A Pilot Study to Examine the Efficacy and Tolerability of Sirolimus on Epidermal Nevi Lesions in Patients 6 Years Old and Above.
Aperçu de l'étude
Statut
Les conditions
Intervention / Traitement
Description détaillée
Epidermal nevi are skin growths that develop because of a genetic change that occurs after conception, meaning only some skin cells are affected. These growths appear as thickened, often darker areas of skin that form lines or swirls. On the arms and legs, they usually appear as straight streaks, while on the trunk and face they follow curved patterns called the lines of Blaschko, which reflect how skin cells develop before birth.
These skin changes are caused by specific genetic changes that turn on certain cell growth pathways inside skin cells. Most linear verrucous epidermal nevi involve one of two major pathways that control cell growth: the PI3K/AKT/mTOR pathway or the RAS/MAPK pathway. In more than 40% of cases, changes in the FGFR3 or PIK3CA genes cause constant activation of the PI3K/AKT/mTOR pathway, leading to excess skin growth.
Sirolimus (also called rapamycin) is a medication that blocks the mTOR pathway, slowing down cell growth and protein production. Medications like this are already used to treat certain cancers caused by overactive mTOR signaling.
Topical sirolimus is FDA-approved as a 0.2% gel (Hyftor) to treat facial angiofibromas in people with tuberous sclerosis complex (TSC). In TSC, genetic changes cause the mTOR pathway to be overly active, leading to skin growths called angiofibromas. Studies have shown that applying sirolimus to the skin can reduce the size and visibility of these lesions.
Type d'étude
Inscription (Estimé)
Phase
- Première phase 1
Contacts et emplacements
Coordonnées de l'étude
- Nom: Dermatology CTU
- Numéro de téléphone: 312-503-5944
- E-mail: NUderm-research@northwestern.edu
Lieux d'étude
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Illinois
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Chicago, Illinois, États-Unis, 60611
- Northwestern University Feinberg School of Medicine, Department of Dermatology
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Critères de participation
Critère d'éligibilité
Âges éligibles pour étudier
- Enfant
- Adulte
- Adulte plus âgé
Accepte les volontaires sains
La description
Inclusion Criteria:
- Patients 6 years of age or above with a non-inflammatory epidermal nevi
- Lesion(s) that have been present for at least 3 years and are relatively stable
- At least 2 cm in length (ideally continuous, but can be cumulatively)
- The lesions is raised, hyperpigmented compared to the surrounding skin, and is noninflammatory with a physician global assessment (PGA) of at least 3 (moderate).
- Agree to avoid emollient use at the nevus site throughout the study
- Agree to not bath or shower 2 hours post-application, knowing that the gel can be applied after bathing or showering.
Exclusion Criteria:
- Patients currently or recently (within past 4 weeks) using a topical keratolytic or topical anti-inflammatory medication at the lesional site (i.e. topical steroid, topical retinoid, alpha-OH acid)
- Pregnant or planning to become pregnant during the course of the study
Plan d'étude
Comment l'étude est-elle conçue ?
Détails de conception
- Objectif principal: Traitement
- Répartition: N / A
- Modèle interventionnel: Affectation à un seul groupe
- Masquage: Aucun (étiquette ouverte)
Armes et Interventions
Groupe de participants / Bras |
Intervention / Traitement |
|---|---|
|
Expérimental: Patients 6 years of age or older with a non-inflammatory epidermal nevi
|
Topical sirolimus will be applied twice daily to the nevi with a maximum daily dose of 600 mg for patients 6-11 years of age and 800 mg for patients 12 years and older.
Autres noms:
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Que mesure l'étude ?
Principaux critères de jugement
Mesure des résultats |
Description de la mesure |
Délai |
|---|---|---|
|
Improvement of nevi flattening and normalization of skin color
Délai: 12 weeks
|
The extent of nevi improvement will be scored at baseline and week 12 from standardized photographs by two independent asessors with an adjudicator (if needed). The score will be defined as follows: 0 = 100% improvement
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12 weeks
|
Mesures de résultats secondaires
Mesure des résultats |
Description de la mesure |
Délai |
|---|---|---|
|
Satisfaction scores of treatment
Délai: 12 weeks
|
Patients (and parents/LAR) will be asked for their satisfaction of the treatment (not satisfied, satisfied, very satisfied) at week 12
|
12 weeks
|
|
Patient-reported improvement of lesion
Délai: 12 weeks
|
Patients (and parents/LAR) will be asked their self-evaluation of lesion improvment from 0-5 (0= cleared, 1= almost cleared, 2= good improvement, 3= slight improvement, 4= no change, 5= worse) at baseline and week 12
|
12 weeks
|
|
Reduction in peak itch score
Délai: 12 weeks
|
At least 2-point reduction in peak itch NRS score (on a scale of 0-10, 0=no itch and 10=worse itch) from baseline to week 12
|
12 weeks
|
Collaborateurs et enquêteurs
Parrainer
Les enquêteurs
- Chercheur principal: Amy Paller, MD, Northwestern University
Dates d'enregistrement des études
Dates principales de l'étude
Début de l'étude (Estimé)
Achèvement primaire (Estimé)
Achèvement de l'étude (Estimé)
Dates d'inscription aux études
Première soumission
Première soumission répondant aux critères de contrôle qualité
Première publication (Réel)
Mises à jour des dossiers d'étude
Dernière mise à jour publiée (Réel)
Dernière mise à jour soumise répondant aux critères de contrôle qualité
Dernière vérification
Plus d'information
Termes liés à cette étude
Mots clés
Termes MeSH pertinents supplémentaires
Autres numéros d'identification d'étude
- STU00224904
Plan pour les données individuelles des participants (IPD)
Prévoyez-vous de partager les données individuelles des participants (DPI) ?
Informations sur les médicaments et les dispositifs, documents d'étude
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