A Phase 2/3 Study of Efgartigimod PH20 SC in Adult Participants with Bullous Pemphigoid

• Register: ClinicalTrials.gov
• Last refreshed on: 21 October 2024
• Main ID: NCT05267600
• Date of registration: 14/02/2022
• Prospective Registration: Yes
• Primary sponsor: argenx
• Public title: A Phase 2/3 Study of Efgartigimod PH20 SC in Adult Participants with Bullous Pemphigoid BALLAD
• Scientific title: A Phase 2/3, Randomized, Double-Blinded, Placebo-Controlled, Parallel-Group Study to Investigate the Efficacy and Safety of Efgartigimod PH20 SC in Adult Participants with Bullous Pemphigoid
• Date of first enrolment: June 9, 2022
• Target sample size: 98
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/ct2/show/NCT05267600
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Double (Participant, Investigator).
• Phase: Phase 2/Phase 3

Countries of recruitment

Australia, Bulgaria, China, Croatia, Czechia, France, Germany, Greece, Hungary, Israel, Italy, Japan, Latvia, Netherlands, Poland, Romania, Serbia, Slovakia, Spain, United Kingdom, United States

Key inclusion & exclusion criteria

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 exclusion criteria can be found in the protocol.


Age minimum: 18 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Bullous Pemphigoid

Intervention(s)

Drug: Prednisone

Other: placebo

Biological: efgartigimod PH20 SC

Primary Outcome(s)

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 [Time Frame: at week 36]

Secondary Outcome(s)

Changes from baseline in the 24-hour worst itch score from the Itch Numerical Rating Scale (Itch NRS) [Time Frame: up to week 36]

Cumulative oral corticosteroid (OCS) dose for the participant at the time points when they exhibit control of disease activity (CDA) [Time Frame: up to week 36]

Time to achieve complete remission (CR) while on minimal oral corticosteroid (OCS) therapy for =8 weeks [Time Frame: 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 [Time Frame: up to week 36]

Incidence of treatment emergent adverse events (TEAEs) [Time Frame: up to 46 weeks]

Number of participants (or their caregivers) who complete the (self-)administration training at study sites [Time Frame: up to week 32]

Percentage of participants (or their caregivers) who successfully (self-)administer efgartigimod PH20 SC under site staff supervision [Time Frame: up to week 35]

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 [Time Frame: 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 [Time Frame: up to week 36]

Severity of serious adverse events (SAEs) [Time Frame: up to 46 weeks]

Time to achieve complete remission (CR) while off oral corticosteroid (OCS) therapy for =8 weeks [Time Frame: up to week 36]

Cumulative oral corticosteroid (OCS) dose for the participant at the time points when they exhibit complete remission (CR) [Time Frame: up to week 36]

Time to achieve relapse [Time Frame: up to week 36]

Autoimmune Bullous Disease Quality of Life (ABQoL) scores over time [Time Frame: up to week 36]

Changes from baseline in the 24-hour average itch score from the Itch Numerical Rating Scale (Itch NRS) [Time Frame: 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 [Time Frame: up to week 36]

Percent change of Anti-BP180 and anti-BP230 antibodies from baseline over time [Time Frame: up to 46 weeks]

Percent change of total IgG serum levels from baseline over time [Time Frame: up to 46 weeks]

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 [Time Frame: up to week 36]

Cumulative oral corticosteroid (OCS) dose for the participant at the time points when they exhibit relapse [Time Frame: up to week 36]

Changes from baseline in the Bullous Pemphigoid Disease Area Index (BPDAI) activity score [Time Frame: up to week 36]

Incidence of adverse events of special interest (AESIs) [Time Frame: up to 46 weeks]

EuroQol 5-Dimension 5-Level (EQ-5D-5L) scores over time [Time Frame: up to week 36]

The Aggregate Improvement Score (AIS) from the Glucocorticoid Toxicity Index (GTI) [Time Frame: up to week 36]

The Glucocorticoid Toxicity Index Specific List (GTI-SL) [Time Frame: up to week 36]

Time to achieve complete remission (CR) [Time Frame: up to week 36]

Efgartigimod serum concentrations [Time Frame: up to week 43]

Incidence of serious adverse events (SAEs) [Time Frame: up to 46 weeks]

Number of participants (or their caregivers) who successfully (self-)administer efgartigimod PH20 SC under site staff supervision [Time Frame: up to week 35]

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 [Time Frame: at week 36]

Incidence of Antidrug antibodies (ADA) against efgartigimod (in serum) and antibodies produced against rHuPH20 (in plasma) [Time Frame: up to 46 weeks]

Severity of treatment emergent adverse events (TEAEs) [Time Frame: up to 46 weeks]

The Cumulative Worsening Score (CWS) from the Glucocorticoid Toxicity Index (GTI) [Time Frame: up to week 36]

Time to achieve control of disease activity (CDA) [Time Frame: up to week 36]

Cumulative dose of oral corticosteroid (OCS) from baseline to week 36 [Time Frame: 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 [Time Frame: up to week 36]

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]

Proportion of participants who receive rescue therapy before week 36 [Time Frame: at week 36]

Dermatology Life Quality Index (DLQI) scores over time [Time Frame: up to week 36]

Percentage 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]

Percentage of participants (or their caregivers) who complete the (self-)administration training at study sites [Time Frame: up to week 32]

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 [Time Frame: at week 36]

Severity of adverse events of special interest (AESIs) [Time Frame: up to 46 weeks]

Time to achieve complete remission (CR)/partial remission (PR) while off oral corticosteroid (OCS) therapy for =8 weeks [Time Frame: up to week 36]

Secondary ID(s)

ARGX-113-2009

Source(s) of Monetary Support

Please refer to primary and secondary sponsors