E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
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E.1.1.1 | Medical condition in easily understood language |
Autoimmune disease that causes painful or itchy blisters on the skin |
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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 | 21.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10006567 |
E.1.2 | Term | Bullous pemphigoid |
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 long-term safety and tolerability of treatment with efgartigimod PH20 SC in participants with BP |
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E.2.2 | Secondary objectives of the trial |
To assess the long-term efficacy and durability of response with efgartigimod PH20 SC treatment in participants with BP To evaluate the impact of efgartigimod PH20 SC treatment in reducing long-term glucocorticoid-associated toxicity in participants with BP To evaluate the impact of efgartigimod PH20 SC treatment on QoL in participants with BP To evaluate the PD and immunogenicity of efgartigimod PH20 SC in participants with BP |
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E.2.3 | Trial contains a sub-study | Yes |
E.2.3.1 | Full title, date and version of each sub-study and their related objectives |
Substudy – Vaccination Response Sites may collect additional blood samples for additional/optional/future vaccination research. Such research may include (but not limited to) the following: 1. Humoral (serum) responses to vaccinations received during the study 2. Cellular (peripheral blood mononuclear cells [PBMCs]) responses to vaccinations received during the study Postvaccination samples will be collected at the visits identified in the Summary of Activities only from participants who took part in the vaccination substudy during ARGX-113-2009. Data obtained from this substudy will not be included in the clinical database; the results may be described in a separate report.
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E.3 | Principal inclusion criteria |
1. Has completed the week 36 visit of ARGX-113-2009 2. Is capable of providing signed informed consent and complying with protocol requirements 3. Agrees to use contraceptive measures consistent with local regulations and the following: a. Male participants: An acceptable method of contraception is a condom. All nonsterilized male participants must use this method from signing of the ICF until the date of the last dose of IMP. b. Women of childbearing potential must have a negative urine pregnancy test at baseline before receiving IMP. WOCBP must use one of the contraception methods described in the protocol from signing the ICF until the last dose of IMP |
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E.4 | Principal exclusion criteria |
1. Clinically significant disease, recent major surgery (within 3 months of baseline), or intention to have surgery during the study; or any other medical condition that, in the investigator’s opinion would confound the results of the study or put the participant at undue risk 2. Known hypersensitivity to IMP or 1 of its excipients 3. Permanently discontinued IMP in ARGX-113-2009 due to an AE considered related to IMP and for whom the benefit/risk balance is not considered positive |
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E.5 End points |
E.5.1 | Primary end point(s) |
Incidence and severity of treatment-emergent adverse events (TEAEs), serious AEs (SAEs), and AEs of special interest (AESIs) Rate of treatment discontinuation because of safety concerns
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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. Proportions of participants achieving: - complete remission (CR) while off oral corticosteroids (OCS) for ≥8 weeks - CR or partial remission (PR) while off OCS for ≥8 weeks - CR while on minimal OCS therapy for ≥8 weeks. (Minimal OCS therapy is defined as ≤0.10 mg/kg/day of prednisone [or an equivalent dose of another OCS]) - CR while off both OCS and efgartigimod PH20 SC for ≥8 weeks - CR or PR while off both OCS and efgartigimod PH20 SC for ≥8 weeks 2. Duration of sustained remission 3. Proportion of participants who relapse 4. Time to relapse 5. Incidence and severity of relapse 6. Bullous Pemphigoid Disease Area Index (BPDAI) activity scores, Investigator Global Assessment of Bullous Pemphigoid (IGA-BP) scores, and itch numerical rating scale (NRS) over time 7. Rate of treatment failure 8. GTI-related scores, including the GTI-AIS, GTI CWS, and GTI SL over time 9. EQ-5D-5L, DLQI, and ABQoL scores over time 10. Percent change from baseline over time for anti BP180 and anti BP230 antibody levels 11. Incidence and prevalence of ADA against efgartigimod (serum levels) |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
1-5 & 7: Up to wk 56 6: At wks 0, 2, 4, 8, 16, 24, 32, 40, 48 & 56 when EFG is NA. When EFG continues/starts its evaluated at wks 0, 2, 4, 8, then every 4 wks to EFG stop, every 8 wks after & at wks 48, 52 & 56 8 & 9: At wks 0, 24 & 48 when EFG is NA. When EFG continues/starts its evaluated at wks 0, 8, every 16 wks up to & after EFG stop, then at wk 48 10: At wks 0, 2, 4, 8, 16, 24, 32, 40, 48 & 56 when EFG is NA. When EFG continues/starts its evaluated at wks 0, 2, 4, 8, then every 4 wks to EFG stop, every 8 wks after, then at wks 48, 52 & 56 11: At wks 0, 2, 4, 8, 16, 24, 32, 40, 48 & 56 when EFG is NA. When EFG continues/starts its evaluated at wks 0, 4, 8, then every 8 wks to EFG stop, every 8 wks after, then at wks 48, 52 & 56 |
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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 | No |
E.6.7 | Pharmacodynamic | No |
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 | Yes |
E.8.4.1 | Number of sites anticipated in Member State concerned | 5 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 70 |
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 |
Canada |
China |
Israel |
Japan |
Russian Federation |
Serbia |
United Kingdom |
United States |
Bulgaria |
Croatia |
Czechia |
France |
Germany |
Greece |
Hungary |
Italy |
Latvia |
Netherlands |
Poland |
Romania |
Slovakia |
Spain |
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E.8.7 | Trial has a data monitoring committee | No |
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 | 7 |
E.8.9.1 | In the Member State concerned days | 15 |
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 | 7 |
E.8.9.2 | In all countries concerned by the trial days | 15 |