Novel Treatment for Syndromic Ichthyoses

CHILD Syndrome Clinical Trial

Official title:

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

Clinical Trial Details — Status: Withdrawn

Administrative data

• NCT number: NCT01110642
• Other study ID #: 2010-04-15
• Status: Withdrawn
• Phase: Phase 2
• First received: April 22, 2010
• Last updated: May 15, 2015
• Start date: July 2011

Study information

• Verified date: May 2015
• Source: Northwestern University
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Food and Drug Administration
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Withdrawn
• Enrollment: 0
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 1 Year and older

Eligibility

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

Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• CHILD Syndrome
• Conradi Syndrome
• Ichthyosis
• Smith Lemli Opitz Syndrome
• Smith-Lemli-Opitz Syndrome
• Syndrome
• Syndromic Ichthyoses

Intervention

Drug:
• Lovastatin
Topical lovastatin applied to red, rashy areas two times daily for 12 months

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
Northwestern University

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Physician global assessment of severity (PGAS)	Difference in physician global assessment of severity at baseline compared to month 12	12 months	No
Secondary	Total body surface area (TBSA)	Percent change in TBSA affected at baseline compared to at month 12	12 months	No