2w, adjusted according to their platelet counts. From visits 17 to 24, participants were fixed on the dosing schedule at visit 16 or at the last visit at which IMP was administered (ie, either weekly or q2w).
Incidence and Severity of the WHO-classified Bleeding Events	4.0 bleeding events Interval 1.0 to 10.0	5.0 bleeding events Interval 2.0 to 14.0

SECONDARY outcome

Timeframe: From Week 17 up to Week 24

Population: Full Analysis Set: all randomized participants.

Percentage of participants in the overall population achieving platelet counts of at least 50 × 10\^9/L for at least 6 of the 8 visits between Week 17 and 24 of the study.

Outcome measures

Outcome measures
Measure	Efgartigimod n=86 Participants Participants receiving efgartigimod Efgartigimod: The participants entered a 24-week treatment period and were randomized to receive efgartigimod 10mg/kg IV, weekly from visits 1 to 4 and then from visits 5 to 16 either weekly or q2w, adjusted according to their platelet counts. From visits 17 to 24, subjects were fixed on the dosing schedule at visit 16 or at the last visit at which IMP was administered (ie, either weekly or q2w).	Placebo n=45 Participants Participants receiving placebo Placebo: The participants entered a 24-week treatment period and were randomized to receive placebo IV, weekly from visits 1 to 4 and then from visits 5 to 16 either weekly or q2w, adjusted according to their platelet counts. From visits 17 to 24, participants were fixed on the dosing schedule at visit 16 or at the last visit at which IMP was administered (ie, either weekly or q2w).
Percentage of Participants in the Overall Population Achieving Platelet Counts of at Least 50×10^9/L for at Least 6 of the 8 Visits Between Week 17 and Week 24	22.1 Percentage of participants	6.7 Percentage of participants

Adverse Events

Efgartigimod

Serious events: 7 serious events

Other events: 80 other events

Deaths: 0 deaths

Placebo

Serious events: 7 serious events

Other events: 43 other events

Deaths: 0 deaths

Serious adverse events

Other adverse events

Additional Information

Regulatory Manager

argenx BV

Phone: +32 9 310 3400

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place

Restriction type: OTHER

Measure	Efgartigimod n=86 participants at risk Patient receiving efgartigimod efgartigimod: Intravenous infusion of efgartigimod	Placebo n=45 participants at risk Patients receiving placebo Placebo: Intravenous infusion of placebo
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Chronic myelomonocytic leukaemia	1.2% 1/86 • Number of events 1 • Full study duration (31 Weeks). Treatment emergent adverse events are defined as adverse events starting on or after first administration of any study drug. Safety analyses were performed on the safety analysis set which included all participants in the randomized population who received at least 1 dose or part of a dose of IMP.	0.00% 0/45 • Full study duration (31 Weeks). Treatment emergent adverse events are defined as adverse events starting on or after first administration of any study drug. Safety analyses were performed on the safety analysis set which included all participants in the randomized population who received at least 1 dose or part of a dose of IMP.
Injury, poisoning and procedural complications Road traffic accident	0.00% 0/86 • Full study duration (31 Weeks). Treatment emergent adverse events are defined as adverse events starting on or after first administration of any study drug. Safety analyses were performed on the safety analysis set which included all participants in the randomized population who received at least 1 dose or part of a dose of IMP.	2.2% 1/45 • Number of events 1 • Full study duration (31 Weeks). Treatment emergent adverse events are defined as adverse events starting on or after first administration of any study drug. Safety analyses were performed on the safety analysis set which included all participants in the randomized population who received at least 1 dose or part of a dose of IMP.
Nervous system disorders Headache	1.2% 1/86 • Number of events 1 • Full study duration (31 Weeks). Treatment emergent adverse events are defined as adverse events starting on or after first administration of any study drug. Safety analyses were performed on the safety analysis set which included all participants in the randomized population who received at least 1 dose or part of a dose of IMP.	0.00% 0/45 • Full study duration (31 Weeks). Treatment emergent adverse events are defined as adverse events starting on or after first administration of any study drug. Safety analyses were performed on the safety analysis set which included all participants in the randomized population who received at least 1 dose or part of a dose of IMP.
Blood and lymphatic system disorders Anaemia	0.00% 0/86 • Full study duration (31 Weeks). Treatment emergent adverse events are defined as adverse events starting on or after first administration of any study drug. Safety analyses were performed on the safety analysis set which included all participants in the randomized population who received at least 1 dose or part of a dose of IMP.	2.2% 1/45 • Number of events 1 • Full study duration (31 Weeks). Treatment emergent adverse events are defined as adverse events starting on or after first administration of any study drug. Safety analyses were performed on the safety analysis set which included all participants in the randomized population who received at least 1 dose or part of a dose of IMP.
Blood and lymphatic system disorders Immune thrombocytopenia	0.00% 0/86 • Full study duration (31 Weeks). Treatment emergent adverse events are defined as adverse events starting on or after first administration of any study drug. Safety analyses were performed on the safety analysis set which included all participants in the randomized population who received at least 1 dose or part of a dose of IMP.	2.2% 1/45 • Number of events 1 • Full study duration (31 Weeks). Treatment emergent adverse events are defined as adverse events starting on or after first administration of any study drug. Safety analyses were performed on the safety analysis set which included all participants in the randomized population who received at least 1 dose or part of a dose of IMP.
Blood and lymphatic system disorders Iron deficiency anaemia	1.2% 1/86 • Number of events 1 • Full study duration (31 Weeks). Treatment emergent adverse events are defined as adverse events starting on or after first administration of any study drug. Safety analyses were performed on the safety analysis set which included all participants in the randomized population who received at least 1 dose or part of a dose of IMP.	0.00% 0/45 • Full study duration (31 Weeks). Treatment emergent adverse events are defined as adverse events starting on or after first administration of any study drug. Safety analyses were performed on the safety analysis set which included all participants in the randomized population who received at least 1 dose or part of a dose of IMP.
Blood and lymphatic system disorders Thrombocytopenia	2.3% 2/86 • Number of events 2 • Full study duration (31 Weeks). Treatment emergent adverse events are defined as adverse events starting on or after first administration of any study drug. Safety analyses were performed on the safety analysis set which included all participants in the randomized population who received at least 1 dose or part of a dose of IMP.	0.00% 0/45 • Full study duration (31 Weeks). Treatment emergent adverse events are defined as adverse events starting on or after first administration of any study drug. Safety analyses were performed on the safety analysis set which included all participants in the randomized population who received at least 1 dose or part of a dose of IMP.
Gastrointestinal disorders Abdominal pain	1.2% 1/86 • Number of events 1 • Full study duration (31 Weeks). Treatment emergent adverse events are defined as adverse events starting on or after first administration of any study drug. Safety analyses were performed on the safety analysis set which included all participants in the randomized population who received at least 1 dose or part of a dose of IMP.	0.00% 0/45 • Full study duration (31 Weeks). Treatment emergent adverse events are defined as adverse events starting on or after first administration of any study drug. Safety analyses were performed on the safety analysis set which included all participants in the randomized population who received at least 1 dose or part of a dose of IMP.
Gastrointestinal disorders Mouth haemorrhage	0.00% 0/86 • Full study duration (31 Weeks). Treatment emergent adverse events are defined as adverse events starting on or after first administration of any study drug. Safety analyses were performed on the safety analysis set which included all participants in the randomized population who received at least 1 dose or part of a dose of IMP.	2.2% 1/45 • Number of events 1 • Full study duration (31 Weeks). Treatment emergent adverse events are defined as adverse events starting on or after first administration of any study drug. Safety analyses were performed on the safety analysis set which included all participants in the randomized population who received at least 1 dose or part of a dose of IMP.
Reproductive system and breast disorders Vaginal haemorrhage	1.2% 1/86 • Number of events 1 • Full study duration (31 Weeks). Treatment emergent adverse events are defined as adverse events starting on or after first administration of any study drug. Safety analyses were performed on the safety analysis set which included all participants in the randomized population who received at least 1 dose or part of a dose of IMP.	0.00% 0/45 • Full study duration (31 Weeks). Treatment emergent adverse events are defined as adverse events starting on or after first administration of any study drug. Safety analyses were performed on the safety analysis set which included all participants in the randomized population who received at least 1 dose or part of a dose of IMP.
Renal and urinary disorders Acute kidney injury	0.00% 0/86 • Full study duration (31 Weeks). Treatment emergent adverse events are defined as adverse events starting on or after first administration of any study drug. Safety analyses were performed on the safety analysis set which included all participants in the randomized population who received at least 1 dose or part of a dose of IMP.	2.2% 1/45 • Number of events 1 • Full study duration (31 Weeks). Treatment emergent adverse events are defined as adverse events starting on or after first administration of any study drug. Safety analyses were performed on the safety analysis set which included all participants in the randomized population who received at least 1 dose or part of a dose of IMP.
Renal and urinary disorders Haematuria	0.00% 0/86 • Full study duration (31 Weeks). Treatment emergent adverse events are defined as adverse events starting on or after first administration of any study drug. Safety analyses were performed on the safety analysis set which included all participants in the randomized population who received at least 1 dose or part of a dose of IMP.	2.2% 1/45 • Number of events 1 • Full study duration (31 Weeks). Treatment emergent adverse events are defined as adverse events starting on or after first administration of any study drug. Safety analyses were performed on the safety analysis set which included all participants in the randomized population who received at least 1 dose or part of a dose of IMP.
Musculoskeletal and connective tissue disorders Musculoskeletal chest pain	1.2% 1/86 • Number of events 1 • Full study duration (31 Weeks). Treatment emergent adverse events are defined as adverse events starting on or after first administration of any study drug. Safety analyses were performed on the safety analysis set which included all participants in the randomized population who received at least 1 dose or part of a dose of IMP.	0.00% 0/45 • Full study duration (31 Weeks). Treatment emergent adverse events are defined as adverse events starting on or after first administration of any study drug. Safety analyses were performed on the safety analysis set which included all participants in the randomized population who received at least 1 dose or part of a dose of IMP.
Infections and infestations Appendicitis	0.00% 0/86 • Full study duration (31 Weeks). Treatment emergent adverse events are defined as adverse events starting on or after first administration of any study drug. Safety analyses were performed on the safety analysis set which included all participants in the randomized population who received at least 1 dose or part of a dose of IMP.	2.2% 1/45 • Number of events 1 • Full study duration (31 Weeks). Treatment emergent adverse events are defined as adverse events starting on or after first administration of any study drug. Safety analyses were performed on the safety analysis set which included all participants in the randomized population who received at least 1 dose or part of a dose of IMP.
Infections and infestations COVID-19	0.00% 0/86 • Full study duration (31 Weeks). Treatment emergent adverse events are defined as adverse events starting on or after first administration of any study drug. Safety analyses were performed on the safety analysis set which included all participants in the randomized population who received at least 1 dose or part of a dose of IMP.	2.2% 1/45 • Number of events 1 • Full study duration (31 Weeks). Treatment emergent adverse events are defined as adverse events starting on or after first administration of any study drug. Safety analyses were performed on the safety analysis set which included all participants in the randomized population who received at least 1 dose or part of a dose of IMP.
Infections and infestations Cytomegalovirus infection	1.2% 1/86 • Number of events 1 • Full study duration (31 Weeks). Treatment emergent adverse events are defined as adverse events starting on or after first administration of any study drug. Safety analyses were performed on the safety analysis set which included all participants in the randomized population who received at least 1 dose or part of a dose of IMP.	0.00% 0/45 • Full study duration (31 Weeks). Treatment emergent adverse events are defined as adverse events starting on or after first administration of any study drug. Safety analyses were performed on the safety analysis set which included all participants in the randomized population who received at least 1 dose or part of a dose of IMP.
Infections and infestations Erysipelas	1.2% 1/86 • Number of events 1 • Full study duration (31 Weeks). Treatment emergent adverse events are defined as adverse events starting on or after first administration of any study drug. Safety analyses were performed on the safety analysis set which included all participants in the randomized population who received at least 1 dose or part of a dose of IMP.	0.00% 0/45 • Full study duration (31 Weeks). Treatment emergent adverse events are defined as adverse events starting on or after first administration of any study drug. Safety analyses were performed on the safety analysis set which included all participants in the randomized population who received at least 1 dose or part of a dose of IMP.

Measure	Efgartigimod n=86 participants at risk Patient receiving efgartigimod efgartigimod: Intravenous infusion of efgartigimod	Placebo n=45 participants at risk Patients receiving placebo Placebo: Intravenous infusion of placebo
Vascular disorders Haematoma	9.3% 8/86 • Number of events 17 • Full study duration (31 Weeks). Treatment emergent adverse events are defined as adverse events starting on or after first administration of any study drug. Safety analyses were performed on the safety analysis set which included all participants in the randomized population who received at least 1 dose or part of a dose of IMP.	24.4% 11/45 • Number of events 34 • Full study duration (31 Weeks). Treatment emergent adverse events are defined as adverse events starting on or after first administration of any study drug. Safety analyses were performed on the safety analysis set which included all participants in the randomized population who received at least 1 dose or part of a dose of IMP.
Vascular disorders Hypertension	5.8% 5/86 • Number of events 6 • Full study duration (31 Weeks). Treatment emergent adverse events are defined as adverse events starting on or after first administration of any study drug. Safety analyses were performed on the safety analysis set which included all participants in the randomized population who received at least 1 dose or part of a dose of IMP.	0.00% 0/45 • Full study duration (31 Weeks). Treatment emergent adverse events are defined as adverse events starting on or after first administration of any study drug. Safety analyses were performed on the safety analysis set which included all participants in the randomized population who received at least 1 dose or part of a dose of IMP.
General disorders Asthenia	7.0% 6/86 • Number of events 8 • Full study duration (31 Weeks). Treatment emergent adverse events are defined as adverse events starting on or after first administration of any study drug. Safety analyses were performed on the safety analysis set which included all participants in the randomized population who received at least 1 dose or part of a dose of IMP.	0.00% 0/45 • Full study duration (31 Weeks). Treatment emergent adverse events are defined as adverse events starting on or after first administration of any study drug. Safety analyses were performed on the safety analysis set which included all participants in the randomized population who received at least 1 dose or part of a dose of IMP.
Reproductive system and breast disorders Heavy menstrual bleeding	5.8% 5/86 • Number of events 9 • Full study duration (31 Weeks). Treatment emergent adverse events are defined as adverse events starting on or after first administration of any study drug. Safety analyses were performed on the safety analysis set which included all participants in the randomized population who received at least 1 dose or part of a dose of IMP.	6.7% 3/45 • Number of events 4 • Full study duration (31 Weeks). Treatment emergent adverse events are defined as adverse events starting on or after first administration of any study drug. Safety analyses were performed on the safety analysis set which included all participants in the randomized population who received at least 1 dose or part of a dose of IMP.
Respiratory, thoracic and mediastinal disorders Epistaxis	9.3% 8/86 • Number of events 24 • Full study duration (31 Weeks). Treatment emergent adverse events are defined as adverse events starting on or after first administration of any study drug. Safety analyses were performed on the safety analysis set which included all participants in the randomized population who received at least 1 dose or part of a dose of IMP.	17.8% 8/45 • Number of events 12 • Full study duration (31 Weeks). Treatment emergent adverse events are defined as adverse events starting on or after first administration of any study drug. Safety analyses were performed on the safety analysis set which included all participants in the randomized population who received at least 1 dose or part of a dose of IMP.
Investigations Blood urine present	36.0% 31/86 • Number of events 66 • Full study duration (31 Weeks). Treatment emergent adverse events are defined as adverse events starting on or after first administration of any study drug. Safety analyses were performed on the safety analysis set which included all participants in the randomized population who received at least 1 dose or part of a dose of IMP.	37.8% 17/45 • Number of events 32 • Full study duration (31 Weeks). Treatment emergent adverse events are defined as adverse events starting on or after first administration of any study drug. Safety analyses were performed on the safety analysis set which included all participants in the randomized population who received at least 1 dose or part of a dose of IMP.
Injury, poisoning and procedural complications Contusion	19.8% 17/86 • Number of events 52 • Full study duration (31 Weeks). Treatment emergent adverse events are defined as adverse events starting on or after first administration of any study drug. Safety analyses were performed on the safety analysis set which included all participants in the randomized population who received at least 1 dose or part of a dose of IMP.	13.3% 6/45 • Number of events 42 • Full study duration (31 Weeks). Treatment emergent adverse events are defined as adverse events starting on or after first administration of any study drug. Safety analyses were performed on the safety analysis set which included all participants in the randomized population who received at least 1 dose or part of a dose of IMP.
Nervous system disorders Headache	16.3% 14/86 • Number of events 20 • Full study duration (31 Weeks). Treatment emergent adverse events are defined as adverse events starting on or after first administration of any study drug. Safety analyses were performed on the safety analysis set which included all participants in the randomized population who received at least 1 dose or part of a dose of IMP.	13.3% 6/45 • Number of events 22 • Full study duration (31 Weeks). Treatment emergent adverse events are defined as adverse events starting on or after first administration of any study drug. Safety analyses were performed on the safety analysis set which included all participants in the randomized population who received at least 1 dose or part of a dose of IMP.
Blood and lymphatic system disorders Anaemia	7.0% 6/86 • Number of events 8 • Full study duration (31 Weeks). Treatment emergent adverse events are defined as adverse events starting on or after first administration of any study drug. Safety analyses were performed on the safety analysis set which included all participants in the randomized population who received at least 1 dose or part of a dose of IMP.	6.7% 3/45 • Number of events 4 • Full study duration (31 Weeks). Treatment emergent adverse events are defined as adverse events starting on or after first administration of any study drug. Safety analyses were performed on the safety analysis set which included all participants in the randomized population who received at least 1 dose or part of a dose of IMP.
Blood and lymphatic system disorders Neutropenia	2.3% 2/86 • Number of events 7 • Full study duration (31 Weeks). Treatment emergent adverse events are defined as adverse events starting on or after first administration of any study drug. Safety analyses were performed on the safety analysis set which included all participants in the randomized population who received at least 1 dose or part of a dose of IMP.	6.7% 3/45 • Number of events 4 • Full study duration (31 Weeks). Treatment emergent adverse events are defined as adverse events starting on or after first administration of any study drug. Safety analyses were performed on the safety analysis set which included all participants in the randomized population who received at least 1 dose or part of a dose of IMP.
Gastrointestinal disorders Diarrhoea	3.5% 3/86 • Number of events 4 • Full study duration (31 Weeks). Treatment emergent adverse events are defined as adverse events starting on or after first administration of any study drug. Safety analyses were performed on the safety analysis set which included all participants in the randomized population who received at least 1 dose or part of a dose of IMP.	8.9% 4/45 • Number of events 6 • Full study duration (31 Weeks). Treatment emergent adverse events are defined as adverse events starting on or after first administration of any study drug. Safety analyses were performed on the safety analysis set which included all participants in the randomized population who received at least 1 dose or part of a dose of IMP.
Gastrointestinal disorders Gingival bleeding	4.7% 4/86 • Number of events 6 • Full study duration (31 Weeks). Treatment emergent adverse events are defined as adverse events starting on or after first administration of any study drug. Safety analyses were performed on the safety analysis set which included all participants in the randomized population who received at least 1 dose or part of a dose of IMP.	13.3% 6/45 • Number of events 12 • Full study duration (31 Weeks). Treatment emergent adverse events are defined as adverse events starting on or after first administration of any study drug. Safety analyses were performed on the safety analysis set which included all participants in the randomized population who received at least 1 dose or part of a dose of IMP.
Gastrointestinal disorders Mouth haemorrhage	8.1% 7/86 • Number of events 13 • Full study duration (31 Weeks). Treatment emergent adverse events are defined as adverse events starting on or after first administration of any study drug. Safety analyses were performed on the safety analysis set which included all participants in the randomized population who received at least 1 dose or part of a dose of IMP.	15.6% 7/45 • Number of events 10 • Full study duration (31 Weeks). Treatment emergent adverse events are defined as adverse events starting on or after first administration of any study drug. Safety analyses were performed on the safety analysis set which included all participants in the randomized population who received at least 1 dose or part of a dose of IMP.
Gastrointestinal disorders Oral blood blister	2.3% 2/86 • Number of events 3 • Full study duration (31 Weeks). Treatment emergent adverse events are defined as adverse events starting on or after first administration of any study drug. Safety analyses were performed on the safety analysis set which included all participants in the randomized population who received at least 1 dose or part of a dose of IMP.	6.7% 3/45 • Number of events 7 • Full study duration (31 Weeks). Treatment emergent adverse events are defined as adverse events starting on or after first administration of any study drug. Safety analyses were performed on the safety analysis set which included all participants in the randomized population who received at least 1 dose or part of a dose of IMP.
Gastrointestinal disorders Nausea	5.8% 5/86 • Number of events 9 • Full study duration (31 Weeks). Treatment emergent adverse events are defined as adverse events starting on or after first administration of any study drug. Safety analyses were performed on the safety analysis set which included all participants in the randomized population who received at least 1 dose or part of a dose of IMP.	4.4% 2/45 • Number of events 2 • Full study duration (31 Weeks). Treatment emergent adverse events are defined as adverse events starting on or after first administration of any study drug. Safety analyses were performed on the safety analysis set which included all participants in the randomized population who received at least 1 dose or part of a dose of IMP.
Skin and subcutaneous tissue disorders Ecchymosis	5.8% 5/86 • Number of events 10 • Full study duration (31 Weeks). Treatment emergent adverse events are defined as adverse events starting on or after first administration of any study drug. Safety analyses were performed on the safety analysis set which included all participants in the randomized population who received at least 1 dose or part of a dose of IMP.	13.3% 6/45 • Number of events 13 • Full study duration (31 Weeks). Treatment emergent adverse events are defined as adverse events starting on or after first administration of any study drug. Safety analyses were performed on the safety analysis set which included all participants in the randomized population who received at least 1 dose or part of a dose of IMP.
Skin and subcutaneous tissue disorders Petechiae	15.1% 13/86 • Number of events 21 • Full study duration (31 Weeks). Treatment emergent adverse events are defined as adverse events starting on or after first administration of any study drug. Safety analyses were performed on the safety analysis set which included all participants in the randomized population who received at least 1 dose or part of a dose of IMP.	26.7% 12/45 • Number of events 20 • Full study duration (31 Weeks). Treatment emergent adverse events are defined as adverse events starting on or after first administration of any study drug. Safety analyses were performed on the safety analysis set which included all participants in the randomized population who received at least 1 dose or part of a dose of IMP.
Skin and subcutaneous tissue disorders Purpura	8.1% 7/86 • Number of events 12 • Full study duration (31 Weeks). Treatment emergent adverse events are defined as adverse events starting on or after first administration of any study drug. Safety analyses were performed on the safety analysis set which included all participants in the randomized population who received at least 1 dose or part of a dose of IMP.	8.9% 4/45 • Number of events 12 • Full study duration (31 Weeks). Treatment emergent adverse events are defined as adverse events starting on or after first administration of any study drug. Safety analyses were performed on the safety analysis set which included all participants in the randomized population who received at least 1 dose or part of a dose of IMP.
Renal and urinary disorders Haematuria	16.3% 14/86 • Number of events 30 • Full study duration (31 Weeks). Treatment emergent adverse events are defined as adverse events starting on or after first administration of any study drug. Safety analyses were performed on the safety analysis set which included all participants in the randomized population who received at least 1 dose or part of a dose of IMP.	13.3% 6/45 • Number of events 11 • Full study duration (31 Weeks). Treatment emergent adverse events are defined as adverse events starting on or after first administration of any study drug. Safety analyses were performed on the safety analysis set which included all participants in the randomized population who received at least 1 dose or part of a dose of IMP.
Musculoskeletal and connective tissue disorders Pain in extremity	1.2% 1/86 • Number of events 1 • Full study duration (31 Weeks). Treatment emergent adverse events are defined as adverse events starting on or after first administration of any study drug. Safety analyses were performed on the safety analysis set which included all participants in the randomized population who received at least 1 dose or part of a dose of IMP.	11.1% 5/45 • Number of events 5 • Full study duration (31 Weeks). Treatment emergent adverse events are defined as adverse events starting on or after first administration of any study drug. Safety analyses were performed on the safety analysis set which included all participants in the randomized population who received at least 1 dose or part of a dose of IMP.
Infections and infestations COVID-19	8.1% 7/86 • Number of events 7 • Full study duration (31 Weeks). Treatment emergent adverse events are defined as adverse events starting on or after first administration of any study drug. Safety analyses were performed on the safety analysis set which included all participants in the randomized population who received at least 1 dose or part of a dose of IMP.	4.4% 2/45 • Number of events 2 • Full study duration (31 Weeks). Treatment emergent adverse events are defined as adverse events starting on or after first administration of any study drug. Safety analyses were performed on the safety analysis set which included all participants in the randomized population who received at least 1 dose or part of a dose of IMP.