A Phase 3 interventional study of efgartigimod PH20 SC and Placebo in Pemphigus Vulgaris and Pemphigus Foliaceus, sponsored by argenx. Completed at 134 sites in 21 countries. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2024-10-01.
Sponsored by argenx · Phase 3, Interventional, and Treatment
This is a prospective, multicenter, randomized, double-blinded, placebo-controlled trial to investigate the efficacy, safety, patient outcome measures, tolerability, immunogenicity, PK, and PD of efgartigimod PH20 SC in adult participants aged from 18 years with PV or PF. The trial comprises a screening period of up to 3 weeks, a treatment period of up to 30 weeks, and an 8-week follow-up period for participants who do not enroll into the open-label extension (OLE) trial ARGX-113-1905. The primary objective of the ARGX-113-1904 trial is to demonstrate the efficacy of subcutaneous administration of efgartigimod co-formulated with recombinant human hyaluronidase PH20 (Efgartigimod PH20 SC) compared to placebo in the treatment of participants with Pemphigus Vulgaris (PV). Secondary objectives are to also demonstrate the efficacy of efgartigimod PH20 SC in the treatment of participants with Pemphigus Foliaceus (PF), and to demonstrate early onset of action and a prednisone-sparing effect. After confirmation of eligibility, participants will be randomized in a 2: 1 ratio to receive efgartigimod PH20 SC or placebo
The participant meets one of the following profiles:
Contraceptive use by men and women should be consistent with local regulations regarding the methods of contraception for those participating clinical trials and:
Female participants: Women of childbearing potential must:
Exclusion Criteria:
Participants who have a history of malignancy unless deemed cured by adequate treatment with no evidence of recurrence for ≥3 years before first IMP administration. Participants with any of the following cancers can be included at any time, provided they are adequately treated prior to their participation in the study:
patients receiving efgartigimod PH20 SC on top of prednisone
Biological: efgartigimod PH20 SC · Drug: prednisone
patients receiving placebo on top of prednisone
Other: Placebo · Drug: prednisone
Subcutaneous injection of efgartigimod using rHuPH20 (PH20) as a permeation enhancer
Subcutaneous injection of placebo
Oral prednisone tablets
Number of Pemphigus Vulgaris (PV) Participants Who Achieve Complete Clinical Remission (CR) on Minimal Prednisone Therapy
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.
Time frame: up to 30 weeks treatment period
Number of Pemphigus Vulgaris (PV) and Pemphigus Foliaceus (PF) Participants Who Achieve Complete Clinical Remission (CR) on Minimal Prednisone Therapy Within 30 Weeks
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.
Time frame: up to 30 weeks treatment period
Normalized Cumulative Prednisone Dose During the Treatment Period in Pemphigus Vulgaris Participants
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 30 weeks
Time to Complete Clinical Remission (CR) in Pemphigus Vulgaris Participants
Time to complete remission (absence of new lesions and complete healing of established lesions) in participants with pemphigus vulgaris
Time frame: Up to 30 weeks
Time to Disease Control (DC) in Pemphigus Vulgaris (PV) Participants
Time to disease control in participants with pemphigus vulgaris (Absence of new lesions and the start of healing of established lesions)
Time frame: Up to 30 weeks
Normalized Cumulative Prednisone Dose During the Treatment Period in Pemphigus Vulgaris and Pemphigus Foliaceus Participants
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 30 weeks
Time to Complete Clinical Remission (CR) in Pemphigus Vulgaris and Pemphigus Foliaceus Participants
Time to complete remission (absence of new lesions and complete healing of established lesions) in participants with pemphigus vulgaris and pemphigus foliaceus
Time frame: Up to 30 weeks
Time to Disease Control in Pemphigus Vulgaris and Pemphigus Foliaceus Participants
Time to disease control in participants with pemphigus vulgaris and pemphigus foliaceus (Absence of new lesions and the start of healing of established lesions)
Time frame: Up to 30 weeks
| Milestone | Efgartigimod PH20 SC | Placebo PH20 SC |
|---|---|---|
| Started | 147 | 75 |
| Completed | 104 | 46 |
| Not completed | 43 | 29 |
| Withdrew: Adverse event | 3 | 2 |
| Withdrew: Withdrawal by subject | 3 | 7 |
| Withdrew: Lost to follow-up | 2 | 0 |
| Withdrew: Physician decision | 4 | 1 |
| Withdrew: Requires prohibited medication | 1 | 0 |
| Withdrew: Lack of efficacy, geopolitical situation in ukraine, flares after crmin, sae due to prednisone, etc | 30 | 19 |
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.
| Participants | Efgartigimod PH20 SC | Placebo PH20 SC |
|---|---|---|
| Number of Pemphigus Vulgaris (PV) Participants Who Achieve Complete Clinical Remission (CR) on Minimal Prednisone Therapy | 44 | 20 |
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.
| Participants | Efgartigimod PH20 SC | 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 | 55 | 24 |
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
| mg/kg/day | Efgartigimod PH20 SC | Placebo PH20 SC |
|---|---|---|
| Normalized Cumulative Prednisone Dose During the Treatment Period in Pemphigus Vulgaris Participants | 0.416 ± 0.215 | 0.444 ± 0.232 |
Time to complete remission (absence of new lesions and complete healing of established lesions) in participants with pemphigus vulgaris
| days | Efgartigimod PH20 SC | Placebo PH20 SC |
|---|---|---|
| Time to Complete Clinical Remission (CR) in Pemphigus Vulgaris Participants | 106 (79 to 161) | 120 (85 to 188) |
Time to disease control in participants with pemphigus vulgaris (Absence of new lesions and the start of healing of established lesions)
| days | Efgartigimod PH20 SC | Placebo PH20 SC |
|---|---|---|
| Time to Disease Control (DC) in Pemphigus Vulgaris (PV) Participants | 16 (15 to 21) | 15 (8 to 17) |
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
| mg/kg/day | Efgartigimod PH20 SC | Placebo PH20 SC |
|---|---|---|
| Normalized Cumulative Prednisone Dose During the Treatment Period in Pemphigus Vulgaris and Pemphigus Foliaceus Participants | 0.403 ± 0.210 | 0.431 ± 0.225 |
Time to complete remission (absence of new lesions and complete healing of established lesions) in participants with pemphigus vulgaris and pemphigus foliaceus
| days | Efgartigimod PH20 SC | Placebo PH20 SC |
|---|---|---|
| Time to Complete Clinical Remission (CR) in Pemphigus Vulgaris and Pemphigus Foliaceus Participants | 106 (84 to 142) | 113 (81 to 149) |
Time to disease control in participants with pemphigus vulgaris and pemphigus foliaceus (Absence of new lesions and the start of healing of established lesions)
| days | Efgartigimod PH20 SC | Placebo PH20 SC |
|---|---|---|
| Time to Disease Control in Pemphigus Vulgaris and Pemphigus Foliaceus Participants | 15 (15 to 17) | 15 (9 to 16) |
Collected over Up to 38 weeks. Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Efgartigimod PH20 SC | 0/147 (0%) | 18/147 (12.2%) | 113/147 (76.9%) |
| Placebo PH20 SC | 0/75 (0%) | 10/75 (13.3%) | 47/75 (62.7%) |
| Event | Efgartigimod PH20 SC | Placebo PH20 SC |
|---|---|---|
| COVID-19Infections and infestations | 2/147 | 2/75 |
| SPINAL COMPRESSION FRACTUREInjury, poisoning and procedural complications | 2/147 | 0/75 |
| HYPERTENSIONVascular disorders | 2/147 | 0/75 |
| ANAEMIABlood and lymphatic system disorders | 0/147 | 1/75 |
| VISION BLURREDEye disorders | 1/147 | 1/75 |
| GASTRITIS EROSIVEGastrointestinal disorders | 0/147 | 1/75 |
| URINARY TRACT INFECTIONInfections and infestations | 1/147 | 1/75 |
| DIABETES MELLITUSMetabolism and nutrition disorders | 1/147 | 1/75 |
| DIABETES MELLITUS INADEQUATE CONTROLMetabolism and nutrition disorders | 0/147 | 1/75 |
| HYPERGLYCAEMIAMetabolism and nutrition disorders | 0/147 | 1/75 |
| Event | Efgartigimod PH20 SC | Placebo PH20 SC |
|---|---|---|
| HYPERTENSIONVascular disorders | 21/147 | 3/75 |
| INSOMNIAPsychiatric disorders | 18/147 | 9/75 |
| COVID-19Infections and infestations | 17/147 | 3/75 |
| MYOPATHYMusculoskeletal and connective tissue disorders | 9/147 | 8/75 |
| ORAL CANDIDIASISInfections and infestations | 6/147 | 6/75 |
| URINARY TRACT INFECTIONInfections and infestations | 4/147 | 6/75 |
| DERMATITIS ACNEIFORMSkin and subcutaneous tissue disorders | 7/147 | 6/75 |
| HYPERTRIGLYCERIDAEMIAMetabolism and nutrition disorders | 11/147 | 2/75 |
| BLOOD LACTATE DEHYDROGENASE INCREASEDInvestigations | 10/147 | 3/75 |
| INCREASED TENDENCY TO BRUISEBlood and lymphatic system disorders | 5/147 | 5/75 |
| Age, Continuous(years) | Efgartigimod PH20 SC | Placebo PH20 SC | Total |
|---|---|---|---|
| Mean | 48.9 ± 12.55 | 52.3 ± 13.37 | 50.1 ± 12.90 |
| Sex: Female, Male(Participants) | Efgartigimod PH20 SC | Placebo PH20 SC | Total |
|---|---|---|---|
| Female | 73 | 42 | 115 |
| Male | 74 | 33 | 107 |
| Race/Ethnicity, Customized(Participants) | Efgartigimod PH20 SC | Placebo PH20 SC | Total |
|---|---|---|---|
| Race — Asian | 49 | 16 | 65 |
| Race — Black or African American | 1 | 2 | 3 |
| Race — White | 96 | 53 | 149 |
| Race — Other | 1 | 4 | 5 |
| Race/Ethnicity, Customized(Participants) | Efgartigimod PH20 SC | Placebo PH20 SC | Total |
|---|---|---|---|
| Ethnicity — Hispanic or Latino | 3 | 5 | 8 |
| Ethnicity — Not Hispanic or Latino | 144 | 70 | 214 |
| Pemphigus Vulgaris (PV)(Participants) | Efgartigimod PH20 SC | Placebo PH20 SC | Total |
|---|---|---|---|
| Count of participants | 124 | 66 | 190 |
| Pemphigus Foliaceus (PF)(Participants) | Efgartigimod PH20 SC | Placebo PH20 SC | Total |
|---|---|---|---|
| Count of participants | 23 | 9 | 32 |
Showing the first 100 of 134 sites across 21 countries.
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