Novel Treatment for Syndromic Ichthyoses

Northwestern University

Overview

This is an open label-pilot study to assess the efficacy and safety of a novel cholesterol-lovastatin topical solution in children with rare syndromic ichthyoses. Often times, these children have difficulty in finding easily applied treatments to make their psoriasiform and ichthyotic plaques more manageable. We propose the use of a cholesterol-lovastatin topical solution as a treatment option with the hypothesis that it will lead to regression of involved areas and decreased erythema and warty-like appearance of the plaques. We plan to enroll children with syndromic ichthyoses over the age of 1 year for a 12 month study with a total of 5 visits and 5 phone calls.

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Conditions

Syndromic Ichthyoses CHILD Syndrome Smith Lemli Opitz Syndrome Conradi Syndrome

Interventions

LovastatinDRUG

Topical lovastatin applied to red, rashy areas two times daily for 12 months

Eligibility

Sex: ALLMin age: 1 Year

Medical Language ↔ Plain English

Inclusion Criteria: * Informed Consent form signed by the subject or subject's legal representative; also, if the subject is under the age of majority but capable of providing assent, signed assent from the subject. * Diagnosis of a syndromic ichthyoses (verified by Dr Amy S. Paller). * Children and adults 12 months of age and above Exclusion Criteria: * Use of any investigational drug within the 30 days before enrollment. * Current malignancy. * Pregnancy or breastfeeding during the study. (All female subjects of childbearing potential will be assessed for pregnancy at all visits.)

Outcomes

Primary Outcomes

Physician global assessment of severity (PGAS)

Difference in physician global assessment of severity at baseline compared to month 12

Time frame: 12 months

Secondary Outcomes

Total body surface area (TBSA)

Percent change in TBSA affected at baseline compared to at month 12

Time frame: 12 months

Data from ClinicalTrials.gov

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