A Study to Assess the Long-term Safety and Efficacy of a Subcutaneous Formulation of Efgartigimod PH20 SC in Adults With Pemphigus (Vulgaris or Foliaceus)

Phase 3TerminatedNCT04598477

argenx183 enrolled

Overview

This was a prospective, multicenter, open label extension (OLE) trial on the efficacy, safety, patient outcome measures, tolerability, immunogenicity, PK and PD of efgartigimod PH20 SC in adult PV or PF participants, who participated in antecedent trial ARGX-113-1904. This trial provided extension of efgartigimod PH20 SC treatment and retreatment options for participants who had been randomized to efgartigimod PH20 SC treatment arm in the trial ARGX-113-1904, and first treatment of efgartigimod PH20 SC and retreatment options for participants who had been randomized to the placebo arm in trial ARGX-113-1904. The participants could also receive concomitant prednisone therapy. Investigators could increase or decrease the prednisone dose based on protocol-specified criteria. Trial ARGX-113-1905 evaluated the ability to (further) taper prednisone therapy and achieve Clinical Remission (CR) off therapy (CRoff), the ability to achieve CR and CR on minimal therapy (CRmin) for participants who had not yet achieved CR or CRmin, and the ability to treat flare; it also assessed patient outcome measures and the safety, PD, PK and immunogenicity of efgartigimod PH20 SC over the duration of trial. Study duration: Up to 60 weeks for participants who receive IMP administration up to 52 weeks and with a follow-up period of 8 weeks after the last IMP administration

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

183

Conditions

Pemphigus Vulgaris

Eligibility

Sex: ALLMin age: 18 Years

Medical Language ↔ Plain English

Inclusion Criteria: 1. Ability to understand the requirements of the trial, to provide written informed consent (including consent for the use and disclosure of research-related health information), willingness and ability to comply with the trial protocol procedures (including required trial visits). 2. The participant participated in trial ARGX-113-1904 and completed the study or has the defined criteria for rollover. 3. Contraceptive use by men and women should be consistent with local regulations regarding the methods for contraception for those participating in clinical trials and: 1. Male participants: Male participants must agree to use an acceptable method of contraception as described in the protocol, from signing the ICF until the last dose of the study drug. 2. Female participants Women of childbearing potential (WOCBP) must: * have a negative urine pregnancy test at baseline before the IMP can be administered, * agree to use a highly effective or acceptable contraception method (as described in the protocol), which should be maintained at minimum until after the last dose of IMP Exclusion Criteria: 1. Pregnant and lactating women and those intending to become pregnant during the trial. 2. Participants with clinical evidence of other significant serious disease or participants who recently underwent or have planned a major surgery during the period of the trial, or any other condition in the opinion of the investigator, that could confound the results of the trial or put the participant at undue risk. 3. Known hypersensitivity to any of the components of the administered treatments.

Outcomes

Primary Outcomes

Incidence of Treatment-Emergent Adverse Events (TEAE), Adverse Events of Special Interest (AESI), and Serious Adverse Events (SAE)

Incidence rates were calculated as 100 × n/PYFU. PYFU=participant-years of follow-up. The safety data sets includes participants with pemphigus vulgaris (PV) and pemphigus foliaceus (PF).

Time frame: Up to 60 weeks

Secondary Outcomes

Proportion of Participants With Pemphigus Vulgaris (PV) and Pemphigus Foliaceus (PF) Who Achieve CRmin

CRmin defined as the absence of new lesions and complete healing of established lesions while the participant was receiving prednisone at ≤10 mg/day for at least 8 weeks.

Time frame: Up to 60 weeks

Proportion of Participants With Pemphigus Vulgaris (PV) Who Achieve CRmin

CRmin (complete clinical remission on minimal prednisone therapy) defined as the absence of new lesions and complete healing of established lesions while the participant was receiving prednisone at ≤10 mg/day for at least 8 weeks.

Time frame: Up to 60 weeks

Time to DC in Participants With PV and PF

Disease Control (DC) defined as absence of new lesions and the start of healing of established lesions

Time frame: Up to 52 weeks

Time to CR in Participants With PV and PF

CR (Complete clinical remission) defined as the absence of new lesions and complete healing of established lesions

Time frame: Up to 52 weeks

Time to CRmin in Participants With PV and PF

CRmin defined as the absence of new lesions and complete healing of established lesions while the participant was receiving prednisone at ≤10 mg/day for at least 8 weeks.

Time frame: Up to 52 weeks

Time to CRoff in Participants With PV and PF

Complete remission off therapy (CRoff) is defined as the absence of new and established lesions completely healed while the patient is receiving no prednisone therapy for at least 8 weeks.

Time frame: Up to 52 weeks

Time to Flare After CRmin in Participants With PV and PF

CRmin defined as defined as the absence of new lesions and complete healing of established lesions while the participant was receiving prednisone at ≤10 mg/day for at least 8 weeks.

Time frame: Up to 52 weeks

Rate of Treatment Failure in Participants With PV and PF

The absence of DC with oral prednisone 1.5 mg/kg/day for a minimum of 3 weeks, or absence of DC due to prednisone-related SAE, or flare before CRmin resulting in withdrawal of the participant.

Time frame: Up to 52 weeks

Number of Flares in Participants With PV and PF

A flare is defined as the appearance of 3 or more new lesions in a 4-week period that do not heal spontaneously within 1 week or the extension, of established lesions in a participant who had achieved DC.

Time frame: Up to 60 weeks

Normalized Cumulative Prednisone Dose in Participants With PV and PF

Normalized Cumulative prednisone dose (NCPD, mg/kg/day) is the average daily intake of all weight-adjusted prednisone doses received during the study, taking into account the number of days in study

Time frame: Up to 60 weeks

A Study to Assess the Long-term Safety and Efficacy of a Subcutaneous Formulation of Efgartigimod PH20 SC in Adults With Pemphigus (Vulgaris or Foliaceus)

Overview

Conditions

Interventions

Eligibility

Locations (127)

Outcomes

Primary Outcomes

Secondary Outcomes