A Phase 2/3 Study of Efgartigimod PH20 SC in Adult Partic... | NCT05267600

NCT05267600

Status Module

Description Module

Label	Type	Description	Intervention Names
efgartigimod PH20 SC	Experimental	participants receiving efgartigimod PH20 SC on top of Prednisone	Biological: efgartigimod PH20 SC Drug: Prednisone
placebo PH20 SC	Placebo Comparator	participants receiving placebo PH20 SC on top of Prednisone	Other: placebo Drug: Prednisone

Name	Type	Description	Arm Group Labels	Other Names
efgartigimod PH20 SC	Biological	Subcutaneous injection of efgartigimod coformulated with rHuPH20, a permeation enhancer	efgartigimod PH20 SC

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
Number of Participants With CRoff at Week 36	CRoff = complete remission while receiving efgartigimod PH20 SC or placebo and being off oral corticosteroid therapy for at least 8 weeks. Complete remission is defined as the absence of new lesions, complete healing of existing lesions and absence of pruritus.	at week 36

Secondary Outcomes

Measure	Description	Time Frame
Cumulative OCS Dose	OCS = oral corticosteroid	up to week 36
Number of Participants Who Achieve an IGA-BP Score of 0 While Off OCS Therapy for ≥8 Weeks at Week 36	IGA-BP = Investigator Global Assessment of Bullous Pemphigoid. IGA-BP scores range from 0-4, with a higher score representing severe BP. OCS = oral corticosteroid

Eligibility Module

Eligibility Criteria

Inclusion Criteria:

The participant is willing and able to do the following:
1. understand the requirements of the study
2. provide written informed consent
3. comply with the study protocol procedures.
The participant is male or female and has reached the age of consent at the time of signing the informed consent form (ICF).
Participants have clinical signs of BP.
Contraceptive use should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies and: Women of childbearing potential must have a negative serum pregnancy test at screening and a negative urine pregnancy test at baseline before study intervention can be administered.

The full list of inclusion criteria can be found in the protocol.

Exclusion Criteria:

Other forms of pemphigoid or other autoimmune bullous diseases (AIBDs).
Received unstable dose of treatments known to cause or exacerbate BP for at least 4 weeks prior to the baseline visit
Use of BP treatments other than oral corticosteroids (OCS), topical corticosteroids (TCS), conventional immunosuppressants or dapsone.
Known contraindication to OCS therapy
Active, chronic or latent infection at screening
Positive COVID-19 test result at screening (testing performed if required per local regulations).
History of malignancy unless deemed cured by adequate treatment with no evidence of recurrence for ≥3 years before the first administration of the IMP. Participants with the following cancers can be included at any time, provided they are adequately treated prior to their participation in the study: Basal cell or squamous cell skin cancer, Carcinoma in situ of the cervix, Carcinoma in situ of the breast, Incidental histological finding of prostate cancer
Clinical evidence of other significant serious diseases, have had a recent surgery, or who have any other condition that, in the opinion of the investigator, could confound the results of the study or put the patient at undue risk or prevent participants from complying with protocol requirements
Use of an investigational product within 3 months before the first dose of IMP
Previously participated in a clinical study with efgartigimod or currently participating in another interventional clinical study
Known hypersensitivity to any of the components of the administered treatments
Positive serum test at screening for an active infection: HBV, HCV, HIV
Current or history (ie, within 12 months of screening) of alcohol, drug, or medication abuse as assessed by the investigator
Pregnant or lactating females and those who intend to become pregnant during the study
Live or live-attenuated vaccine received <4 weeks before baseline visit

The full list of exclusion criteria can be found in the protocol.

Facility	Status	City	State	ZIP	Country	Contacts
Investigator site 74 - US0010178		Phoenix	Arizona	85006	United States
Investigator site 6 - US0010138

Results Section

Participant Flow Module

Arm/Group Information

ID	Title	Description
FG000	Efgartigimod PH20 SC	All participants from Part A and Part B receiving efgartigimod PH20 SC. Participants received concurrent oral prednisone (or equivalent) at a starting dose of 0.5 mg/kg/day. The dose of prednisone was adjusted according to recommendations as per protocol.
FG001	Placebo PH20 SC

Baseline Characteristics Module

Outcome Measures Module

Adverse Events Module

More Info Module

Type	Includes Protocol	Includes SAP	Includes ICF	Document Label	Document Date	Document Uploaded Date	Document File Name
Prot	Yes	No	No	Study Protocol	Feb 27, 2024	Sep 1, 2025

Arm/Group Information

ID	Title	Description
BG000	Efgartigimod PH20 SC	All participants from Part A and Part B receiving efgartigimod PH20 SC. Participants received concurrent oral prednisone (or equivalent) at a starting dose of 0.5 mg/kg/day. The dose of prednisone was adjusted according to recommendations as per protocol.
BG001	Placebo PH20 SC	All participants from Part A and Part B receiving placebo PH20 SC. Participants received concurrent oral prednisone (or equivalent) at a starting dose of 0.5 mg/kg/day. The dose of prednisone was adjusted according to recommendations as per protocol.
BG002	Total	Total of all reporting groups

Baseline Measures

Title

Description

Population Description

Parameter Type

Dispersion Type

Unit of Measure

Calculate Percentage

Denominator Units Selected

Denominators

Classes

Age, Continuous

Mean

Standard Deviation

years

Title

Denominators

Categories

Title	Measurements
	BG00071.0± 12.02 BG00170.9± 9.37 BG00270.9± 10.75

Age, Customized

Count of Participants

Participants

Title

Denominators

Categories

18 - <65 years

Title	Measurements
	BG00012 BG00111 BG002

Sex: Female, Male

Count of Participants

Participants

Title

Denominators

Categories

Title	Measurements
Female	BG00037 BG00135 BG002

Race/Ethnicity, Customized

Count of Participants

Participants

Title

Denominators

Categories

AMERICAN INDIAN OR ALASKA NATIVE

Title	Measurements
	BG0001 BG0010 BG002

Race/Ethnicity, Customized

Count of Participants

Participants

Title

Denominators

Categories

HISPANIC OR LATINO

Title	Measurements
	BG0001 BG0010 BG002

Type

Title

Description

Population Description

Reporting Status

Anticipated Posting Date

Parameter Type

Dispersion Type

Unit of Measure

Calculate Percentage

Time Frame

Units Analyzed

Denominator Units Selected

Arm/Group Information

Denominators

Classes

Analyses

Primary

Number of Participants With CRoff at Week 36

CRoff = complete remission while receiving efgartigimod PH20 SC or placebo and being off oral corticosteroid therapy for at least 8 weeks. Complete remission is defined as the absence of new lesions, complete healing of existing lesions and absence of pruritus.

Posted

Count of Participants

Participants

at week 36

ID	Title	Description
OG000	Efgartigimod PH20 SC	All participants from Part A and Part B receiving efgartigimod PH20 SC. Participants received concurrent oral prednisone (or equivalent) at a starting dose of 0.5 mg/kg/day. The dose of prednisone was adjusted according to recommendations as per protocol.
OG001	Placebo PH20 SC	All participants from Part A and Part B receiving placebo PH20 SC. Participants received concurrent oral prednisone (or equivalent) at a starting dose of 0.5 mg/kg/day. The dose of prednisone was adjusted according to recommendations as per protocol.

Units	Counts
Participants	OG00050 OG00148

Title

Denominators

Categories

Title	Measurements
	OG0008 OG0012

Secondary

Cumulative OCS Dose

OCS = oral corticosteroid

Posted

Mean

Standard Deviation

mg/kg/day

up to week 36

ID	Title	Description
OG000	Efgartigimod PH20 SC	All participants from Part A and Part B receiving efgartigimod PH20 SC. Participants received concurrent oral prednisone (or equivalent) at a starting dose of 0.5 mg/kg/day. The dose of prednisone was adjusted according to recommendations as per protocol.
OG001	Placebo PH20 SC	All participants from Part A and Part B receiving placebo PH20 SC. Participants received concurrent oral prednisone (or equivalent) at a starting dose of 0.5 mg/kg/day. The dose of prednisone was adjusted according to recommendations as per protocol.

Units	Counts
Participants	OG000

Secondary

Number of Participants Who Achieve an IGA-BP Score of 0 While Off OCS Therapy for ≥8 Weeks at Week 36

IGA-BP = Investigator Global Assessment of Bullous Pemphigoid. IGA-BP scores range from 0-4, with a higher score representing severe BP. OCS = oral corticosteroid

Posted

Count of Participants

Participants

at week 36

ID	Title	Description
OG000	Efgartigimod PH20 SC	All participants from Part A and Part B receiving efgartigimod PH20 SC. Participants received concurrent oral prednisone (or equivalent) at a starting dose of 0.5 mg/kg/day. The dose of prednisone was adjusted according to recommendations as per protocol.
OG001	Placebo PH20 SC	All participants from Part A and Part B receiving placebo PH20 SC. Participants received concurrent oral prednisone (or equivalent) at a starting dose of 0.5 mg/kg/day. The dose of prednisone was adjusted according to recommendations as per protocol.

Units	Counts
Participants	OG000

Secondary

Number of Participants With CDA Who Remained Free of Relapse Through Week 36

Control of disease activity (CDA) is defined as the point at which new lesions ceased to form, established lesions began to heal, and pruritic symptoms started to abate. Relapse is defined as the appearance of 3 or more new lesions a month or at least 1 large lesion that did not heal within 1 week, or extension of established lesions or daily pruritus in a participant who had achieved CDA.

Posted

Count of Participants

Participants

up to week 36

ID	Title	Description
OG000	Efgartigimod PH20 SC	All participants from Part A and Part B receiving efgartigimod PH20 SC. Participants received concurrent oral prednisone (or equivalent) at a starting dose of 0.5 mg/kg/day. The dose of prednisone was adjusted according to recommendations as per protocol.
OG001	Placebo PH20 SC	All participants from Part A and Part B receiving placebo PH20 SC. Participants received concurrent oral prednisone (or equivalent) at a starting dose of 0.5 mg/kg/day. The dose of prednisone was adjusted according to recommendations as per protocol.

Units	Counts
Participants

Secondary

Number of Participants With CRmin at Week 36

CRmin = complete remission receiving minimal oral corticosteroids for at least 8 weeks. Minimal oral corticosteroid (OCS) therapy is defined as ≤0.1 mg/kg/day of prednisone (or equivalent).

Posted

Count of Participants

Participants

at week 36

ID	Title	Description
OG000	Efgartigimod PH20 SC	All participants from Part A and Part B receiving efgartigimod PH20 SC. Participants received concurrent oral prednisone (or equivalent) at a starting dose of 0.5 mg/kg/day. The dose of prednisone was adjusted according to recommendations as per protocol.
OG001	Placebo PH20 SC	All participants from Part A and Part B receiving placebo PH20 SC. Participants received concurrent oral prednisone (or equivalent) at a starting dose of 0.5 mg/kg/day. The dose of prednisone was adjusted according to recommendations as per protocol.

Units	Counts
Participants	OG000

Secondary

Change From Baseline to Week 36 in the 24-Hour Average Itch NRS Score

Itch NRS = Itch Numerical Rating Scale. The Itch NRS scores range from 0-10 with 10 representing the worst imaginable itch.

Only participants with an assessment at baseline and at week 36 are reported.

Posted

Mean

Standard Deviation

score on a scale

up to week 36

ID	Title	Description
OG000	Efgartigimod PH20 SC	All participants from Part A and Part B receiving efgartigimod PH20 SC. Participants received concurrent oral prednisone (or equivalent) at a starting dose of 0.5 mg/kg/day. The dose of prednisone was adjusted according to recommendations as per protocol.
OG001	Placebo PH20 SC	All participants from Part A and Part B receiving placebo PH20 SC. Participants received concurrent oral prednisone (or equivalent) at a starting dose of 0.5 mg/kg/day. The dose of prednisone was adjusted according to recommendations as per protocol.

Units	Counts
Participants

Secondary

Changes From Baseline in the BPDAI Total Activity Score

BPDAI = Bullous Pemphigoid Disease Area Index (BPDAI) Total Activity Score is a tool to objectively measure disease activity. BPDAI scores range from 0 to 360 with a higher score indicating more severe disease (max 240 for total skin activity and 120 for mucosal activity).

Only participants with an assessment at baseline and week 36 are reported here.

Posted

Mean

Standard Deviation

score on a scale

up to week 36

ID	Title	Description
OG000	Efgartigimod PH20 SC	All participants from Part A and Part B receiving efgartigimod PH20 SC. Participants received concurrent oral prednisone (or equivalent) at a starting dose of 0.5 mg/kg/day. The dose of prednisone was adjusted according to recommendations as per protocol.
OG001	Placebo PH20 SC	All participants from Part A and Part B receiving placebo PH20 SC. Participants received concurrent oral prednisone (or equivalent) at a starting dose of 0.5 mg/kg/day. The dose of prednisone was adjusted according to recommendations as per protocol.

Units	Counts
Participants

Secondary

Time to CDA

CDA = control of disease activity, the point at which new lesions ceased to form, established lesions began to heal, and pruritic symptoms started to abate.

Posted

Median

95% Confidence Interval

days

up to week 36

ID	Title	Description
OG000	Efgartigimod PH20 SC	All participants from Part A and Part B receiving efgartigimod PH20 SC. Participants received concurrent oral prednisone (or equivalent) at a starting dose of 0.5 mg/kg/day. The dose of prednisone was adjusted according to recommendations as per protocol.
OG001	Placebo PH20 SC	All participants from Part A and Part B receiving placebo PH20 SC. Participants received concurrent oral prednisone (or equivalent) at a starting dose of 0.5 mg/kg/day. The dose of prednisone was adjusted according to recommendations as per protocol.

Units	Counts
Participants	OG000

Secondary

Time to CR

CR = complete remission, the absence of new lesions, complete healing of existing lesions, and absence of pruritus.

Posted

Median

95% Confidence Interval

days

up to week 36

ID	Title	Description
OG000	Efgartigimod PH20 SC	All participants from Part A and Part B receiving efgartigimod PH20 SC. Participants received concurrent oral prednisone (or equivalent) at a starting dose of 0.5 mg/kg/day. The dose of prednisone was adjusted according to recommendations as per protocol.
OG001	Placebo PH20 SC	All participants from Part A and Part B receiving placebo PH20 SC. Participants received concurrent oral prednisone (or equivalent) at a starting dose of 0.5 mg/kg/day. The dose of prednisone was adjusted according to recommendations as per protocol.

Units	Counts
Participants	OG000

Secondary

Time to CRmin

CRmin = complete remission, the absence of new lesions, complete healing of existing lesions, and absence of pruritus while being on minimal dose of OCS for ≥8 Weeks. OCS = oral corticosteroid. Minimal OCS therapy is defined as an oral prednisone dosage of ≤0.10 mg/kg/day (or equivalent).

Posted

Median

95% Confidence Interval

days

up to week 36

ID	Title	Description
OG000	Efgartigimod PH20 SC	All participants from Part A and Part B receiving efgartigimod PH20 SC. Participants received concurrent oral prednisone (or equivalent) at a starting dose of 0.5 mg/kg/day. The dose of prednisone was adjusted according to recommendations as per protocol.
OG001	Placebo PH20 SC	All participants from Part A and Part B receiving placebo PH20 SC. Participants received concurrent oral prednisone (or equivalent) at a starting dose of 0.5 mg/kg/day. The dose of prednisone was adjusted according to recommendations as per protocol.

Units	Counts
Participants	OG000

Secondary

Time to CRoff/PRoff

CRoff= complete remission (the absence of new lesions, complete healing of existing lesions, and absence of pruritus) while receiving efgartigimod PH20 SC or placebo and being off oral corticosteroid therapy for at least 8 weeks. PRoff = partial remission (the presence of only new transient lesions) while receiving efgartigimod PH20 SC or placebo and being off oral corticosteroid therapy for at least 8 weeks.

Posted

Median

95% Confidence Interval

days

up to week 36

ID	Title	Description
OG000	Efgartigimod PH20 SC	All participants from Part A and Part B receiving efgartigimod PH20 SC. Participants received concurrent oral prednisone (or equivalent) at a starting dose of 0.5 mg/kg/day. The dose of prednisone was adjusted according to recommendations as per protocol.
OG001	Placebo PH20 SC	All participants from Part A and Part B receiving placebo PH20 SC. Participants received concurrent oral prednisone (or equivalent) at a starting dose of 0.5 mg/kg/day. The dose of prednisone was adjusted according to recommendations as per protocol.

Units	Counts
Participants

Secondary

Time to CRoff

CRoff is defined as the absence of new lesions, complete healing of existing lesions, and absence of pruritus while being off OCS therapy for ≥8 weeks.

Posted

Median

95% Confidence Interval

days

up to week 36

ID	Title	Description
OG000	Efgartigimod PH20 SC	All participants from Part A and Part B receiving efgartigimod PH20 SC. Participants received concurrent oral prednisone (or equivalent) at a starting dose of 0.5 mg/kg/day. The dose of prednisone was adjusted according to recommendations as per protocol.
OG001	Placebo PH20 SC	All participants from Part A and Part B receiving placebo PH20 SC. Participants received concurrent oral prednisone (or equivalent) at a starting dose of 0.5 mg/kg/day. The dose of prednisone was adjusted according to recommendations as per protocol.

Units	Counts
Participants	OG000

Secondary

Time From CDA to Achieve Relapse

CDA is defined as the point at which new lesions ceased to form, established lesions began to heal, and pruritic symptoms started to abate. Relapse is defined as the appearance of 3 or more new lesions a month or at least 1 large lesion that did not heal within 1 week, or extension of established lesions or daily pruritus in a participant who had achieved CDA.

Only participants achieving CDA are included in this section.

Posted

Median

95% Confidence Interval

days

up to week 36

ID	Title	Description
OG000	Efgartigimod PH20 SC	All participants from Part A and Part B receiving efgartigimod PH20 SC. Participants received concurrent oral prednisone (or equivalent) at a starting dose of 0.5 mg/kg/day. The dose of prednisone was adjusted according to recommendations as per protocol.
OG001	Placebo PH20 SC	All participants from Part A and Part B receiving placebo PH20 SC. Participants received concurrent oral prednisone (or equivalent) at a starting dose of 0.5 mg/kg/day. The dose of prednisone was adjusted according to recommendations as per protocol.

Units	Counts
Participants

Secondary

Number of Participants Who Receive Rescue Therapy Before Week 36

Posted

Count of Participants

Participants

at week 36

ID	Title	Description
OG000	Efgartigimod PH20 SC	All participants from Part A and Part B receiving efgartigimod PH20 SC. Participants received concurrent oral prednisone (or equivalent) at a starting dose of 0.5 mg/kg/day. The dose of prednisone was adjusted according to recommendations as per protocol.
OG001	Placebo PH20 SC	All participants from Part A and Part B receiving placebo PH20 SC. Participants received concurrent oral prednisone (or equivalent) at a starting dose of 0.5 mg/kg/day. The dose of prednisone was adjusted according to recommendations as per protocol.

Units	Counts
Participants	OG000

Secondary

The AIS From the GTI

The Aggregate Improvement Score (AIS) from the Glucocorticoid Toxicity Index (GTI) can range from -346 to 439 with a higher score representing greater corticosteroid toxicity. If a study drug is effective at lowering greater corticosteroid toxicity over time, the score will be lower.

Only participants with a GTI assessment at baseline and at week 36 are reported here.

Posted

Mean

Standard Deviation

score on a scale

up to week 36

ID	Title	Description
OG000	Efgartigimod PH20 SC	All participants from Part A and Part B receiving efgartigimod PH20 SC. Participants received concurrent oral prednisone (or equivalent) at a starting dose of 0.5 mg/kg/day. The dose of prednisone was adjusted according to recommendations as per protocol.
OG001	Placebo PH20 SC	All participants from Part A and Part B receiving placebo PH20 SC. Participants received concurrent oral prednisone (or equivalent) at a starting dose of 0.5 mg/kg/day. The dose of prednisone was adjusted according to recommendations as per protocol.

Units	Counts
Participants

Secondary

The CWS From the GTI

The Cumulative Worsening Score (CWS) from the Glucocorticoid Toxicity Index (GTI) can range from 0 to 439 with a higher score representing greater corticosteroid toxicity.

Only participants with a GTI assessment at baseline and at week 36 are reported here.

Posted

Mean

Standard Deviation

score on a scale

up to week 36

ID	Title	Description
OG000	Efgartigimod PH20 SC	All participants from Part A and Part B receiving efgartigimod PH20 SC. Participants received concurrent oral prednisone (or equivalent) at a starting dose of 0.5 mg/kg/day. The dose of prednisone was adjusted according to recommendations as per protocol.
OG001	Placebo PH20 SC	All participants from Part A and Part B receiving placebo PH20 SC. Participants received concurrent oral prednisone (or equivalent) at a starting dose of 0.5 mg/kg/day. The dose of prednisone was adjusted according to recommendations as per protocol.

Units	Counts
Participants

Secondary

EQ-5D-5L VAS Scores Over Time

The EuroQol 5-Dimension 5-Level Visual Analog Scale (EQ-5D-5L VAS) scores range from 0-100 with 0 representing the worst health.

Only participants with an assessment at baseline and at week 36 are reported here.

Posted

Mean

Standard Deviation

score on a scale

up to week 36

ID	Title	Description
OG000	Efgartigimod PH20 SC	All participants from Part A and Part B receiving efgartigimod PH20 SC. Participants received concurrent oral prednisone (or equivalent) at a starting dose of 0.5 mg/kg/day. The dose of prednisone was adjusted according to recommendations as per protocol.
OG001	Placebo PH20 SC	All participants from Part A and Part B receiving placebo PH20 SC. Participants received concurrent oral prednisone (or equivalent) at a starting dose of 0.5 mg/kg/day. The dose of prednisone was adjusted according to recommendations as per protocol.

Units	Counts
Participants	OG000

Secondary

DLQI Scores Over Time

Dermatology Life Quality Index (DLQI) assess the participant's perception of the impact of skin diseases on different aspects of their health-related QoL. Scores range from 0 to 30 with a higher score representing a worse quality of life.

Only participants with an assessment at baseline and at week 36 are reported here.

Posted

Mean

Standard Deviation

score on a scale

up to week 36

ID	Title	Description
OG000	Efgartigimod PH20 SC	All participants from Part A and Part B receiving efgartigimod PH20 SC. Participants received concurrent oral prednisone (or equivalent) at a starting dose of 0.5 mg/kg/day. The dose of prednisone was adjusted according to recommendations as per protocol.
OG001	Placebo PH20 SC	All participants from Part A and Part B receiving placebo PH20 SC. Participants received concurrent oral prednisone (or equivalent) at a starting dose of 0.5 mg/kg/day. The dose of prednisone was adjusted according to recommendations as per protocol.

Units	Counts
Participants

Secondary

ABQoL Scores Over Time

Autoimmune Bullous Disease Quality of Life Index (ABQoL) questionnaire assesses the impact of Autoimmune Bullous Disease and their therapies on the daily life of patients. Scores range from 0 to 51 with a higher score representing a worse quality of life.

Only participants with an assessment at baseline and at week 36 are analyzed.

Posted

Mean

Standard Deviation

score on a scale

up to week 36

ID	Title	Description
OG000	Efgartigimod PH20 SC	All participants from Part A and Part B receiving efgartigimod PH20 SC. Participants received concurrent oral prednisone (or equivalent) at a starting dose of 0.5 mg/kg/day. The dose of prednisone was adjusted according to recommendations as per protocol.
OG001	Placebo PH20 SC	All participants from Part A and Part B receiving placebo PH20 SC. Participants received concurrent oral prednisone (or equivalent) at a starting dose of 0.5 mg/kg/day. The dose of prednisone was adjusted according to recommendations as per protocol.

Units	Counts
Participants

Secondary

Efgartigimod Serum Concentrations

The number analyzed differs from overall number analyzed because the following samples were excluded from analysis: Pre-dose samples that were taken after dosing on that day, samples taken outside the visit window, and when the study drug administration prior to the sample is missed.

Posted

Mean

Standard Deviation

ug/mL

up to week 36

ID	Title	Description
OG000	Efgartigimod PH20 SC	All participants from Part A and Part B receiving efgartigimod PH20 SC. Participants received concurrent oral prednisone (or equivalent) at a starting dose of 0.5 mg/kg/day. The dose of prednisone was adjusted according to recommendations as per protocol.

Units	Counts
Participants	OG000

Secondary

Percent Change of Total IgG Serum Levels From Baseline Over Time

Only participants with an assessment at baseline and at week 36 are reported here.

Posted

Mean

Standard Deviation

percent change

up to 36 weeks

ID	Title	Description
OG000	Efgartigimod PH20 SC	All participants from Part A and Part B receiving efgartigimod PH20 SC. Participants received concurrent oral prednisone (or equivalent) at a starting dose of 0.5 mg/kg/day. The dose of prednisone was adjusted according to recommendations as per protocol.
OG001	Placebo PH20 SC	All participants from Part A and Part B receiving placebo PH20 SC. Participants received concurrent oral prednisone (or equivalent) at a starting dose of 0.5 mg/kg/day. The dose of prednisone was adjusted according to recommendations as per protocol.

Units	Counts
Participants	OG000

Secondary

Percent Change of Anti-BP180 and Anti-BP230 Antibodies From Baseline Over Time

Only participants with an assessment at baseline and at week 36 are reported here (31+34 with values for anti-BP180 and 11+12 with values for anti-BP230).

Posted

Mean

Standard Deviation

Percent change in RU/mL

up to 36 weeks

ID	Title	Description
OG000	Efgartigimod PH20 SC	All participants from Part A and Part B receiving efgartigimod PH20 SC. Participants received concurrent oral prednisone (or equivalent) at a starting dose of 0.5 mg/kg/day. The dose of prednisone was adjusted according to recommendations as per protocol.
OG001	Placebo PH20 SC	All participants from Part A and Part B receiving placebo PH20 SC. Participants received concurrent oral prednisone (or equivalent) at a starting dose of 0.5 mg/kg/day. The dose of prednisone was adjusted according to recommendations as per protocol.

Units	Counts
Participants	OG000

Secondary

Incidence of Antidrug Antibodies Against Efgartigimod and Antibodies Produced Against rHuPH20

Posted

Count of Participants

Participants

up to 46 weeks

ID	Title	Description
OG000	Efgartigimod PH20 SC	All participants from Part A and Part B receiving efgartigimod PH20 SC. Participants received concurrent oral prednisone (or equivalent) at a starting dose of 0.5 mg/kg/day. The dose of prednisone was adjusted according to recommendations as per protocol.
OG001	Placebo PH20 SC	All participants from Part A and Part B receiving placebo PH20 SC. Participants received concurrent oral prednisone (or equivalent) at a starting dose of 0.5 mg/kg/day. The dose of prednisone was adjusted according to recommendations as per protocol.

Units	Counts
Participants	OG000

Secondary

Number of Participants (or Their Caregivers) Who Are Determined by Site Staff to be Sufficiently Competent in (Self-)Administering Efgartigimod PH20 SC

The number analyzed for participants and caregivers is the total number who received training and therefore were eligible to be assessed for this outcome (5+4 participants received training and 2+3 caregivers received training).

Posted

Count of Participants

Participants

up to week 32

ID	Title	Description
OG000	Efgartigimod PH20 SC	All participants from Part A and Part B receiving efgartigimod PH20 SC. Participants received concurrent oral prednisone (or equivalent) at a starting dose of 0.5 mg/kg/day. The dose of prednisone was adjusted according to recommendations as per protocol.
OG001	Placebo PH20 SC	All participants from Part A and Part B receiving placebo PH20 SC. Participants received concurrent oral prednisone (or equivalent) at a starting dose of 0.5 mg/kg/day. The dose of prednisone was adjusted according to recommendations as per protocol.

Units	Counts
Participants

Arm/Groups

ID	Title	Description	Deaths (Affected)	Deaths (At Risk)	Serious Events (Affected)	Serious Events (At Risk)	Other Events (Affected)	Other Events (At Risk)
EG000	Efgartigimod PH20 SC	All participants from Part A and Part B receiving efgartigimod PH20 SC. Participants received concurrent oral prednisone (or equivalent) at a starting dose of 0.5 mg/kg/day. The dose of prednisone was adjusted according to recommendations as per protocol.	1	50	12	50	47	50
EG001	Placebo PH20 SC	All participants from Part A and Part B receiving placebo PH20 SC. Participants received concurrent oral prednisone (or equivalent) at a starting dose of 0.5 mg/kg/day. The dose of prednisone was adjusted according to recommendations as per protocol.	2	48	12	48	46	48

Serious Adverse Events

Term	Organ System	Source Vocabulary	Assessment Type	Statistical Information
ANAEMIA	Blood and lymphatic system disorders	MedDRA 27.1	Systematic Assessment	EG0001 affected50 at risk EG0010 affected48 at risk
ACUTE MYOCARDIAL INFARCTION	Cardiac disorders	MedDRA 27.1	Systematic Assessment	EG0000 affected50 at risk EG0011 affected48 at risk
ATRIAL FIBRILLATION	Cardiac disorders	MedDRA 27.1	Systematic Assessment	EG0001 affected50 at risk EG0011 affected48 at risk
CARDIAC ARREST	Cardiac disorders	MedDRA 27.1	Systematic Assessment	EG0001 affected50 at risk EG0010 affected48 at risk
CARDIAC FAILURE	Cardiac disorders	MedDRA 27.1	Systematic Assessment	EG0000 affected50 at risk EG0011 affected48 at risk
CARDIAC FAILURE ACUTE	Cardiac disorders	MedDRA 27.1	Systematic Assessment	EG0001 affected50 at risk EG0010 affected48 at risk
CARDIAC FAILURE CONGESTIVE	Cardiac disorders	MedDRA 27.1	Systematic Assessment	EG0001 affected50 at risk EG0010 affected48 at risk
COR PULMONALE	Cardiac disorders	MedDRA 27.1	Systematic Assessment	EG0001 affected50 at risk EG0010 affected48 at risk
CORONARY ARTERY STENOSIS	Cardiac disorders	MedDRA 27.1	Systematic Assessment	EG0000 affected50 at risk EG0011 affected48 at risk
SINUS NODE DYSFUNCTION	Cardiac disorders	MedDRA 27.1	Systematic Assessment	EG0000 affected50 at risk EG0011 affected48 at risk
CUSHING'S SYNDROME	Endocrine disorders	MedDRA 27.1	Systematic Assessment	EG0000 affected50 at risk EG0011 affected48 at risk
LENS DISLOCATION	Eye disorders	MedDRA 27.1	Systematic Assessment	EG0001 affected50 at risk EG0010 affected48 at risk
INGUINAL HERNIA	Gastrointestinal disorders	MedDRA 27.1	Systematic Assessment	EG0001 affected50 at risk EG0010 affected48 at risk
INTESTINAL MASS	Gastrointestinal disorders	MedDRA 27.1	Systematic Assessment	EG0001 affected50 at risk EG0010 affected48 at risk
ASTHENIA	General disorders	MedDRA 27.1	Systematic Assessment	EG0001 affected50 at risk EG0010 affected48 at risk
CONDITION AGGRAVATED	General disorders	MedDRA 27.1	Systematic Assessment	EG0002 affected50 at risk EG0010 affected48 at risk
NON-CARDIAC CHEST PAIN	General disorders	MedDRA 27.1	Systematic Assessment	EG0000 affected50 at risk EG0011 affected48 at risk
CELLULITIS GANGRENOUS	Infections and infestations	MedDRA 27.1	Systematic Assessment	EG0000 affected50 at risk EG0011 affected48 at risk
PNEUMONIA	Infections and infestations	MedDRA 27.1	Systematic Assessment	EG0002 affected50 at risk EG0010 affected48 at risk
PNEUMONIA ASPIRATION	Infections and infestations	MedDRA 27.1	Systematic Assessment	EG0000 affected50 at risk EG0011 affected48 at risk
SEPTIC SHOCK	Infections and infestations	MedDRA 27.1	Systematic Assessment	EG0000 affected50 at risk EG0011 affected48 at risk
ANKLE FRACTURE	Injury, poisoning and procedural complications	MedDRA 27.1	Systematic Assessment	EG0001 affected50 at risk EG0010 affected48 at risk
FALL	Injury, poisoning and procedural complications	MedDRA 27.1	Systematic Assessment	EG0000 affected50 at risk EG0011 affected48 at risk
FEMUR FRACTURE	Injury, poisoning and procedural complications	MedDRA 27.1	Systematic Assessment	EG0001 affected50 at risk EG0010 affected48 at risk
MULTIPLE INJURIES	Injury, poisoning and procedural complications	MedDRA 27.1	Systematic Assessment	EG0000 affected50 at risk EG0011 affected48 at risk
SKIN LACERATION	Injury, poisoning and procedural complications	MedDRA 27.1	Systematic Assessment	EG0001 affected50 at risk EG0010 affected48 at risk
STRESS FRACTURE	Injury, poisoning and procedural complications	MedDRA 27.1	Systematic Assessment	EG0001 affected50 at risk EG0010 affected48 at risk
THORACIC VERTEBRAL FRACTURE	Injury, poisoning and procedural complications	MedDRA 27.1	Systematic Assessment	EG0001 affected50 at risk EG0010 affected48 at risk
VASCULAR GRAFT OCCLUSION	Injury, poisoning and procedural complications	MedDRA 27.1	Systematic Assessment	EG0001 affected50 at risk EG0010 affected48 at risk
DEHYDRATION	Metabolism and nutrition disorders	MedDRA 27.1	Systematic Assessment	EG0000 affected50 at risk EG0011 affected48 at risk
DIABETIC KETOACIDOSIS	Metabolism and nutrition disorders	MedDRA 27.1	Systematic Assessment	EG0000 affected50 at risk EG0011 affected48 at risk
OVARIAN EPITHELIAL CANCER	Neoplasms benign, malignant and unspecified (incl cysts and polyps)	MedDRA 27.1	Systematic Assessment	EG0000 affected50 at risk EG0011 affected48 at risk
CAROTID ARTERY OCCLUSION	Nervous system disorders	MedDRA 27.1	Systematic Assessment	EG0001 affected50 at risk EG0010 affected48 at risk
CEREBROVASCULAR DISORDER	Nervous system disorders	MedDRA 27.1	Systematic Assessment	EG0000 affected50 at risk EG0011 affected48 at risk
ENCEPHALOPATHY	Nervous system disorders	MedDRA 27.1	Systematic Assessment	EG0001 affected50 at risk EG0010 affected48 at risk
HYPOTONIA	Nervous system disorders	MedDRA 27.1	Systematic Assessment	EG0000 affected50 at risk EG0011 affected48 at risk
ISCHAEMIC STROKE	Nervous system disorders	MedDRA 27.1	Systematic Assessment	EG0001 affected50 at risk EG0010 affected48 at risk
ACUTE RESPIRATORY FAILURE	Respiratory, thoracic and mediastinal disorders	MedDRA 27.1	Systematic Assessment	EG0001 affected50 at risk EG0010 affected48 at risk
CHRONIC OBSTRUCTIVE PULMONARY DISEASE	Respiratory, thoracic and mediastinal disorders	MedDRA 27.1	Systematic Assessment	EG0001 affected50 at risk EG0010 affected48 at risk
PULMONARY EMBOLISM	Respiratory, thoracic and mediastinal disorders	MedDRA 27.1	Systematic Assessment	EG0000 affected50 at risk EG0011 affected48 at risk
RESPIRATORY FAILURE	Respiratory, thoracic and mediastinal disorders	MedDRA 27.1	Systematic Assessment	EG0001 affected50 at risk EG0010 affected48 at risk
SKIN ULCER	Skin and subcutaneous tissue disorders	MedDRA 27.1	Systematic Assessment	EG0001 affected50 at risk EG0010 affected48 at risk
DEEP VEIN THROMBOSIS	Vascular disorders	MedDRA 27.1	Systematic Assessment	EG0000 affected50 at risk EG0012 affected48 at risk

Other Adverse Events

Term	Organ System	Source Vocabulary	Assessment Type	Statistical Information
INCREASED TENDENCY TO BRUISE	Blood and lymphatic system disorders	MedDRA 27.1	Systematic Assessment	EG0005 affected50 at risk EG0017 affected48 at risk
LEUKOCYTOSIS	Blood and lymphatic system disorders	MedDRA 27.1	Systematic Assessment	EG0000 affected50 at risk EG0014 affected48 at risk
LYMPHOPENIA	Blood and lymphatic system disorders	MedDRA 27.1	Systematic Assessment	EG0005 affected50 at risk EG0013 affected48 at risk
CUSHINGOID	Endocrine disorders	MedDRA 27.1	Systematic Assessment	EG0004 affected50 at risk EG0010 affected48 at risk
CONSTIPATION	Gastrointestinal disorders	MedDRA 27.1	Systematic Assessment	EG0003 affected50 at risk EG0010 affected48 at risk
DIARRHOEA	Gastrointestinal disorders	MedDRA 27.1	Systematic Assessment	EG0005 affected50 at risk EG0013 affected48 at risk
NAUSEA	Gastrointestinal disorders	MedDRA 27.1	Systematic Assessment	EG0003 affected50 at risk EG0011 affected48 at risk
VOMITING	Gastrointestinal disorders	MedDRA 27.1	Systematic Assessment	EG0002 affected50 at risk EG0014 affected48 at risk
CONDITION AGGRAVATED	General disorders	MedDRA 27.1	Systematic Assessment	EG0003 affected50 at risk EG0010 affected48 at risk
FATIGUE	General disorders	MedDRA 27.1	Systematic Assessment	EG0004 affected50 at risk EG0011 affected48 at risk
INJECTION SITE ERYTHEMA	General disorders	MedDRA 27.1	Systematic Assessment	EG0004 affected50 at risk EG0012 affected48 at risk
INJECTION SITE PARAESTHESIA	General disorders	MedDRA 27.1	Systematic Assessment	EG0003 affected50 at risk EG0012 affected48 at risk
OEDEMA	General disorders	MedDRA 27.1	Systematic Assessment	EG0003 affected50 at risk EG0010 affected48 at risk
OEDEMA PERIPHERAL	General disorders	MedDRA 27.1	Systematic Assessment	EG0005 affected50 at risk EG0015 affected48 at risk
COVID-19	Infections and infestations	MedDRA 27.1	Systematic Assessment	EG0002 affected50 at risk EG0014 affected48 at risk
NASOPHARYNGITIS	Infections and infestations	MedDRA 27.1	Systematic Assessment	EG0003 affected50 at risk EG0011 affected48 at risk
PNEUMONIA	Infections and infestations	MedDRA 27.1	Systematic Assessment	EG0003 affected50 at risk EG0010 affected48 at risk
URINARY TRACT INFECTION	Infections and infestations	MedDRA 27.1	Systematic Assessment	EG0005 affected50 at risk EG0016 affected48 at risk
FALL	Injury, poisoning and procedural complications	MedDRA 27.1	Systematic Assessment	EG0002 affected50 at risk EG0014 affected48 at risk
ALANINE AMINOTRANSFERASE INCREASED	Investigations	MedDRA 27.1	Systematic Assessment	EG0001 affected50 at risk EG0013 affected48 at risk
GLYCOSYLATED HAEMOGLOBIN INCREASED	Investigations	MedDRA 27.1	Systematic Assessment	EG0004 affected50 at risk EG0013 affected48 at risk
DIABETES MELLITUS	Metabolism and nutrition disorders	MedDRA 27.1	Systematic Assessment	EG0003 affected50 at risk EG0012 affected48 at risk
HYPOKALAEMIA	Metabolism and nutrition disorders	MedDRA 27.1	Systematic Assessment	EG0000 affected50 at risk EG0013 affected48 at risk
ARTHRALGIA	Musculoskeletal and connective tissue disorders	MedDRA 27.1	Systematic Assessment	EG0004 affected50 at risk EG0013 affected48 at risk
BACK PAIN	Musculoskeletal and connective tissue disorders	MedDRA 27.1	Systematic Assessment	EG0005 affected50 at risk EG0015 affected48 at risk
MUSCLE SPASMS	Musculoskeletal and connective tissue disorders	MedDRA 27.1	Systematic Assessment	EG0003 affected50 at risk EG0011 affected48 at risk
MUSCULAR WEAKNESS	Musculoskeletal and connective tissue disorders	MedDRA 27.1	Systematic Assessment	EG0003 affected50 at risk EG0010 affected48 at risk
MYOPATHY	Musculoskeletal and connective tissue disorders	MedDRA 27.1	Systematic Assessment	EG0003 affected50 at risk EG0014 affected48 at risk
DIZZINESS	Nervous system disorders	MedDRA 27.1	Systematic Assessment	EG0004 affected50 at risk EG0012 affected48 at risk
HEADACHE	Nervous system disorders	MedDRA 27.1	Systematic Assessment	EG0007 affected50 at risk EG0012 affected48 at risk
DEPRESSION	Psychiatric disorders	MedDRA 27.1	Systematic Assessment	EG0005 affected50 at risk EG0011 affected48 at risk
INSOMNIA	Psychiatric disorders	MedDRA 27.1	Systematic Assessment	EG0007 affected50 at risk EG0018 affected48 at risk
MANIA	Psychiatric disorders	MedDRA 27.1	Systematic Assessment	EG0001 affected50 at risk EG0013 affected48 at risk
CHRONIC KIDNEY DISEASE	Renal and urinary disorders	MedDRA 27.1	Systematic Assessment	EG0000 affected50 at risk EG0013 affected48 at risk
GLYCOSURIA	Renal and urinary disorders	MedDRA 27.1	Systematic Assessment	EG0000 affected50 at risk EG0013 affected48 at risk
COUGH	Respiratory, thoracic and mediastinal disorders	MedDRA 27.1	Systematic Assessment	EG0006 affected50 at risk EG0010 affected48 at risk
DRY SKIN	Skin and subcutaneous tissue disorders	MedDRA 27.1	Systematic Assessment	EG0002 affected50 at risk EG0013 affected48 at risk
HIRSUTISM	Skin and subcutaneous tissue disorders	MedDRA 27.1	Systematic Assessment	EG0001 affected50 at risk EG0013 affected48 at risk
INTERTRIGO	Skin and subcutaneous tissue disorders	MedDRA 27.1	Systematic Assessment	EG0004 affected50 at risk EG0010 affected48 at risk
PRURITUS	Skin and subcutaneous tissue disorders	MedDRA 27.1	Systematic Assessment	EG0004 affected50 at risk EG0015 affected48 at risk
HYPERTENSION	Vascular disorders	MedDRA 27.1	Systematic Assessment	EG0006 affected50 at risk EG0012 affected48 at risk

ID	Term
D012872	Skin Diseases, Vesiculobullous
D012871	Skin Diseases
D017437	Skin and Connective Tissue Diseases
D001327	Autoimmune Diseases
D007154	Immune System Diseases

ID	Term
D011244	Pregnadienediols
D011245	Pregnadienes
D011278	Pregnanes
D013256	Steroids
D000072473	Fused-Ring Compounds
D011083	Polycyclic Compounds