E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Discoid lupus erythematosus |
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E.1.1.1 | Medical condition in easily understood language |
Discoid lupus erythematosus (DLE) |
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E.1.1.2 | Therapeutic area | Diseases [C] - Skin and Connective Tissue Diseases [C17] |
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 | 10013072 |
E.1.2 | Term | Discoid lupus erythematosus |
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 investigate the efficacy of delgocitinib cream 20 mg/g twice daily on active DLE target lesions. |
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E.2.2 | Secondary objectives of the trial |
To evaluate the safety of delgocitinib cream 20 mg/g twice daily on active DLE target lesions
To further investigate the efficacy of delgocitinib cream 20 mg/g twice daily on active DLE target lesions |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
• Age 18-70 years. • Histopathological findings (current or previous) consistent with clinical diagnosis of DLE. • Unequivocal clinical diagnosis of 2 active DLE target lesions that are <6 months old and amenable for clinical evaluation. This includes lesions located on the scalp if they fulfil all lesion-specific eligibility criteria. • Target lesion Investigator Global Assessment score of at least moderate (≥3) at screening and baseline. • Target lesion erythema score ≥2 at screening and baseline.
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E.4 | Principal exclusion criteria |
• Target lesion dyspigmentation score of 2 at screening or baseline. • Target lesion scarring/atrophy score of 2 at screening or baseline. • Target lesion scarring alopecia score of >0 in scalp lesions at screening or baseline. • Medical history of systemic lupus erythematosus (SLE) with clinically significant organ involvement (American College of Rheumatology SLE classification criteria no. 6-9) including LE-related pleuritis or pericarditis (by clinical evaluation, i.e. no electrocardiogram or X-ray required), and neurologic, renal, and/or other major SLE-related organ system involvement. SLE joint involvement is acceptable. • Subjects with unstable or significant SLE disease activity findings that would, by its progressive nature and/or severity, interfere with the trial evaluation, completion, and/or procedures per the investigator's discretion. • Other skin conditions at screening or baseline that would interfere with the evaluation of DLE. • Immunosuppressive/immunomodulating therapy with e.g. methotrexate, cyclosporine, azathioprine, retinoids (both topical and systemic), or dapsone within 4 weeks prior to baseline. • Systemic prednisolone >7.5 mg/day or changed dose within 4 weeks prior to baseline (nasal and inhaled corticosteroids are allowed). • Treatment with the following medications: • Oral antimalarial treatment with hydroxychloroquine >6.5 mg/kg body weight/day, or chloroquine >4 mg/kg body weight/day, or changed dose within 12 weeks prior to baseline. • Quinacrine combined with either hydroxychloroquine or chloroquine within 12 weeks prior to baseline. • Drugs known to interact with antimalarials (e.g. digoxin, cimetidine) within 12 weeks prior to baseline. • Treatment with topical corticosteroids, calcineurin inhibitors, and phosphodiesterase-4 (PDE-4) inhibitors within 2 weeks prior to baseline. • Use of systemic antibiotics or cutaneously applied antibiotics on the target lesions within 2 weeks prior to baseline. • Ultraviolet (UV) therapy within 2 weeks prior to baseline. • Any procedure impairing the skin barrier (e.g. incision) within 2 cm from the border of any of the target lesions within 4 weeks prior to baseline. • Receipt of live (attenuated) vaccines within 4 weeks prior to baseline. • Treatment with any marketed biological therapy or investigational biologic agents: • Any cell-depleting agents including but not limited to rituximab: within 6 months prior to baseline, or until lymphocyte count returns to normal, whichever is longer. • Other biologics: within 3 months or 5 half-lives, whichever is longer, prior to baseline. • Smoking of >70 cigarettes/week or >70 g of tobacco content/week within 1 month prior to screening. • History of any active skin infection within 1 week prior to baseline. • Clinically significant infection within 4 weeks prior to baseline which, in the opinion of the investigator, may compromise the safety of the subject in the trial, interfere with evaluation of the IMP, or reduce the subject’s ability to participate in the trial. Clinically significant infections are defined as: • A systemic infection. • A serious skin infection requiring parenteral (intravenous or intramuscular) antibiotics, antiviral, or antifungal medication. • Tuberculosis requiring treatment within 12 months prior to screening and/or subjects with a positive blood test for tuberculosis at screening. Subjects with high risk of latent tuberculosis (e.g. prior residence in or travel to countries with high prevalence of tuberculosis, close contact with a person with active tuberculosis, or a history of active or latent tuberculosis where an adequate course of treatment cannot be confirmed) must be tested at screening.
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E.5 End points |
E.5.1 | Primary end point(s) |
• Target lesions with Investigator’s Global Assessment (IGA) score of 0 or 1 at Week 6. |
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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) |
• Number of adverse events (AEs) up to Week 6 • Number of subjects with AEs up to Week 6 • Number of lesion-specific, treatment-related AEs up to Week 6. • A ≥2-point reduction in IGA score at Week 6 compared to baseline. • A ≥2-point reduction in erythema score at Week 6 compared to baseline. • Erythema score at Week 6. • Total skin disease activity score (sum of scores for erythema, scaling/hyperkeratosis, and oedema/infiltration at Week 6.
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
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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 | No |
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) | Yes |
E.7.3 | Therapeutic confirmatory (Phase III) | No |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | Yes |
E.8.1.1 | Randomised | Yes |
E.8.1.2 | Open | No |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | Yes |
E.8.1.5 | Parallel group | No |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | Yes |
E.8.1.7.1 | Other trial design description |
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E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | No |
E.8.2.2 | Placebo | Yes |
E.8.2.3 | Other | No |
E.8.2.4 | Number of treatment arms in the trial | 2 |
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 | 4 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 13 |
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 |
Denmark |
France |
Germany |
United States |
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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 | 0 |
E.8.9.1 | In the Member State concerned months | 7 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 0 |
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 | 0 |