Better Evidence and Translation for Calciphylaxis
CalciphylaxisThis global platform study will evaluate multiple interventions, across several domains of therapeutic care, in adult patients with kidney failure and newly diagnosed calciphylaxis.
Rheopheresis as Adjuvant Treatment of Calciphylaxis
Metabolic DisorderEnd Stage Renal Disease1 moreWe propose to set up a prospective randomized controlled trial to control the security and assess the efficacy of adjuvant treatment by rheopheresis in necrotizing-ulcered calciphylaxis in the hemodialysis population.
Phase 3 Study of SNF472 for Calciphylaxis
CalciphylaxisCalcific Uremic ArteriolopathyThe primary objectives are to assess the efficacy, safety, and tolerability of SNF472 compared to placebo when added to background care for the treatment of calciphylaxis (CUA).
Stem Cells for Uremic Calciphylaxis Patients
Chronic Kidney DiseasesCalciphylaxis2 moreTreatment for Uremic Calciphylaxis Patients with Human Amniotic Mesenchymal Stem Cells
Partners Calciphylaxis Biobank
CalciphylaxisCalcific Uremic Arteriolopathy2 moreCalciphylaxis, a vascular calcification disorder, is a rare and serious disorder characterized by calcification of dermal arterioles. There are significant gaps in the understanding of the pathophysiology and risk factors for calciphylaxis. At present, there is no effective treatment. Uncertain pathobiology, rare incidence and lack of collaborative approach have been some of the major limiting factors towards treating calciphylaxis. The Partners Calciphylaxis Biorepository (PCB) aims to address these gaps within calciphylaxis research by utilizing existing and, when necessary, developing new infrastructure to support the consent of patients and the collection of dedicated samples for a calciphylaxis repository.
National Registry of Rare Kidney Diseases
Adenine Phosphoribosyltransferase DeficiencyAH Amyloidosis85 moreThe goal of this National Registry is to is to collect information from patients with rare kidney diseases, so that it that can be used for research. The purpose of this research is to: Develop Clinical Guidelines for specific rare kidney diseases. These are written recommendations on how to diagnose and treat a medical condition. Audit treatments and outcomes. An audit makes checks to see if what should be done is being done and asks if it could be done better. Further the development of future treatments. Participants will be invited to participate on clinical trials and other studies. The registry has the capacity to feedback relevant information to patients and in conjunction with Patient Knows Best (Home - Patients Know Best), allows patients to provide information themselves, including their own reported quality of life and outcome measures.
Retrospective Study Evaluating the Contribution of Rheopheresis in the Treatment of Uremic Calciphylaxis...
RheopheresisCalcifying Uremic Arteriolopathy1 moreCalcifying uremic arteriolopathy (or uremic calciphylaxis) is a rare disease (prevalence <1% of dialysis patients), but the prognosis is often catastrophic. The main non-modifiable risk factors are age, female gender, diabetes, obesity and length of time on dialysis. Today, there is no specific treatment for this pathology, and the therapeutic management is poorly codified. However, it is commonly accepted that the treatment is based on the control of risk factors, local care, and the possible addition of treatment with sodium thiosulfate. Hyperbaric oxygen therapy has also been proposed by some authors, but remains not very accessible in practice. Recently, it has been proposed to use Rheopheresis as an adjuvant treatment for severe forms of uraemic calciphylaxis. It is a technique of apheresis in double filtration, allowing the extraction of molecules of high molecular weight, and thus the improvement of the rheological conditions of microcirculation. The expected effect is the improvement of tissue oxygenation and the acceleration of the healing of skin lesions, with the consequent reduction of infectious complications. The aim of this study is to propose a large national retrospective study, studying the evolution of patients with uremic calciphylaxis and treated by rheopheresis, compared to a control group. This will allow to have a more precise idea of the contribution of Rheopheresis in this indication.
A Phase 3 Clinical Trial of Intravenous Sodium Thiosulfate in Acute Calciphylaxis Patients
CalciphylaxisThis multicenter, randomized, double-blind, placebo-controlled clinical trial will evaluate the efficacy and safety of intravenous Sodium Thiosulfate Injection for treatment of acute calciphylaxis-associated pain in chronic hemodialysis patients.
Randomized Double Blind Placebo Controlled Trial of Sodium Thiosulfate for the Treatment of Pain...
CalciphylaxisCalcific Uremic ArteriolopathyTo evaluate the efficacy of Sodium Thiosulfate (STS) compared to placebo, in reducing analgesic requirement in subjects with calcific uremic arteriolopathy (CUA) during an initial 28-day treatment phase.
Efficacy of Lanthanum Carbonate in Calciphylaxis
CalciphylaxisThe research question and primary aim is to determine if lanthanum carbonate is effective in treating calciphylaxis by measurement of complete or partial remission of skin lesions. Secondary endpoints will be measured to determine if lanthanum carbonate can lower calcium-phosphorus product levels, intact PTH and mortality. Albumin levels will be measured as a marker of nutritional status and inflammation.