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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Withdrawn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
Northwestern University
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Syndromic Ichthyoses

Eligibility Criteria

1 Year - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

    First Posted
    April 22, 2010
    Last Updated
    May 15, 2015
    Sponsor
    Northwestern University
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    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    Withdrawn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    Study was withdrawn due to lack of eligible population for study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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