CTRI/2021/01/030594进行中（未招募）3 期
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
试验速览
- 阶段
- 3 期
- 状态
- 进行中（未招募）
- 发起方
- argenx BV
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
入排标准
入选标准
- •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):
- •a. Must have a negative serum pregnancy test at screening and a negative urine pregnancy test at baseline before trial medication can be administered.
- •b. Must be on a stable regimen for at least 1 month of at least 1 highly effective method of contraception (ie, failure rate of less than 1% per year; see Highly effective methods of female contraception in Section 10.5) during the trial and for 90 days after the last administration of IMP.
- •6. Non-sterilized male patients who are sexually active with a female partner of childbearing potential must use effective contraception from first administration of IMP through 90 days after the last administration of the IMP. Male patients practicing true sexual abstinence (as consistent with preferred and usual life style) can be included. Sterilized male patients who have had a vasectomy and with documented absence of sperm post-procedure can be included. Male patients are not allowed to donate sperm from first administration of IMP through 90 days after the last dose of IMP.
排除标准
- •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.
- •8. The patient has a history of refractory disease, as defined by a failure to respond to firstline and second-line
- •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
- •b. Carcinoma in situ of the cervix
- •c. Carcinoma in situ of the breast
- •d. Incidental histological finding of prostate cancer (TNM stage T1a or T1b)
- •10. Patients with clinical evidence of other significant serious disease or patients who recently underwent or have planned a major surgery during the period of the trial, or any other condition in the opinion of the investigator, that could confound the results of the trial or put the patient at undue risk.
- •11. Pregnant and lactating women and those intending to become pregnant during the trial or within 90 days after the last administration of IMP.
- •12. Current or history (ie, within 12 months of screening) of alcohol, drug, or medication abuse.
- •13. Any other known autoimmune disease that, in the opinion of the investigator, would interfere with an accurate assessment of clinical symptoms of PV or PF or put the participant at undue risk.
- •14. The patient has a Karnofsky performance score <60% (see Section 10.9).
- •15. Vaccination with live/attenuated viral vaccines within 28 days prior to randomization (see Section 8.3.7).
- •16. The patient has clinically significant uncontrolled active or chronic bacterial, viral, or
- •fungal infection.
- •17. Positive serum test at screening for an active viral infection with any of the following conditions:
- •a. Hepatitis B Virus (HBV) that is indicative of an acute or chronic infection (https://www.cdc.gov/hepatitis/HBV/PDFs/SerologicChartv8.pdf; see Sect
研究者
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