Novel Treatment for Syndromic Ichthyoses

Open-label, Pilot Study to Assess Cholesterol-Lovastatin Solution in the Treatment of Syndromic Ichthyoses

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 Overview

• Status: Withdrawn
• Conditions: CHILD Syndrome; Smith Lemli Opitz Syndrome; Syndromic Ichthyoses; Conradi Syndrome
• Intervention / Treatment: Drug: Lovastatin

• Study Type: Interventional
• Phase: Phase 2

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 1 year and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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.)

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Lovastatin solution; All patients will receive lovastatin solution	Drug: Lovastatin; Topical lovastatin applied to red, rashy areas two times daily for 12 months

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Physician global assessment of severity (PGAS)	Difference in physician global assessment of severity at baseline compared to month 12	12 months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Total body surface area (TBSA)	Percent change in TBSA affected at baseline compared to at month 12	12 months

Collaborators and Investigators

• Sponsor: Northwestern University

Study record dates

Study Major Dates

• Study Start: July 1, 2011

Study Registration Dates

• First Submitted: April 22, 2010
• First Submitted That Met QC Criteria: April 23, 2010
• First Posted (Estimate): April 26, 2010

Study Record Updates

• Last Update Posted (Estimate): May 19, 2015
• Last Update Submitted That Met QC Criteria: May 15, 2015
• Last Verified: May 1, 2015

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Pathologic Processes; Metabolic Diseases; Skin Diseases; Disease; Congenital Abnormalities; Infant, Newborn, Diseases; Genetic Diseases, Inborn; Skin Diseases, Genetic; Metabolism, Inborn Errors; Lipid Metabolism Disorders; Dyslipidemias; Skin Abnormalities; Abnormalities, Multiple; Lipid Metabolism, Inborn Errors; Steroid Metabolism, Inborn Errors; Keratosis; Ichthyosiform Erythroderma, Congenital; Syndrome; Ichthyosis; Ichthyosis, Lamellar; Smith-Lemli-Opitz Syndrome; Molecular Mechanisms of Pharmacological Action; Enzyme Inhibitors; Antimetabolites; Anticholesteremic Agents; Hypolipidemic Agents; Lipid Regulating Agents; Hydroxymethylglutaryl-CoA Reductase Inhibitors; Lovastatin; L 647318; Dihydromevinolin
• Other Study ID Numbers: 2010-04-15