Trial Outcomes & Findings for A Study to Evaluate the Efficacy and Safety of Efgartigimod PH20 Subcutaneous in Adult Patients With Primary Immune Thrombocytopenia (NCT NCT04687072)

Last Updated: 2024-10-31

Results Overview

A participant was considered a responder for this endpoint (i.e., had a sustained platelet count response) if the participant had platelet counts of ≥50 × 10\^9/L for ≥4 of the 6 analysis visits between Weeks 19 and 24.

Recruitment status

COMPLETED

Study phase

PHASE3

Target enrollment

207 participants

Primary outcome timeframe

Up to 6 weeks (between Weeks 19 and 24)

Results posted on

2024-10-31

Participant Flow

A regionally diverse participant population was enrolled (including but not limited to North America, East Asia, Europe, and the rest of world \[ROW\]). A total of 207 participants were randomized to IMP: 137 participants in the efgartigimod PH20 subcutaneous (SC) arm and 70 participants in the placebo PH20 SC arm.

Participant milestones

Participant milestones
Measure	Efgartigimod PH20 SC Participants receiving efgartigimod PH20 SC treatment.	Placebo PH20 SC Participants receiving placebo PH20 SC treatment.
Overall Study STARTED	137	70
Overall Study COMPLETED	113	62
Overall Study NOT COMPLETED	24	8

Reasons for withdrawal

Reasons for withdrawal
Measure	Efgartigimod PH20 SC Participants receiving efgartigimod PH20 SC treatment.	Placebo PH20 SC Participants receiving placebo PH20 SC treatment.
Overall Study Adverse Event	2	0
Overall Study Death	1	0
Overall Study Lack of Efficacy	2	1
Overall Study Physician Decision	1	0
Overall Study Pregnancy	1	0
Overall Study Requires Prohibited Medication	2	0
Overall Study Withdrawal of Consent	11	6
Overall Study Miscellaneous	4	1

Baseline Characteristics

A Study to Evaluate the Efficacy and Safety of Efgartigimod PH20 Subcutaneous in Adult Patients With Primary Immune Thrombocytopenia

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Efgartigimod PH20 SC n=137 Participants Participants receiving efgartigimod PH20 SC treatment.	Placebo PH20 SC n=70 Participants Participants receiving placebo PH20 SC treatment.	Total n=207 Participants Total of all reporting groups
Age, Continuous	47.3 years STANDARD_DEVIATION 17.22 • n=5 Participants	50.0 years STANDARD_DEVIATION 15.29 • n=7 Participants	48.2 years STANDARD_DEVIATION 16.60 • n=5 Participants
Sex: Female, Male Female	88 Participants n=5 Participants	43 Participants n=7 Participants	131 Participants n=5 Participants
Sex: Female, Male Male	49 Participants n=5 Participants	27 Participants n=7 Participants	76 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	8 Participants n=5 Participants	2 Participants n=7 Participants	10 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	129 Participants n=5 Participants	68 Participants n=7 Participants	197 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race/Ethnicity, Customized Asian	44 Participants n=5 Participants	18 Participants n=7 Participants	62 Participants n=5 Participants
Race/Ethnicity, Customized Black or African American	4 Participants n=5 Participants	2 Participants n=7 Participants	6 Participants n=5 Participants
Race/Ethnicity, Customized White	88 Participants n=5 Participants	49 Participants n=7 Participants	137 Participants n=5 Participants
Race/Ethnicity, Customized Other	1 Participants n=5 Participants	1 Participants n=7 Participants	2 Participants n=5 Participants

PRIMARY outcome

Timeframe: Up to 6 weeks (between Weeks 19 and 24)

Population: Full Analysis Set (FAS)-Chronic: Participants from the FAS (all randomized participants) including only participants with chronic ITP.

Outcome measures

Outcome measures
Measure	Efgartigimod PH20 SC n=124 Participants Participants receiving efgartigimod PH20 SC treatment.	Placebo PH20 SC n=68 Participants Participants receiving placebo PH20 SC treatment.
Percentage of Participants With Chronic Immune Thrombocytopenia (ITP) With a Sustained Platelet Count Response Between Weeks 19 and 24	13.7 percentage of participants	16.2 percentage of participants

SECONDARY outcome

Timeframe: Up to 24 weeks

Population: FAS-Chronic: Participants from the FAS (all randomized participants) including only participants with chronic ITP.

Extent of disease control was 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.

Outcome measures

Outcome measures
Measure	Efgartigimod PH20 SC n=124 Participants Participants receiving efgartigimod PH20 SC treatment.	Placebo PH20 SC n=68 Participants Participants receiving placebo PH20 SC treatment.
Extent of Disease Control Over the 24-Week Treatment Period in the Chronic ITP Population	0.5 weeks Interval 0.0 to 24.0	0.0 weeks Interval 0.0 to 23.0

SECONDARY outcome

Timeframe: Up to 6 weeks (between Weeks 19 and 24)

Population: FAS: All randomized participants.

Outcome measures

Outcome measures
Measure	Efgartigimod PH20 SC n=137 Participants Participants receiving efgartigimod PH20 SC treatment.	Placebo PH20 SC n=70 Participants Participants receiving placebo PH20 SC treatment.
Percentage of Participants in the Overall Population (Chronic and Persistent ITP) With a Sustained Platelet Count Response Between Weeks 19 and 24	16.1 percentage of participants	15.7 percentage of participants

SECONDARY outcome

Timeframe: Up to 8 weeks (between Weeks 17 and 24)

Population: FAS: All randomized participants.

A participant was considered a responder for this endpoint (i.e., had a sustained platelet count response) if the participant had platelet counts of ≥50 × 10\^9/L for ≥6 of the 8 analysis visits between weeks 17 and 24.

Outcome measures

Outcome measures
Measure	Efgartigimod PH20 SC n=137 Participants Participants receiving efgartigimod PH20 SC treatment.	Placebo PH20 SC n=70 Participants Participants receiving placebo PH20 SC treatment.
Percentage of Participants in the Overall Population With Sustained Platelet Count Response Between Weeks 17 and 24	12.4 percentage of participants	14.3 percentage of participants

SECONDARY outcome

Timeframe: Up to 24 weeks

Population: FAS: All randomized participants.

A participant was considered a responder for this endpoint (i.e., had an overall platelet count response) if the participant had platelet counts of ≥50 × 10\^9/L for ≥4 analysis visits at any time during the 24-week treatment period.

Outcome measures

Outcome measures
Measure	Efgartigimod PH20 SC n=137 Participants Participants receiving efgartigimod PH20 SC treatment.	Placebo PH20 SC n=70 Participants Participants receiving placebo PH20 SC treatment.
Percentage of Participants in the Overall Population Achieving Overall Platelet Count Response at Any Time During the 24-week Treatment Period	29.9 percentage of participants	25.7 percentage of participants

SECONDARY outcome

Timeframe: Up to 12 weeks

Population: FAS: All randomized participants.

Extent of disease control was defined as the number of cumulative weeks until Week 12 with platelet counts of ≥50×10\^9/L in the overall population.

Outcome measures

Outcome measures
Measure	Efgartigimod PH20 SC n=137 Participants Participants receiving efgartigimod PH20 SC treatment.	Placebo PH20 SC n=70 Participants Participants receiving placebo PH20 SC treatment.
Extent of Disease Control Until Week 12 in the Overall Population	0.00 weeks Interval 0.0 to 12.0	0.00 weeks Interval 0.0 to 11.0

SECONDARY outcome

Timeframe: Up to 12 weeks

Population: FAS: All randomized participants.

Outcome measures

Outcome measures
Measure	Efgartigimod PH20 SC n=137 Participants Participants receiving efgartigimod PH20 SC treatment.	Placebo PH20 SC n=70 Participants Participants receiving placebo PH20 SC treatment.
Percentage of Participants in the Overall Population Achieving Overall Platelet Count Response at Any Time Until Week 12	18.2 percentage of participants	15.7 percentage of participants

SECONDARY outcome

Timeframe: Baseline and Weeks 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12, 13, 14, 15, 16, 17, 18, 19, 20, 21, 22, 23, 24, Safety and Efficacy Follow-up Visit 1 (SEFU1) (up to Week 29), and SEFU2 (up to Week 33)

Population: FAS: All randomized participants with available data.

Change from Baseline at time point t = value at time point t - Baseline value. Baseline was defined as the last available value prior to first administration of the investigational medicinal product (IMP).

Outcome measures

Outcome measures
Measure	Efgartigimod PH20 SC n=136 Participants Participants receiving efgartigimod PH20 SC treatment.	Placebo PH20 SC n=69 Participants Participants receiving placebo PH20 SC treatment.
Mean Change From Baseline in Platelet Count at Each Visit in the Overall Population Week 8	21.77 platelets x10^9/L Standard Deviation 52.308	16.57 platelets x10^9/L Standard Deviation 42.508
Mean Change From Baseline in Platelet Count at Each Visit in the Overall Population Week 14	23.60 platelets x10^9/L Standard Deviation 47.521	24.04 platelets x10^9/L Standard Deviation 55.678
Mean Change From Baseline in Platelet Count at Each Visit in the Overall Population Week 1	13.84 platelets x10^9/L Standard Deviation 41.150	8.82 platelets x10^9/L Standard Deviation 34.355
Mean Change From Baseline in Platelet Count at Each Visit in the Overall Population Week 2	16.62 platelets x10^9/L Standard Deviation 49.408	23.54 platelets x10^9/L Standard Deviation 85.005
Mean Change From Baseline in Platelet Count at Each Visit in the Overall Population Week 3	17.07 platelets x10^9/L Standard Deviation 39.855	19.22 platelets x10^9/L Standard Deviation 71.947
Mean Change From Baseline in Platelet Count at Each Visit in the Overall Population Week 4	12.39 platelets x10^9/L Standard Deviation 37.886	9.31 platelets x10^9/L Standard Deviation 26.813
Mean Change From Baseline in Platelet Count at Each Visit in the Overall Population Week 5	18.14 platelets x10^9/L Standard Deviation 54.335	12.24 platelets x10^9/L Standard Deviation 30.059
Mean Change From Baseline in Platelet Count at Each Visit in the Overall Population Week 6	34.61 platelets x10^9/L Standard Deviation 112.204	16.35 platelets x10^9/L Standard Deviation 40.891
Mean Change From Baseline in Platelet Count at Each Visit in the Overall Population Week 7	28.25 platelets x10^9/L Standard Deviation 107.932	13.46 platelets x10^9/L Standard Deviation 41.876
Mean Change From Baseline in Platelet Count at Each Visit in the Overall Population Week 9	19.90 platelets x10^9/L Standard Deviation 54.542	20.42 platelets x10^9/L Standard Deviation 53.488
Mean Change From Baseline in Platelet Count at Each Visit in the Overall Population Week 10	21.73 platelets x10^9/L Standard Deviation 47.685	15.42 platelets x10^9/L Standard Deviation 33.119
Mean Change From Baseline in Platelet Count at Each Visit in the Overall Population Week 11	20.13 platelets x10^9/L Standard Deviation 42.193	14.54 platelets x10^9/L Standard Deviation 25.588
Mean Change From Baseline in Platelet Count at Each Visit in the Overall Population Week 12	21.02 platelets x10^9/L Standard Deviation 43.882	22.28 platelets x10^9/L Standard Deviation 56.173
Mean Change From Baseline in Platelet Count at Each Visit in the Overall Population Week 13	22.29 platelets x10^9/L Standard Deviation 44.654	15.51 platelets x10^9/L Standard Deviation 37.651
Mean Change From Baseline in Platelet Count at Each Visit in the Overall Population Week 15	21.30 platelets x10^9/L Standard Deviation 45.776	28.05 platelets x10^9/L Standard Deviation 67.897
Mean Change From Baseline in Platelet Count at Each Visit in the Overall Population Week 16	30.32 platelets x10^9/L Standard Deviation 71.553	23.09 platelets x10^9/L Standard Deviation 46.154
Mean Change From Baseline in Platelet Count at Each Visit in the Overall Population Week 17	25.12 platelets x10^9/L Standard Deviation 54.747	30.05 platelets x10^9/L Standard Deviation 56.581
Mean Change From Baseline in Platelet Count at Each Visit in the Overall Population Week 18	28.54 platelets x10^9/L Standard Deviation 53.115	31.64 platelets x10^9/L Standard Deviation 57.309
Mean Change From Baseline in Platelet Count at Each Visit in the Overall Population Week 19	33.86 platelets x10^9/L Standard Deviation 59.323	37.26 platelets x10^9/L Standard Deviation 68.832
Mean Change From Baseline in Platelet Count at Each Visit in the Overall Population Week 20	30.87 platelets x10^9/L Standard Deviation 55.342	25.85 platelets x10^9/L Standard Deviation 48.409
Mean Change From Baseline in Platelet Count at Each Visit in the Overall Population Week 21	30.04 platelets x10^9/L Standard Deviation 61.438	26.23 platelets x10^9/L Standard Deviation 50.323
Mean Change From Baseline in Platelet Count at Each Visit in the Overall Population Week 22	33.26 platelets x10^9/L Standard Deviation 61.558	29.23 platelets x10^9/L Standard Deviation 55.175
Mean Change From Baseline in Platelet Count at Each Visit in the Overall Population Week 23	29.51 platelets x10^9/L Standard Deviation 53.437	24.89 platelets x10^9/L Standard Deviation 41.103
Mean Change From Baseline in Platelet Count at Each Visit in the Overall Population Week 24	25.51 platelets x10^9/L Standard Deviation 52.203	24.91 platelets x10^9/L Standard Deviation 42.972
Mean Change From Baseline in Platelet Count at Each Visit in the Overall Population SEFU1	40.44 platelets x10^9/L Standard Deviation 64.860	48.70 platelets x10^9/L Standard Deviation NA Standard deviation could not be calculated as a single participant was analyzed.
Mean Change From Baseline in Platelet Count at Each Visit in the Overall Population SEFU2	30.00 platelets x10^9/L Standard Deviation 35.011	56.25 platelets x10^9/L Standard Deviation 85.206

SECONDARY outcome

Timeframe: Up to 24 weeks

Population: FAS: All randomized participants. Participants with no occurrence of platelet count response, early discontinuation of treatment, or dose and/or frequency of concurrent ITP therapy increased or a new ITP therapy were censored. If multiple censoring conditions apply, the earliest censoring date is considered.

Time to platelet count response, defined as the time to have 2 consecutive platelet counts of ≥50 × 10\^9/L via Kaplan-Meier estimates.

Outcome measures

Outcome measures
Measure	Efgartigimod PH20 SC n=137 Participants Participants receiving efgartigimod PH20 SC treatment.	Placebo PH20 SC n=70 Participants Participants receiving placebo PH20 SC treatment.
Time to Platelet Count Response in the Overall Population	NA days Interval 155.0 to Median and upper confidence interval were not calculable due to fewer than 50% events.	NA days Median and confidence intervals were not calculable due to insufficient number of events.

SECONDARY outcome

Timeframe: Up to 24 weeks

Population: FAS: All randomized participants.

Extent of disease control was defined as the number of cumulative weeks over the planned 24-week treatment period with platelet counts of ≥30×10\^9/L with at least ≥20×10\^9/L above Baseline in the overall population.

Outcome measures

Outcome measures
Measure	Efgartigimod PH20 SC n=137 Participants Participants receiving efgartigimod PH20 SC treatment.	Placebo PH20 SC n=70 Participants Participants receiving placebo PH20 SC treatment.
Extent of Disease Control Over the 24-Week Treatment Period in the Overall Population	1.0 weeks Interval 0.0 to 24.0	1.0 weeks Interval 0.0 to 24.0

SECONDARY outcome

Timeframe: Up to 24 weeks

Population: FAS: All randomized participants with Baseline Platelet Count of \<15×10\^9/L.

Outcome measures

Outcome measures
Measure	Efgartigimod PH20 SC n=59 Participants Participants receiving efgartigimod PH20 SC treatment.	Placebo PH20 SC n=35 Participants Participants receiving placebo PH20 SC treatment.
Extent of Disease Control Over the 24-Week Treatment Period in the Overall Population for Participants With Baseline Platelet Count of <15×10^9/L	1.0 weeks Interval 0.0 to 23.0	0.0 weeks Interval 0.0 to 24.0

SECONDARY outcome

Timeframe: Up to 24 weeks

Population: FAS: All randomized participants.

Assessed using the WHO bleeding scale. The WHO bleeding scale is a five-point scale where Grade 0 = no bleeding; Grade 1 = petechial bleeding; Grade 2 = mild blood loss; Grade 3 = gross blood loss (requires transfusion); and Grade 4 = debilitating blood loss, associated with fatality.

Outcome measures

Outcome measures
Measure	Efgartigimod PH20 SC n=137 Participants Participants receiving efgartigimod PH20 SC treatment.	Placebo PH20 SC n=70 Participants Participants receiving placebo PH20 SC treatment.
Number of the World Health Organization (WHO)-Classified Bleeding Events (Grade ≥1) in the Overall Population	9.0 bleeding events Interval 0.0 to 24.0	10.0 bleeding events Interval 0.0 to 24.0

SECONDARY outcome

Timeframe: Up to 24 weeks

Population: FAS: All randomized participants.

IWG complete response was defined as platelet counts of ≥100 × 10\^9/L and the absence of bleeding events (WHO Grading = 0 \[no bleeding\]) for at least 2 separate, consecutive analysis visits at least 7 days apart. IWG response was defined as platelet counts of ≥30 × 10\^9/L and a 2-fold increase of platelet count from Baseline and the absence of bleeding events (WHO grading = 0) for at least 2 separate, consecutive analysis visits that were at least 7 days apart. Initial response was defined as platelet counts of ≥30 × 10\^9/L and a 2-fold increase from the Baseline platelet count at analysis visit 5.

Outcome measures

Outcome measures
Measure	Efgartigimod PH20 SC n=137 Participants Participants receiving efgartigimod PH20 SC treatment.	Placebo PH20 SC n=70 Participants Participants receiving placebo PH20 SC treatment.
Percentage of Participants With a Platelet Count International Working Group (IWG) Response IWG Complete Response	10.2 percentage of participants	11.4 percentage of participants
Percentage of Participants With a Platelet Count International Working Group (IWG) Response IWG Response	28.5 percentage of participants	20.0 percentage of participants
Percentage of Participants With a Platelet Count International Working Group (IWG) Response Initial Response	19.7 percentage of participants	17.1 percentage of participants

SECONDARY outcome

Timeframe: Up to 24 weeks

Population: FAS: All randomized participants.

Rescue therapy was defined as an occurrence where the participant needed treatment with 1 or more rescue treatments. An occurrence was defined as a period of maximum 5 days where 1 or more rescue treatments were administered simultaneously or consecutively to the trial participant. The following rescue treatments were permitted: methylprednisolone, dexamethasone, prednisone, normal immunoglobulins, anti-D (Rho) immunoglobins, or platelet transfusions.

Outcome measures

Outcome measures
Measure	Efgartigimod PH20 SC n=137 Participants Participants receiving efgartigimod PH20 SC treatment.	Placebo PH20 SC n=70 Participants Participants receiving placebo PH20 SC treatment.
Rate of Receipt of Rescue Therapy (Rescue Per Participant Per Month) in the Overall Population	0.25 rescues per participant per month Standard Deviation 0.553	0.17 rescues per participant per month Standard Deviation 0.363

SECONDARY outcome

Timeframe: Up to 13 weeks (between Weeks 12 and 24)

Population: FAS: All randomized participants.

A change in ITP therapy was defined as either an increase in the dose and/or frequency of a concurrent ITP therapy relative to Baseline or the initiation of a new concurrent ITP therapy.

Outcome measures

Outcome measures
Measure	Efgartigimod PH20 SC n=137 Participants Participants receiving efgartigimod PH20 SC treatment.	Placebo PH20 SC n=70 Participants Participants receiving placebo PH20 SC treatment.
Percentage of Participants for Whom Dose and/or Frequency of Concurrent ITP Therapies Have Increased at Week 12 or Later in the Overall Population	11.7 percentage of participants	15.7 percentage of participants

SECONDARY outcome

Timeframe: Baseline and Week 24

Population: FAS: All randomized participants.

The FACIT-fatigue scale is a short, 13-item, easy-to-administer tool that measures an individual's level of fatigue during his/her usual daily activities over the past week. The level of fatigue was measured by recording item responses on a 5-point Likert scale ranging from 0 "not at all" to 4 "very much". All items were summed to create a single fatigue score with a range from 0 to 52, where a higher FACIT-F score indicated more severe symptoms. A negative change score from Baseline indicated improvement in quality of life (QoL).

Outcome measures

Outcome measures
Measure	Efgartigimod PH20 SC n=137 Participants Participants receiving efgartigimod PH20 SC treatment.	Placebo PH20 SC n=70 Participants Participants receiving placebo PH20 SC treatment.
Change From Baseline in Functional Assessment of Chronic Illness Therapy Fatigue Scale (FACIT-Fatigue) at Week 24 in the Overall Population	-0.025 score on a scale Standard Error 0.712	0.741 score on a scale Standard Error 0.985

SECONDARY outcome

Timeframe: Baseline and Weeks 4, 8, 12, 16, 20, and 24

Population: FAS: All randomized participants.

The FACT-Th6 uses a 5-level Likert scale (0=not at all to 4=very much), with participants rating their degree of concern in the past 7 days. The 6 selected items pertain to ability to do usual activities, worry about problems with bleeding or bruising, worry about the possibility of serious bleeding, avoidance of physical or social activity because of concern with bleeding or bruising and frustration due to the inability to carry out usual activities. All items were summed to create a single score with a range from 0 to 24, where a higher score indicated less severe symptoms. A positive change score from Baseline indicated improvement in QoL.

Outcome measures

Outcome measures
Measure	Efgartigimod PH20 SC n=137 Participants Participants receiving efgartigimod PH20 SC treatment.	Placebo PH20 SC n=70 Participants Participants receiving placebo PH20 SC treatment.
Change From Baseline in Functional Assessment of Cancer Therapy Questionnaire-Th6 (Fact-Th6) at Week 24 in the Overall Population	0.225 score on a scale Standard Error 0.360	0.359 score on a scale Standard Error 0.498

SECONDARY outcome

Timeframe: Baseline and Week 24

Population: FAS: All randomized participants.

The SF-36 is a 36-item scale constructed to survey health-related QoL on 8 domains: limitations in physical activities due to health problems; limitations in social activities due to physical or emotional problems; limitations in usual role activities due to physical health problems; bodily pain; general mental health (psychological distress and well-being); limitations in usual role activities due to emotional problems; vitality (energy and fatigue); and general health perceptions. The scores from the 8 domains were evaluated independently and aggregated into 2 norm-based summary component measures of physical and mental health. The summary component scores could range from 0 to 100, where a higher score indicated improvement in QoL. A positive change score from Baseline indicated improvement in QoL.

Outcome measures

Outcome measures
Measure	Efgartigimod PH20 SC n=137 Participants Participants receiving efgartigimod PH20 SC treatment.	Placebo PH20 SC n=70 Participants Participants receiving placebo PH20 SC treatment.
Change From Baseline in Short Form-36 (SF-36) at Week 24 in the Overall Population Week 24: Mental Component	1.215 score on a scale Standard Error 0.620	0.957 score on a scale Standard Error 0.841
Change From Baseline in Short Form-36 (SF-36) at Week 24 in the Overall Population Week 24: Physical Component	-0.848 score on a scale Standard Error 0.525	-0.850 score on a scale Standard Error 0.712

SECONDARY outcome

Timeframe: Up to 35 weeks

Population: SAF: All participants who received at least 1 dose or part of a dose including only antibody-evaluable participants.

Anti-drug antibody (ADA) incidence was defined as the percentage of participants with treatment-induced or treatment boosted ADA (denominator: number of evaluable participants). ADA prevalence was defined as the percentage of participants with treatment-unaffected ADA, treatment-induced ADA or treatment-boosted ADA (denominator: number of evaluable participants).

Outcome measures

Outcome measures
Measure	Efgartigimod PH20 SC n=136 Participants Participants receiving efgartigimod PH20 SC treatment.	Placebo PH20 SC n=70 Participants Participants receiving placebo PH20 SC treatment.
Incidence and Prevalence of Antibodies to Efgartigimod and/or rHuPH20 in the Overall Population Incidence of Antibodies to Efgartigimod	5.1 percentage of participants	2.9 percentage of participants
Incidence and Prevalence of Antibodies to Efgartigimod and/or rHuPH20 in the Overall Population Prevalence of Antibodies to Efgartigimod	16.2 percentage of participants	7.1 percentage of participants
Incidence and Prevalence of Antibodies to Efgartigimod and/or rHuPH20 in the Overall Population Incidence of Antibodies to rHuPH20	41.2 percentage of participants	20.0 percentage of participants
Incidence and Prevalence of Antibodies to Efgartigimod and/or rHuPH20 in the Overall Population Prevalence of Antibodies to rHuPH20	50.0 percentage of participants	31.4 percentage of participants

SECONDARY outcome

Timeframe: Weeks 3, 7, 11, 15, 19, 23, 24, SEFU1 (up to Week 29), and SEFU2 (up to Week 33)

Population: SAF: All participants who received at least 1 dose or part of a dose with available data.

A titer was determined in the samples with a positive assay response.

Outcome measures

Outcome measures
Measure	Efgartigimod PH20 SC n=45 Participants Participants receiving efgartigimod PH20 SC treatment.	Placebo PH20 SC n=16 Participants Participants receiving placebo PH20 SC treatment.
Titers of Antibodies to Efgartigimod and/or rHuPH20 in the Overall Population Week 7: ADA Against Efgartigimod Titer	1.0 titer Standard Deviation NA Standard deviation could not be calculated as a single participant was analyzed.	11.3 titer Standard Deviation 10.33
Titers of Antibodies to Efgartigimod and/or rHuPH20 in the Overall Population Week 11: ADA Against Efgartigimod Titer	—	6.3 titer Standard Deviation 4.84
Titers of Antibodies to Efgartigimod and/or rHuPH20 in the Overall Population Week 15: ADA Against Efgartigimod Titer	—	8.5 titer Standard Deviation 7.50
Titers of Antibodies to Efgartigimod and/or rHuPH20 in the Overall Population Week 15: ADA Against rHuPH20 Titer	3209.3 titer Standard Deviation 1325.96	15.7 titer Standard Deviation 2.02
Titers of Antibodies to Efgartigimod and/or rHuPH20 in the Overall Population Week 19: ADA Against rHuPH20 Titer	3742.7 titer Standard Deviation 1414.15	49.1 titer Standard Deviation 27.87
Titers of Antibodies to Efgartigimod and/or rHuPH20 in the Overall Population Week 3: ADA Against Efgartigimod Titer	—	16.5 titer Standard Deviation 15.50
Titers of Antibodies to Efgartigimod and/or rHuPH20 in the Overall Population Week 23: ADA Against Efgartigimod Titer	—	2.0 titer Standard Deviation NA Standard deviation could not be calculated as a single participant was analyzed.
Titers of Antibodies to Efgartigimod and/or rHuPH20 in the Overall Population Week 24: ADA Against Efgartigimod Titer	16.3 titer Standard Deviation 8.95	1.0 titer Standard Deviation 0.00
Titers of Antibodies to Efgartigimod and/or rHuPH20 in the Overall Population SEFU1: ADA Against Efgartigimod Titer	2.5 titer Standard Deviation 1.50	—
Titers of Antibodies to Efgartigimod and/or rHuPH20 in the Overall Population SEFU2: ADA Against Efgartigimod Titer	17.0 titer Standard Deviation 15.00	—
Titers of Antibodies to Efgartigimod and/or rHuPH20 in the Overall Population Week 3: ADA Against rHuPH20 Titer	12.7 titer Standard Deviation 2.53	14.1 titer Standard Deviation 3.15
Titers of Antibodies to Efgartigimod and/or rHuPH20 in the Overall Population Week 7: ADA Against rHuPH20 Titer	645.9 titer Standard Deviation 314.79	14.4 titer Standard Deviation 4.27
Titers of Antibodies to Efgartigimod and/or rHuPH20 in the Overall Population Week 11: ADA Against rHuPH20 Titer	2204.8 titer Standard Deviation 1297.43	11.4 titer Standard Deviation 2.37
Titers of Antibodies to Efgartigimod and/or rHuPH20 in the Overall Population Week 23: ADA Against rHuPH20 Titer	5936.6 titer Standard Deviation 2180.33	58.3 titer Standard Deviation 27.83
Titers of Antibodies to Efgartigimod and/or rHuPH20 in the Overall Population Week 24: ADA Against rHuPH20 Titer	4731.3 titer Standard Deviation 1898.96	69.1 titer Standard Deviation 26.60
Titers of Antibodies to Efgartigimod and/or rHuPH20 in the Overall Population SEFU1: ADA Against rHuPH20 Titer	5122.5 titer Standard Deviation 5117.50	—
Titers of Antibodies to Efgartigimod and/or rHuPH20 in the Overall Population SEFU2: ADA Against rHuPH20 Titer	5160.0 titer Standard Deviation 5080.00	—

SECONDARY outcome

Timeframe: Up to 35 weeks

Population: SAF: All participants who received at least 1 dose or part of a dose with available data.

Samples were tested for the presence of NAb against efgartigimod and/or rHuPH20 and titers for NAb against rHuPH20. NAb incidence is defined as the total percentage of participants with participant classification "baseline negative-postbaseline positive" and "baseline positive-postbaseline positive". NAb prevalence is defined as the total percentage of participants with participant classification "baseline negative-postbaseline positive," "baseline positive-postbaseline positive," or "baseline positive-postbaseline negative".

Outcome measures

Outcome measures
Measure	Efgartigimod PH20 SC n=136 Participants Participants receiving efgartigimod PH20 SC treatment.	Placebo PH20 SC n=70 Participants Participants receiving placebo PH20 SC treatment.
Incidence and Prevalence of Neutralizing Antibodies (NAb) to Efgartigimod and/or rHuPH20 in the Overall Population Incidence of NAb to Efgartigimod	0.7 percentage of participants	0.0 percentage of participants
Incidence and Prevalence of Neutralizing Antibodies (NAb) to Efgartigimod and/or rHuPH20 in the Overall Population Prevalence of NAb to Efgartigimod	5.1 percentage of participants	0.0 percentage of participants
Incidence and Prevalence of Neutralizing Antibodies (NAb) to Efgartigimod and/or rHuPH20 in the Overall Population Incidence of NAb to rHuPH20	0.0 percentage of participants	0.0 percentage of participants
Incidence and Prevalence of Neutralizing Antibodies (NAb) to Efgartigimod and/or rHuPH20 in the Overall Population Prevalence of NAb to rHuPH20	0.0 percentage of participants	0.0 percentage of participants

SECONDARY outcome

Timeframe: Predose on Weeks 1, 2, 3, 17, 19, 21, 23, and 24

Population: PK Analysis Set: Safety analysis set excluding placebo participants and including participants with at least one serum post dose PK measurement.

All pharmacokinetic (PK) samples were collected predose, on the day of IMP administration.

Outcome measures

Outcome measures
Measure	Efgartigimod PH20 SC n=135 Participants Participants receiving efgartigimod PH20 SC treatment.	Placebo PH20 SC Participants receiving placebo PH20 SC treatment.
Serum Efgartigimod Trough Concentration (Ctrough) in the Overall Population Week 1	13.2 μg/mL Standard Deviation 6.46	—
Serum Efgartigimod Trough Concentration (Ctrough) in the Overall Population Week 2	16.6 μg/mL Standard Deviation 8.17	—
Serum Efgartigimod Trough Concentration (Ctrough) in the Overall Population Week 3	17.0 μg/mL Standard Deviation 7.95	—
Serum Efgartigimod Trough Concentration (Ctrough) in the Overall Population Week 17	15.1 μg/mL Standard Deviation 8.54	—
Serum Efgartigimod Trough Concentration (Ctrough) in the Overall Population Week 19	15.4 μg/mL Standard Deviation 7.43	—
Serum Efgartigimod Trough Concentration (Ctrough) in the Overall Population Week 21	15.1 μg/mL Standard Deviation 8.86	—
Serum Efgartigimod Trough Concentration (Ctrough) in the Overall Population Week 23	14.9 μg/mL Standard Deviation 7.79	—
Serum Efgartigimod Trough Concentration (Ctrough) in the Overall Population Week 24	14.7 μg/mL Standard Deviation 8.10	—

SECONDARY outcome

Timeframe: Baseline and Weeks 1, 2, 3, 17, 19, 21, 23, and 24

Population: Pharmacodynamic (PD) Analysis Set: Safety analysis set including participants with at least one serum post dose PD measurement.

Samples were collected predose, on the day of IMP administration.

Outcome measures

Outcome measures
Measure	Efgartigimod PH20 SC n=133 Participants Participants receiving efgartigimod PH20 SC treatment.	Placebo PH20 SC n=67 Participants Participants receiving placebo PH20 SC treatment.
Percentage Change From Baseline in Total IgG in the Overall Population Week 1	-33.64 percentage of total IgG Standard Deviation 26.018	0.60 percentage of total IgG Standard Deviation 14.065
Percentage Change From Baseline in Total IgG in the Overall Population Week 2	-50.68 percentage of total IgG Standard Deviation 34.973	10.31 percentage of total IgG Standard Deviation 44.858
Percentage Change From Baseline in Total IgG in the Overall Population Week 3	-56.80 percentage of total IgG Standard Deviation 39.133	4.97 percentage of total IgG Standard Deviation 28.484
Percentage Change From Baseline in Total IgG in the Overall Population Week 17	-64.76 percentage of total IgG Standard Deviation 11.478	1.27 percentage of total IgG Standard Deviation 16.749
Percentage Change From Baseline in Total IgG in the Overall Population Week 19	-60.98 percentage of total IgG Standard Deviation 26.460	9.28 percentage of total IgG Standard Deviation 44.989
Percentage Change From Baseline in Total IgG in the Overall Population Week 21	-62.38 percentage of total IgG Standard Deviation 15.176	4.62 percentage of total IgG Standard Deviation 25.406
Percentage Change From Baseline in Total IgG in the Overall Population Week 23	-62.45 percentage of total IgG Standard Deviation 20.286	2.45 percentage of total IgG Standard Deviation 21.271
Percentage Change From Baseline in Total IgG in the Overall Population Week 24	-61.97 percentage of total IgG Standard Deviation 18.200	-0.36 percentage of total IgG Standard Deviation 17.408

SECONDARY outcome

Timeframe: Weeks 7, 15, 23, and 24

Population: PD Analysis Set: Safety analysis set including participants with at least one serum post dose PD measurement and tested positive for antiplatelet antibodies at Baseline.

The antiplatelet antibody was positive if optical density value \>0.129.

Outcome measures

Outcome measures
Measure	Efgartigimod PH20 SC n=36 Participants Participants receiving efgartigimod PH20 SC treatment.	Placebo PH20 SC n=16 Participants Participants receiving placebo PH20 SC treatment.
Number of Participants With Antiplatelet Antibodies in the Overall Population Week 15	18 Participants	10 Participants
Number of Participants With Antiplatelet Antibodies in the Overall Population Week 23	17 Participants	9 Participants
Number of Participants With Antiplatelet Antibodies in the Overall Population Week 24	19 Participants	8 Participants
Number of Participants With Antiplatelet Antibodies in the Overall Population Week 7	20 Participants	10 Participants

Adverse Events

Efgartigimod PH20 SC

Serious events: 14 serious events

Other events: 130 other events

Deaths: 1 deaths

Placebo PH20 SC

Serious events: 10 serious events

Other events: 65 other events

Deaths: 0 deaths

Serious adverse events

Other adverse events

Additional Information

Regulatory manager

Argenx

Phone: Please email

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place

Measure	Efgartigimod PH20 SC n=137 participants at risk Participants receiving efgartigimod PH20 SC treatment.	Placebo PH20 SC n=70 participants at risk Participants receiving placebo PH20 SC treatment.
Blood and lymphatic system disorders Immune thrombocytopenia	0.73% 1/137 • Up to 35 weeks The total number of participants affected by other (not including serious) adverse events (AEs) include participants who experienced other AEs under the frequency threshold of 5%. SAF: All participants who received at least 1 dose or part of a dose.	0.00% 0/70 • Up to 35 weeks The total number of participants affected by other (not including serious) adverse events (AEs) include participants who experienced other AEs under the frequency threshold of 5%. SAF: All participants who received at least 1 dose or part of a dose.
Blood and lymphatic system disorders Thrombocytopenia	0.73% 1/137 • Up to 35 weeks The total number of participants affected by other (not including serious) adverse events (AEs) include participants who experienced other AEs under the frequency threshold of 5%. SAF: All participants who received at least 1 dose or part of a dose.	2.9% 2/70 • Up to 35 weeks The total number of participants affected by other (not including serious) adverse events (AEs) include participants who experienced other AEs under the frequency threshold of 5%. SAF: All participants who received at least 1 dose or part of a dose.
Endocrine disorders Autoimmune thyroiditis	0.00% 0/137 • Up to 35 weeks The total number of participants affected by other (not including serious) adverse events (AEs) include participants who experienced other AEs under the frequency threshold of 5%. SAF: All participants who received at least 1 dose or part of a dose.	1.4% 1/70 • Up to 35 weeks The total number of participants affected by other (not including serious) adverse events (AEs) include participants who experienced other AEs under the frequency threshold of 5%. SAF: All participants who received at least 1 dose or part of a dose.
Eye disorders Amaurosis fugax	0.73% 1/137 • Up to 35 weeks The total number of participants affected by other (not including serious) adverse events (AEs) include participants who experienced other AEs under the frequency threshold of 5%. SAF: All participants who received at least 1 dose or part of a dose.	0.00% 0/70 • Up to 35 weeks The total number of participants affected by other (not including serious) adverse events (AEs) include participants who experienced other AEs under the frequency threshold of 5%. SAF: All participants who received at least 1 dose or part of a dose.
Eye disorders Vitreous haemorrhage	0.73% 1/137 • Up to 35 weeks The total number of participants affected by other (not including serious) adverse events (AEs) include participants who experienced other AEs under the frequency threshold of 5%. SAF: All participants who received at least 1 dose or part of a dose.	0.00% 0/70 • Up to 35 weeks The total number of participants affected by other (not including serious) adverse events (AEs) include participants who experienced other AEs under the frequency threshold of 5%. SAF: All participants who received at least 1 dose or part of a dose.
Gastrointestinal disorders Abdominal distension	0.73% 1/137 • Up to 35 weeks The total number of participants affected by other (not including serious) adverse events (AEs) include participants who experienced other AEs under the frequency threshold of 5%. SAF: All participants who received at least 1 dose or part of a dose.	0.00% 0/70 • Up to 35 weeks The total number of participants affected by other (not including serious) adverse events (AEs) include participants who experienced other AEs under the frequency threshold of 5%. SAF: All participants who received at least 1 dose or part of a dose.
Gastrointestinal disorders Haematochezia	0.73% 1/137 • Up to 35 weeks The total number of participants affected by other (not including serious) adverse events (AEs) include participants who experienced other AEs under the frequency threshold of 5%. SAF: All participants who received at least 1 dose or part of a dose.	0.00% 0/70 • Up to 35 weeks The total number of participants affected by other (not including serious) adverse events (AEs) include participants who experienced other AEs under the frequency threshold of 5%. SAF: All participants who received at least 1 dose or part of a dose.
Gastrointestinal disorders Inguinal hernia strangulated	0.00% 0/137 • Up to 35 weeks The total number of participants affected by other (not including serious) adverse events (AEs) include participants who experienced other AEs under the frequency threshold of 5%. SAF: All participants who received at least 1 dose or part of a dose.	1.4% 1/70 • Up to 35 weeks The total number of participants affected by other (not including serious) adverse events (AEs) include participants who experienced other AEs under the frequency threshold of 5%. SAF: All participants who received at least 1 dose or part of a dose.
Gastrointestinal disorders Melaena	0.73% 1/137 • Up to 35 weeks The total number of participants affected by other (not including serious) adverse events (AEs) include participants who experienced other AEs under the frequency threshold of 5%. SAF: All participants who received at least 1 dose or part of a dose.	0.00% 0/70 • Up to 35 weeks The total number of participants affected by other (not including serious) adverse events (AEs) include participants who experienced other AEs under the frequency threshold of 5%. SAF: All participants who received at least 1 dose or part of a dose.
Hepatobiliary disorders Cholecystitis acute	0.73% 1/137 • Up to 35 weeks The total number of participants affected by other (not including serious) adverse events (AEs) include participants who experienced other AEs under the frequency threshold of 5%. SAF: All participants who received at least 1 dose or part of a dose.	0.00% 0/70 • Up to 35 weeks The total number of participants affected by other (not including serious) adverse events (AEs) include participants who experienced other AEs under the frequency threshold of 5%. SAF: All participants who received at least 1 dose or part of a dose.
Infections and infestations Arthritis infective	0.00% 0/137 • Up to 35 weeks The total number of participants affected by other (not including serious) adverse events (AEs) include participants who experienced other AEs under the frequency threshold of 5%. SAF: All participants who received at least 1 dose or part of a dose.	1.4% 1/70 • Up to 35 weeks The total number of participants affected by other (not including serious) adverse events (AEs) include participants who experienced other AEs under the frequency threshold of 5%. SAF: All participants who received at least 1 dose or part of a dose.
Infections and infestations COVID-19	2.2% 3/137 • Up to 35 weeks The total number of participants affected by other (not including serious) adverse events (AEs) include participants who experienced other AEs under the frequency threshold of 5%. SAF: All participants who received at least 1 dose or part of a dose.	0.00% 0/70 • Up to 35 weeks The total number of participants affected by other (not including serious) adverse events (AEs) include participants who experienced other AEs under the frequency threshold of 5%. SAF: All participants who received at least 1 dose or part of a dose.
Infections and infestations COVID-19 pneumonia	0.73% 1/137 • Up to 35 weeks The total number of participants affected by other (not including serious) adverse events (AEs) include participants who experienced other AEs under the frequency threshold of 5%. SAF: All participants who received at least 1 dose or part of a dose.	0.00% 0/70 • Up to 35 weeks The total number of participants affected by other (not including serious) adverse events (AEs) include participants who experienced other AEs under the frequency threshold of 5%. SAF: All participants who received at least 1 dose or part of a dose.
Infections and infestations Meningitis aseptic	0.73% 1/137 • Up to 35 weeks The total number of participants affected by other (not including serious) adverse events (AEs) include participants who experienced other AEs under the frequency threshold of 5%. SAF: All participants who received at least 1 dose or part of a dose.	0.00% 0/70 • Up to 35 weeks The total number of participants affected by other (not including serious) adverse events (AEs) include participants who experienced other AEs under the frequency threshold of 5%. SAF: All participants who received at least 1 dose or part of a dose.
Infections and infestations Urosepsis	0.73% 1/137 • Up to 35 weeks The total number of participants affected by other (not including serious) adverse events (AEs) include participants who experienced other AEs under the frequency threshold of 5%. SAF: All participants who received at least 1 dose or part of a dose.	0.00% 0/70 • Up to 35 weeks The total number of participants affected by other (not including serious) adverse events (AEs) include participants who experienced other AEs under the frequency threshold of 5%. SAF: All participants who received at least 1 dose or part of a dose.
Injury, poisoning and procedural complications Head injury	0.00% 0/137 • Up to 35 weeks The total number of participants affected by other (not including serious) adverse events (AEs) include participants who experienced other AEs under the frequency threshold of 5%. SAF: All participants who received at least 1 dose or part of a dose.	1.4% 1/70 • Up to 35 weeks The total number of participants affected by other (not including serious) adverse events (AEs) include participants who experienced other AEs under the frequency threshold of 5%. SAF: All participants who received at least 1 dose or part of a dose.
Investigations Haemoglobin decreased	0.73% 1/137 • Up to 35 weeks The total number of participants affected by other (not including serious) adverse events (AEs) include participants who experienced other AEs under the frequency threshold of 5%. SAF: All participants who received at least 1 dose or part of a dose.	0.00% 0/70 • Up to 35 weeks The total number of participants affected by other (not including serious) adverse events (AEs) include participants who experienced other AEs under the frequency threshold of 5%. SAF: All participants who received at least 1 dose or part of a dose.
Investigations Platelet count decreased	0.00% 0/137 • Up to 35 weeks The total number of participants affected by other (not including serious) adverse events (AEs) include participants who experienced other AEs under the frequency threshold of 5%. SAF: All participants who received at least 1 dose or part of a dose.	1.4% 1/70 • Up to 35 weeks The total number of participants affected by other (not including serious) adverse events (AEs) include participants who experienced other AEs under the frequency threshold of 5%. SAF: All participants who received at least 1 dose or part of a dose.
Musculoskeletal and connective tissue disorders Osteonecrosis	0.00% 0/137 • Up to 35 weeks The total number of participants affected by other (not including serious) adverse events (AEs) include participants who experienced other AEs under the frequency threshold of 5%. SAF: All participants who received at least 1 dose or part of a dose.	1.4% 1/70 • Up to 35 weeks The total number of participants affected by other (not including serious) adverse events (AEs) include participants who experienced other AEs under the frequency threshold of 5%. SAF: All participants who received at least 1 dose or part of a dose.
Nervous system disorders Cerebral infarction	0.73% 1/137 • Up to 35 weeks The total number of participants affected by other (not including serious) adverse events (AEs) include participants who experienced other AEs under the frequency threshold of 5%. SAF: All participants who received at least 1 dose or part of a dose.	0.00% 0/70 • Up to 35 weeks The total number of participants affected by other (not including serious) adverse events (AEs) include participants who experienced other AEs under the frequency threshold of 5%. SAF: All participants who received at least 1 dose or part of a dose.
Nervous system disorders Headache	1.5% 2/137 • Up to 35 weeks The total number of participants affected by other (not including serious) adverse events (AEs) include participants who experienced other AEs under the frequency threshold of 5%. SAF: All participants who received at least 1 dose or part of a dose.	0.00% 0/70 • Up to 35 weeks The total number of participants affected by other (not including serious) adverse events (AEs) include participants who experienced other AEs under the frequency threshold of 5%. SAF: All participants who received at least 1 dose or part of a dose.
Renal and urinary disorders Haematuria	0.00% 0/137 • Up to 35 weeks The total number of participants affected by other (not including serious) adverse events (AEs) include participants who experienced other AEs under the frequency threshold of 5%. SAF: All participants who received at least 1 dose or part of a dose.	2.9% 2/70 • Up to 35 weeks The total number of participants affected by other (not including serious) adverse events (AEs) include participants who experienced other AEs under the frequency threshold of 5%. SAF: All participants who received at least 1 dose or part of a dose.
Reproductive system and breast disorders Heavy menstrual bleeding	0.73% 1/137 • Up to 35 weeks The total number of participants affected by other (not including serious) adverse events (AEs) include participants who experienced other AEs under the frequency threshold of 5%. SAF: All participants who received at least 1 dose or part of a dose.	0.00% 0/70 • Up to 35 weeks The total number of participants affected by other (not including serious) adverse events (AEs) include participants who experienced other AEs under the frequency threshold of 5%. SAF: All participants who received at least 1 dose or part of a dose.
Reproductive system and breast disorders Intermenstrual bleeding	0.73% 1/137 • Up to 35 weeks The total number of participants affected by other (not including serious) adverse events (AEs) include participants who experienced other AEs under the frequency threshold of 5%. SAF: All participants who received at least 1 dose or part of a dose.	0.00% 0/70 • Up to 35 weeks The total number of participants affected by other (not including serious) adverse events (AEs) include participants who experienced other AEs under the frequency threshold of 5%. SAF: All participants who received at least 1 dose or part of a dose.
Respiratory, thoracic and mediastinal disorders Epistaxis	0.00% 0/137 • Up to 35 weeks The total number of participants affected by other (not including serious) adverse events (AEs) include participants who experienced other AEs under the frequency threshold of 5%. SAF: All participants who received at least 1 dose or part of a dose.	1.4% 1/70 • Up to 35 weeks The total number of participants affected by other (not including serious) adverse events (AEs) include participants who experienced other AEs under the frequency threshold of 5%. SAF: All participants who received at least 1 dose or part of a dose.

Measure	Efgartigimod PH20 SC n=137 participants at risk Participants receiving efgartigimod PH20 SC treatment.	Placebo PH20 SC n=70 participants at risk Participants receiving placebo PH20 SC treatment.
Blood and lymphatic system disorders Anaemia	7.3% 10/137 • Up to 35 weeks The total number of participants affected by other (not including serious) adverse events (AEs) include participants who experienced other AEs under the frequency threshold of 5%. SAF: All participants who received at least 1 dose or part of a dose.	8.6% 6/70 • Up to 35 weeks The total number of participants affected by other (not including serious) adverse events (AEs) include participants who experienced other AEs under the frequency threshold of 5%. SAF: All participants who received at least 1 dose or part of a dose.
Gastrointestinal disorders Diarrhoea	5.1% 7/137 • Up to 35 weeks The total number of participants affected by other (not including serious) adverse events (AEs) include participants who experienced other AEs under the frequency threshold of 5%. SAF: All participants who received at least 1 dose or part of a dose.	1.4% 1/70 • Up to 35 weeks The total number of participants affected by other (not including serious) adverse events (AEs) include participants who experienced other AEs under the frequency threshold of 5%. SAF: All participants who received at least 1 dose or part of a dose.
Gastrointestinal disorders Dyspepsia	2.2% 3/137 • Up to 35 weeks The total number of participants affected by other (not including serious) adverse events (AEs) include participants who experienced other AEs under the frequency threshold of 5%. SAF: All participants who received at least 1 dose or part of a dose.	7.1% 5/70 • Up to 35 weeks The total number of participants affected by other (not including serious) adverse events (AEs) include participants who experienced other AEs under the frequency threshold of 5%. SAF: All participants who received at least 1 dose or part of a dose.
Gastrointestinal disorders Gingival bleeding	10.2% 14/137 • Up to 35 weeks The total number of participants affected by other (not including serious) adverse events (AEs) include participants who experienced other AEs under the frequency threshold of 5%. SAF: All participants who received at least 1 dose or part of a dose.	14.3% 10/70 • Up to 35 weeks The total number of participants affected by other (not including serious) adverse events (AEs) include participants who experienced other AEs under the frequency threshold of 5%. SAF: All participants who received at least 1 dose or part of a dose.
Gastrointestinal disorders Mouth haemorrhage	13.9% 19/137 • Up to 35 weeks The total number of participants affected by other (not including serious) adverse events (AEs) include participants who experienced other AEs under the frequency threshold of 5%. SAF: All participants who received at least 1 dose or part of a dose.	8.6% 6/70 • Up to 35 weeks The total number of participants affected by other (not including serious) adverse events (AEs) include participants who experienced other AEs under the frequency threshold of 5%. SAF: All participants who received at least 1 dose or part of a dose.
Gastrointestinal disorders Oral blood blister	7.3% 10/137 • Up to 35 weeks The total number of participants affected by other (not including serious) adverse events (AEs) include participants who experienced other AEs under the frequency threshold of 5%. SAF: All participants who received at least 1 dose or part of a dose.	1.4% 1/70 • Up to 35 weeks The total number of participants affected by other (not including serious) adverse events (AEs) include participants who experienced other AEs under the frequency threshold of 5%. SAF: All participants who received at least 1 dose or part of a dose.
General disorders Fatigue	5.8% 8/137 • Up to 35 weeks The total number of participants affected by other (not including serious) adverse events (AEs) include participants who experienced other AEs under the frequency threshold of 5%. SAF: All participants who received at least 1 dose or part of a dose.	0.00% 0/70 • Up to 35 weeks The total number of participants affected by other (not including serious) adverse events (AEs) include participants who experienced other AEs under the frequency threshold of 5%. SAF: All participants who received at least 1 dose or part of a dose.
General disorders Injection site bruising	9.5% 13/137 • Up to 35 weeks The total number of participants affected by other (not including serious) adverse events (AEs) include participants who experienced other AEs under the frequency threshold of 5%. SAF: All participants who received at least 1 dose or part of a dose.	2.9% 2/70 • Up to 35 weeks The total number of participants affected by other (not including serious) adverse events (AEs) include participants who experienced other AEs under the frequency threshold of 5%. SAF: All participants who received at least 1 dose or part of a dose.
General disorders Injection site erythema	6.6% 9/137 • Up to 35 weeks The total number of participants affected by other (not including serious) adverse events (AEs) include participants who experienced other AEs under the frequency threshold of 5%. SAF: All participants who received at least 1 dose or part of a dose.	2.9% 2/70 • Up to 35 weeks The total number of participants affected by other (not including serious) adverse events (AEs) include participants who experienced other AEs under the frequency threshold of 5%. SAF: All participants who received at least 1 dose or part of a dose.
General disorders Injection site haemorrhage	10.2% 14/137 • Up to 35 weeks The total number of participants affected by other (not including serious) adverse events (AEs) include participants who experienced other AEs under the frequency threshold of 5%. SAF: All participants who received at least 1 dose or part of a dose.	20.0% 14/70 • Up to 35 weeks The total number of participants affected by other (not including serious) adverse events (AEs) include participants who experienced other AEs under the frequency threshold of 5%. SAF: All participants who received at least 1 dose or part of a dose.
General disorders Injection site pain	5.8% 8/137 • Up to 35 weeks The total number of participants affected by other (not including serious) adverse events (AEs) include participants who experienced other AEs under the frequency threshold of 5%. SAF: All participants who received at least 1 dose or part of a dose.	2.9% 2/70 • Up to 35 weeks The total number of participants affected by other (not including serious) adverse events (AEs) include participants who experienced other AEs under the frequency threshold of 5%. SAF: All participants who received at least 1 dose or part of a dose.
General disorders Injection site rash	5.1% 7/137 • Up to 35 weeks The total number of participants affected by other (not including serious) adverse events (AEs) include participants who experienced other AEs under the frequency threshold of 5%. SAF: All participants who received at least 1 dose or part of a dose.	1.4% 1/70 • Up to 35 weeks The total number of participants affected by other (not including serious) adverse events (AEs) include participants who experienced other AEs under the frequency threshold of 5%. SAF: All participants who received at least 1 dose or part of a dose.
General disorders Injection site reaction	7.3% 10/137 • Up to 35 weeks The total number of participants affected by other (not including serious) adverse events (AEs) include participants who experienced other AEs under the frequency threshold of 5%. SAF: All participants who received at least 1 dose or part of a dose.	1.4% 1/70 • Up to 35 weeks The total number of participants affected by other (not including serious) adverse events (AEs) include participants who experienced other AEs under the frequency threshold of 5%. SAF: All participants who received at least 1 dose or part of a dose.
General disorders Vessel puncture site haemorrhage	2.2% 3/137 • Up to 35 weeks The total number of participants affected by other (not including serious) adverse events (AEs) include participants who experienced other AEs under the frequency threshold of 5%. SAF: All participants who received at least 1 dose or part of a dose.	7.1% 5/70 • Up to 35 weeks The total number of participants affected by other (not including serious) adverse events (AEs) include participants who experienced other AEs under the frequency threshold of 5%. SAF: All participants who received at least 1 dose or part of a dose.
Infections and infestations COVID-19	13.1% 18/137 • Up to 35 weeks The total number of participants affected by other (not including serious) adverse events (AEs) include participants who experienced other AEs under the frequency threshold of 5%. SAF: All participants who received at least 1 dose or part of a dose.	8.6% 6/70 • Up to 35 weeks The total number of participants affected by other (not including serious) adverse events (AEs) include participants who experienced other AEs under the frequency threshold of 5%. SAF: All participants who received at least 1 dose or part of a dose.
Infections and infestations Upper respiratory tract infection	8.8% 12/137 • Up to 35 weeks The total number of participants affected by other (not including serious) adverse events (AEs) include participants who experienced other AEs under the frequency threshold of 5%. SAF: All participants who received at least 1 dose or part of a dose.	8.6% 6/70 • Up to 35 weeks The total number of participants affected by other (not including serious) adverse events (AEs) include participants who experienced other AEs under the frequency threshold of 5%. SAF: All participants who received at least 1 dose or part of a dose.
Infections and infestations Urinary tract infection	5.8% 8/137 • Up to 35 weeks The total number of participants affected by other (not including serious) adverse events (AEs) include participants who experienced other AEs under the frequency threshold of 5%. SAF: All participants who received at least 1 dose or part of a dose.	8.6% 6/70 • Up to 35 weeks The total number of participants affected by other (not including serious) adverse events (AEs) include participants who experienced other AEs under the frequency threshold of 5%. SAF: All participants who received at least 1 dose or part of a dose.
Injury, poisoning and procedural complications Contusion	16.1% 22/137 • Up to 35 weeks The total number of participants affected by other (not including serious) adverse events (AEs) include participants who experienced other AEs under the frequency threshold of 5%. SAF: All participants who received at least 1 dose or part of a dose.	17.1% 12/70 • Up to 35 weeks The total number of participants affected by other (not including serious) adverse events (AEs) include participants who experienced other AEs under the frequency threshold of 5%. SAF: All participants who received at least 1 dose or part of a dose.
Investigations Blood urine present	31.4% 43/137 • Up to 35 weeks The total number of participants affected by other (not including serious) adverse events (AEs) include participants who experienced other AEs under the frequency threshold of 5%. SAF: All participants who received at least 1 dose or part of a dose.	28.6% 20/70 • Up to 35 weeks The total number of participants affected by other (not including serious) adverse events (AEs) include participants who experienced other AEs under the frequency threshold of 5%. SAF: All participants who received at least 1 dose or part of a dose.
Investigations C-reactive protein increased	2.2% 3/137 • Up to 35 weeks The total number of participants affected by other (not including serious) adverse events (AEs) include participants who experienced other AEs under the frequency threshold of 5%. SAF: All participants who received at least 1 dose or part of a dose.	11.4% 8/70 • Up to 35 weeks The total number of participants affected by other (not including serious) adverse events (AEs) include participants who experienced other AEs under the frequency threshold of 5%. SAF: All participants who received at least 1 dose or part of a dose.
Metabolism and nutrition disorders Hyperuricaemia	5.1% 7/137 • Up to 35 weeks The total number of participants affected by other (not including serious) adverse events (AEs) include participants who experienced other AEs under the frequency threshold of 5%. SAF: All participants who received at least 1 dose or part of a dose.	4.3% 3/70 • Up to 35 weeks The total number of participants affected by other (not including serious) adverse events (AEs) include participants who experienced other AEs under the frequency threshold of 5%. SAF: All participants who received at least 1 dose or part of a dose.
Musculoskeletal and connective tissue disorders Arthralgia	2.2% 3/137 • Up to 35 weeks The total number of participants affected by other (not including serious) adverse events (AEs) include participants who experienced other AEs under the frequency threshold of 5%. SAF: All participants who received at least 1 dose or part of a dose.	5.7% 4/70 • Up to 35 weeks The total number of participants affected by other (not including serious) adverse events (AEs) include participants who experienced other AEs under the frequency threshold of 5%. SAF: All participants who received at least 1 dose or part of a dose.
Nervous system disorders Dizziness	5.8% 8/137 • Up to 35 weeks The total number of participants affected by other (not including serious) adverse events (AEs) include participants who experienced other AEs under the frequency threshold of 5%. SAF: All participants who received at least 1 dose or part of a dose.	4.3% 3/70 • Up to 35 weeks The total number of participants affected by other (not including serious) adverse events (AEs) include participants who experienced other AEs under the frequency threshold of 5%. SAF: All participants who received at least 1 dose or part of a dose.
Nervous system disorders Headache	11.7% 16/137 • Up to 35 weeks The total number of participants affected by other (not including serious) adverse events (AEs) include participants who experienced other AEs under the frequency threshold of 5%. SAF: All participants who received at least 1 dose or part of a dose.	10.0% 7/70 • Up to 35 weeks The total number of participants affected by other (not including serious) adverse events (AEs) include participants who experienced other AEs under the frequency threshold of 5%. SAF: All participants who received at least 1 dose or part of a dose.
Renal and urinary disorders Haematuria	23.4% 32/137 • Up to 35 weeks The total number of participants affected by other (not including serious) adverse events (AEs) include participants who experienced other AEs under the frequency threshold of 5%. SAF: All participants who received at least 1 dose or part of a dose.	32.9% 23/70 • Up to 35 weeks The total number of participants affected by other (not including serious) adverse events (AEs) include participants who experienced other AEs under the frequency threshold of 5%. SAF: All participants who received at least 1 dose or part of a dose.
Respiratory, thoracic and mediastinal disorders Epistaxis	14.6% 20/137 • Up to 35 weeks The total number of participants affected by other (not including serious) adverse events (AEs) include participants who experienced other AEs under the frequency threshold of 5%. SAF: All participants who received at least 1 dose or part of a dose.	21.4% 15/70 • Up to 35 weeks The total number of participants affected by other (not including serious) adverse events (AEs) include participants who experienced other AEs under the frequency threshold of 5%. SAF: All participants who received at least 1 dose or part of a dose.
Skin and subcutaneous tissue disorders Ecchymosis	24.8% 34/137 • Up to 35 weeks The total number of participants affected by other (not including serious) adverse events (AEs) include participants who experienced other AEs under the frequency threshold of 5%. SAF: All participants who received at least 1 dose or part of a dose.	28.6% 20/70 • Up to 35 weeks The total number of participants affected by other (not including serious) adverse events (AEs) include participants who experienced other AEs under the frequency threshold of 5%. SAF: All participants who received at least 1 dose or part of a dose.
Skin and subcutaneous tissue disorders Petechiae	24.8% 34/137 • Up to 35 weeks The total number of participants affected by other (not including serious) adverse events (AEs) include participants who experienced other AEs under the frequency threshold of 5%. SAF: All participants who received at least 1 dose or part of a dose.	27.1% 19/70 • Up to 35 weeks The total number of participants affected by other (not including serious) adverse events (AEs) include participants who experienced other AEs under the frequency threshold of 5%. SAF: All participants who received at least 1 dose or part of a dose.
Skin and subcutaneous tissue disorders Purpura	10.9% 15/137 • Up to 35 weeks The total number of participants affected by other (not including serious) adverse events (AEs) include participants who experienced other AEs under the frequency threshold of 5%. SAF: All participants who received at least 1 dose or part of a dose.	15.7% 11/70 • Up to 35 weeks The total number of participants affected by other (not including serious) adverse events (AEs) include participants who experienced other AEs under the frequency threshold of 5%. SAF: All participants who received at least 1 dose or part of a dose.
Vascular disorders Haematoma	5.1% 7/137 • Up to 35 weeks The total number of participants affected by other (not including serious) adverse events (AEs) include participants who experienced other AEs under the frequency threshold of 5%. SAF: All participants who received at least 1 dose or part of a dose.	4.3% 3/70 • Up to 35 weeks The total number of participants affected by other (not including serious) adverse events (AEs) include participants who experienced other AEs under the frequency threshold of 5%. SAF: All participants who received at least 1 dose or part of a dose.
Vascular disorders Hypertension	5.1% 7/137 • Up to 35 weeks The total number of participants affected by other (not including serious) adverse events (AEs) include participants who experienced other AEs under the frequency threshold of 5%. SAF: All participants who received at least 1 dose or part of a dose.	5.7% 4/70 • Up to 35 weeks The total number of participants affected by other (not including serious) adverse events (AEs) include participants who experienced other AEs under the frequency threshold of 5%. SAF: All participants who received at least 1 dose or part of a dose.