Novel Treatment for Syndromic Ichthyoses

Phase 2

Withdrawn

• Conditions: Smith Lemli Opitz Syndrome; Conradi Syndrome; Syndromic Ichthyoses; CHILD Syndrome
• Interventions: Drug: Lovastatin

• Registration Number: NCT01110642
• Lead Sponsor: Northwestern University

Brief Summary

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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2010-04-22
• Study First Submitted QC: 2010-04-23
• Study First Posted: 2010-04-26
• Study Start: 2011-07
• Status Verified: 2015-05
• Last Update Submitted: 2015-05-15
• Last Update Posted: 2015-05-19

Recruitment & Eligibility

• Status: WITHDRAWN
• Sex: All
• Target Recruitment: Not specified

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 & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Lovastatin solution	Lovastatin	All patients will receive lovastatin solution

Primary Outcome Measures

Name	Time	Method
Physician global assessment of severity (PGAS)	12 months	Difference in physician global assessment of severity at baseline compared to month 12

Secondary Outcome Measures

Name	Time	Method
Total body surface area (TBSA)	12 months	Percent change in TBSA affected at baseline compared to at month 12