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.
Autologous Stem Cell Transplantation for Patients With AL Amyloidosis
AL AmyloidosisPlasma Cell DyscrasiaThis study mainly evaluated the efficacy and safety of autologous stem cell transplantation for the treatment of AL amyloidosis, the role of induction and maintenance therapy in autologous stem cell transplantation, and the long-term efficacy and prognosis risk factors of autologous stem cell transplantation for the treatment of AL amyloidosis.
A Registry of Chinese AL Amyloidosis Patients Treated With Subcutaneous or Intravenous Daratumumab...
AL AmyloidosisThe purpose of this protocol is to generate a registry of Chinese patients with AL amyloidosis treated with subcutaneous/intravenous daratumumab alone or subcutaneous/intravenous daratumumab combined with chemotherapy.
A Registry of AL Amyloidosis (ReAL)
AL AmyloidosisThe purpose of this protocol is to generate a large registry of patients with AL amyloidosis.
Screening to Improve Survival in AL Amyloidosis
Smoldering Multiple MyelomaMonoclonal Gammopathy of Undetermined SignificanceThe purpose of this study is to see whether certain genes may be linked with the development of AL amyloidosis in subjects 60 years of age or older with the blood disorders SMM and MGUS. A limited repertoire of immunoglobulin (Ig) variable region genes have been associated with AL amyloidosis. The clonal plasma cells of subjects with SMM and MGUS may express one of these Ig variable region genes indicating a risk of progression to AL amyloidosis and potentially enabling early diagnosis. We hope this study will help us begin to understand whether Ig variable region gene identification can be a useful tool for assessing a subject's risk of progression to AL amyloidosis.
Probiotic Supplementation Reduces Gastrointestinal Symptoms During the Therapy and Improves Therapeutic...
Immunoglobulin Light-chain AmyloidosisThe purpose of this clinical trial is to evaluate whether specific probiotic can reduce gastrointestinal symptoms and improves therapeutic response, on a background of Bortezomib+dexamethasone or Bortezomib+dexamethasone combined with daratumumab therapy, for naive AL amyloidosis patients.
Study in Subjects With Light Chain (AL) Amyloidosis
AL AmyloidosisThe objective of this study is to evaluate the long-term safety and efficacy of NEOD001 in subjects with AL amyloidosis who have completed Study NEOD001-201.
Monitoring of Early Disease Progression in Hereditary Transthyretin Amyloidosis
AmyloidosisAmyloid6 moreThis study measures circulating, misfolded ATTR oligomers in asymptomatic ATTRm amyloidosis genetic carriers longitudinally over five years.
Pomalidomide With Melphalan and Dexamethasone for Untreated Systemic AL Amyloidosis
MyelomaThe goal of this clinical research study is to find the highest tolerable dose of pomalidomide that can be given in combination with melphalan and dexamethasone that can be given to patients with AL amyloidosis. The safety of this drug combination will also be studied. Pomalidomide is designed to change the body's immune system. It may also interfere with the development of tiny blood vessels that help support tumor growth. This may decrease the growth of cancer cells. Melphalan is designed to damage the DNA (genetic material) of cells, which may cause cancer cells to die. Dexamethasone is a corticosteroid that is similar to a natural hormone made by your body. Dexamethasone is often given to Multiple Myeloma (MM) patients in combination with other chemotherapy to treat cancer. Planned Phase I/II Study terminated early during Phase I portion without continuation to Phase II.
Amifostine and Melphalan in Treating Patients With Primary Systemic Amyloidosis Who Are Undergoing...
Drug/Agent Toxicity by Tissue/OrganMultiple Myeloma and Plasma Cell NeoplasmRATIONALE: Drugs used in chemotherapy work in different ways to stop the growth of plasma cells, either by killing the cells or by stopping them from dividing. Having a peripheral stem cell transplant to replace the blood-forming cells destroyed by chemotherapy, allows higher dose of chemotherapy to be given so that more plasma cells are killed. Giving a chemoprotective drug such as amifostine may protect kidney cells from the side effects of chemotherapy. PURPOSE: This phase I trial is studying the side effects and best dose of melphalan given together with amifostine in treating patients who are undergoing peripheral stem cell transplant for primary systemic amyloidosis.