Efficacy and Safety of Two Doses of Liarozole vs. Placebo for the Treatment of Lamellar Ichthyosis...
IchthyosisLamellarLamellar ichthyosis is a congenital disease of the skin with a generalized scaling. The primary activity of liarozole is considered to be the inhibition of the degradation of a substance called retinoic acid, which is the principal endogenous regulator of growth and differentiation of epithelial tissues in mammals. The current study intends to evaluate the efficacy and safety in patients with lamellar ichthyosis.
A Vehicle Controlled Study to Evaluate Safety and Efficacy of Topical TMB-001 for Treatment of Congenital...
Congenital IchthyosisThe purpose of this study is to investigate the efficacy and safety of two concentrations of topically applied ointment formulation of isotretinoin called TMB-001 (0.05% and 0.1% isotretinoin) in subjects 9 years of age and older for the treatment of congenital ichthyosis (CI), including recessive X-linked ichthyosis (RXLI) and autosomal recessive congenital ichthyosis-lamellar ichthyosis (ARCI-LI) subtypes. Funding Source FDA-OOPD
Fish Skin Compared to Cadaver Skin as Temporary Cover for Full Thickness Burns
BurnsObjectives and rationale: Optimal burn management involves removing all the dead or burned tissue as early as feasible and cover with an autograft called split thickness skin graft (STSG) taken from the patient. This procedure creates a new wound on the patient and sometimes, when the burn covers very large portion of the patient body, there is a lack of healthy skin to use for this purpose. Under those circumstances, donated cadaver skin is used as a temporary coverage until the patient´s own donor site wound has healed enough to be used again. The proposed clinical study aims to determine if treatment with fish skin is an alternative to cadaver skin as a temporary coverage for debrided full-thickness burns prior to STSG in terms of autograft take, time to heal, quality of healing (scarring), pain and adverse effects.
A Six Week Topical Cream Study for Subjects With Ichthyosis Vulgaris
Ichthyosis VulgarisThe objective of this study is to determine and compare the safety and efficacy of topical 146-9251 cream and vehicle cream applied twice daily for 6 weeks in subjects with moderate to severe ichthyosis vulgaris (IV).
A Study of Topical NS2 Cream to Treat Ichthyosis in Sjögren-Larsson Syndrome (SLS)
Sjögren-Larsson SyndromeThis is a multi-center, randomized, double-blind, vehicle-controlled, parallel-group study designed to evaluate the safety, pharmacokinetic (PK), and exploratory activity of topically-applied NS2 dermatologic cream administered once-daily (QD) to subjects with ichthyosis secondary to Sjögren- Larsson Syndrome (SLS). NS2 is expected to trap fatty aldehydes that are pathogenic in SLS patients, and thereby diminish the lipid-aldehyde adduct formation that likely results in ichthyosis associated with SLS, and potentially reduce the mild dermal inflammation characteristic of SLS.
Phase III Study of Monolaurin Cream Therapy for Patients With Congenital Ichthyosis
IchthyosisOBJECTIVES: I. Compare the safety and efficacy of monolaurin cream versus a placebo vehicle cream in suppressing the signs of nonbullous congenital ichthyosiform erythroderma. II. Assess the incidence of posttreatment rebound or recrudescence in patients with congenital ichthyosis. III. Evaluate the long term safety of monolaurin cream with whole body application in these patients.
Study Determining Safety and Efficacy of Avena Sativa (Oat) Skincare Products for Treating Skin...
IchthyosisPruritusThis study will test the safety and efficacy of three topical agents containing oat kernel flour to determine how well they relieve skin dryness and itching related to cancer therapies. Participants will receive a body wash, a body cream, and an anti-itch balm to use at home for 4-6 weeks.
A Safety and Tolerability Study of Topical PAT-001 in Congenital Ichthyosis
Congenital IchthyosisCongenital ichthyosis (CI) is a large, heterogeneous family of inherited skin disorders of cornification resulting from an abnormality of skin keratinization, such as scaling and thickening of the skin. Treatment options include keratolytic agents, which can abruptly lead to extensive shedding or peeling of scales. PAT-001 primarily acts as a keratolytic agent; thus, making it a potential drug candidate for the treatment of skin disorders associated with hyperkeratinization, such as CI. The current study intends to evaluate the safety and tolerability of PAT-001 in patients with CI of either the Lamellar or X-Linked subtypes.
Cancer Risk in Carriers of the Gene for Xeroderma Pigmentosum
Xeroderma PigmentosumBasal Cell Carcinoma3 moreThis study will determine if family members of patients with xeroderma pigmentosum (XP) have various abnormalities, including: skin abnormalities; nervous system abnormalities, such as hearing problems; skin, eye, or internal cancers, or other changes. XP is a rare inherited disease that involves an inability to repair damage to cell DNA (genetic material). It can affect several organ systems, including the skin, eye, nervous system, and bones. Patients have a more than thousand-fold increase in frequency in all major skin cancers. Parents of patients with XP are carriers of the abnormal XP gene. Other family members may also be carriers of the abnormal XP gene. Carriers do not develop the disease themselves; symptoms develop only in children who have inherited the faulty gene from both parents. This study will try to clarify the genetic basis for XP and to understand the increased frequency of cancer in the disease. XP patients who have been evaluated at the NIH Clinical Center and their relatives are eligible for this study. Newly diagnosed XP patients are also eligible. Spouses of relatives will also be included as control subjects. Patients and their family members will undergo some or all of the following procedures: Parental permission to review the child s relevant medical records and pathology material from treatments or surgery for cancer or other related illnesses Medical history and physical examination, with particular attention to the skin and possible eye, hearing or neurological examinations Photographs to document skin and other physical findings Nuclear medicine scans to evaluate the brain and nervous system X-rays of the skull or other parts of the body Nervous system testing with an electroencephalogram (EEG), electroretinogram (ERG), electromyogram (EMG) or nerve conduction velocity measurement Collection of blood and skin samples for gene studies Establishment of cell lines from collected blood or tissues to study DNA repair, skin cancer, cancers related to XP, immune defects, and related studies. Biopsy (surgical removal of a small piece of tissue) of suspicious skin lesions for examination under a microscope Collection of a cheek cell sample, obtained by twirling a soft brush against the inside of the cheek Collection of a hair sample for microscopic examination and composition analysis Surgery to treat skin cancers or other lesions
Novel Treatment for Syndromic Ichthyoses
Syndromic IchthyosesCHILD Syndrome2 moreThis 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.