E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Patients with the chronic inflammatory skin disease Lichen planus will be treated with the JAK inhibitor upadacitinib. |
Patienten, die an der chronisch inflammatorischen Hauterkrankung Lichen planus leiden, werden mit dem JAK Inhibitor Upadacitinib behandelt. |
|
E.1.1.1 | Medical condition in easily understood language |
Patients with a skin disease (Lichen planus) will be treated with a substance that reduces the action of the immune system |
Patienten mit einer Hauterkrankung (Lichen planus) werden mit einer Substanz behandelt, die die Aktivität des Immunsystems reduziert. |
|
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 | 20.1 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10030983 |
E.1.2 | Term | Oral lichen planus |
E.1.2 | System Organ Class | 10017947 - Gastrointestinal disorders |
|
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 evaluate the efficacy of upadacitinib (Rinvoq®) therapy in patients with Lichen planus. |
Bewertung der Immunantwort, Wirksamkeit und Sicherheit von Upadacitinib (Rinvoq®) Behandlung bei Patienten mit Lichen planus. |
|
E.2.2 | Secondary objectives of the trial |
To evaluate the immunological response and safety of upadacitinib (Rinvoq®) therapy in patients with Lichen planus. |
Bewertung der Immunantwort und Sicherheit von Upadacitinib (Rinvoq®) Behandlung bei Patienten mit Lichen planus. |
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. Patients with diagnosis of acute or chronic (>3 months) Lichen planus and with a clinical presentation and histopathology consistent (performed within 6 months prior to screening) with Lichen planus. 2. Patients with moderate to severe disease and Investigator Global Assessment (IGA) score ≥ 3 (scale of 0 to 4) at screening and baseline visit. 3. Patient with involvement of the oral cavity (with at least one target lesion of oral lichen planus). 4. Male or female patient aged 18 to 65 years old. 5. Patients with body weight ≥ 40 kg and ≤ 120 kg. |
1. Patienten mit einem diagnostizierten akuten oder chronischen Lichen planus (>3 Monate), histopathologisch mit Lichen planus übereinstimmend (innerhalb 6 Monate vor dem Screening). 2. Patienten mit moderater bis schwerer Erkrankung und einer IGA (Investigators global assessment) Punktzahl von ≥ 3 (Skala 0-4) bei Screening und Baseline Visite. 3. Patienten, bei denen die Mundhöhle betroffen ist (mindestens eine Zielläsion von oralem Lichen planus). 4. Männliche oder weibliche Patienten im Alter von 18 bis 65 Jahren 5. Patienten mit einem Körpergewicht zwischen ≥ 40 kg und ≤ 120 kg.
|
|
E.4 | Principal exclusion criteria |
1. Presence of skin comorbidities that may interfere with the study assessments. 2. Evidence of acute contact dermatitis at screening. 3. Evidence of other Lichen planus variants including but not limited to hypertrophic, atrophic, follicular (including lichen planopilaris), and bullous cutaneous forms. Evidence of asymptomatic reticulate or hypertrophic oral Lichen planus. 4. History of allergy to any component of the study medication. 5. Women of childbearing potential who are unwilling to practice highly effective contraception prior to the initial dose/start of the first treatment, during the study, and for at least 12 weeks after the last dose. 6. Pregnant or breastfeeding women or planning to become pregnant or breastfeed during the patient’s participation in this study. |
1. Vorliegen von Haut Komorbiditäten, die eventuell die Studienbewertung beeinträchtigen könnten. 2. Nachweis einer akuten Kontakt-Dermatitis beim Screening 3. Nachweis anderer Lichen planus Varianten einschließlich aber nicht begrenzt auf hypertrophe, atrophe, follikuläre (einschließlich Lichen planopilaris), und bullöse kutane Formen. Nachweis von asymptomatischen netzartigen oder hypertrophen oralen Lichen planus. 4. Allergie gegen jegliche Komponenten der Prüfmedikation. 5. Frauen im gebärfähigen Alter, die nicht in die Einnahme von hochwirksamen Verhütungsmethoden vor der ersten Dosisgabe/Beginn der Behandlung, während der Studie und mindestens 12 Wochen nach der letzten Dosis einwilligen. 6. Schwangere oder stillende Frauen oder Frauen, die planen schwanger zu werden oder während der Studienteilnahme zu stillen. 7. Teilnehmer, die gegenwärtig an einer anderen Studie mit Gerätestudie oder Arzneimittelprüfung teilnehmen und das Arzneimittel innerhalb der letzten 90 Tage vor Screening erhalten haben.
|
|
E.5 End points |
E.5.1 | Primary end point(s) |
Therapeutic efficacy defined as the percentage of patients achieving a clinical response (IGA 0 or 1) in mucosal disease at week 12. |
Therapeutische Wirksamkeit definiert als der Anteil an Patienten, welche eine klinische Verbesserung, gemessen am IGA (0-1) in mukosaler Erkrankung in Woche 12, erzielt haben. |
|
E.5.1.1 | Timepoint(s) of evaluation of this end point |
|
E.5.2 | Secondary end point(s) |
Improvement of the Participant self-assessment (PSA) from baseline. Improvement of the Physician Assessment of Surface Area of Disease (PSAD) from baseline. Improvement of the pain on Visual Analogue Scale (VAS) from baseline. The Improvement of pruritus (itching) on Visual Analogue Scale (VAS) from baseline. The Improvement of immunological effects of upadacitinib from baseline. Improvement of DLQI from baseline.
|
Verbesserung der Teilnehmer-Selbsteinschätzung (PSA) zur Baseline. Verbesserung der ärztlichen Beurteilung der Krankheitsoberfläche (PSAD) zur Baseline. Verbesserung des Schmerzes auf einer visuellen Analogskala (VAS) zur Baseline Verbesserung des Juckreizes auf einer visuellen Analogskala (VAS) zur Baseline. Verbesserung der immunologischen Effekte zur Baseline Verbesserung des DLQI zur Baseline. |
|
E.5.2.1 | Timepoint(s) of evaluation of this end point |
PSA: week 2, 4, 6, 8, and 12 PSAD: week 4, 8, and 12 Pain VAS: week 4, 8, and 12 Itch VAS: week 4, 8, and 12 Immunological effects: week 12 DLQI: week 4, 8, and 12 |
PSA: Woche 2, 4, 6, 8, and 12 PSAD: Woche 4, 8, and 12 Pain VAS: Woche 4, 8, and 12 Itch VAS: Woche 4, 8, and 12 Immunological effects: Woche 12 DLQI: Woche 4, 8, and 12 |
|
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 | Yes |
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
|
|
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 | 10 |
E.8.9.1 | In the Member State concerned days | 0 |