A Single-Site Tissue Repository Providing Annotated Biospecimens for Approved Investigator-directed...
Age-Related Macular DegenerationAllergies45 moreTo collect, preserve, and/or distribute annotated biospecimens and associated medical data to institutionally approved, investigator-directed biomedical research to discover and develop new treatments, diagnostics, and preventative methods for specific and complex conditions.
FARD (RaDiCo Cohort) (RaDiCo-FARD)
Inherited Epidermolysis BullosaIchthyosis7 moreThe goal of this observational study is to conduct a prospective assessment of the individual Burden of 9 rare skin diseases to assess disability in the broadest sense of the term (psychological, social, economic and physical) for patients and/or families. Two types of indicators will be used to reach this objective : an individual burden score calculated based on a burden questionnaire created specifically, approved and designed to understand the tendency to changes in care and lifestyles. The burden questionnaire should be used by patients and/or their family themselves in self-assessment. a descriptive analysis of all resources (medical and non-medical) used by the family unit to manage the disease.
Autoimmune Blistering Diseases Study
Pemphigus VulgarisPemphigus Foliaceus1 morePemphigus and bullous pemphigoid (BP) are severe autoimmune blistering diseases (AIBD) that pose a critical need for new therapeutic approaches. Clinical trials in pemphigus and BP will require the availability of validated disease severity measures that can be used to define primary outcomes.
A Safety and Dose-Finding Study of SYNT001 in Subjects With Pemphigus (Vulgaris or Foliaceus)
PemphigusPemphigus Vulgaris1 moreThis was a multicenter, open-label safety study to determine the dose regimen of SYNT001 (ALXN1830) administered intravenously in participants with pemphigus (vulgaris or foliaceus).
Evaluating Sirolimus to Treat Autoimmune Blistering Dermatosis Pemphigus
PemphigusThe purpose of this research is to study alternative treatments for the skin disease pemphigus (a rare autoimmune blistering disorder of the skin) by using sirolimus, an immunosuppressive drug. Immunosuppressive drugs inhibit or prevent the activity of the immune system and are commonly used to treat autoimmune diseases, inflammatory diseases, and organ transplantation rejection.
Efficacy and Safety of Ofatumumab in Treatment of Pemphigus Vulgaris
Pemphigus VulgarisPemphigus vulgaris (PV) is a rare, chronic, debilitating, and potentially life-threatening autoimmune disorder that is characterized by mucocutaneous blisters. Ofatumumab is a novel monoclonal antibody (mAb) that specifically binds to the human CD20 antigen, which is expressed only in B lymphocytes. The purpose of this study was to evaluate the efficacy, tolerability, and safety of ofatumumab injection for subcutaneous use (ofatumumab SC) 20 milligrams (mg) administered once in every 4 weeks, (with an additional 20 mg loading dose [i.e. 40 mg total] at both Week 0 and Week 4) in subjects with PV. It was anticipated that with sustained B-cell depletion in the presence of ofatumumab SC, and the resultant reduction of pathogenic anti Dsg (desmoglein) autoantibodies in PV, that clinical remission of the disease would result.
Randomized Trial of IVIg With or Without Cyclophosphamide in Pemphigus
Pemphigus VulgarisThe purpose of this study is to compare two standard treatments for pemphigus to determine which more effectively improves the clinical manifestations of the disease and decreases serum level of the autoantibodies which cause the disease.
Efficacy of Dapsone as a Steroid Sparing Agent in Pemphigus Vulgaris
Pemphigus VulgarisThe purpose of this 12-month study was to determine the efficacy of dapsone as a glucocorticoid-sparing agent in maintenance phase pemphigus vulgaris.
Study of Efficacy and Safety of VAY736 in Patients With Pemphigus Vulgaris
Pemphigus VulgarisThe study evaluated the efficacy, safety and pharmacokinetics of VAY736 in the treatment of patients with pemphigus vulagaris (PV).
A Study of PRN1008 in Patients With Pemphigus
PemphigusThis was a Phase 3 randomized, parallel-group, double-blind, placebo-controlled trial (blinded treatment [BT] period) followed by an open-label extension [OLE] period intended to evaluate the efficacy and safety of oral PRN1008 in moderate to severe pemphigus. After completing the open-label extension period, eligible participants might continue in a long term extension (LTE) Period of 48 weeks.