Novel Treatment for Syndromic Ichthyoses | NCT01110642

Studies
Novel Treatment for Syndromic Ichthyoses

NCT01110642

Sponsor

Northwestern University

Status

Withdrawn

Last Update Posted

May 19, 2015Estimated

Enrollment

0Actual

Phase

Phase 2

Conditions

Syndromic Ichthyoses
CHILD Syndrome
Smith Lemli Opitz Syndrome
Conradi Syndrome

Interventions

Lovastatin

Countries

Not provided

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

Status Module

Record Verification Date

May 2015

Overall Recruitment Status or Expanded Access Status

Withdrawn

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

Study was withdrawn due to lack of eligible population for study

Expanded Access Info

Start Date

Jul 2011

Primary Completion Date

Not provided

Completion Date

Not provided

First Submitted Date

Apr 22, 2010

First Submission Date that Met QC Criteria

Apr 23, 2010

First Posted Date

Apr 26, 2010Estimated

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

May 15, 2015

Last Update Posted Date

May 19, 2015Estimated

Sponsor/Collaborators Module

Responsible Party, by Official Title

Amy Paller, Professor and Chair of Department of Dermatology, Professor of Pediatrics, Northwestern UniversityPrincipal Investigator

Lead Sponsor

Northwestern UniversityOTHER

Collaborators

Not provided

Oversight Module

Has Data Monitoring Committee (DMC)

Is FDA Regulated Drug

Not provided

Is FDA Regulated Device

Not provided

Is Unapproved Device

Not provided

Pediatric Postmarket Surveillance of a Device Product

Not provided

Product Exported from US

Not provided

FDAAA801 Violation

Not provided

Description Module

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.

Detailed Description

Not provided

Design Module

Study Type

Interventional

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Not provided

Target Follow-Up Duration

Not provided

Phases

Phase 2

Interventional Study Design

Allocation

Biospecimen

No data available

No data is available for this block.

Enrollment

0Actual

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
Lovastatin solution	Experimental	All patients will receive lovastatin solution	Drug: Lovastatin

Interventions

Name	Type	Description	Arm Group Labels	Other Names
Lovastatin	Drug	Topical lovastatin applied to red, rashy areas two times daily for 12 months	Lovastatin solution

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
Physician global assessment of severity (PGAS)	Difference in physician global assessment of severity at baseline compared to month 12	12 months

Secondary Outcomes

Measure	Description	Time Frame
Total body surface area (TBSA)	Percent change in TBSA affected at baseline compared to at month 12	12 months

Other Outcomes

Not provided

Eligibility Module

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.)

Accepts Healthy Volunteers

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

1 Year

Maximum Age

Not provided

Standard Ages

ChildAdultOlder Adult

Study Population

Not provided

Sampling Method

Not provided

Name	Affiliation	Role
Amy S Paller, MD	Northwestern University	Principal Investigator

Condition Browse Module

MeSH Terms

ID	Term
C562515	Congenital Hemidysplasia with Ichthyosiform Erythroderma and Limb Defects
D019082	Smith-Lemli-Opitz Syndrome

Ancestor Terms

ID	Term
D000015	Abnormalities, Multiple
D000013	Congenital Abnormalities
D009358	Congenital, Hereditary, and Neonatal Diseases and Abnormalities
D008052	Lipid Metabolism, Inborn Errors

Browse Leaves

Not provided

Browse Branches

Not provided

Intervention Browse Module

MeSH Terms

ID	Term
D008148	Lovastatin

Ancestor Terms

ID	Term
D009281	Naphthalenes
D011084	Polycyclic Aromatic Hydrocarbons
D006841	Hydrocarbons, Aromatic
D006844	Hydrocarbons, Cyclic

Browse Leaves

Not provided

Browse Branches

Not provided