Safety Study of Elidel (Pimecrolimus) 1% Cream to Treat Netherton Syndrome

Status Completed

Clinical Trial Summary

Netherton syndrome is a genetic condition that can result in abnormal skin functioning. People with this condition often have red and scaling skin; sparse or short hair; and problems with absorption of medicines or chemicals that are applied to the skin. If these chemicals are absorbed at a high level, they may cause health problems. Elidel (pimecrolimus) is a new medicine that is available as a cream. It has been shown to help improve the appearance of the skin in patients with another skin condition known as atopic dermatitis, and is approved by the United States (US) Food and Drug Administration for use in children with mild to moderate atopic dermatitis. The purpose of this study is to determine if Elidel is safe, to see whether the medication is absorbed through the skin, and to see if side effects are associated with its use in children with Netherton syndrome.

Clinical Trial Description

Patients with Netherton syndrome, a rare genodermatosis, manifest a chronic, eczematous dermatitis with erythema and scaling that is often recalcitrant to conventional therapy with emollients and topical corticosteroids. These patients display an altered epidermal barrier with increased permeability to topical agents and are therefore susceptible to evaporative transepidermal water loss and infection. Topical therapy with the calcineurin inhibitors tacrolimus and pimecrolimus has been demonstrated to improve the skin integrity and the quality of life of patients with several chronic dermatoses, including atopic dermatitis. As a result of the underlying skin barrier dysfunction, however, the possibility of significant systemic absorption and resultant side effects is a concern when these agents are used in patients with Netherton syndrome. Experience with topical tacrolimus 0.1% ointment for patients with Netherton syndrome has demonstrated both marked efficacy as well as significant systemic absorption of the drug in this patient population. Use of topical pimecrolimus in patients with Netherton syndrome has not been reported to date. Investigation of the extent of systemic absorption and side effects will help to define the safety and efficacy profile of topical pimecrolimus in patients with Netherton syndrome. ;

Study Design

Related Conditions & MeSH terms

Clinical Trial Details

NCT number	NCT00208026
Study type	Interventional
Source	Children's Hospital of Philadelphia
Contact
Status	Completed
Phase	Phase 1/Phase 2
Start date	September 2005
Completion date	March 2008

View Details

See also
Status	Clinical Trial	Phase
Recruiting	`NCT05979831` - A Study to Explore Safety, Pharmacokinetics, and Early Clinical Signal of Efficacy of DS-2325a in Patients With Netherton Syndrome	Phase 1/Phase 2
Completed	`NCT05583669` - A Study to Assess the Safety and Pharmacokinetics of Multiple Ascending Subcutaneous Doses of DS-2325a in Healthy Subjects	Phase 1
Completed	`NCT01428297` - A Study Evaluating the Safety and Efficacy of Topical BPR277 for the Treatment of Atopic Dermatitis and Netherton Syndrome	Phase 1
Recruiting	`NCT05856526` - A Study to Test Whether Spesolimab Helps People With a Skin Disease Called Netherton Syndrome	Phase 2/Phase 3
Recruiting	`NCT05789056` - Open Label, Safety and Efficacy Study of QRX003 Lotion in Subjects With Netherton Syndrome	Phase 2/Phase 3
Recruiting	`NCT05521438` - Safety, Tolerability and Efficacy of QRX003 Lotion in Subjects With Netherton Syndrome	Phase 2/Phase 3
Completed	`NCT05388903` - A Study to Assess the Safety and Pharmacokinetics of Single Ascending Subcutaneous and Intravenous Doses of DS-2325a in Healthy Subjects	Phase 1
Recruiting	`NCT06137157` - Evaluation of Topical ATR12-351 in Adults With Netherton Syndrome	Phase 1
Not yet recruiting	`NCT05902663` - Natural History of Netherton Syndrome
Recruiting	`NCT04244006` - A Pilot Study of the Efficacy and Safety of Dupilumab Versus Placebo in Patients With Netherton Syndrome	Phase 2/Phase 3
Recruiting	`NCT03417856` - Defining the Skin and Blood Biomarkers of Ichthyosis
Completed	`NCT02113904` - Clinical Trial Using Humira in Netherton Syndrome	Phase 2
Recruiting	`NCT05211830` - A Study to Evaluate Topically Applied SXR1096 Cream in Patients With Netherton Syndrome	Phase 1/Phase 2
Completed	`NCT03041038` - The Efficacy and Safety of Secukinumab in Patients With Ichthyoses	Phase 2
Recruiting	`NCT01545323` - Gene Therapy for Netherton Syndrome	Phase 1
Recruiting	`NCT02081313` - Natural History and Biological Study of Netherton Syndrome	N/A

Clinical trials are conducted in a series of steps, called phases - each phase is designed to answer a separate research question.

Phase 1: Researchers test a new drug or treatment in a small group of people for the first time to evaluate its safety, determine a safe dosage range, and identify side effects.
Phase 2: The drug or treatment is given to a larger group of people to see if it is effective and to further evaluate its safety.
Phase 3: The drug or treatment is given to large groups of people to confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the drug or treatment to be used safely.
Phase 4: Studies are done after the drug or treatment has been marketed to gather information on the drug's effect in various populations and any side effects associated with long-term use.