A Phase 2/3 Study of Efgartigimod PH20 SC in Adult Participants with Bullous Pemphigoid
- Conditions
- Bullous Pemphigoid
- Interventions
- Registration Number
- NCT05267600
- Lead Sponsor
- argenx
- 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).
Other than differences in main goals, endpoints, and statistical analyses, parts A and B are identical in schedule, structure, assessments, and conduct.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 98
-
The participant is willing and able to do the following:
- understand the requirements of the study
- provide written informed consent
- 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.
- 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.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description efgartigimod PH20 SC Prednisone participants receiving efgartigimod PH20 SC on top of Prednisone placebo PH20 SC placebo participants receiving placebo PH20 SC on top of Prednisone efgartigimod PH20 SC efgartigimod PH20 SC participants receiving efgartigimod PH20 SC on top of Prednisone placebo PH20 SC Prednisone participants receiving placebo PH20 SC on top of Prednisone
- Primary Outcome Measures
Name Time Method Proportion of participants who are in complete remission (CR) while receiving efgartigimod PH20 SC or placebo and have been off oral corticosteroid (OCS) therapy for ≥8 weeks at week 36 at week 36
- Secondary Outcome Measures
Name Time Method Cumulative dose of oral corticosteroid (OCS) from baseline to week 36 up to week 36 EuroQol 5-Dimension 5-Level (EQ-5D-5L) scores over time up to week 36 Proportion of participants who achieve an Investigator Global Assessment of Bullous Pemphigoid (IGA-BP) score of 0 while receiving efgartigimod PH20 SC or placebo and have been off oral corticosteroid (OCS) therapy for ≥8 weeks at week 36 at week 36 Proportion of participants who achieve an Investigator Global Assessment of Bullous Pemphigoid (IGA-BP) score of 0 or 1 while receiving efgartigimod PH20 SC or placebo and have been off oral corticosteroid (OCS) therapy for ≥8 weeks at week 36 at week 36 Proportion of participants who achieve an Investigator Global Assessment of Bullous Pemphigoid (IGA-BP) score of 0 or 1 while receiving efgartigimod PH20 SC or placebo at any time through week 36 up to week 36 Changes from baseline in the Bullous Pemphigoid Disease Area Index (BPDAI) activity score up to week 36 Proportion of participants who are in complete remission (CR) while receiving efgartigimod PH20 SC or placebo and have been receiving minimal oral corticosteroid (OCS) therapy for ≥8 weeks at week 36 at week 36 Minimal oral corticosteroid (OCS) therapy is defined as ≤0.1 mg/kg/day of prednisone (or an equivalent dose of another OCS).
Time to achieve control of disease activity (CDA) up to week 36 Time to achieve complete remission (CR) up to week 36 Time to achieve complete remission (CR) while on minimal oral corticosteroid (OCS) therapy for ≥8 weeks up to week 36 Time to achieve complete remission (CR)/partial remission (PR) while off oral corticosteroid (OCS) therapy for ≥8 weeks up to week 36 Time to achieve complete remission (CR) while off oral corticosteroid (OCS) therapy for ≥8 weeks up to week 36 Time to achieve relapse up to week 36 Cumulative oral corticosteroid (OCS) dose for the participant at the time points when they exhibit control of disease activity (CDA) up to week 36 Cumulative oral corticosteroid (OCS) dose for the participant at the time points when they exhibit complete remission (CR) up to week 36 Cumulative oral corticosteroid (OCS) dose for the participant at the time points when they exhibit complete remission (CR) while on minimal oral corticosteroid (OCS) therapy for ≥8 weeks up to week 36 Cumulative oral corticosteroid (OCS) dose for the participant at the time points when they exhibit complete remission (CR)/partial remission (PR) while off oral corticosteroid (OCS) therapy for ≥8 weeks up to week 36 Cumulative oral corticosteroid (OCS) dose for the participant at the time points when they exhibit complete remission (CR) while off oral corticosteroid (OCS) therapy for ≥8 weeks up to week 36 Cumulative oral corticosteroid (OCS) dose for the participant at the time points when they exhibit relapse up to week 36 Proportion of participants who receive rescue therapy before week 36 at week 36 Proportion of participants who achieve control of disease activity (CDA) while receiving efgartigimod PH20 SC or placebo and remain free of relapse through week 36 up to week 36 Changes from baseline in the 24-hour average itch score from the Itch Numerical Rating Scale (Itch NRS) up to week 36 Changes from baseline in the 24-hour worst itch score from the Itch Numerical Rating Scale (Itch NRS) up to week 36 Incidence of treatment emergent adverse events (TEAEs) up to 46 weeks Severity of treatment emergent adverse events (TEAEs) up to 46 weeks Incidence of adverse events of special interest (AESIs) up to 46 weeks Severity of adverse events of special interest (AESIs) up to 46 weeks Incidence of serious adverse events (SAEs) up to 46 weeks Severity of serious adverse events (SAEs) up to 46 weeks The Aggregate Improvement Score (AIS) from the Glucocorticoid Toxicity Index (GTI) up to week 36 The Cumulative Worsening Score (CWS) from the Glucocorticoid Toxicity Index (GTI) up to week 36 The Glucocorticoid Toxicity Index Specific List (GTI-SL) up to week 36 Dermatology Life Quality Index (DLQI) scores over time up to week 36 Autoimmune Bullous Disease Quality of Life (ABQoL) scores over time up to week 36 Efgartigimod serum concentrations up to week 43 Percent change of total IgG serum levels from baseline over time up to 46 weeks Percent change of Anti-BP180 and anti-BP230 antibodies from baseline over time up to 46 weeks Incidence of Antidrug antibodies (ADA) against efgartigimod (in serum) and antibodies produced against rHuPH20 (in plasma) up to 46 weeks Number of participants (or their caregivers) who complete the (self-)administration training at study sites up to week 32 Percentage of participants (or their caregivers) who complete the (self-)administration training at study sites up to week 32 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 Percentage 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 (or their caregivers) who successfully (self-)administer efgartigimod PH20 SC under site staff supervision up to week 35 Percentage of participants (or their caregivers) who successfully (self-)administer efgartigimod PH20 SC under site staff supervision up to week 35
Trial Locations
- Locations (129)
Investigator site 121 - US0010092
🇺🇸Redwood City, California, United States
Investigator site 115 - US0010157
🇺🇸Jackson, Mississippi, United States
Investigator site 34 - US0010182
🇺🇸Houston, Texas, United States
Investigator site 108 - CN0860023
🇨🇳Fujian, China
Investigator site 91 - CN0860017
🇨🇳Beijing, China
Investigator site 107 - CN0860021
🇨🇳Guangzhou, China
Investigator site 128 - CN0860097
🇨🇳Hefei, China
Investigator site 109 - CN0860025
🇨🇳Wuhan, China
Investigator site 15 - BG3590020
🇧🇬Stara Zagora, Bulgaria
Investigator site 11 - HU0360003
🇭🇺Debrecen, Hungary
Investigator site 84 - RS3810010
🇷🇸Belgrad, Serbia
Investigator site 113 - SK4210002
🇸🇰Bratislava, Slovakia
Investigator site 59 - GR0300005
🇬🇷Thessaloníki, Greece
Investigator 68 - RS381011
🇷🇸Belgrade, Serbia
Investigator site 67 - RS3810012
🇷🇸Niš, Serbia
Investigator site 76 - GR030003
🇬🇷Chaïdári, Greece
Investigator site 88 - LV3710004
🇱🇻Riga, Latvia
Investigator site 120 - SK4210004
🇸🇰Košice, Slovakia
Investigator site 19 - PL0480046
🇵🇱Wrocław, Poland
Investigator site 2 - US0010087
🇺🇸Boca Raton, Florida, United States
Investigator site 10 - US0010153
🇺🇸Castle Rock, Colorado, United States
Investigator site 25 - US0010158
🇺🇸Pittsburgh, Pennsylvania, United States
Investigator site 5 - US0010088
🇺🇸Buffalo, New York, United States
Investigator site 85 - US0010159
🇺🇸Lebanon, New Hampshire, United States
Investigator site 4 - US0010137
🇺🇸Fairborn, Ohio, United States
Investigator site 28 - BG3590018
🇧🇬Sofia, Bulgaria
Investigator site 14 - BG3590010
🇧🇬Sofia, Bulgaria
Investigator site 3 - US0010151
🇺🇸Morgantown, West Virginia, United States
Investigator site 27 - AU0610006
🇦🇺Kogarah, Australia
Investigator site 125 - AU0610019
🇦🇺Woolloongabba, Australia
Investigator site 116 - CN0860027
🇨🇳Chongqing, China
Investigator site 118 - CZ4200016
🇨🇿Hradec Králové, Czechia
Investigator site 124 - CN0860098
🇨🇳Nanchang, China
Investigator site 94 - CN0860066
🇨🇳Nanyang, China
Investigator site 127 - CN860020
🇨🇳Shangai, China
Investigator site 123 - CN0860095
🇨🇳Shanghai, China
Investigator site 126 - CN0860026
🇨🇳Zhengzhou, China
Investigator site 37 - HR3850001
🇭🇷Zagreb, Croatia
Investigator site 38 - FR0330040
🇫🇷Nice, France
Investigator site 29 - FR0330029
🇫🇷Rouen, France
Investigator site 97 - CZ4200015
🇨🇿Brno, Czechia
Investigator site 45 - DE0490001
🇩🇪Marburg, Germany
Investigator site 60 - GE0300004
🇬🇷Athens, Greece
Investigator site 58 - GR0300001
🇬🇷Athens, Greece
Investigator site 61 - GE0300002
🇬🇷Thessaloníki, Greece
Investigator site 55 - DE0490026
🇩🇪Würzburg, Germany
Investigator site 51 - DE0490008
🇩🇪Essen, Germany
Investigator site 75 - DE0490047
🇩🇪München, Germany
Investigator site 63 - IL9720018
🇮🇱Haifa, Israel
Investigator site 39 - IL9720003
🇮🇱Afula, Israel
Investigator site 129 - JP0810073
🇯🇵Niigata, Japan
Investigator site 81 - IT0390030
🇮🇹Genova, Italy
Investigator site 77 - IT0390062
🇮🇹Milano, Italy
Investigator site 99 - JP0810050
🇯🇵Kurume, Japan
Investigator site 87 - LV3710005
🇱🇻Riga, Latvia
Investigator site 82 - LV3710003
🇱🇻Riga, Latvia
Investigator site 66 - NL0310015
🇳🇱Groningen, Netherlands
Investigator site 102 - JP0810069
🇯🇵Ōsaka-sayama, Japan
Investigator site 79 - PL0480025
🇵🇱Rzeszów, Poland
Investigator site 83 - PL0480050
🇵🇱Warsaw, Poland
Investigator site 18 - PL0480048
🇵🇱Wrocław, Poland
Investigator site 17 - PL0480047
🇵🇱Łódź, Poland
Investigator site 90 - RO0400014
🇷🇴Cluj-Napoca, Romania
Investigator site 89 - RO0400015
🇷🇴Iaşi, Romania
Investigator site 12 - ES0340025
🇪🇸Madrid, Spain
Investigator 20 - ES0340029
🇪🇸Madrid, Spain
Investigator site 44 - UK0440037
🇬🇧Southampton, United Kingdom
Investigator site 49 - ES0340058
🇪🇸Manises, Spain
Investigator site 70 - ES0340061
🇪🇸Valencia, Spain
Investigator site 9 - ES0340052
🇪🇸Sevilla, Spain
Investigator site 43 - IT0390060
🇮🇹Brescia, Italy
Investigator site 64 - IT0390061
🇮🇹Pavia, Italy
Investigator site 30 - IT0390040
🇮🇹Siena, Italy
Investigator site 23 - IT0390006
🇮🇹Roma, Italy
Investigator site 42 - IT0390005
🇮🇹Roma, Italy
Investigator site 33 - UK0440022
🇬🇧Bristol, United Kingdom
Investigator site 103 - JP0810070
🇯🇵Kumamoto, Japan
Investigator site 104 - JP0810071
🇯🇵Maebashi, Japan
Investigator site 100 - JP0810046
🇯🇵Nagakute, Japan
Investigator site 101 - JP0810049
🇯🇵Osaka, Japan
Investigator site 112 - JP0810045
🇯🇵Sapporo, Japan
Investigator site 105 - JP0810067
🇯🇵Sendai, Japan
Investigator site 74 - US0010178
🇺🇸Phoenix, Arizona, United States
Investigator site 6 - US0010138
🇺🇸Fountain Valley, California, United States
Investigator site 72 - US0010186
🇺🇸Santa Monica, California, United States
Investigator site 21 - US0010152
🇺🇸Clearwater, Florida, United States
Investigator site 13 - US0010155
🇺🇸West Lafayette, Indiana, United States
Investigator site 1 - US0010017
🇺🇸Miami, Florida, United States
Investigator site 73 - US0010098
🇺🇸Saint Louis, Missouri, United States
Investigator site 93 - US0010169
🇺🇸New York, New York, United States
Investigator site 92 - US0010150
🇺🇸Murray, Utah, United States
Investigator site 111 - CN0860064
🇨🇳Beijing, China
Investigator site 95 - CN0860018
🇨🇳Chengdu, China
Investigator site 110 - CN0860053
🇨🇳Guanzhou, China
Investigator site 122 - CN0860065
🇨🇳Ürümqi, China
Investigator site 22 - HR3850003
🇭🇷Split, Croatia
Investigator site 16 - HR3850002
🇭🇷Zagreb, Croatia
Investigator site 117 - CZ4200013
🇨🇿Plzen-Bory, Czechia
Investigator site 56 - DE0490028
🇩🇪Kiel, Germany
Investigator site 96 - CZ4200012
🇨🇿Prague, Czechia
Investigator site 80 - DE0490041
🇩🇪Düsseldorf, Germany
Investigator site 46 - DE0490039
🇩🇪Berlin, Germany
Investigator site 52 - DE0490024
🇩🇪Frankfurt am main, Germany
Investigator site 57 - DE0490046
🇩🇪Erlangen, Germany
Investigator site 54 - DE0490030
🇩🇪Dresden, Germany
Investigator site 53 - DE0490023
🇩🇪Freiburg, Germany
Investigator site 62 - GE0300006
🇬🇷Athens, Greece
Investigator site 26 - HU0360023
🇭🇺Budapest, Hungary
Investigator site 7 - HU0360008
🇭🇺Pécs, Hungary
Investigator site 41 - IL9720001
🇮🇱Ramat Gan, Israel
Investigator site 65 - IT0390055
🇮🇹Bologna, Italy
Investigator site 40 - IL9720002
🇮🇱Tel Aviv, Israel
Investigator site 78 - IT0390039
🇮🇹Catania, Italy
Investigator site 47 - IT0390031
🇮🇹Firenze, Italy
Investigator site 86 - IT0390067
🇮🇹Firenze, Italy
Investigator site 114 - SK4210003
🇸🇰Trnava, Slovakia
Investigator site 119 - IT0390066
🇮🇹Parma, Italy
Investigator site 98 - JP0810043
🇯🇵Tokyo, Japan
Investigator site 106 - JP0810068
🇯🇵Yokohama, Japan
Investigator site 32 - ES0340050
🇪🇸Badalona, Spain
Investigator site 69 - RS3810009
🇷🇸Novi Sad, Serbia
Investigator 24 - ES0340051
🇪🇸Barcelona, Spain
Investigator site 8 - ES0340053
🇪🇸Granada, Spain
Investigator site 48 - ES0340059
🇪🇸Mieres, Spain
Investigator site 31 - ES0340057
🇪🇸Málaga, Spain
Investigator site 71 - UK0440036
🇬🇧London, United Kingdom
Investigator site 36 - AU0610013
🇦🇺Fitzroy, Australia
Investigator site 50 - US0010149
🇺🇸Ann Arbor, Michigan, United States
Investigator site 35 - US0010156
🇺🇸Louisville, Kentucky, United States