E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Genital erosive lichen planus |
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E.1.1.1 | Medical condition in easily understood language |
Lichen planus in the genital area |
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E.1.1.2 | Therapeutic area | Diseases [C] - Skin and Connective Tissue Diseases [C17] |
MedDRA Classification |
E.1.3 | Condition being studied is a rare disease | No |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
To assess the effect of apremilast treatment on GELP
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E.2.2 | Secondary objectives of the trial |
- To assess quality of life and sexual function in women with GELP before and during apremilast treatment - To describe immunohistochemical changes in GELP lesions after treatment with apremilast
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
- >18 years of age - Female - Moderate to severe GELP at inclusion with the diagnosis based on characteristic clinical and/or histological features. Minimum GELP score 5/30 in vagina and/or vulva (scored separately), of which erythema and pain ≥1 are mandatory - Informed consent from the patient to the protocol and clinical procedures.
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E.4 | Principal exclusion criteria |
- Patients receiving other systemic immune modulating therapy - Concomitant use of strong CYP3A4 enzyme inducers - Inadequate birth control, pregnancy and/or breast-feeding - Depression and suicidal ideation - Patients with severe renal impairment - Patients with active tuberculosis, serious infections or cancer - Unexplained and clinically significant weight loss in underweight patients - Hypersensitivity to the active substance(s) or to any of the excipients - Participating in another trial that might affect the current study or there should be minimum 90 days between participation in another intervention trial
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E.5 End points |
E.5.1 | Primary end point(s) |
Mean GELP score at week 16 in apremilast-treated patients versus placebo-treated patients |
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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) |
• Mean GELP score improvement from week 0 to week 32 in all patients • Weekly use of topical steroid, collected from patient diary • Weekly VAS pain score, collected from patient diary • Number of patients with GELP score improvement at week 16 and 32. • Separate GELP score assessments: Area of involvement (in cm2), number of erosions, erythema, striae and pain (VAS) at week 4, 16 and 32 • Physician Global Assessment (PGA) and Patient Global Assessment (PtGA) scores at week 0, 16 and 32 • Selected QoL measures (DLQI, GHQ-28) at week 0, 16 and 32 • Sexual function assessments (FSDS-R) at week 0, 16 and 32 • Description of immune histochemical changes and expression of selected cytokines before and after apremilast therapy, assessed in vulvar or vaginal biopsies at week 0 and 16 • Description of immune histochemical findings in oral biopsies at baseline (week 0) • Immunological markers before and after apremilast therapy, assessed in blood samples collected at week 0 and 16 • Assessment of extragenital lichen planus at week 0, 16 and 32 • Evaluation of clinical photos taken at week 0 and week 32 • Adverse events |
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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 | Yes |
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 |
- Immunohistochemical response in affected tissue - Immunological markers in blood before and during treatment
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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) | 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 | No |
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
| Yes |
E.8.4 | The trial involves multiple sites in the Member State concerned | No |
E.8.5 | The trial involves multiple Member States | No |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | No |
E.8.6.2 | Trial being conducted completely outside of the EEA | No |
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 | |
E.8.9.1 | In the Member State concerned days | |