A Pilot Study to Examine the Efficacy and Tolerability of Sirolimus on Epidermal Nevi Lesions in Patients 6 Years Old and Above.
Studie Overzicht
Toestand
Conditie
Interventie / Behandeling
Gedetailleerde beschrijving
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.
Studietype
Inschrijving (Geschat)
Fase
- Vroege fase 1
Contacten en locaties
Studiecontact
- Naam: Dermatology CTU
- Telefoonnummer: 312-503-5944
- E-mail: NUderm-research@northwestern.edu
Studie Locaties
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Illinois
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Chicago, Illinois, Verenigde Staten, 60611
- Northwestern University Feinberg School of Medicine, Department of Dermatology
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Deelname Criteria
Geschiktheidscriteria
Leeftijden die in aanmerking komen voor studie
- Kind
- Volwassen
- Oudere volwassene
Accepteert gezonde vrijwilligers
Beschrijving
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
Studie plan
Hoe is de studie opgezet?
Ontwerpdetails
- Primair doel: Behandeling
- Toewijzing: NVT
- Interventioneel model: Opdracht voor een enkele groep
- Masker: Geen (open label)
Wapens en interventies
Deelnemersgroep / Arm |
Interventie / Behandeling |
|---|---|
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Experimenteel: Patients 6 years of age or older with a non-inflammatory epidermal nevi
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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.
Andere namen:
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Wat meet het onderzoek?
Primaire uitkomstmaten
Uitkomstmaat |
Maatregel Beschrijving |
Tijdsspanne |
|---|---|---|
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Improvement of nevi flattening and normalization of skin color
Tijdsspanne: 12 weeks
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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
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Secundaire uitkomstmaten
Uitkomstmaat |
Maatregel Beschrijving |
Tijdsspanne |
|---|---|---|
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Satisfaction scores of treatment
Tijdsspanne: 12 weeks
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Patients (and parents/LAR) will be asked for their satisfaction of the treatment (not satisfied, satisfied, very satisfied) at week 12
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12 weeks
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Patient-reported improvement of lesion
Tijdsspanne: 12 weeks
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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
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12 weeks
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Reduction in peak itch score
Tijdsspanne: 12 weeks
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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
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12 weeks
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Medewerkers en onderzoekers
Sponsor
Onderzoekers
- Hoofdonderzoeker: Amy Paller, MD, Northwestern University
Studie record data
Bestudeer belangrijke data
Studie start (Geschat)
Primaire voltooiing (Geschat)
Studie voltooiing (Geschat)
Studieregistratiedata
Eerst ingediend
Eerst ingediend dat voldeed aan de QC-criteria
Eerst geplaatst (Werkelijk)
Updates van studierecords
Laatste update geplaatst (Werkelijk)
Laatste update ingediend die voldeed aan QC-criteria
Laatst geverifieerd
Meer informatie
Termen gerelateerd aan deze studie
Trefwoorden
Aanvullende relevante MeSH-voorwaarden
Andere studie-ID-nummers
- STU00224904
Plan Individuele Deelnemersgegevens (IPD)
Bent u van plan om gegevens van individuele deelnemers (IPD) te delen?
Informatie over medicijnen en apparaten, studiedocumenten
Bestudeert een door de Amerikaanse FDA gereguleerd geneesmiddel
Bestudeert een door de Amerikaanse FDA gereguleerd apparaatproduct
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