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 enrollmentAugust 27, 2020

Overview

Phase: Phase 1
Intervention: Not specified
Conditions: Not specified
Sponsor: argenx BV
Enrollment: 150
Status: Active, not recruiting
Last Updated: 3 years ago

Overview Investigators Eligibility Criteria Outcomes Similar Trials

Overview

Brief Summary

No summary available.

Registry

who.int

Start Date

August 27, 2020

End Date

TBD

Last Updated

3 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 participant is male or female, and aged from 18 years at the time of signing the informed consent form (ICF).
•3\. The participant has a clinical diagnosis of PV (mucosal, cutaneous, mucocutaneous) or PF that has been confirmed by cutaneous histology, positive direct immunofluorescence (IF), and positive indirect IF and/or ELISA.
•4\. The participant 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 participant 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 disease onset, and off prednisone therapy ± a conventional immunosuppressant (eg, azathioprine, cyclophosphamide, methotrexate, mycophenolate mofetil) or dapsone. Note: conventional immunosuppressants and dapsone must be discontinued before baseline.
•d. Experiencing flare with PDAI \=15, a maximum of 4 years since disease onset, 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) or dapsone for at least 2 weeks and participants are fit to start prednisone treatment at 0\.5 mg/kg qd at baseline. Note: conventional
•immunosuppressants and dapsone must be discontinued before baseline.
•5\. Contraceptive use by men and women should be consistent with local regulations regarding the methods of contraception for those participating clinical trials and:

Exclusion Criteria

•1\. Participant 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\. Participants with mild disease severity as defined by PDAI \<15 at baseline.
•3\. Participants who show a significant improvement of PV or PF in the period from screening to baseline according to clinical judgment (eg, the participant has achieved DC or a substantial reduction in PDAI activity score during screening period).
•4\. The participants has been administered therapy other than oral prednisone or conventional immunosuppressants (eg, azathioprine, cyclophosphamide, methotrexate, mycophenolate mofetil) or dapsone within 2 months before the baseline visit and that can affect clinical disease activity. For example, excluded medications are intravenous methylprednisolone, sulfasalazine, tetracyclines, nicotinamide at doses above the recommended daily allowance (RDA)/dietary reference intake (DRI), plasmapheresis/plasma exchange, immunoadsorption, and IVIg. Note: conventional immunosuppressants and dapsone must be discontinued before baseline. Nicotinamide doses at or below RDA/DRI from oral supplements is allowed.
•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 participant has a known contraindication to oral prednisone.
•8\. The participant has a history of refractory disease, as defined by a failure to respond to first\-line and second\-line therapies.
•9\. 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:
•a. Basal cell or squamous cell skin cancer