A study to assess the safety and efficacy of a subcutaneous formulation of efgartigimod PH20 SC in adults with Pemphigus (Vulgaris or Foliaceus)
- Conditions
- Pemphigus Vulgaris or Pemphigus FoliaceusMedDRA version: 20.0Level: LLTClassification code 10052802Term: Pemphigus vulgarisSystem Organ Class: 100000004858MedDRA version: 20.0Level: LLTClassification code 10057069Term: Pemphigus foliaceusSystem Organ Class: 100000004858Therapeutic area: Diseases [C] - Immune System Diseases [C20]
- Registration Number
- EUCTR2020-002915-23-GR
- Lead Sponsor
- argenx BV
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 213
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:
a. Male participants:
-Male participants must agree to use acceptable method of contraception, from signing the ICF until the last dose of IMP.
b. Female participants:
- Women of childbearing potential must:
--have a negative serum pregnancy test at screening and a negative urine pregnancy test at baseline before the IMP can be
administered.
-- agree to use a highly effective or acceptable contraception method, which should be maintained at minimum until the last dose of IMP.
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 149
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 64
.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 Participant has been administered therapy other than oral prednisone, 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, dapsone, sulfasalazine, tetracyclines, nicotinamide
at doses above the recommended daily allowance (RDA)/dietaryreference 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 antiCD20 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
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. Participants with clinical evidence of other significant serious disease or participants 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 participant at undue risk.
11. Pregnant and lactating women and those intending to become pregnant during the trial.
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 participant has a Karnofsky performance score <60%.
15. Vaccination with live/attenuated viral vaccines within 28 days prior to randomization.
16. The participant 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)
b. Hepatitis C Virus (HCV)
c. HIV based on test results that are associated with an AIDS- defining condition or a CD4 count < =200 cells/mm3
18. The participant has total immunoglobulin G (IgG) <6 g/L at screening.
19.
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method