Novel Treatment for Syndromic Ichthyoses
Primary Purpose
Syndromic Ichthyoses, CHILD Syndrome, Smith Lemli Opitz Syndrome
Status
Withdrawn
Phase
Phase 2
Locations
Study Type
Interventional
Intervention
Lovastatin
Sponsored by
About this trial
This is an interventional treatment trial for Syndromic Ichthyoses
Eligibility Criteria
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.)
Sites / Locations
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Lovastatin solution
Arm Description
All patients will receive lovastatin solution
Outcomes
Primary Outcome Measures
Physician global assessment of severity (PGAS)
Difference in physician global assessment of severity at baseline compared to month 12
Secondary Outcome Measures
Total body surface area (TBSA)
Percent change in TBSA affected at baseline compared to at month 12
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT01110642
Brief Title
Novel Treatment for Syndromic Ichthyoses
Official Title
Open-label, Pilot Study to Assess Cholesterol-Lovastatin Solution in the Treatment of Syndromic Ichthyoses
Study Type
Interventional
2. Study Status
Record Verification Date
May 2015
Overall Recruitment Status
Withdrawn
Why Stopped
Study was withdrawn due to lack of eligible population for study
Study Start Date
July 2011 (undefined)
Primary Completion Date
undefined (undefined)
Study Completion Date
undefined (undefined)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
Northwestern University
4. Oversight
Data Monitoring Committee
No
5. Study Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Syndromic Ichthyoses, CHILD Syndrome, Smith Lemli Opitz Syndrome, Conradi Syndrome
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
0 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Lovastatin solution
Arm Type
Experimental
Arm Description
All patients will receive lovastatin solution
Intervention Type
Drug
Intervention Name(s)
Lovastatin
Intervention Description
Topical lovastatin applied to red, rashy areas two times daily for 12 months
Primary Outcome Measure Information:
Title
Physician global assessment of severity (PGAS)
Description
Difference in physician global assessment of severity at baseline compared to month 12
Time Frame
12 months
Secondary Outcome Measure Information:
Title
Total body surface area (TBSA)
Description
Percent change in TBSA affected at baseline compared to at month 12
Time Frame
12 months
10. Eligibility
Sex
All
Minimum Age & Unit of Time
1 Year
Accepts Healthy Volunteers
No
Eligibility Criteria
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.)
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Amy S Paller, MD
Organizational Affiliation
Northwestern University
Official's Role
Principal Investigator
12. IPD Sharing Statement
Learn more about this trial
Novel Treatment for Syndromic Ichthyoses
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