A Phase 3 Study to Evaluate the Long-term Safety, Tolerability and Efficacy of Efgartigimod PH20 SC in Adult Participants With Bullous Pemphigoid

Phase 3TerminatedNCT05681481

argenx64 enrolled

Overview

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

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Bullous Pemphigoid

Interventions

Eligibility

Sex: ALLMin age: 18 Years

Medical Language ↔ Plain English

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

Locations (40)

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

St Louis, Missouri, United States

Outcomes

Primary Outcomes

Incidence of treatment-emergent adverse events, serious adverse events and adverse events of special interest

Time frame: Up to 56 weeks

Rate of treatment discontinuation because of safety concerns

Time frame: Up to 56 weeks

Secondary Outcomes

Proportion of participants achieving complete remission while off oral corticosteroids for ≥ 8 weeks

Time frame: Up to 56 weeks

Proportion of participants achieving complete remission or partial remission while off oral corticosteroids for ≥ 8 weeks

Time frame: Up to 56 weeks

Proportion of participants achieving complete remission while on minimal oral corticosteroids therapy for ≥ 8 weeks

Minimal oral corticosteroid therapy is defined as ≤0.10 mg/kg/day of prednisone (or an equivalent dose of another oral corticosteroid)

Time frame: Up to 56 weeks

Proportion of participants achieving complete remission while off both oral corticosteroids and efgartigimod PH20 SC for ≥ 8 weeks

Time frame: 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

Time frame: Up to 56 weeks

Duration of sustained remission

Time frame: Up to 56 weeks

Proportion of participants who relapse

Time frame: Up to 56 weeks

Time to relapse

Time frame: Up to 56 weeks

Incidence of relapse

Time frame: Up to 56 weeks

BPDAI activity scores over time

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

Time frame: For participants not requiring treatment with efgartigimod PH20 SC at rollover: at weeks 0, 2, 4, 8, 16, 24, 32, 40, 48 and 56.

BPDAI activity scores over time

Time frame: 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.

IGA-BP scores over time

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).

Time frame: For participants not requiring treatment with efgartigimod PH20 SC at rollover: at weeks 0, 2, 4, 8, 16, 24, 32, 40, 48 and 56.

IGA-BP scores over time

Itch NRS over time

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)

Time frame: For participants not requiring treatment with efgartigimod PH20 SC at rollover: at weeks 0, 2, 4, 8, 16, 24, 32, 40, 48 and 56.

Itch NRS over time

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

Time frame: Up to 56 weeks

Glucocorticoid Toxicity Index (GTI)-related scores, including the Glucocorticoid Toxicity Index - Aggregate Improvement Score (GTI-AIS) over time

Time frame: For participants not requiring treatment with efgartigimod PH20 SC at rollover: at weeks 0, 24 and 48.

Glucocorticoid Toxicity Index (GTI)-related scores, including the Glucocorticoid Toxicity Index - Aggregate Improvement Score (GTI-AIS) over time

Time frame: 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

Time frame: For participants not requiring treatment with efgartigimod PH20 SC at rollover: at weeks 0, 24 and 48.

Glucocorticoid Toxicity Index (GTI)-related scores, including the Glucocorticoid Toxicity Index Cumulative Worsening Score (GTI-CWS) over time

Time frame: 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

Time frame: For participants not requiring treatment with efgartigimod at rollover: at weeks 0, 24 and 48.

Glucocorticoid Toxicity Index (GTI)-related scores, including the Glucocorticoid Toxicity Index Specific List (GTI-SL) over time

Time frame: 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.

EQ-5D-5L scores over time

The EQ-5D-5L questionnaire is a patient-reported outcome measure, ranging 0 to 100 (lower score, worse outcome).

Time frame: For participants not requiring treatment with efgartigimod PH20 SC at rollover: at weeks 0, 24 and 48.

EQ-5D-5L scores over time

The EQ-5D-5L questionnaire is a patient-reported outcome measure, ranging 0 to 100 (lower score, worse outcome).

Time frame: 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.

ABQoL scores over time

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.

Time frame: For participants not requiring treatment with efgartigimod PH20 SC at rollover: at weeks 0, 24 and 48.

ABQoL scores over time

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.

Time frame: 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.

DLQI scores over time

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."

Time frame: For participants not requiring treatment with efgartigimod PH20 SC at rollover: at weeks 0, 24 and 48.

DLQI scores over time

Time frame: 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.

Percent changes from baseline over time for anti-BP180 and anti-BP-230 antibody levels

Time frame: For participants not requiring treatment with efgartigimod PH20 SC at rollover: at weeks 0, 2, 4, 8, 16, 24, 32, 40, 48 and 56.

Percent changes from baseline over time for anti-BP180 and anti-BP-230 antibody levels

Time frame: 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.

Incidence of antidrug antibody(ies) (ADA) against efgartigimod (serum levels)

Time frame: For participants not requiring treatment with efgartigimod PH20 SC at rollover: at weeks 0, 4, 8, 16, 24, 32, 40, 48 and 56.

Incidence of antidrug antibody(ies) (ADA) against efgartigimod (serum levels)

Time frame: 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.