Novel Treatment for Syndromic Ichthyoses

NCT01110642 | Withdrawn Phase 2

• 1 other identifier: 2010-04-15
• Study Type: interventional
• Target: N/A
• Locations: 0 countries
• Sites: N/A

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.

Conditions

Syndromic Ichthyoses; CHILD Syndrome; Smith Lemli Opitz Syndrome; Conradi Syndrome

Outcome Measures

Primary Outcomes (1)

• Physician global assessment of severity (PGAS)

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

  12 months

Secondary Outcomes (1)

• Total body surface area (TBSA)

  12 months

Study Arms (1)

Lovastatin solution

EXPERIMENTAL

All patients will receive lovastatin solution

Drug: Lovastatin

Interventions

Lovastatin DRUG

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

Lovastatin solution

Eligibility Criteria

• Age: 1 Year+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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.)

Sponsors & Collaborators

• Northwestern University: lead

MeSH Terms

Conditions

Congenital Hemidysplasia with Ichthyosiform Erythroderma and Limb Defects; Smith-Lemli-Opitz Syndrome

Interventions

Lovastatin

Condition Hierarchy (Ancestors)

Abnormalities, Multiple; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Lipid Metabolism, Inborn Errors; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Steroid Metabolism, Inborn Errors; Dyslipidemias; Lipid Metabolism Disorders; Metabolic Diseases; Nutritional and Metabolic Diseases

Intervention Hierarchy (Ancestors)

Naphthalenes; Polycyclic Aromatic Hydrocarbons; Hydrocarbons, Aromatic; Hydrocarbons, Cyclic; Hydrocarbons; Organic Chemicals; Polycyclic Compounds

Study Officials

• Amy S Paller, MD

  Northwestern University

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor and Chair of Department of Dermatology, Professor of Pediatrics

Study Record Dates

• First Submitted: April 22, 2010
• First Posted: April 26, 2010
• Study Start: July 1, 2011
• Last Updated: May 19, 2015

Record last verified: 2015-05