Trial record 1 of 1 for:
"X-linked chondrodysplasia punctata 2" OR "Conradi Hunermann Syndrome" OR "Happle Syndrome"
Novel Treatment for Syndromic Ichthyoses
This study has been withdrawn prior to enrollment.
(Study was withdrawn due to lack of eligible population for study)
Information provided by (Responsible Party):
Amy Paller, Northwestern University
First received: April 22, 2010
Last updated: November 29, 2012
Last verified: November 2012
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.
Smith Lemli Opitz Syndrome
||Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
||Open-label, Pilot Study to Assess Cholesterol-Lovastatin Solution in the Treatment of Syndromic Ichthyoses
Primary Outcome Measures:
Secondary Outcome Measures:
| Study Start Date:
Experimental: Lovastatin solution
All patients will receive lovastatin solution
Topical lovastatin applied to red, rashy areas two times daily for 12 months
|Ages Eligible for Study:
||1 Year and older
|Genders Eligible for Study:
|Accepts Healthy Volunteers:
- 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
- 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.)
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study.
To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below.
For general information, see Learn About Clinical Studies.
Please refer to this study by its ClinicalTrials.gov identifier: NCT01110642
||Amy S Paller, MD
No publications provided
||Amy Paller, Professor and Chair of Department of Dermatology, Professor of Pediatrics, Northwestern University
History of Changes
|Other Study ID Numbers:
|Study First Received:
||April 22, 2010
||November 29, 2012
||United States: Food and Drug Administration
Additional relevant MeSH terms:
ClinicalTrials.gov processed this record on July 26, 2014
Infant, Newborn, Diseases
Ichthyosiform Erythroderma, Congenital
Skin Diseases, Genetic
Genetic Diseases, Inborn
Lipid Metabolism, Inborn Errors
Metabolism, Inborn Errors
Steroid Metabolism, Inborn Errors
Lipid Metabolism Disorders
Bone Diseases, Developmental
Hydroxymethylglutaryl-CoA Reductase Inhibitors
Molecular Mechanisms of Pharmacological Action