E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Hidradenitis suppurativa, Hurley stage I-III |
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E.1.1.1 | Medical condition in easily understood language |
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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 | Yes |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
To document whether LTX-109 is an effective compound on hidradenitis |
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E.2.2 | Secondary objectives of the trial |
Safety documentation Duration of clinical response (survival of effect) |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
Patients with active hidradenitis in the stage I-III according to Hurley’s classification. (24). Patients are referred to a dermatology out-patient clinic or patients already in an established treatment program, where there is indication for new or different treatment, or surgical intervention. Patients must have typical affection of the disease of either axillae, groins, and/or perigenital/perianal area Patients must be at least 18 years of age.
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E.4 | Principal exclusion criteria |
Patients in need of emergency medical or surgical treatment of hidradenitis will be excluded until the disease is in a quiet, controlled phase. Subjects must not have used the following HS treatments within the specified timeframe prior to Baseline Visit: o Systemic therapy for HS, including but not limited to corticosteroids, antibiotics, dapsone or retinoids within 4 weeks o Targeted biologic treatments (refer to within 5 half-lives [if known]) or within 12 weeks, whichever is longer; o Topical treatments with antibiotics, including but not limited to clindamycin within 4 weeks Pregnant or lactating women Any reason why, in the opinion of the investigator, the patient should not participate
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E.5 End points |
E.5.1 | Primary end point(s) |
Patient reported improvement after invention with LTX-109. Outcome measured by Dermatological Life Quality Index (DLQI)-scores and self-reported disease activity and pain experience.
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
[ Time Frame: Interim analysis after 3 weeks. End point analysis 6 weeks]. |
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E.5.2 | Secondary end point(s) |
• Identification of clinical subgroups with best response to intervention as assessed by clinically scored measures . Registration and analysis on investigator scored measures assessed by Hidradenitis suppurativa score (HiSCR) in relation to clinical phenotypes (location, Hurley stage)
• Identify if covariates such as age, disease duration, smoking state, BMI and sweating influence patient reported improvement measures Covariate analysis on patient recorded DLQI and self-reported disease activity and pain experience in relation to pre-registered variables
Identification of clinical subgroups with best response to intervention as assessed by patient reported improvement Registration and analysis on patient recorded DLQI and self-reported disease activity and pain experience in relation to clinical phenotypes (location specific score (Sartorius), Hurley stage, IHS-4)
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
[Time Frame: Interim analysis after 3 weeks. End point analysis 6 weeks and sub group analysis 3 months] |
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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 | 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 | 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
| 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 | 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 | 0 |
E.8.9.1 | In the Member State concerned months | 6 |
E.8.9.1 | In the Member State concerned days | 0 |