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 | 10040785 - Skin and subcutaneous tissue disorders |
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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 | 10040785 - Skin and subcutaneous tissue disorders |
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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 treatment, extended treatment and re-treatment with efgartigimod PH20 SC in participants 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 assess the health impact of glucocorticoid (GC) use in participants with PV or PF To evaluate the effects of efgartigimod PH20 SC on quality of life (QoL) in participants with PV or PF To evaluate the pharmacokinetics (PK) of efgartigimod PH20 SC in participants with PV or PF To evaluate the PD of efgartigimod PH20 SC in participants with PV or PF To evaluate the immunogenicity of efgartigimod PH20 SC in participants with PV or PF To explore feasibility of self-administration of efgartigimod PH20 SC
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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 participant participated in trial ARGX-113-1904 and completed the study or has the defined criteria for rollover. 3A. Contraceptive use by men and women should be consistent with local regulations regarding the methods of contraception for those participating clinical trials and: Women of childbearing potential: i. Male participants: - Male participants must agree to use an acceptable method of contraception from signing the ICF (informed consent form) until the last dose the study drug. b. Female participants: - Women of childbearing potential must: - have 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 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: 1A. Pregnant and lactating women and those intending to become pregnant during the trial. 2. 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. 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 participants who achieve CR min 2. Proportion of PV and PF participants who achieve CR min. 3. Time to DC 4. Time to CR 5. Time to CR min 6. Time to CR off 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. Composite Glucocorticoid Toxicity Index (CGTI) comprising the Aggregate Improvement Score (AIS) and the Cumulative Worsening Score (CWS) 13. EuroQol 5-Dimension 5-Level (EQ-5D-5L) score 14. Autoimmune Bullous Disease Quality of Life (ABQOL) score 15. Efgartigimod serum concentrations 16. Total IgG and subtype (IgG1, IgG2, IgG3, IgG4) serum levels 17. Anti-Dsg-1 and -3 autoantibodies serum levels 18. Incidence and prevalence of antidrug antibodies (ADA) against efgartigimod (serum levels) 19. Percentage of participants who performed self-administration 20. Percentage of caregivers who administered the injection to the participant 21. Number of visits needed for the participant or caregiver to be competent to start administering efgartigimod PH20 SC 22. Frequency of self- or caregiver-supported administration at home |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
1,2,10,12,13,14,19,20,21 and 22: up to 52 weeks treatment period 3,4,5,6,7,8,9,11,15,16,17 and 18: 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 |
Immunogenicity; Tolerability |
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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 | Yes |
E.8.4.1 | Number of sites anticipated in Member State concerned | 6 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 60 |
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 |
Ukraine |
Australia |
China |
Georgia |
India |
Israel |
Japan |
Russian Federation |
Serbia |
United Kingdom |
United States |
Bulgaria |
Croatia |
France |
Germany |
Greece |
Hungary |
Italy |
Netherlands |
Poland |
Portugal |
Romania |
Spain |
Türkiye |
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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 | 3 |
E.8.9.1 | In the Member State concerned months | 3 |
E.8.9.1 | In the Member State concerned days | 23 |
E.8.9.2 | In all countries concerned by the trial years | 3 |
E.8.9.2 | In all countries concerned by the trial months | 3 |
E.8.9.2 | In all countries concerned by the trial days | 23 |