A Phase 3 Study to Evaluate the Long-term Safety, Tolerability and Efficacy of Efgartigimod PH20 SC in Adult Participants With Bullous Pemphigoid
- Conditions
- Bullous Pemphigoid
- Interventions
- Biological: efgartigimod PH20 SC
- Registration Number
- NCT05681481
- Lead Sponsor
- argenx
- Brief Summary
The purpose of this study is to evaluate the safety of efgartigimod PH20 SC over a longer period of time in adult participants with moderate-to-severe bullous pemphigoid (BP) who have completed ARGX-113-2009 study. The study will also evaluate the efficacy of efgartigimod PH20 SC.
Eligible participants can roll over from the main study (ARGX-113-2009) to this open-label extension study (ARGX-113-2010). The study consists of a treatment period of up to 48 weeks in which participants receive efgartigimod PH20 SC. After the first 5 visits, the participants will visit the study centers at least once every 4 weeks. The participants who are not receiving efgartigimod PH20 SC (after the main study or currently on the study), will enter an observation period with study visits at least once every 8 weeks. If the participant relapses, they can re-enter the treatment period where they will receive efgartigimod PH20 SC. The treatment and observation period is followed by a follow-up period of 8 weeks. Oral or topical corticosterioids can be administered at the investigator's indiscretion
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- TERMINATED
- Sex
- All
- Target Recruitment
- 64
- Has completed the week 36 visit of ARGX-113-2009
- Is capable of providing signed informed consent and complying with protocol requirements
- Agrees to use contraceptive measures consistent with local regulations and the following: Women of childbearing potential must have a negative urine pregnancy test at baseline before receiving the study drug and must use one of the contraception methods described in the protocol from signing the ICF until the last dose of the study drug
- Clinically significant disease, recent major surgery (within 3 months of baseline), or intends to have surgery during the study; or any other medical condition that, in the investigator's opinion would confound the results of the study or put the participant at undue risk
- Known hypersensitivity to the study drug or 1 of its excipients
- Permanently discontinued IMP in ARGX-113-2009 due to an adverse event (AE) considered related to the study drug and for whom the benefit/risk balance is not considered positive
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description efgartigimod PH20 SC efgartigimod PH20 SC participants receiving efgartigimod PH20 SC on top of Prednisone efgartigimod PH20 SC Prednisone participants receiving efgartigimod PH20 SC on top of Prednisone
- Primary Outcome Measures
Name Time Method Incidence of treatment-emergent adverse events, serious adverse events and adverse events of special interest Up to 56 weeks Rate of treatment discontinuation because of safety concerns Up to 56 weeks Rate of treatment discontinuation because of safety concerns
- Secondary Outcome Measures
Name Time Method Proportion of participants achieving complete remission while off oral corticosteroids for ≥ 8 weeks Up to 56 weeks Proportion of participants achieving complete remission while off oral corticosteroids for ≥ 8 weeks
Proportion of participants achieving complete remission or partial remission while off oral corticosteroids for ≥ 8 weeks Up to 56 weeks Proportion of participants achieving complete remission or partial remission while off oral corticosteroids for ≥ 8 weeks
Incidence of antidrug antibody(ies) (ADA) against efgartigimod (serum levels) For participants continuing/starting efgartigimod treatment at rollover or relapse: at weeks 0, 2, 4 and 8 and then every 8 weeks until and after efgartigimod PH20 SC stop and at weeks 48, 52 and 56. Incidence of antidrug antibody(ies) (ADA) against efgartigimod (serum levels)
Proportion of participants achieving complete remission while on minimal oral corticosteroids therapy for ≥ 8 weeks Up to 56 weeks Minimal oral corticosteroid therapy is defined as ≤0.10 mg/kg/day of prednisone (or an equivalent dose of another oral corticosteroid)
Proportion of participants achieving complete remission while off both oral corticosteroids and efgartigimod PH20 SC for ≥ 8 weeks Up to 56 weeks Proportion of participants achieving complete remission while off both oral corticosteroids and efgartigimod PH20 SC for ≥ 8 weeks
Proportion of participants achieving complete remission or partial remission while off both oral corticosteroids and efgartigimod PH20 SC for ≥ 8 weeks Up to 56 weeks Proportion of participants achieving complete remission or partial remission while off both oral corticosteroids and efgartigimod PH20 SC for ≥ 8 weeks
Duration of sustained remission Up to 56 weeks Duration of sustained remission
Proportion of participants who relapse Up to 56 weeks Proportion of participants who relapse
Time to relapse Up to 56 weeks Time to relapse
Incidence of relapse Up to 56 weeks Incidence of relapse
BPDAI activity scores over time For participants continuing/starting efgartigimod PH20 SC treatment at rollover or relapse: at weeks 0, 2, 4 and 8 and then every 4 weeks until efgartigimod stop, every 8 weeks after efgartigimod stop and at weeks 48, 52 and 56. The Bullous Pemphigoid Disease Area Index (BPDAI) is an internationally validated tool to objectively measure disease activity. The BPDAI differentiates scores for skin (erosions/blisters and urticaria/erythema) and mucous membrane activity in several anatomical locations
IGA-BP scores over time For participants continuing/starting efgartigimod PH20 SC treatment at rollover or relapse: at weeks 0, 2, 4 and 8 and then every 4 weeks until efgartigimod stop, every 8 weeks after efgartigimod stop and at weeks 48, 52 and 56. The Investigator Global Assessment of Bullous Pemphigoid (IGA-BP) is a tool used to asses BP disease activity and severity. The IGA-BP categorizes the severity of BP on a numerical scale of 0 (clear) to 4 (severe).
Itch NRS over time For participants continuing/starting efgartigimod PH20 SC treatment at rollover or relapse: at weeks 0, 2, 4 and 8 and then every 4 weeks until efgartigimod stop, every 8 weeks after efgartigimod stop and at weeks 48, 52 and 56. The Itch Numerical Rating Scale (NRS) is used to indicate pruritic symptoms of BP. The score varies between 0 (best outcome) to 10 (worst outcome)
Rate of treatment failure Up to 56 weeks Rate of treatment failure
Glucocorticoid Toxicity Index (GTI)-related scores, including the Glucocorticoid Toxicity Index - Aggregate Improvement Score (GTI-AIS) over time For participants continuing/starting efgartigimod treatment at rollover or relapse: weeks 0, 8, every 16 weeks until and after efgartigimod treatment stop and at week 48. Glucocorticoid Toxicity Index (GTI)-related scores, including the Glucocorticoid Toxicity Index - Aggregate Improvement Score (GTI-AIS) over time
Glucocorticoid Toxicity Index (GTI)-related scores, including the Glucocorticoid Toxicity Index Cumulative Worsening Score (GTI-CWS) over time For participants continuing/starting efgartigimod treatment at rollover or relapse: weeks 0, 8, every 16 weeks until and after efgartigimod treatment stop and at week 48. Glucocorticoid Toxicity Index (GTI)-related scores, including the Glucocorticoid Toxicity Index Cumulative Worsening Score (GTI-CWS) over time
Glucocorticoid Toxicity Index (GTI)-related scores, including the Glucocorticoid Toxicity Index Specific List (GTI-SL) over time For participants continuing/starting efgartigimod treatment at rollover or relapse: weeks 0, 8, every 16 weeks until and after efgartigimod treatment stop and at week 48. Glucocorticoid Toxicity Index (GTI)-related scores, including the Glucocorticoid Toxicity Index Specific List (GTI-SL) over time
EQ-5D-5L scores over time For participants continuing/starting efgartigimod treatment at rollover or relapse: at weeks 0, 8, every 16 weeks until and after efgartigimod PH20 SC treatment stop and at week 48. The EQ-5D-5L questionnaire is a patient-reported outcome measure, ranging 0 to 100 (lower score, worse outcome).
ABQoL scores over time For participants continuing/starting efgartigimod treatment at rollover or relapse: at weeks 0, 8, every 16 weeks until and after efgartigimod PH20 SC treatment stop and at week 48. The Autoimmune Bullous Disease Quality of Life (ABQoL) was developed and validated for determining the impact of AIBDs and their therapies on the daily lives of patients.
DLQI scores over time For participants continuing/starting efgartigimod treatment at rollover or relapse: at weeks 0, 8, every 16 weeks until and after efgartigimod PH20 SC treatment stop and at week 48. The Dermatology Life Quality Index (DLQI) consists of 10 questions concerning the participant's perception of the impact of skin diseases on different aspects of their health-related QoL the previous week. The impact of each aspect on the QoL assessment is scored qualitatively, ranging from "not at all" to "very much."
Percent changes from baseline over time for anti-BP180 and anti-BP-230 antibody levels For participants continuing/starting efgartigimod PH20 SC treatment at rollover or relapse: at weeks 0, 2, 4 and 8 and then every 4 weeks to efgartigimod stop, every 8 weeks after efgartigimod stop and at weeks 48, 52 and 56. Percent changes from baseline over time for anti-BP180 and anti-BP-230 antibody levels
Related Research Topics
Explore scientific publications, clinical data analysis, treatment approaches, and expert-compiled information related to the mechanisms and outcomes of this trial. Click any topic for comprehensive research insights.
Trial Locations
- Locations (40)
The First Affiliated Hospital of Chongqing Medical University
🇨🇳Chongqing, China
Ruijin Hospital Shanghai Jiaotong University School of Medicine
🇨🇳Shanghai, China
Medical Dermatology Specialists
🇺🇸Phoenix, Arizona, United States
First OC Dermatology
🇺🇸Fountain Valley, California, United States
Miami Dermatology and Laser Institute
🇺🇸Miami, Florida, United States
University of Michigan Hospital
🇺🇸Ann Arbor, Michigan, United States
Saint Louis University
🇺🇸Saint Louis, Missouri, United States
Wright State Physicians
🇺🇸Fairborn, Ohio, United States
Premier Specialists
🇦🇺Kogarah, Australia
Diagnostic and Consulting Center Aleksandrovska EOOD
🇧🇬Sofia, Bulgaria
West China Hospital of Sichuan University
🇨🇳Chengdu, China
Poliklinika Solmed
🇭🇷Zagreb, Croatia
Fakultni nemocnice Bulovka
🇨🇿Praha, Czechia
Charité - Universitätsmedizin Berlin
🇩🇪Berlin, Germany
Universitätsklinikum Carl Gustav Carus an der TU Dresden
🇩🇪Dresden, Germany
Universitatsklinikum Dusseldorf
🇩🇪Düsseldorf, Germany
Universitatsklinikum Schleswig-Holstein
🇩🇪Kiel, Germany
LMU Klinikum der Universität
🇩🇪München, Germany
Universitätsklinikum Würzburg
🇩🇪Würzburg, Germany
Hospital of Venereal and Skin Diseases A.Syggros
🇬🇷Athens, Greece
Hospital Of Skin And Venereal Diseases of Thessaloniki
🇬🇷Thessaloníki, Greece
Semmelweis Egyetem
🇭🇺Budapest, Hungary
Sheba Medical Center - PPDS
🇮🇱Ramat Gan, Israel
Azienda Ospedaliero Universitaria Policlinico Vittorio Emanuele
🇮🇹Catania, Italy
Azienda USL Toscana Centro - Ospidale Piero Palagi
🇮🇹Firenze, Italy
Azienda Sanitaria Di Firenze
🇮🇹Firenze, Italy
Fondazione IRCCS Cà Granda Ospedale Maggiore Policlinico
🇮🇹Milano, Italy
Ospedale Policlinico San Martino
🇮🇹Genova, Italy
Fondazione IRCCS Policlinico San Matteo di Pavia
🇮🇹Pavia, Italy
IDI IRCCS - Istituto Dermopatico dell'Immacolata
🇮🇹Roma, Italy
Fondazione Policlinico Universitario A. Gemelli
🇮🇹Rome, Italy
Hokkaido University Hospital
🇯🇵Sapporo, Japan
Universitair Medisch Centrum Groningen
🇳🇱Groningen, Netherlands
University Clinical Center of Serbia - PPDS
🇷🇸Belgrade, Serbia
Univerzitna nemocnica Bratislava
🇸🇰Bratislava, Slovakia
Fakultna nemocnica Trnava
🇸🇰Trnava, Slovakia
Hospital Universitario Clínico San Cecilio
🇪🇸Granada, Spain
Hospital Universitario 12 de Octubre
🇪🇸Madrid, Spain
Hospital Universitario Doctor Peset
🇪🇸Valencia, Spain
Guy's and St Thomas' NHS Foundation Trust
🇬🇧London, United Kingdom