A Randomized, Double-Blinded, Placebo-Controlled Trial to Investigate the Efficacy, Safety, and Tolerability of Efgartigimod PH20 SC in Adult Patients With Pemphigus (Vulgaris or Foliaceus) (ADDRESS) - ADDRESS

ARGENX BV0 sites150 target enrollmentMay 24, 2021

ConditionsPemphigus Vulgaris or Pemphigus Foliaceus MedDRA version: 20.0Level: LLTClassification code 10057069Term: Pemphigus foliaceusSystem Organ Class: 100000004858 MedDRA version: 20.0Level: LLTClassification code 10052802Term: Pemphigus vulgarisSystem Organ Class: 100000004858 Therapeutic area: Diseases [C] - Immune System Diseases [C20]

DrugsPrednison 50 mg GALEN Prednison 5 mg GALEN Prednison20 mg GALEN Prednison 10 mg GALEN

Overview

Phase: Phase 1
Intervention: Not specified
Conditions: Pemphigus Vulgaris or Pemphigus Foliaceus
Sponsor: ARGENX BV
Enrollment: 150
Status: Active, not recruiting
Last Updated: 2 years ago

Overview Investigators Eligibility Criteria Outcomes Similar Trials

Overview

Brief Summary

No summary available.

Registry

who.int

Start Date

May 24, 2021

End Date

TBD

Last Updated

2 years ago

Study Type

Interventional clinical trial of medicinal product

Sex

All

Investigators

Sponsor

ARGENX BV

Eligibility Criteria

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 patient is male or female, and aged 18 years to 80 years at the time of signing the informed consent form (ICF).
•3\. The patient has a clinical diagnosis of PV (mucosal, cutaneous, mucocutaneous) or PF which has been confirmed by cutaneous histology, positive direct immunofluorescence (IF), and positive indirect IF and/or ELISA.
•4\. The patient meets 1 of the following profiles:
•a. Newly diagnosed disease with PDAI \=15 at baseline and naïve to treatment.
•b. Newly diagnosed disease with PDAI \=15 while receiving a first course of oral prednisone (or equivalent). According to clinical judgment, the patient has shown no significant improvement of PV or PF signs for at least 2 weeks before baseline and is considered fit to start prednisone
•treatment at 0\.5 mg/kg qd at baseline.
•c. Experiencing flare with PDAI \=15, a maximum of 4 years since diagnosis, and off prednisone therapy ± a conventional immunosuppressant (eg, azathioprine, cyclophosphamide, methotrexate, mycophenolate mofetil). Note: conventional immunosuppressants must be discontinued before baseline.
•d. Experiencing flare with PDAI \=15, a maximum of 4 years since diagnosis, and receiving a tapered dose of oral prednisone (or equivalent), provided that prednisone has been given at stable dose ± a conventional immunosuppressant (eg, azathioprine, cyclophosphamide, methotrexate, mycophenolate mofetil) for at least 2 weeks and patients are fit to start prednisone treatment at 0\.5 mg/kg qd at baseline. Note: conventional
•immunosuppressants must be discontinued before baseline.

Exclusion Criteria

•1\. Patient has a confirmed diagnosis of paraneoplastic pemphigus, drug\-induced pemphigus, pemphigus vegetans, pemphigus erythematosus, or any other non\-PV/non\-PF autoimmune blistering disease.
•2\. Patients with mild disease severity as defined by PDAI \<15 at baseline.
•3\. Patients who show a significant improvement of PV or PF in the period from screening to baseline according to clinical judgment (eg, the patient has achieved DC or a substantial reduction in PDAI activity score during screening period).
•4\. The patient has been administered therapy other than oral prednisone or conventional immunosuppressants (eg, azathioprine, cyclophosphamide, methotrexate, mycophenolate mofetil) within 2 months before the baseline visit and that can affect clinical disease activity.
•5\. Use of any monoclonal antibody (including rituximab or another anti\-CD20 biologic) within 6 months before the baseline visit.
•6\. Known hypersensitivity to any of the components of the administered treatments.
•7\. The patient has a known contraindication to oral prednisone.
•8\. The patient has a history of refractory disease, as defined by a failure to respond to first\-line and second\-line therapies.
•9\. Patients who have a history of malignancy unless deemed cured by adequate treatment with no evidence of recurrence for \=3 years before first IMP administration. Patients with any of the following cancers can be included at any time:
•a. Adequately treated basal cell or squamous cell skin cancer