E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Hidradenitis suppurativa, acne inversa, ectopic acne |
Hidradenitis suppurativa, acne inversa, ectopisch acne |
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E.1.1.1 | Medical condition in easily understood language |
Hidradenitis suppurativa, acne inversa, ectopic acne |
Hidradenitis suppurativa, acne inversa, ectopisch acne |
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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 |
Primary objectives: To evaluate the effects of apremilast on the expression of inflammatory cytokines in HS lesional skin at week one (t=1), week four (t=4) and week sixteen (t=16) relative to baseline (t=0), in relation to its clinical efficacy. |
Primaire doel: Om de effecten van apremilast op de expressie van inflammatoire cytokines in de door HS aangedane huid te evalueren in relatie tot de klinische werkzaamheid in week één (t= 1), week vier (t = 4) en week zestien (t = 16) ten opzichte van baseline (t = 0 ) . |
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E.2.2 | Secondary objectives of the trial |
Secondary objectives:
- To prospectively evaluate the clinical efficacy of apremilast.
- To assess the effect of apremilast on patient reported outcomes measures.
- To assess the short-term safety and tolerability of apremilast in patients with hidradenitis suppurativa. |
Secundaire doelstellingen:
- Om de klinische werkzaamheid van apremilast prospectief te evalueren.
- Om het effect van apremilast op de door patiënt gerapporteerde uitkomsten te beoordelen.
- Om de korte termijn veiligheid en verdraagzaamheid van apremilast bij patiënten met hidradenitis suppurativa te beoordelen (in het kader van off label gebruik) |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
Key inclusion criteria: adult (≥ 18 years of age) male or female patients with moderate HS according to a PGA of 3 on the 5-point HS-Physician Global Assessment (HS-PGA); HS of more than 6 months duration; have lesions in at least two anatomical locations. |
Belangrijkste inclusiecriteria: volwassen (≥ 18 jaar) mannelijke of vrouwelijke patiënten met matig-ernstige HS volgens een PGA van 3 op de 5-punts HS-Physician Global Assessment (HS-PGA); een duur van HS van meer dan 6 maanden; HS-laesies in ten minste twee anatomische regio's. |
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E.4 | Principal exclusion criteria |
Key exclusion criteria: contraindication for apremilast; previous use of apremilast; have any current and/or recurrent clinically significant skin condition in the treatment area other than HS; presence of other uncontrolled major disease; pregnant or lactating women. |
Belangrijkste exclusiecriteria: contra-indicatie voor apremilast; eerder gebruik van apremilast; een thans aanwezige of recidiverende klinisch significante aandoening van de huid in het te behandelen gebied anders dan HS; aanwezigheid van een ernstige (actieve) zich openbarende ziekte; zwangere vrouwen of vrouwen die borstvoeding geven. |
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E.5 End points |
E.5.1 | Primary end point(s) |
The expression levels of inflammatory cytokine protein and mRNA in HS lesional skin will be analyzed at week one (t=1), week four (t=4) and week sixteen (t=16) relative to baseline (t=0), in relation to its clinical efficacy. The proteins and genes of interest to be measured are IL-1β, IL-6, TNF-α, IL-10, IL-12, IL-23, IL-17, IFN-γ, IL-31. Exactly the same cytokines will be measured using ELISA and qPCR in order to correlate the data. |
De expressieniveaus van inflammatoire cytokines (eiwit en mRNA) in de door HS aangedane huid zal worden geanalyseerd in week één (t = 1), week vier (t = 4) en week zestien (t = 16) en vergeleken ten opzichte van baseline (t = 0), tezamen in relatie tot de klinische werkzaamheid.
De eiwitten en genen van belang te meten zijn IL-1β, IL-6, TNF-α, IL-10, IL-12, IL-23, IL-17, IFN-γ, IL-31. De gebruikte onderzoekstechnieken zijn ELISA en qPCR. |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
at week one (t=1), week four (t=4) and week sixteen (t=16) relative to baseline (t=0), |
week 0, week 1, week 4, week 16 |
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E.5.2 | Secondary end point(s) |
The clinical efficacy will be measured using the following scoring systems:
• Individual inflammatory lesion count (Appendix 3)
• HS-Physician Global Assessment (HS-PGA)(16) (Appendix 4)
• Hidradenitis Suppurativa Clinical Response (HiSCR) (17) (Appendix 5)
The patient reported outcomes will be measured using the following scoring sytems:
• NRS to assess pain, pruritus and patient disease global assessment score (Appendix 6, 7, 8)
• DLQI (18) (Appendix 9) |
Voor gedetailleerde weergave zie Appendix 3 tot 9 van het studieprotocol
De klinische efficitivieit zal worden gemeten met behulp van de volgende scoring-systemen
• Individuele inflammatoire laesie telling
• HS-Physician Global Assessment (HS-PGA)
• Hidradenitis suppurativa klinische respons score (HiSCR)
De patiënt gerapporteerde uitkomsten zullen worden gemeten aan de hand van de volgende scoresysteem:
• NRS om pijn, jeuk en de patiënt de ziekte globale beoordeling score te beoordelen
• DLQI |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
week 0, 2, 4, 8, 12, 16 |
week 0, 2, 4, 8, 12, 16 |
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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 | No |
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 | No |
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.8.3 |
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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Last visit is visit 8, i.e. at week 16 |
laatste visitie is visite 8, d.w.z. op week 16 |
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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 | |
E.8.9.1 | In the Member State concerned months | |
E.8.9.1 | In the Member State concerned days | |