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)
- Conditions
- Pemphigus FoliaceusPemphigus Vulgaris
- Interventions
- Biological: efgartigimod PH20 SC
- Registration Number
- NCT04598477
- Lead Sponsor
- argenx
- Brief Summary
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
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- TERMINATED
- Sex
- All
- Target Recruitment
- 183
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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).
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The participant participated in trial ARGX-113-1904 and completed the study or has the defined criteria for rollover.
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Contraceptive use by men and women should be consistent with local regulations regarding the methods for contraception for those participating in clinical trials and:
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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.
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Female participants
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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
- Pregnant and lactating women and those intending to become pregnant during the trial.
- 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.
- Known hypersensitivity to any of the components of the administered treatments.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description efgartigimod PH20 SC efgartigimod PH20 SC patients receiving efgartigimod PH20 SC on top of prednisone efgartigimod PH20 SC prednisone patients receiving efgartigimod PH20 SC on top of prednisone
- Primary Outcome Measures
Name Time Method Incidence of Treatment-Emergent Adverse Events (TEAE), Adverse Events of Special Interest (AESI), and Serious Adverse Events (SAE) Up to 60 weeks 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).
- Secondary Outcome Measures
Name Time Method Proportion of Participants With Pemphigus Vulgaris (PV) Who Achieve CRmin Up to 60 weeks 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 to DC in Participants With PV and PF Up to 52 weeks Disease Control (DC) defined as absence of new lesions and the start of healing of established lesions
Time to CR in Participants With PV and PF Up to 52 weeks CR (Complete clinical remission) defined as the absence of new lesions and complete healing of established lesions
Time to CRmin in Participants With PV and PF Up to 52 weeks 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 to CRoff in Participants With PV and PF Up to 52 weeks 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 to Flare After CRmin in Participants With PV and PF Up to 52 weeks 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.
Rate of Treatment Failure in Participants With PV and PF Up to 52 weeks 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.
Number of Flares in Participants With PV and PF Up to 60 weeks 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.
Normalized Cumulative Prednisone Dose in Participants With PV and PF Up to 60 weeks 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
Proportion of Participants With Pemphigus Vulgaris (PV) and Pemphigus Foliaceus (PF) Who Achieve CRmin Up to 60 weeks 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.
Trial Locations
- Locations (127)
Investigator site 115 - US0010086
🇺🇸Birmingham, Alabama, United States
Investigator site 89 - US0010091
🇺🇸Scottsdale, Arizona, United States
Investigator site 124 - US0010092
🇺🇸Redwood City, California, United States
Investigator site 1 - US0010087
🇺🇸Boca Raton, Florida, United States
Investigator site 90 - US0010117
🇺🇸Miami, Florida, United States
Investigator site 91 - US0010109
🇺🇸Orlando, Florida, United States
Investigator site 126 - US0010090
🇺🇸Minneapolis, Minnesota, United States
Investigator site 111 - US0010098
🇺🇸Saint Louis, Missouri, United States
Investigator site 10 - US0010088
🇺🇸Buffalo, New York, United States
Investigator site 76 - US0010096
🇺🇸Durham, North Carolina, United States
Scroll for more (117 remaining)Investigator site 115 - US0010086🇺🇸Birmingham, Alabama, United States