Clinical trial NCT05681481: A Phase 3 Study to Evaluate the Long-term Safety, Tolerability and Efficacy of Efgartigimod PH20 SC in Adult Participants With Bullous Pemphigoid
Official title: An Open-label Extension Study of ARGX-113-2009 to Evaluate the Long Term Safety, Tolerability, and Efficacy of Efgartigimod PH20 SC in Adult Participants With Bullous Pemphigoid
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE3
Conditions: Bullous Pemphigoid
Lead sponsor: argenx
Sponsor class: INDUSTRY
First posted: 2023-01-12
Enrollment: 64
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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


Eligibility criteria: Inclusion Criteria:

* 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

Exclusion Criteria:

* 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

Primary outcomes:
measure: Incidence of treatment-emergent adverse events, serious adverse events and adverse events of special interest
timeFrame: Up to 56 weeks
measure: Rate of treatment discontinuation because of safety concerns
timeFrame: Up to 56 weeks
Secondary outcomes:
measure: Proportion of participants achieving complete remission while off oral corticosteroids for ≥ 8 weeks
timeFrame: Up to 56 weeks
measure: Proportion of participants achieving complete remission or partial remission while off oral corticosteroids for ≥ 8 weeks
timeFrame: Up to 56 weeks
measure: Proportion of participants achieving complete remission while on minimal oral corticosteroids therapy for ≥ 8 weeks
timeFrame: Up to 56 weeks
measure: Proportion of participants achieving complete remission while off both oral corticosteroids and efgartigimod PH20 SC for ≥ 8 weeks
timeFrame: Up to 56 weeks
measure: Proportion of participants achieving complete remission or partial remission while off both oral corticosteroids and efgartigimod PH20 SC for ≥ 8 weeks
timeFrame: Up to 56 weeks
measure: Duration of sustained remission
timeFrame: Up to 56 weeks
measure: Proportion of participants who relapse
timeFrame: Up to 56 weeks
measure: Time to relapse
timeFrame: Up to 56 weeks
measure: Incidence of relapse
timeFrame: Up to 56 weeks
measure: BPDAI activity scores over time
timeFrame: For participants not requiring treatment with efgartigimod PH20 SC at rollover: at weeks 0, 2, 4, 8, 16, 24, 32, 40, 48 and 56.
measure: BPDAI activity scores over time
timeFrame: 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.
measure: IGA-BP scores over time
timeFrame: For participants not requiring treatment with efgartigimod PH20 SC at rollover: at weeks 0, 2, 4, 8, 16, 24, 32, 40, 48 and 56.
measure: IGA-BP scores over time
timeFrame: 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.
measure: Itch NRS over time
timeFrame: For participants not requiring treatment with efgartigimod PH20 SC at rollover: at weeks 0, 2, 4, 8, 16, 24, 32, 40, 48 and 56.
measure: Itch NRS over time
timeFrame: 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.
measure: Rate of treatment failure
timeFrame: Up to 56 weeks
measure: Glucocorticoid Toxicity Index (GTI)-related scores, including the Glucocorticoid Toxicity Index - Aggregate Improvement Score (GTI-AIS) over time
timeFrame: For participants not requiring treatment with efgartigimod PH20 SC at rollover: at weeks 0, 24 and 48.
measure: Glucocorticoid Toxicity Index (GTI)-related scores, including the Glucocorticoid Toxicity Index - Aggregate Improvement Score (GTI-AIS) over time
timeFrame: 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.
measure: Glucocorticoid Toxicity Index (GTI)-related scores, including the Glucocorticoid Toxicity Index Cumulative Worsening Score (GTI-CWS) over time
timeFrame: For participants not requiring treatment with efgartigimod PH20 SC at rollover: at weeks 0, 24 and 48.
measure: Glucocorticoid Toxicity Index (GTI)-related scores, including the Glucocorticoid Toxicity Index Cumulative Worsening Score (GTI-CWS) over time
timeFrame: 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.
measure: Glucocorticoid Toxicity Index (GTI)-related scores, including the Glucocorticoid Toxicity Index Specific List (GTI-SL) over time
timeFrame: For participants not requiring treatment with efgartigimod at rollover: at weeks 0, 24 and 48.
measure: Glucocorticoid Toxicity Index (GTI)-related scores, including the Glucocorticoid Toxicity Index Specific List (GTI-SL) over time
timeFrame: 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.
measure: EQ-5D-5L scores over time
timeFrame: For participants not requiring treatment with efgartigimod PH20 SC at rollover: at weeks 0, 24 and 48.
measure: EQ-5D-5L scores over time
timeFrame: 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.
measure: ABQoL scores over time
timeFrame: For participants not requiring treatment with efgartigimod PH20 SC at rollover: at weeks 0, 24 and 48.
measure: ABQoL scores over time
timeFrame: 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.
measure: DLQI scores over time
timeFrame: For participants not requiring treatment with efgartigimod PH20 SC at rollover: at weeks 0, 24 and 48.
measure: DLQI scores over time
timeFrame: 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.
measure: Percent changes from baseline over time for anti-BP180 and anti-BP-230 antibody levels
timeFrame: For participants not requiring treatment with efgartigimod PH20 SC at rollover: at weeks 0, 2, 4, 8, 16, 24, 32, 40, 48 and 56.
measure: Percent changes from baseline over time for anti-BP180 and anti-BP-230 antibody levels
timeFrame: 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.
measure: Incidence of antidrug antibody(ies) (ADA) against efgartigimod (serum levels)
timeFrame: For participants not requiring treatment with efgartigimod PH20 SC at rollover: at weeks 0, 4, 8, 16, 24, 32, 40, 48 and 56.
measure: Incidence of antidrug antibody(ies) (ADA) against efgartigimod (serum levels)
timeFrame: 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.
Other outcomes:
Locations:
city: Phoenix
country: United States
facility: Medical Dermatology Specialists
state: Arizona
city: Fountain Valley
country: United States
facility: First OC Dermatology
state: California
city: Miami
country: United States
facility: Miami Dermatology and Laser Institute
state: Florida
city: Ann Arbor
country: United States
facility: University of Michigan Hospital
state: Michigan
city: Saint Louis
country: United States
facility: Saint Louis University
state: Missouri
city: Fairborn
country: United States
facility: Wright State Physicians
state: Ohio
city: Kogarah
country: Australia
facility: Premier Specialists
city: Sofia
country: Bulgaria
facility: Diagnostic and Consulting Center Aleksandrovska EOOD
city: Chengdu
country: China
facility: West China Hospital of Sichuan University
city: Chongqing
country: China
facility: The First Affiliated Hospital of Chongqing Medical University
city: Shanghai
country: China
facility: Ruijin Hospital Shanghai Jiaotong University School of Medicine
city: Zagreb
country: Croatia
facility: Poliklinika Solmed
city: Praha
country: Czechia
facility: Fakultni nemocnice Bulovka
city: Berlin
country: Germany
facility: Charité - Universitätsmedizin Berlin
city: Dresden
country: Germany
facility: Universitätsklinikum Carl Gustav Carus an der TU Dresden
city: Düsseldorf
country: Germany
facility: Universitatsklinikum Dusseldorf
city: Kiel
country: Germany
facility: Universitatsklinikum Schleswig-Holstein
city: München
country: Germany
facility: LMU Klinikum der Universität
city: Würzburg
country: Germany
facility: Universitätsklinikum Würzburg
city: Athens
country: Greece
facility: Hospital of Venereal and Skin Diseases A.Syggros
city: Thessaloníki
country: Greece
facility: Hospital Of Skin And Venereal Diseases of Thessaloniki
city: Budapest
country: Hungary
facility: Semmelweis Egyetem
city: Ramat Gan
country: Israel
facility: Sheba Medical Center - PPDS
city: Catania
country: Italy
facility: Azienda Ospedaliero Universitaria Policlinico Vittorio Emanuele
city: Firenze
country: Italy
facility: Azienda USL Toscana Centro - Ospidale Piero Palagi
city: Firenze
country: Italy
facility: Azienda Sanitaria Di Firenze
city: Genova
country: Italy
facility: Ospedale Policlinico San Martino
city: Milano
country: Italy
facility: Fondazione IRCCS Cà Granda Ospedale Maggiore Policlinico
city: Pavia
country: Italy
facility: Fondazione IRCCS Policlinico San Matteo di Pavia
city: Roma
country: Italy
facility: IDI IRCCS - Istituto Dermopatico dell'Immacolata
city: Rome
country: Italy
facility: Fondazione Policlinico Universitario A. Gemelli
city: Sapporo
country: Japan
facility: Hokkaido University Hospital
city: Groningen
country: Netherlands
facility: Universitair Medisch Centrum Groningen
city: Belgrade
country: Serbia
facility: University Clinical Center of Serbia - PPDS
city: Bratislava
country: Slovakia
facility: Univerzitna nemocnica Bratislava
city: Trnava
country: Slovakia
facility: Fakultna nemocnica Trnava
city: Granada
country: Spain
facility: Hospital Universitario Clínico San Cecilio
city: Madrid
country: Spain
facility: Hospital Universitario 12 de Octubre
city: Valencia
country: Spain
facility: Hospital Universitario Doctor Peset
city: London
country: United Kingdom
facility: Guy's and St Thomas' NHS Foundation Trust
Study officials:
References: