Trial · NCT04598477
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)
NCT04598477, Phase 3, Terminated, argenx; results first posted 2025-03-30
3 sourcestrials-2026-11-12-0.4.0
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ConductScience Library. 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). Trials, record NCT04598477. Release trials-2026-11-12-0.4.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04598477
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- 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)
- NCT number
- NCT04598477
- Status
- Terminated
- Phases
- Phase 3
- Enrollment
- 183 participants, actual
- Lead sponsor
- argenx
- Conditions
- Pemphigus Vulgaris, Pemphigus Foliaceus
- Interventions
- efgartigimod PH20 SC, prednisone
- First posted
- 2020-10-22
- Last updated
- 2025-03-30
- Results first posted
- 2025-03-30
- Limitations and caveats
- Study was terminated prematurely by the Sponsor.
Source ClinicalTrials.gov
Registered outcomes11
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | 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 | 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. |
| Secondary | 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. |
| Secondary | 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 |
| Secondary | 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 |
| Secondary | 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. |
| Secondary | 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. |
| Secondary | 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. |
| Secondary | 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. |
| Secondary | 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. |
1 to 10 of 11Source ClinicalTrials.gov
See all 11 registered outcomesOutcomes11
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Incidence of Treatment-Emergent Adverse Events (TEAE), Adverse Events of Special Interest (AESI), and Serious Adverse Events (SAE) | Up to 60 weeks | Safety set | number of events x 100/PYFU | NUMBER |
| SECONDARY | Proportion of Participants With Pemphigus Vulgaris (PV) and Pemphigus Foliaceus (PF) Who Achieve CRmin | Up to 60 weeks | Safety set | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Proportion of Participants With Pemphigus Vulgaris (PV) Who Achieve CRmin | Up to 60 weeks | Safety set - Participants with PV | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Time to DC in Participants With PV and PF | Up to 52 weeks | Safety set - Participants with status DC at the roll-over visit were not included in the analysis. | days | MEDIAN |
| SECONDARY | Time to CR in Participants With PV and PF | Up to 52 weeks | Safety set - Participants with status CR at the roll-over visit were not included in this analysis. | Days | MEDIAN |
| SECONDARY | Time to CRmin in Participants With PV and PF | Up to 52 weeks | Safety set - Participants with status CRmin at the roll-over visit were not included in this analysis. | days | MEDIAN |
| SECONDARY | Time to CRoff in Participants With PV and PF | Up to 52 weeks | Safety set - Participants with status CRoff at the roll-over visit were not included in this analysis. | days | MEDIAN |
| SECONDARY | Time to Flare After CRmin in Participants With PV and PF | Up to 52 weeks | Safety set - Only participants who achieved CRmin were considered for the analysis. | days | MEDIAN |
| SECONDARY | Rate of Treatment Failure in Participants With PV and PF | Up to 52 weeks | Safety set | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Number of Flares in Participants With PV and PF | Up to 60 weeks | Roll-over set - Only participants who achieved DC were considered for the analysis. | Flares | MEAN |
1 to 10 of 11Source ClinicalTrials.gov
See all 11 outcomesResults by group26
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Incidence of Treatment-Emergent Adverse Events (TEAE), Adverse Events of Special Interest (AESI), and Serious Adverse Events (SAE) | Efgartigimod-efgartigimod PH20 SC | Treatment-Emergent Adverse Event (TEAE) | 116.4 | number of events x 100/PYFU | |
| Incidence of Treatment-Emergent Adverse Events (TEAE), Adverse Events of Special Interest (AESI), and Serious Adverse Events (SAE) | Placebo-efgartigimod PH20 SC | Treatment-Emergent Adverse Event (TEAE) | 113.0 | number of events x 100/PYFU | |
| Incidence of Treatment-Emergent Adverse Events (TEAE), Adverse Events of Special Interest (AESI), and Serious Adverse Events (SAE) | Efgartigimod-efgartigimod PH20 SC | Adverse Event of Special Interest (AESI) | 72.0 | number of events x 100/PYFU | |
| Incidence of Treatment-Emergent Adverse Events (TEAE), Adverse Events of Special Interest (AESI), and Serious Adverse Events (SAE) | Placebo-efgartigimod PH20 SC | Adverse Event of Special Interest (AESI) | 69.3 | number of events x 100/PYFU | |
| Incidence of Treatment-Emergent Adverse Events (TEAE), Adverse Events of Special Interest (AESI), and Serious Adverse Events (SAE) | Efgartigimod-efgartigimod PH20 SC | Serious Adverse Event (SAE) | 24.5 | number of events x 100/PYFU | |
| Incidence of Treatment-Emergent Adverse Events (TEAE), Adverse Events of Special Interest (AESI), and Serious Adverse Events (SAE) | Placebo-efgartigimod PH20 SC | Serious Adverse Event (SAE) | 14.6 | number of events x 100/PYFU | |
| Proportion of Participants With Pemphigus Vulgaris (PV) and Pemphigus Foliaceus (PF) Who Achieve CRmin | Efgartigimod-efgartigimod PH20 SC | 55 | Participants | ||
| Proportion of Participants With Pemphigus Vulgaris (PV) and Pemphigus Foliaceus (PF) Who Achieve CRmin | Placebo-efgartigimod PH20 SC | 26 | Participants | ||
| Proportion of Participants With Pemphigus Vulgaris (PV) Who Achieve CRmin | Efgartigimod-efgartigimod PH20 SC | 47 | Participants | ||
| Proportion of Participants With Pemphigus Vulgaris (PV) Who Achieve CRmin | Placebo-efgartigimod PH20 SC | 22 | Participants |
1 to 10 of 26Source ClinicalTrials.gov
See all 26 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-efgartigimod PH20 SC | 123 | |
| Overall Study | STARTED | Placebo-efgartigimod PH20 SC | 60 | |
| Overall Study | COMPLETED | Efgartigimod-efgartigimod PH20 SC | 64 | |
| Overall Study | COMPLETED | Placebo-efgartigimod PH20 SC | 23 | |
| Overall Study | NOT COMPLETED | Efgartigimod-efgartigimod PH20 SC | 59 | |
| Overall Study | NOT COMPLETED | Placebo-efgartigimod PH20 SC | 37 |
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-efgartigimod PH20 SC | 0 | 101 | |
| other | INCREASED TENDENCY TO BRUISE | Blood and lymphatic system disorders | Placebo-efgartigimod PH20 SC | 3 | 48 | |
| other | TACHYCARDIA | Cardiac disorders | Efgartigimod-efgartigimod PH20 SC | 1 | 101 | |
| other | TACHYCARDIA | Cardiac disorders | Placebo-efgartigimod PH20 SC | 3 | 48 | |
| other | INJECTION SITE ERYTHEMA | General disorders | Efgartigimod-efgartigimod PH20 SC | 4 | 101 | |
| other | INJECTION SITE ERYTHEMA | General disorders | Placebo-efgartigimod PH20 SC | 3 | 48 | |
| other | COVID-19 | Infections and infestations | Efgartigimod-efgartigimod PH20 SC | 12 | 101 | |
| other | COVID-19 | Infections and infestations | Placebo-efgartigimod PH20 SC | 5 | 48 | |
| other | FOLLICULITIS | Infections and infestations | Efgartigimod-efgartigimod PH20 SC | 5 | 101 | |
| other | FOLLICULITIS | Infections and infestations | Placebo-efgartigimod PH20 SC | 1 | 48 |
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-efgartigimod PH20 SC | 0 | 101 |
| Total, all-cause mortality | Placebo-efgartigimod PH20 SC | 1 | 48 |
| Total, other adverse events | Efgartigimod-efgartigimod PH20 SC | 46 | 101 |
| Total, other adverse events | Placebo-efgartigimod PH20 SC | 19 | 48 |
| Total, serious adverse events | Efgartigimod-efgartigimod PH20 SC | 16 | 101 |
| Total, serious adverse events | Placebo-efgartigimod PH20 SC | 4 | 48 |
Source ClinicalTrials.gov
Papers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
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 comparisons5
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
| Efgartigimod-efgartigimod PH20 SC vs Placebo-efgartigimod PH20 SC: Proportion of Participants With Pemphigus Vulgaris (PV) Who Achieve CRmin | Trial comparisons |
| Efgartigimod-efgartigimod PH20 SC vs Placebo-efgartigimod PH20 SC: Proportion of Participants With Pemphigus Vulgaris (PV) and Pemphigus Foliaceus (PF) Who Achieve CRmin | Trial comparisons |
| Efgartigimod-efgartigimod PH20 SC vs Placebo-efgartigimod PH20 SC: Rate of Treatment Failure in Participants With PV and PF | Trial comparisons |
| Efgartigimod-efgartigimod PH20 SC vs Placebo-efgartigimod PH20 SC: deaths from any cause | Trial comparisons |
| Efgartigimod-efgartigimod PH20 SC vs Placebo-efgartigimod 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/NCT04598477
GET https://conductscience.org/api/trials/v1/NCT04598477/results?page=2