Bullous Pemphigoid
Conditions
Brief summary
ARGX-113-2009 is an operationally seamless 2-part, phase 2/3, prospective, global, multicenter, randomized, double-blinded, placebo-controlled study to investigate the efficacy, safety, tolerability, immunogenicity, participant-reported outcome measures (including those assessing participant QoL), PK, and PD of efgartigimod PH20 SC administered via subcutaneous (SC) injection in adult participants with moderate to severe BP. This study intends to demonstrate that efgartigimod is an effective and safe treatment for BP, providing participants with control of disease activity (CDA) and eventually remission while reducing their cumulative exposure to OCS. study will consist of 2 parts: * Part A of the study is a phase 2 evaluation that intends to provide proof of concept for the therapeutic activity of efgartigimod PH20 SC in participants with BP. * Part B of the study is a phase 3 evaluation that intends to confirm the results obtained from part A in a separate, larger group of participants with BP. An interim analysis will be performed during part A (on data obtained through week 26 for all Part A participants) to assess the primary endpoint and several secondary endpoints, confirm the appropriate sample size for part B of the study, and determine whether the efficacy results observed through week 26 of part A warrant continued study of efgartigimod PH20 SC for the treatment of participants with BP (futility analysis).
Interventions
BIOLOGICALefgartigimod PH20 SC
Subcutaneous injection of efgartigimod coformulated with rHuPH20, a permeation enhancer
OTHERplacebo
Subcutaneous injection of placebo coformulated with rHuPH20, a permeation enhancer
DRUGPrednisone
Oral Prednisone
Sponsors
argenx
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
DOUBLE (Subject, Investigator)
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Participants With CRoff at Week 36 | 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. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Number of Participants Who Achieve an IGA-BP Score of 0 While Off OCS Therapy for ≥8 Weeks at Week 36 | 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 |
| Number of Participants With CDA Who Remained Free of Relapse Through Week 36 | up to 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. |
| Number of Participants With CRmin at Week 36 | 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). |
| Change From Baseline to Week 36 in the 24-Hour Average Itch NRS Score | up to week 36 | Itch NRS = Itch Numerical Rating Scale. The Itch NRS scores range from 0-10 with 10 representing the worst imaginable itch. |
| Changes From Baseline in the BPDAI Total Activity Score | up to week 36 | 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). |
| Time to CDA | up to week 36 | 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. |
| Time to CR | up to week 36 | CR = complete remission, the absence of new lesions, complete healing of existing lesions, and absence of pruritus. |
| Time to CRmin | up to week 36 | 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). |
| Time to CRoff/PRoff | up to week 36 | 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. |
| Time to CRoff | up to week 36 | 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. |
| Time From CDA to Achieve Relapse | up to week 36 | 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. |
| Cumulative OCS Dose | up to week 36 | OCS = oral corticosteroid |
| The AIS From the GTI | up to week 36 | 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. |
| The CWS From the GTI | up to week 36 | 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. |
| EQ-5D-5L VAS Scores Over Time | up to week 36 | The EuroQol 5-Dimension 5-Level Visual Analog Scale (EQ-5D-5L VAS) scores range from 0-100 with 0 representing the worst health. |
| DLQI Scores Over Time | up to week 36 | 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. |
| ABQoL Scores Over Time | up to week 36 | 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. |
| Efgartigimod Serum Concentrations | up to week 36 | — |
| Percent Change of Total IgG Serum Levels From Baseline Over Time | up to 36 weeks | — |
| Percent Change of Anti-BP180 and Anti-BP230 Antibodies From Baseline Over Time | up to 36 weeks | — |
| Incidence of Antidrug Antibodies Against Efgartigimod and Antibodies Produced Against rHuPH20 | up to 46 weeks | — |
| Number of Participants (or Their Caregivers) Who Are Determined by Site Staff to be Sufficiently Competent in (Self-)Administering Efgartigimod PH20 SC | up to week 32 | — |
| Number of Participants Who Receive Rescue Therapy Before Week 36 | at week 36 | — |
Countries
Australia, Bulgaria, China, Croatia, Czechia, France, Germany, Greece, Hungary, Israel, Italy, Japan, Latvia, Netherlands, Poland, Romania, Serbia, Slovakia, Spain, United Kingdom, United States
Participant flow
Pre-assignment details
Parts A and B were identical in schedule, structure, eligibility requirements, assessments, and conduct therefore the results are shown with the pooled data from part A and part B.
Participants by arm
| Arm | Count |
|---|---|
| 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. | 50 |
| 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. | 48 |
| Total | 98 |
Withdrawals & dropouts
| Period | Reason | FG000 | FG001 |
|---|---|---|---|
| Overall Study | Adverse Event | 1 | 4 |
| Overall Study | Death | 1 | 2 |
| Overall Study | Lack of Efficacy | 1 | 2 |
| Overall Study | Lost to Follow-up | 3 | 0 |
| Overall Study | Physician Decision | 4 | 0 |
| Overall Study | Treatment Unblinded | 0 | 1 |
| Overall Study | Withdrawal by Subject | 5 | 4 |
Baseline characteristics
| Characteristic | Efgartigimod PH20 SC | Placebo PH20 SC | Total |
|---|---|---|---|
| Age, Continuous | 71.0 years STANDARD_DEVIATION 12.02 | 70.9 years STANDARD_DEVIATION 9.37 | 70.9 years STANDARD_DEVIATION 10.75 |
| Age, Customized 18 - <65 years | 12 Participants | 11 Participants | 23 Participants |
| Age, Customized 65 - <75 years | 18 Participants | 17 Participants | 35 Participants |
| Age, Customized 75 - <85 years | 15 Participants | 17 Participants | 32 Participants |
| Age, Customized >=85 years | 5 Participants | 3 Participants | 8 Participants |
| Race/Ethnicity, Customized AMERICAN INDIAN OR ALASKA NATIVE | 1 Participants | 0 Participants | 1 Participants |
| Race/Ethnicity, Customized ASIAN | 8 Participants | 9 Participants | 17 Participants |
| Race/Ethnicity, Customized HISPANIC OR LATINO | 1 Participants | 0 Participants | 1 Participants |
| Race/Ethnicity, Customized MULTIPLE | 1 Participants | 0 Participants | 1 Participants |
| Race/Ethnicity, Customized NOT HISPANIC OR LATINO | 49 Participants | 48 Participants | 97 Participants |
| Race/Ethnicity, Customized WHITE | 40 Participants | 39 Participants | 79 Participants |
| Sex: Female, Male Female | 37 Participants | 35 Participants | 72 Participants |
| Sex: Female, Male Male | 13 Participants | 13 Participants | 26 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | 1 / 50 | 2 / 48 |
| other Total, other adverse events | 47 / 50 | 46 / 48 |
| serious Total, serious adverse events | 12 / 50 | 12 / 48 |
Outcome results
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.
Time frame: at week 36
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Efgartigimod PH20 SC | Number of Participants With CRoff at Week 36 | 8 Participants |
| Placebo PH20 SC | Number of Participants With CRoff at Week 36 | 2 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.
Time frame: up to week 36
Population: Only participants with an assessment at baseline and at week 36 are analyzed.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Efgartigimod PH20 SC | ABQoL Scores Over Time | -12.3 score on a scale | Standard Deviation 6.44 |
| Placebo PH20 SC | ABQoL Scores Over Time | -12.0 score on a scale | Standard Deviation 10.92 |
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.
Time frame: up to week 36
Population: Only participants with an assessment at baseline and at week 36 are reported.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Efgartigimod PH20 SC | Change From Baseline to Week 36 in the 24-Hour Average Itch NRS Score | -4.4 score on a scale | Standard Deviation 3.35 |
| Placebo PH20 SC | Change From Baseline to Week 36 in the 24-Hour Average Itch NRS Score | -2.4 score on a scale | Standard Deviation 4.26 |
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).
Time frame: up to week 36
Population: Only participants with an assessment at baseline and week 36 are reported here.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Efgartigimod PH20 SC | Changes From Baseline in the BPDAI Total Activity Score | -43.13 score on a scale | Standard Deviation 19.633 |
| Placebo PH20 SC | Changes From Baseline in the BPDAI Total Activity Score | -43.56 score on a scale | Standard Deviation 32.584 |
Secondary
Cumulative OCS Dose
OCS = oral corticosteroid
Time frame: up to week 36
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Efgartigimod PH20 SC | Cumulative OCS Dose | 0.242 mg/kg/day | Standard Deviation 0.138 |
| Placebo PH20 SC | Cumulative OCS Dose | 0.289 mg/kg/day | Standard Deviation 0.173 |
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.
Time frame: up to week 36
Population: Only participants with an assessment at baseline and at week 36 are reported here.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Efgartigimod PH20 SC | DLQI Scores Over Time | -7.3 score on a scale | Standard Deviation 6.25 |
| Placebo PH20 SC | DLQI Scores Over Time | -6.7 score on a scale | Standard Deviation 7.14 |
Secondary
Efgartigimod Serum Concentrations
Time frame: up to week 36
Population: 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.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Efgartigimod PH20 SC | Efgartigimod Serum Concentrations | Week 1 | 35.7 ug/mL | Standard Deviation 13.6 |
| Efgartigimod PH20 SC | Efgartigimod Serum Concentrations | Day 10 | 96.4 ug/mL | Standard Deviation 23.9 |
| Efgartigimod PH20 SC | Efgartigimod Serum Concentrations | Week 2 | 37.3 ug/mL | Standard Deviation 12.5 |
| Efgartigimod PH20 SC | Efgartigimod Serum Concentrations | Week 4 | 26.0 ug/mL | Standard Deviation 11 |
| Efgartigimod PH20 SC | Efgartigimod Serum Concentrations | Week 8 | 25.8 ug/mL | Standard Deviation 10.5 |
| Efgartigimod PH20 SC | Efgartigimod Serum Concentrations | Week 12 | 24.4 ug/mL | Standard Deviation 8.92 |
| Efgartigimod PH20 SC | Efgartigimod Serum Concentrations | Week 16 | 24.3 ug/mL | Standard Deviation 8.51 |
| Efgartigimod PH20 SC | Efgartigimod Serum Concentrations | Week 20 | 23.9 ug/mL | Standard Deviation 9.12 |
| Efgartigimod PH20 SC | Efgartigimod Serum Concentrations | Week 24 | 24.3 ug/mL | Standard Deviation 9.98 |
| Efgartigimod PH20 SC | Efgartigimod Serum Concentrations | Week 26 | 24.6 ug/mL | Standard Deviation 10.2 |
| Efgartigimod PH20 SC | Efgartigimod Serum Concentrations | Week 28 | 23.3 ug/mL | Standard Deviation 10.3 |
| Efgartigimod PH20 SC | Efgartigimod Serum Concentrations | Week 32 | 26.1 ug/mL | Standard Deviation 11.3 |
| Efgartigimod PH20 SC | Efgartigimod Serum Concentrations | Week 36 | 22.2 ug/mL | Standard Deviation 8.59 |
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.
Time frame: up to week 36
Population: Only participants with an assessment at baseline and at week 36 are reported here.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Efgartigimod PH20 SC | EQ-5D-5L VAS Scores Over Time | 12.7 score on a scale | Standard Deviation 15.94 |
| Placebo PH20 SC | EQ-5D-5L VAS Scores Over Time | 4.5 score on a scale | Standard Deviation 24.44 |
Secondary
Incidence of Antidrug Antibodies Against Efgartigimod and Antibodies Produced Against rHuPH20
Time frame: up to 46 weeks
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Efgartigimod PH20 SC | Incidence of Antidrug Antibodies Against Efgartigimod and Antibodies Produced Against rHuPH20 | ADA against efgartigimod | 2 Participants |
| Efgartigimod PH20 SC | Incidence of Antidrug Antibodies Against Efgartigimod and Antibodies Produced Against rHuPH20 | Antibodies against rHuPH20 | 9 Participants |
| Placebo PH20 SC | Incidence of Antidrug Antibodies Against Efgartigimod and Antibodies Produced Against rHuPH20 | ADA against efgartigimod | 2 Participants |
| Placebo PH20 SC | Incidence of Antidrug Antibodies Against Efgartigimod and Antibodies Produced Against rHuPH20 | Antibodies against rHuPH20 | 0 Participants |
Secondary
Number of Participants (or Their Caregivers) Who Are Determined by Site Staff to be Sufficiently Competent in (Self-)Administering Efgartigimod PH20 SC
Time frame: up to week 32
Population: 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).
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Efgartigimod PH20 SC | Number of Participants (or Their Caregivers) Who Are Determined by Site Staff to be Sufficiently Competent in (Self-)Administering Efgartigimod PH20 SC | Caregivers | 2 Participants |
| Efgartigimod PH20 SC | Number of Participants (or Their Caregivers) Who Are Determined by Site Staff to be Sufficiently Competent in (Self-)Administering Efgartigimod PH20 SC | Participants | 5 Participants |
| Placebo PH20 SC | Number of Participants (or Their Caregivers) Who Are Determined by Site Staff to be Sufficiently Competent in (Self-)Administering Efgartigimod PH20 SC | Participants | 4 Participants |
| Placebo PH20 SC | Number of Participants (or Their Caregivers) Who Are Determined by Site Staff to be Sufficiently Competent in (Self-)Administering Efgartigimod PH20 SC | Caregivers | 3 Participants |
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
Time frame: at week 36
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Efgartigimod PH20 SC | Number of Participants Who Achieve an IGA-BP Score of 0 While Off OCS Therapy for ≥8 Weeks at Week 36 | 9 Participants |
| Placebo PH20 SC | Number of Participants Who Achieve an IGA-BP Score of 0 While Off OCS Therapy for ≥8 Weeks at Week 36 | 3 Participants |
Secondary
Number of Participants Who Receive Rescue Therapy Before Week 36
Time frame: at week 36
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Efgartigimod PH20 SC | Number of Participants Who Receive Rescue Therapy Before Week 36 | 19 Participants |
| Placebo PH20 SC | Number of Participants Who Receive Rescue Therapy Before Week 36 | 18 Participants |
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.
Time frame: up to week 36
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Efgartigimod PH20 SC | Number of Participants With CDA Who Remained Free of Relapse Through Week 36 | 7 Participants |
| Placebo PH20 SC | Number of Participants With CDA Who Remained Free of Relapse Through Week 36 | 8 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).
Time frame: at week 36
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Efgartigimod PH20 SC | Number of Participants With CRmin at Week 36 | 8 Participants |
| Placebo PH20 SC | Number of Participants With CRmin at Week 36 | 3 Participants |
Secondary
Percent Change of Anti-BP180 and Anti-BP230 Antibodies From Baseline Over Time
Time frame: up to 36 weeks
Population: 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).
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Efgartigimod PH20 SC | Percent Change of Anti-BP180 and Anti-BP230 Antibodies From Baseline Over Time | Percent change of Anti-BP180 antibodies from baseline over time | -58.58 Percent change in RU/mL | Standard Deviation 56.983 |
| Efgartigimod PH20 SC | Percent Change of Anti-BP180 and Anti-BP230 Antibodies From Baseline Over Time | Percent change of Anti-BP230 antibodies from baseline over time | -46.72 Percent change in RU/mL | Standard Deviation 35.95 |
| Placebo PH20 SC | Percent Change of Anti-BP180 and Anti-BP230 Antibodies From Baseline Over Time | Percent change of Anti-BP180 antibodies from baseline over time | -57.98 Percent change in RU/mL | Standard Deviation 33.47 |
| Placebo PH20 SC | Percent Change of Anti-BP180 and Anti-BP230 Antibodies From Baseline Over Time | Percent change of Anti-BP230 antibodies from baseline over time | -50.95 Percent change in RU/mL | Standard Deviation 28.617 |
Secondary
Percent Change of Total IgG Serum Levels From Baseline Over Time
Time frame: up to 36 weeks
Population: Only participants with an assessment at baseline and at week 36 are reported here.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Efgartigimod PH20 SC | Percent Change of Total IgG Serum Levels From Baseline Over Time | -46.91 percent change | Standard Deviation 42.244 |
| Placebo PH20 SC | Percent Change of Total IgG Serum Levels From Baseline Over Time | -7.65 percent change | Standard Deviation 25.374 |
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.
Time frame: up to week 36
Population: Only participants with a GTI assessment at baseline and at week 36 are reported here.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Efgartigimod PH20 SC | The AIS From the GTI | 5.7 score on a scale | Standard Deviation 56.98 |
| Placebo PH20 SC | The AIS From the GTI | 10.9 score on a scale | Standard Deviation 45.42 |
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.
Time frame: up to week 36
Population: Only participants with a GTI assessment at baseline and at week 36 are reported here.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Efgartigimod PH20 SC | The CWS From the GTI | 56.1 score on a scale | Standard Deviation 40.71 |
| Placebo PH20 SC | The CWS From the GTI | 47.5 score on a scale | Standard Deviation 46.68 |
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.
Time frame: up to week 36
Population: Only participants achieving CDA are included in this section.
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Efgartigimod PH20 SC | Time From CDA to Achieve Relapse | 114.0 days |
| Placebo PH20 SC | Time From CDA to Achieve Relapse | 105.0 days |
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.
Time frame: up to week 36
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Efgartigimod PH20 SC | Time to CDA | 16.0 days |
| Placebo PH20 SC | Time to CDA | 15.0 days |
Secondary
Time to CR
CR = complete remission, the absence of new lesions, complete healing of existing lesions, and absence of pruritus.
Time frame: up to week 36
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Efgartigimod PH20 SC | Time to CR | 134.0 days |
| Placebo PH20 SC | Time to CR | 99.0 days |
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).
Time frame: up to week 36
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Efgartigimod PH20 SC | Time to CRmin | NA days |
| Placebo PH20 SC | Time to CRmin | NA days |
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.
Time frame: up to week 36
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Efgartigimod PH20 SC | Time to CRoff | NA days |
| Placebo PH20 SC | Time to CRoff | NA days |
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.
Time frame: up to week 36
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Efgartigimod PH20 SC | Time to CRoff/PRoff | NA days |
| Placebo PH20 SC | Time to CRoff/PRoff | NA days |