Trial · NCT04598451
A Study to Assess the Efficacy and Safety of a Subcutaneous Formulation of Efgartigimod PH20 SC in Adults With Pemphigus (Vulgaris or Foliaceus)
NCT04598451, Phase 3, Completed, argenx; results first posted 2024-10-01
3 sourcestrials-2026-11-12-0.4.0
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ConductScience Library. A Study to Assess the Efficacy and Safety of a Subcutaneous Formulation of Efgartigimod PH20 SC in Adults With Pemphigus (Vulgaris or Foliaceus). Trials, record NCT04598451. Release trials-2026-11-12-0.4.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04598451
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Study to Assess the Efficacy and Safety of a Subcutaneous Formulation of Efgartigimod PH20 SC in Adults With Pemphigus (Vulgaris or Foliaceus)
- NCT number
- NCT04598451
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 222 participants, actual
- Lead sponsor
- argenx
- Conditions
- Pemphigus Vulgaris, Pemphigus Foliaceus
- Interventions
- efgartigimod PH20 SC, Placebo, prednisone
- First posted
- 2020-10-22
- Last updated
- 2024-10-01
- Results first posted
- 2024-10-01
Source ClinicalTrials.gov
Registered outcomes8
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Number of Pemphigus Vulgaris (PV) Participants Who Achieve Complete Clinical Remission (CR) on Minimal Prednisone Therapy | up to 30 weeks treatment period | Proportion of participants with pemphigus vulgaris who had CRmin within 30 weeks, 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. |
| Secondary | Number of Pemphigus Vulgaris (PV) and Pemphigus Foliaceus (PF) Participants Who Achieve Complete Clinical Remission (CR) on Minimal Prednisone Therapy Within 30 Weeks | up to 30 weeks treatment period | Proportion of participants with pemphigus vulgaris and pemphigus foliaceus who had CRmin within 30 weeks, 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. |
| Secondary | Normalized Cumulative Prednisone Dose During the Treatment Period in Pemphigus Vulgaris Participants | Up to 30 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 |
| Secondary | Time to Complete Clinical Remission (CR) in Pemphigus Vulgaris Participants | Up to 30 weeks | Time to complete remission (absence of new lesions and complete healing of established lesions) in participants with pemphigus vulgaris |
| Secondary | Time to Disease Control (DC) in Pemphigus Vulgaris (PV) Participants | Up to 30 weeks | Time to disease control in participants with pemphigus vulgaris (Absence of new lesions and the start of healing of established lesions) |
| Secondary | Normalized Cumulative Prednisone Dose During the Treatment Period in Pemphigus Vulgaris and Pemphigus Foliaceus Participants | Up to 30 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 |
| Secondary | Time to Complete Clinical Remission (CR) in Pemphigus Vulgaris and Pemphigus Foliaceus Participants | Up to 30 weeks | Time to complete remission (absence of new lesions and complete healing of established lesions) in participants with pemphigus vulgaris and pemphigus foliaceus |
| Secondary | Time to Disease Control in Pemphigus Vulgaris and Pemphigus Foliaceus Participants | Up to 30 weeks | Time to disease control in participants with pemphigus vulgaris and pemphigus foliaceus (Absence of new lesions and the start of healing of established lesions) |
Source ClinicalTrials.gov
Outcomes8
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Number of Pemphigus Vulgaris (PV) Participants Who Achieve Complete Clinical Remission (CR) on Minimal Prednisone Therapy | up to 30 weeks treatment period | Randomized participants with pemphigus vulgaris (PV) who received at least part of a dose of efgartigimod PH20 SC or placebo PH20 SC. These participants also received prednisone with a starting dosage of 0.5 mg/kg/day. The dose of prednisone was adjusted per protocol | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Number of Pemphigus Vulgaris (PV) and Pemphigus Foliaceus (PF) Participants Who Achieve Complete Clinical Remission (CR) on Minimal Prednisone Therapy Within 30 Weeks | up to 30 weeks treatment period | Randomized participants with pemphigus vulgaris or pemphigus foliaceus who received at least part of a dose of efgartigimod PH20 SC or placebo PH20 SC. These participants also received prednisone with a starting dosage of 0.5 mg/kg/day. The dose of prednisone was adjusted per protocol. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Normalized Cumulative Prednisone Dose During the Treatment Period in Pemphigus Vulgaris Participants | Up to 30 weeks | Randomized participants with pemphigus vulgaris (PV) who received at least part of a dose of efgartigimod PH20 SC or placebo PH20 SC. These participants also received prednisone with a starting dosage of 0.5 mg/kg/day. The dose of prednisone was adjusted per protocol | mg/kg/day | MEAN |
| SECONDARY | Time to Complete Clinical Remission (CR) in Pemphigus Vulgaris Participants | Up to 30 weeks | Randomized participants with pemphigus vulgaris (PV) who received at least part of a dose of efgartigimod PH20 SC or placebo PH20 SC. These participants also received prednisone with a starting dosage of 0.5 mg/kg/day. The dose of prednisone was adjusted per protocol | days | MEDIAN |
| SECONDARY | Time to Disease Control (DC) in Pemphigus Vulgaris (PV) Participants | Up to 30 weeks | Randomized participants with pemphigus vulgaris (PV) who received at least part of a dose of efgartigimod PH20 SC or placebo PH20 SC. These participants also received prednisone with a starting dosage of 0.5 mg/kg/day. The dose of prednisone was adjusted per protocol | days | MEDIAN |
| SECONDARY | Normalized Cumulative Prednisone Dose During the Treatment Period in Pemphigus Vulgaris and Pemphigus Foliaceus Participants | Up to 30 weeks | Randomized participants with pemphigus vulgaris or pemphigus foliaceus who received at least part of a dose of efgartigimod PH20 SC or placebo PH20 SC. These participants also received prednisone with a starting dosage of 0.5 mg/kg/day. The dose of prednisone was adjusted per protocol. | mg/kg/day | MEAN |
| SECONDARY | Time to Complete Clinical Remission (CR) in Pemphigus Vulgaris and Pemphigus Foliaceus Participants | Up to 30 weeks | Randomized participants with pemphigus vulgaris or pemphigus foliaceus who received at least part of a dose of efgartigimod PH20 SC or placebo PH20 SC. These participants also received prednisone with a starting dosage of 0.5 mg/kg/day. The dose of prednisone was adjusted per protocol. | days | MEDIAN |
| SECONDARY | Time to Disease Control in Pemphigus Vulgaris and Pemphigus Foliaceus Participants | Up to 30 weeks | Randomized participants with pemphigus vulgaris or pemphigus foliaceus who received at least part of a dose of efgartigimod PH20 SC or placebo PH20 SC. These participants also received prednisone with a starting dosage of 0.5 mg/kg/day. The dose of prednisone was adjusted per protocol. | days | MEDIAN |
Source ClinicalTrials.gov
Results by group16
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Number of Pemphigus Vulgaris (PV) Participants Who Achieve Complete Clinical Remission (CR) on Minimal Prednisone Therapy | Efgartigimod PH20 SC | 44 | Participants | ||
| Number of Pemphigus Vulgaris (PV) Participants Who Achieve Complete Clinical Remission (CR) on Minimal Prednisone Therapy | Placebo PH20 SC | 20 | Participants | ||
| Number of Pemphigus Vulgaris (PV) and Pemphigus Foliaceus (PF) Participants Who Achieve Complete Clinical Remission (CR) on Minimal Prednisone Therapy Within 30 Weeks | Efgartigimod PH20 SC | 55 | Participants | ||
| Number of Pemphigus Vulgaris (PV) and Pemphigus Foliaceus (PF) Participants Who Achieve Complete Clinical Remission (CR) on Minimal Prednisone Therapy Within 30 Weeks | Placebo PH20 SC | 24 | Participants | ||
| Normalized Cumulative Prednisone Dose During the Treatment Period in Pemphigus Vulgaris Participants | Efgartigimod PH20 SC | 0.416 | Standard Deviation 0.215 | mg/kg/day | |
| Normalized Cumulative Prednisone Dose During the Treatment Period in Pemphigus Vulgaris Participants | Placebo PH20 SC | 0.444 | Standard Deviation 0.232 | mg/kg/day | |
| Time to Complete Clinical Remission (CR) in Pemphigus Vulgaris Participants | Efgartigimod PH20 SC | 106 | 95% Confidence Interval 79 to 161 | days | |
| Time to Complete Clinical Remission (CR) in Pemphigus Vulgaris Participants | Placebo PH20 SC | 120 | 95% Confidence Interval 85 to 188 | days | |
| Time to Disease Control (DC) in Pemphigus Vulgaris (PV) Participants | Efgartigimod PH20 SC | 16 | 95% Confidence Interval 15 to 21 | days | |
| Time to Disease Control (DC) in Pemphigus Vulgaris (PV) Participants | Placebo PH20 SC | 15 | 95% Confidence Interval 8 to 17 | days |
1 to 10 of 16Source ClinicalTrials.gov
See all 16 results by groupParticipant flow6
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Efgartigimod PH20 SC | 147 | |
| Overall Study | STARTED | Placebo PH20 SC | 75 | |
| Overall Study | COMPLETED | Efgartigimod PH20 SC | 104 | |
| Overall Study | COMPLETED | Placebo PH20 SC | 46 | |
| Overall Study | NOT COMPLETED | Efgartigimod PH20 SC | 43 | |
| Overall Study | NOT COMPLETED | Placebo PH20 SC | 29 |
Source ClinicalTrials.gov
Adverse events84
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | INCREASED TENDENCY TO BRUISE | Blood and lymphatic system disorders | Efgartigimod PH20 SC | 5 | 147 | |
| other | INCREASED TENDENCY TO BRUISE | Blood and lymphatic system disorders | Placebo PH20 SC | 5 | 75 | |
| other | INJECTION SITE ERYTHEMA | General disorders | Efgartigimod PH20 SC | 8 | 147 | |
| other | INJECTION SITE ERYTHEMA | General disorders | Placebo PH20 SC | 1 | 75 | |
| other | COVID-19 | Infections and infestations | Efgartigimod PH20 SC | 17 | 147 | |
| other | COVID-19 | Infections and infestations | Placebo PH20 SC | 3 | 75 | |
| other | NASOPHARYNGITIS | Infections and infestations | Efgartigimod PH20 SC | 4 | 147 | |
| other | NASOPHARYNGITIS | Infections and infestations | Placebo PH20 SC | 5 | 75 | |
| other | ORAL CANDIDIASIS | Infections and infestations | Efgartigimod PH20 SC | 6 | 147 | |
| other | ORAL CANDIDIASIS | Infections and infestations | Placebo PH20 SC | 6 | 75 |
1 to 10 of 84Source ClinicalTrials.gov
See all 84 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Efgartigimod PH20 SC | 0 | 147 |
| Total, all-cause mortality | Placebo PH20 SC | 0 | 75 |
| Total, other adverse events | Efgartigimod PH20 SC | 113 | 147 |
| Total, other adverse events | Placebo PH20 SC | 47 | 75 |
| Total, serious adverse events | Efgartigimod PH20 SC | 18 | 147 |
| Total, serious adverse events | Placebo PH20 SC | 10 | 75 |
Source ClinicalTrials.gov
Papers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 42723554 | DERIVED |
Source PubMed
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons4
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
| Efgartigimod PH20 SC vs Placebo PH20 SC: Number of Pemphigus Vulgaris (PV) Participants Who Achieve Complete Clinical Remission (CR) on Minimal Prednisone Therapy | Trial comparisons |
Efgartigimod PH20 SC vs Placebo PH20 SC: Number of Pemphigus Vulgaris (PV) and Pemphigus Foliaceus (PF) Participants Who Achieve Complete Clinical Remission (CR) on Minimal Prednisone Therapy Within 30 Weeks | Trial comparisons |
| Efgartigimod PH20 SC vs Placebo PH20 SC: deaths from any cause | Trial comparisons |
| Efgartigimod PH20 SC vs Placebo PH20 SC: serious adverse events | Trial comparisons |
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Source
- Release
- trials-2026-11-12-0.4.0
- Release manifest SHA-256
- 0f4a8d8c48aee2a7573ae0fa94ecbeab70c1adbe449bbc95c424bc7d3e97b9d4
- Registry capture
- 2026-11-12
- Results copy, AACT
- 2026-03-23
- NIH clinical studies file
- 2026-11-13
Source ClinicalTrials.gov
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNone stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT04598451
GET https://conductscience.org/api/trials/v1/NCT04598451/events?page=2