Clinical trial NCT01447381: Systemic Therapies for Pediatric Atopic Dermatitis
Official title: Systemic Therapies for Pediatric Atopic Dermatitis
Study type: OBSERVATIONAL
Overall status: UNKNOWN
Conditions: Dermatitis, Atopic
Lead sponsor: Rady Children's Hospital, San Diego
Sponsor class: OTHER
Collaborators: name: University of California, San Diego
First posted: 2011-10-06
Enrollment: 250
Sex: ALL
Minimum age (years): 2
Maximum age (years): 17
Standard ages: CHILD
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: PROBABILITY_SAMPLE
Study population: Ages 2 to 17 (inclusive) with moderate to severe atopic dermatitis and who require treatment with cyclosporine, azathioprine, mycophenolate mofetil, and/or methotrexate. Both male and female. Non-pregnant females and minorities will be included without restriction.

Brief summary: While many patients with atopic dermatitis (eczema) can be managed with topical creams and treatments for itch, some children have such severe, long-standing disease that they need treatment with oral medications that decrease the ability of the immune system to react. However, there is not enough information on the proper use of these medications or how well they work compared with each other. The current study looks at the response of children treated with these medications to provide this information and improve their use.

Detailed description: Severe and/or refractory cases of atopic dermatitis (AD) can require systemic immunosuppressive therapy for disease control, yet there are few studies regarding the appropriate use of these drugs for pediatric AD and even less data comparing them. The current observational study will observe the effect of these therapies on children with moderate to severe atopic dermatitis as they are treated. These drugs being observed will be cyclosporine, azathioprine, mycophenolate mofetil, and methotrexate.

Eligibility criteria: Inclusion Criteria:

* Diagnosis of moderate, severe, or very severe atopic dermatitis meeting the Hanifin and Rajka criteria
* Needs systemic therapy (e.g. cyclosporine, azathioprine, mycophenolate mofetil, or methotrexate) for severe atopic dermatitis
* Subject has severe or recalcitrant disease interrupting daily life as evidenced by fulfilling 2 or more of the following 4 criteria:

  * Failure of multimodal therapy including emollients/barrier repair, topical anti-inflammatory agents (medium to high potency topical corticosteroids, and/or low-potency or topical calcineurin inhibitors if facial/intertriginous areas), and antihistamines
  * Significant impairment in quality of life (physical, psychological, emotional) such as significant sleep disruption, poor school performance, or frequent school absenteeism, per the judgement of the investigator.
  * Inability to receive, prior failure, or the need for more than one course of phototherapy
  * Prior failure or need for more than one course of another oral immunosuppressive medication
* No serious medical condition that precludes the use of oral immunosuppressives based on the subject's medical history, physical examination, and safety laboratory tests.
* Negative PPD within the last year prior to study initiation.
* Female of childbearing potential has a negative pregnancy test at the time of starting the systemic drug and who consents to be abstinent or using an effective method of contraception during the study. Effective contraception is defined as IUD, condom with spermicide, diaphragm with spermicide, or stable use of a hormonal contraceptive (oral, implant, injection or transdermal patch) beginning at least 1 month prior to starting the systemic drug.
* Subject and parent/guardian are willing and able to comply with study instructions and return to the clinic for all required visits.

Exclusion Criteria:

* Female who is pregnant, nursing, or planning a pregnancy during the study period.
* Concurrent participation in another clinical trial with an investigational drug or device that may impact on the individual's atopic dermatitis.
* Subjects with clinically significant hepatic disease, history of lymphoma or myelosuppression, low TMPT activity (if starting azathioprine), renal disease (if starting cyclosporine or azathioprine), hypertension (if starting cyclosporine), blood dyscrasias, or central nervous system disorders, such as uncontrolled seizures or peripheral neuropathy, or taking systemic medications that could interact adversely with the study medicine (e.g. erythromycin use with cyclosporine).
* Subjects with any underlying disease that the Investigator deems uncontrolled, and poses a concern for subject safety while participating on the study.
* Subject has a history of clinically significant drug or alcohol abuse in the last year.
* Subject is considered by the Investigator, for any reason, to be an unsuitable candidate for the study.
* Active systemic infection that could worsen with systemic therapy for AD.

Primary outcomes:
measure: Reduction in Investigator Global Severity Scale (IGSS)
timeFrame: 10 years
measure: Reduction in SCORing Atopic Dermatitis (SCORAD) Index
timeFrame: 10 years
measure: Reduction in Eczema Area and Severity Index (EASI)
timeFrame: 10 years
Secondary outcomes:
measure: Time to Response
timeFrame: 10 years
measure: Rate of Taper
timeFrame: 10 years
measure: Length of time before recurrent flare
timeFrame: 10 years
Other outcomes:
Locations:
city: San Diego
country: United States
facility: Rady Children's Hospital
state: California
city: San Francisco
country: United States
facility: University of California, San Francisco
state: California
city: Chicago
country: United States
facility: Children's Memorial Hospital
state: Illinois
Study officials:
affiliation: Rady Children's Hospital and UC San Diego
name: Wynnis Tom, MD
role: PRINCIPAL_INVESTIGATOR
References:
citation: Meagher LJ, Wines NY, Cooper AJ. Atopic dermatitis: review of immunopathogenesis and advances in immunosuppressive therapy. Australas J Dermatol. 2002 Nov;43(4):247-54. doi: 10.1046/j.1440-0960.2002.00610.x.
citation: Akhavan A, Rudikoff D. The treatment of atopic dermatitis with systemic immunosuppressive agents. Clin Dermatol. 2003 May-Jun;21(3):225-40. doi: 10.1016/s0738-081x(02)00362-0. No abstract available.