E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Pemphigus Vulgaris or Pemphigus Foliaceus |
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E.1.1.1 | Medical condition in easily understood language |
Blistering autoimmune diseases that affect the skin and mucous membranes |
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E.1.1.2 | Therapeutic area | Diseases [C] - Immune System Diseases [C20] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 20.0 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10052802 |
E.1.2 | Term | Pemphigus vulgaris |
E.1.2 | System Organ Class | 100000004858 |
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E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 20.0 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10057069 |
E.1.2 | Term | Pemphigus foliaceus |
E.1.2 | System Organ Class | 100000004858 |
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E.1.3 | Condition being studied is a rare disease | Yes |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
To assess the safety of extended treatment and re-treatment with efgartigimod PH20 SC in patients with PV or PF |
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E.2.2 | Secondary objectives of the trial |
To evaluate the efficacy of efgartigimod PH20 SC treatment in PV and PF
To evaluate the effects of efgartigimod PH20 SC on quality of life (QoL) in patients with PV or PF
To evaluate the pharmacokinetics (PK) of efgartigimod PH20 SC in patients with PV or PF
To evaluate the PD of efgartigimod PH20 SC in patients with PV or PF
To evaluate the immunogenicity of efgartigimod PH20 SC in patients with PV or PF
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
Participants must meet all of the following 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 participated in trial ARGX-113-1904 and completed the study or has the defined criteria for rollover.
3. Women of childbearing potential:
a. Must have 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) during the trial and for 90 days after the last administration of IMP.
4. 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. |
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E.4 | Principal exclusion criteria |
Participants are excluded from the trial if any of the following criteria apply:
1. Pregnant and lactating women and those intending to become pregnant during the trial or within 90 days after the last administration of IMP.
2. 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.
3. Known hypersensitivity to any of the components of the administered treatments. |
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E.5 End points |
E.5.1 | Primary end point(s) |
1. Incidence and severity of treatment-emergent adverse events (TEAEs), adverse events of special interest (AESIs), and serious adverse events (SAEs) by System Organ Class (SOC) and Preferred Term (PT)
2. Vital signs, physical examination, electrocardiogram (ECG), and clinical laboratory safety evaluations |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
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E.5.2 | Secondary end point(s) |
1. Proportion of PV patients who achieve CR on minimal prednisone therapy
2. Proportion of PV and PF patients who achieve CR on minimal prednisone therapy in patients with PV and PF 3.
3. Time to DC
4. Time to CR
5. Time to CR on minimal prednisone therapy
6. Time to CR off therapy
7. Time to flare
8. Rate of treatment failure
9. Rate of flare
10. Cumulative prednisone dose over the trial
11. Pemphigus Disease Area Index (PDAI) at each visit
12. EuroQol 5-Dimension 5-Level (EQ-5D-5L) score
13. Autoimmune Bullous Disease Quality of Life (ABQOL) score
14. Efgartigimod serum concentrations
15. Total IgG and subtype (IgG1, IgG2, IgG3, IgG4) serum levels
16. Anti-Dsg-1 and -3 autoantibodies serum levels
17. Anti-drug antibodies (ADAs) to efgartigimod (serum levels) and rHuPH20 (plasma levels)
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
1+2: up to 52 weeks treatment period
3,4,5,6,7,8,9: up to 60 weeks
10,11,12,13: up to 52 weeks treatment period
14,15,16, 17: up to 60 weeks |
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E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | No |
E.6.3 | Therapy | No |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | Yes |
E.6.7 | Pharmacodynamic | Yes |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | No |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | Yes |
E.6.13.1 | Other scope of the trial description |
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E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | No |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | No |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | No |
E.7.3 | Therapeutic confirmatory (Phase III) | Yes |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | No |
E.8.1.1 | Randomised | No |
E.8.1.2 | Open | Yes |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | No |
E.8.1.5 | Parallel group | No |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | No |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | No |
E.8.2.2 | Placebo | No |
E.8.2.3 | Other | No |
E.8.2.4 | Number of treatment arms in the trial | 1 |
E.8.3 |
The trial involves single site in the Member State concerned
| No |
E.8.4 | The trial involves multiple sites in the Member State concerned | No |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 51 |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | Yes |
E.8.6.2 | Trial being conducted completely outside of the EEA | No |
E.8.6.3 | If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned |
Australia |
Bulgaria |
France |
Georgia |
Germany |
Greece |
Hungary |
India |
Israel |
Italy |
Japan |
Poland |
Romania |
Russian Federation |
Spain |
Turkey |
Ukraine |
United Kingdom |
United States |
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E.8.7 | Trial has a data monitoring committee | Yes |
E.8.8 |
Definition of the end of the trial and justification where it is not the last
visit of the last subject undergoing the trial
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E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 2 |
E.8.9.1 | In the Member State concerned months | 9 |
E.8.9.1 | In the Member State concerned days | 25 |
E.8.9.2 | In all countries concerned by the trial years | 2 |
E.8.9.2 | In all countries concerned by the trial months | 9 |
E.8.9.2 | In all countries concerned by the trial days | 25 |