Accepts Healthy Volunteers
Healthy volunteers are participants who do not have a disease or condition, or related conditions or symptoms
An interventional clinical study is where participants are assigned to receive one or more interventions (or no intervention) so that researchers can evaluate the effects of the interventions on biomedical or health-related outcomes.
An observational clinical study is where participants identified as belonging to study groups are assessed for biomedical or health outcomes.
Searching Both is inclusive of interventional and observational studies.
|Eligible Ages||18 Years and Over|
- - INCLUSION CRITERIA 1.1 Females and males, aged greater than or equal to 18.
This trial id was obtained from ClinicalTrials.gov, a service of the U.S. National Institutes of Health, providing information on publicly and privately supported clinical studies of human participants with locations in all 50 States and in 196 countries.
Phase 1: Studies that emphasize safety and how the drug is metabolized and excreted in humans.
Phase 2: Studies that gather preliminary data on effectiveness (whether the drug works in people who have a certain disease or condition) and additional safety data.
Phase 3: Studies that gather more information about safety and effectiveness by studying different populations and different dosages and by using the drug in combination with other drugs.
Phase 4: Studies occurring after FDA has approved a drug for marketing, efficacy, or optimal use.
The sponsor is the organization or person who oversees the clinical study and is responsible for analyzing the study data.
|National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)|
The person who is responsible for the scientific and technical direction of the entire clinical study.
|Edward W Cowen, M.D.|
|Principal Investigator Affiliation||National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)|
Category of organization(s) involved as sponsor (and collaborator) supporting the trial.
The disease, disorder, syndrome, illness, or injury that is being studied.
|Sneddon-Wilkinson, Acrodermatitis Continua of Hallopeau, Pustular Psoriasis, Palmoplantar Pustulosis|
|Study Website:||View Trial Website|
- - Inflammatory disorders that present with neutrophilic pustular skin lesions, including generalized pustular psoriasis, are characterized by severe cutaneous manifestations, generalized inflammation and significant morbidity.
- - Recent studies in patients with phenotypically similar pustular diseases have identified two monogenic forms of neutrophilic pustular psoriasis implicating interleukin (IL)-1 in disease pathogenesis.
- - Deficiency of the IL-1 receptor antagonist (IL1RN, DIRA) is an autosomal recessive condition characterized by severe generalized pustular eruptions in the neonatal period, osteopenia, lytic bone lesions, joint pain, respiratory insufficiency, thrombosis, elevated acute phase reactants and significant mortality.
- - Deficiency of IL-36 receptor antagonist (IL-36RN/IL1F5, DITRA) is an autosomal recessive condition with episodic widespread pustular skin lesions, fevers and systemic inflammation defined by marked leukocytosis and elevated creactive protein.
- - Both IL1RN and IL36RN/IL1F5 are highly expressed in epidermal keratinocytes, suggesting a role for keratinocytes in initiating innate immunity-mediated inflammatory skin diseases, and ultimately manifesting in a pustular phenotype.
- - Patients with inflammatory pustular diseases often respond poorly to conventional treatment with methotrexate, cyclosporine and anti-TNF agents.
- - Two recent case reports describe patients with pustular psoriasis unresponsive to TNF inhibition who responded to anti-IL-1 receptor therapy with anakinra.
- - We propose a phase 2 study that will utilize a collaborative bench-to-bedside approach, applying targeted anti-IL-1 therapy, novel imaging modalities, and laboratory techniques including immunohistochemistry, gene expression and cytokine studies, and in vitro manipulations of skin to dissect and validate pathways in these complex diseases.
- - Age greater than or equal to 18 years.
- - Active macroscopic noninfectious pustular skin lesions involving greater than or equal to 5% of the total body surface area, or palmoplantar involvement.
- - Histopathologic confirmation of epidermal neutrophilic pustulosis.
- - Patients must have maintained a stable dose of immunosuppressant therapy, retinoids or anti-neutrophil therapy for 2 weeks prior to study initiation with resultant stable or worsening skin disease.
- - Use of biologic agents requires a washout period of at least 3 half-lives prior to study initiation.
- - Patients must have organ and marrow function as defined below: - leukocytes >3,000/mcL - absolute neutrophil count >1,500/mcL - platelets >100,000/mcL - creatinine within normal institutional limits OR creatinine clearance >60 mL/min/1.73 m2 for patients with creatinine levels above institutional normal.
- - A 16-week, open-label phase 2 study.
- - Patients will initially receive treatment with anakinra 100 mg/day by self-administered subcutaneous injection.
- - Disease response will be assessed every 4 weeks, and determination of dose escalation will be made based on clinical assessment.
- - If a response is achieved with anakinra, other immunosuppressants administered for the purpose of treatment of pustular skin disease may be tapered per physician discretion.
- - Clinical assessment, and laboratory and subjective data will be collected in-person every 4 weeks to determine disease response.
- - Twenty-five evaluable patients will be enrolled onto this trial.
An initial dose of anakinra 100 mg/day will beadministered daily via self-administered subcutaneousinjection. If pustule formation persists at this dose,anakinra dose may be escalated up to 200 mg/dayinjected subcutaneously daily at week 4
Drug: - Anakinra
An initial dose of anakinra 100 mg/day will be administered daily via self-administered subcutaneous injection. If pustule formation persists at this dose, anakinra dose may be escalated up to 200 mg/day injected subcutaneously daily at week 4
Contact a Trial Team
If you are interested in learning more about this trial, find the trial site nearest to your location and contact the site coordinator via email or phone. We also strongly recommend that you consult with your healthcare provider about the trials that may interest you and refer to our terms of service below.