12 Clinical Trials for Various Conditions
A two-component therapeutic consisting of FCX-013 and veledimex for the treatment of localized scleroderma (or morphea). The first component, FCX-013, is autologous human fibroblasts genetically-modified using lentivirus and encoded for matrix metalloproteinase 1 (MMP-1), a protein responsible for breaking down collagen. FCX-013 is designed to be injected under the skin at the location of the fibrotic lesions where the genetically-modified fibroblast cells will produce MMP-1 to break down excess collagen accumulation. With the FCX-013 therapy, the patient will take an oral compound (Veledimex) to induce MMP-1 protein expression from the injected cells. Once the fibrosis is resolved, the patient will stop taking the oral compound which will stop further MMP-1 production from the injected cells. FCX-013 plus veledimex is being developed in anticipation of improving skin function in patients by resolving fibrotic lesions and normalizing dermal collagen production
This is a pilot study to determine the safety and clinical efficacy of crisaborole 2% ointment in the treatment of morphea.
This study will look into how topical treatment with synthetic Vitamin D3, calcipotriene ointment, used as standard of care, works in patients with morphea. Skin biopsies of morphea lesions before and after treatment with topical calcipotriene 0.005% ointment will be analyzed for changes in RNA and protein. A skin biopsy of unaffected skin will also be obtained and used for a control. This is an initial study to look at the molecular effects of topical calcipotriene on human morphea-involved skin. This study will look at the differences between affected and unaffected skin. This study also will look at clinical outcomes in morphea patients and determine if there are any clinical predictors for improvement with the medication.
The Morphea in Adults and Children (MAC) cohort is the first registry for both children and adults with morphea (also known as localized scleroderma) in the country. The purpose of the registry is to learn more about morphea, specifically: * How morphea behaves over time * How frequently specific problems occur along with morphea (for example, arthritis) * Whether morphea has an autoimmune background
This is a randomized, blinded, and controlled trial to assess the efficacy and safety of UVA1 phototherapy in the treatment of active morphea in adults and children. Forty patients will be randomized to receive either medium dose (70 J/cm2) phototherapy (active UVA1 phototherapy) with an ultraviolet translucent acrylic screen or "sham" UVA1 (0 J/cm2) phototherapy with an ultraviolet opaque acrylic screen 3 times per week for 10 weeks. The phototherapists, patients, and principal investigator will be blinded to whether the patients receive active or sham UVA1 phototherapy. Patients will only be allowed to apply emollients during the study. Patients completing the randomized placebo controlled trial (RPCT) will be followed during an open observation period for 3 months. During the open phase, all outcome measures from the RPCT (LoSSI, PGA-A) will be assessed every 5 weeks as well as adverse events. Patients who received sham UVA1 phototherapy will be invited to receive active UVA1 phototherapy using the same protocol as in the RPCT during the open observation. Adult patients enrolled in the RPCT will also be part of a nested translational study investigating the effect of UVA1 phototherapy on gene expression from whole skin biopsies taken before (Study Visit 1) and after UVA1 phototherapy (Study Visit 3). Gene expression profiles will be compared in lesional skin before and after treatment as well as nonlesional skin.
Compare UVA-1 phototherapy treatment to topical steroid treatment in subjects with morphea.
An open-label single center trial studying the efficacy and safety of sarilumab on morphea patients.
The purpose of this study is to assess the degree of improvement seen patient reported outcomes after 30 sessions of UVA-1 therapy in treating systemic scleroderma, morphea, and sclerodermatous Graft-Versus-Host Disease. While patients have verbally reported improvement of their sclerosing skin disease with UVA-1, patient reported outcomes have not been rigorously studied. In sclerosing skin diseases where clinical change is difficult to measure, patient reported outcomes may offer a better way to study the impact of treatments like UVA-1. This will be a non-blinded, non-randomized prospective trial using UVA-1 phototherapy in patients with established sclerosing skin disease. Patients will report the severity of their condition using multiple patient reported outcomes and will also be analyzed using multiple clinical investigator assessments at the beginning and end of 30 treatment sessions.
Corticosteroid therapy, including intralesional and topical applications, has many indications within the fields of Dermatology, Plastic Surgery, and Orthopedics. However, these injections can be quite painful, which leads many patients to discontinue treatment. Often, the injection involves a mixture of local anesthetic and corticosteroids despite a lack of evidence that the use of lidocaine improves pain. Due to the acidic pH, the lidocaine component of the injection can actually cause a significant burning sensation during the procedure. Lidocaine does not have anti-inflammatory properties and does not treat the underlying pathology. By including another medication, lidocaine also adds cost and risk to the procedure. The purpose of this study is to see if removing lidocaine from intralesional injections decreases the pain of injection.
In this study, Laser Doppler Flowmetry (LDF), Laser Doppler Imaging (LDI), Orthogonal Polarization Spectral Imaging (OPSI), Nail fold video capillaroscopy (NVC) and Optical Coherence Tomography (OCT) will be used to assess differences in microvascular function and density of oral mucosa and skin in subjects with 1) autoimmune diseases with cutaneous involvement: systemic sclerosis (SSc), morphea, dermatomyositis, cutaneous lupus and vasculitis, 2) sickle cell disease (SCD) and 3) chronic graft-versus-host disease (GVHD) compared to healthy subjects. The microvascular changes will be compared to overall treatment response in patients with scleroderma and chronic GVHD as assessments will be made before and after the patients start treatment for their diseases and determine if these imaging techniques provide valuable and reproducible data when assessing a patient's response to treatment for those diseases. In addition, the application of Acoustic Radiation Force Impulse (ARFI) in determining cutaneous thickness in patients with SSc, GVHD and morphea will be evaluated. The investigators hypothesize that the vascular and dermal structures are altered in patients with autoimmune disease, SCD and chronic GVHD. In addition, they hypothesize that imaging modalities such as LDF, LDI, OCT, NVC, OPSI and ARFI can quantify such structural alterations and can be used to 1) detect early disease activity, 2) quantify and assess response to therapy and 3) quantify and correlate with overall disease activity.
The purpose of this investigation is to evaluate the effectiveness of an investigational device which is similar in appearance to a "tanning bed" but which emits ultraviolet irradiation of a specific wavelength known as UVA1. This device has not been approved by the FDA for general use in this country, as yet, but it has been used quite successfully in Europe for several years in treating such conditions as scleroderma, keloids, and other fibrosing conditions of the skin. Your participation in this study may yield important information regarding the safety and effectiveness of this form of light therapy for the treatment of these skin conditions which, at present, are difficult to treat.
The purpose of this investigation is to evaluate the effectiveness of high-dose UVA1 irradiation in the treatment of fibrosing conditions of the skin, e.g., keloid (a thick scar from growth of fibrous tissue), scleroderma (deposits of fibrous tissue in the skin) and acne keloidalis nuchae (keloids on the back of the neck or hairline) old burn scars, granuloma annulare or other similar skin conditions. This UVA1 dosing schedule has been used successfully in Germany for various skin diseases, such as the above mentioned scleroderma.