CTRI/2021/01/030594
Active, not recruiting
Phase 3
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
Overview
- Phase
- Phase 3
- Intervention
- Not specified
- Conditions
- Health Condition 1: L102- Pemphigus foliaceousHealth Condition 2: L100- Pemphigus vulgaris
- Sponsor
- argenx BV
- Status
- Active, not recruiting
- Last Updated
- last year
Overview
Brief Summary
No summary available.
Investigators
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 activity score \=15 (see Section 10\.8\) at baseline and naïve to treatment.
- •b. Newly diagnosed disease with PDAI activity score \=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
- •c. Experiencing flare with PDAI activity score \=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 activity score \=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.
- •5\. Women of childbearing potential (see definition in Section 10\.5\):
Exclusion Criteria
- •Exclusion Criteria:
- •Participants are excluded from the trial if any of the following criteria apply:
- •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 activity score \<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(ies) 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. For example, excluded medications are intravenous methylprednisolone, dapsone, sulfasalazine, tetracyclines, nicotinamide, plasmapheresis/ plasma exchange,
- •immunoadsorption, and IVIg. Note: conventional immunosuppressants must be discontinued before baseline.
- •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.
Outcomes
Primary Outcomes
Not specified
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