25 Clinical Trials for Various Conditions
This is an open-label Phase 2 study evaluating the long term safety and tolerability of GFB-887 in patients with focal segmental glomerulosclerosis (FSGS), and treatment-resistant minimal change disease (TR-MCD)
The objective of this study is as follows: * Perform genetic analysis to define the prevalence of each of the known gene mutations in an unselected cohort of patients with focal segmental glomerulosclerosis (FSGS) * Perform a comprehensive assessment of cardiovascular status to determine the incidence of any cardiac abnormalities in patients with FSGS * Determine if patients with mutations in specific proteins are more likely to have cardiovascular abnormalities * Initiate long-term follow up in all patients to determine whether cardiac prognosis is related to any specific genetic abnormality
This is a phase 2a study evaluating the safety and tolerability of multiple ascending doses of GFB-887 in patients with diabetic nephropathy (DN), focal segmental glomerulosclerosis (FSGS), and treatment-resistant minimal change disease (TR-MCD).
This study will examine the safety and effectiveness of ACTHAR Gel, when used to treat 15 patients diagnosed with "treatment resistant nephrotic syndrome." Nephrotic syndrome is a group of symptoms that includes low levels of protein in the blood, swelling of tissue (edema), especially around the eyes, feet and hands; and high plasma levels of cholesterol. It is caused by a variety of diseases and underlying disorders that damage the kidneys, resulting in excessive excretion of protein in the urine. These diseases damage the glomeruli, which are small blood vessels that filter wastes and excess water from the blood and pass them into the bladder as urine. As a result of protein loss in the urine, the blood is deficient in protein. Normal amounts of blood protein are needed to help regulate fluid throughout the body. Protein in the blood normally draws water from the tissues and into the bloodstream. When blood protein levels are low, the normal movement of water is reversed, and fluid is drawn from the blood and accumulates in the tissues. This excess tissue fluid causes the swelling and puffiness (edema) that is a symptom of nephrotic syndrome. Nephrotic syndrome is described as "treatment resistant" when a patient fails to achieve a sustained partial or complete remission after treatment with at least two first line therapies. The goal of this study is to determine whether injections of ACTHAR Gel (an FDA approved treatment for nephrotic syndrome) over a six month period will lead to a correction of treatment resistant nephrotic syndrome in these patients.
A Clinical Trial Investigating the Safety and Efficacy of ADX-629 in Subjects with Frequently Relapsing and Steroid-Dependent Nephrotic Syndrome
Researchers from the University of Michigan and Northwestern University are studying people's experiences with swelling caused by Nephrotic Syndrome. Interviews with patients (child and adult) and parents of young children will be conducted. The information collected from the interviews will be used to develop a survey to use when testing new medications for Nephrotic Syndrome. Please consider participating in a 1-hour long interview with the Prepare-NS research study to discuss children and adults experiences with swelling.
Adalimumab, a treatment which blocks tumor necrosis factor (TNF), was tested to see if it changed levels of urine biomarker levels, tissue inhibitor of metalloprotease-1 (TIMP1), and monocyte chemoattractant protein-1 (MCP1). Results may help develop individualized treatment options for future patients with TNF-driven focal segmental glomerulosclerosis (FSGS) or minimal change disease (MCD).
An Open Label, Intra-Subject Dose Escalation Study of CCX140 B in Subjects with Primary FSGS and Nephrotic Syndrome
This is a multicenter, open label, randomized study investigating two dose titration regimens of CXA-10 in subjects at least 18 years of age with primary FSGS. The study will be performed at approximately 25 study centers across the United States of America (USA). The recruitment period is anticipated to be up to approximately 16 months. Approximately 30 subjects will be randomized to ensure 26 subjects complete the study. An optional 9 month open label is available
The purpose of this study is evaluate if abatacept is effective and safe in decreasing the level of protein loss in the urine in patients with excessive loss of protein in the urine (nephrotic syndrome) due to either focal segmental glomerulosclerosis (FSGS) or minimal change disease (MCD). Candidates must have a prior kidney biopsy with either diagnosis. Another kidney biopsy will not be required as part of the study. Candidates must have failed or be intolerant of prior therapy for their kidney disease. The failed or intolerant therapy must include corticosteroids and at least one other drug. Candidates can be adults and children over the age of 6. Abatacept will be administered by venous infusion every 4 weeks.
The investigators are trying to learn more about the cause of kidney diseases such as Focal Segmental Glomerulosclerosis (FSGS) and Nephrotic syndrome by studying genetics. The investigators are interested in discovering which genes play a role in causing a predisposition to FSGS/NS. The investigators also want to learn why FSGS/NS can run in families. Participation in our study involves a saliva sample and a urine sample that you can give from home. There is no cost to participate. All information is kept private and confidential. The investigators also like to include healthy volunteers (parents, spouses) if interested/available but of course this is completely optional.
This is a single-arm, multicenter, open-label Phase II, proof-of-mechanism study to evaluate the efficacy, safety, tolerability and pharmacokinetics of losmapimod in approximately 21 subjects with primary (idiopathic) focal segmental glomerulosclerosis (FSGS) and substantive proteinuria as indicated by a Urinary protein/creatinine Up/c ratio \>=2 gram/gram (g/g) or 24 hr urine protein \>=2 g/day. Losmapimod will be orally administered twice daily over a 24-week treatment phase followed by a 12-week follow-up for safety and relapse assessments.
This study will investigate whether RE-021 (Sparsentan), a selective dual-acting receptor antagonist with affinity for endothelin (A type) and angiotensin II receptors (Type 1), is safe and effective in treating patients with focal segmental glomerulosclerosis (FSGS).
Minimal change disease (MCD), focal segmental glomerulosclerosis (FSGS), and Membranous nephropathy (MN), generate an enormous individual and societal financial burden, accounting for approximately 12% of prevalent end stage renal disease (ESRD) cases (2005) at an annual cost in the US of more than $3 billion. However, the clinical classification of these diseases is widely believed to be inadequate by the scientific community. Given the poor understanding of MCD/FSGS and MN biology, it is not surprising that the available therapies are imperfect. The therapies lack a clear biological basis, and as many families have experienced, they are often not beneficial, and in fact may be significantly toxic. Given these observations, it is essential that research be conducted that address these serious obstacles to effectively caring for patients. In response to a request for applications by the National Institutes of Health, Office of Rare Diseases (NIH, ORD) for the creation of Rare Disease Clinical Research Consortia, a number of affiliated universities joined together with The NephCure Foundation the NIDDK, the ORDR, and the University of Michigan in collaboration towards the establishment of a Nephrotic Syndrome (NS) Rare Diseases Clinical Research Consortium. Through this consortium the investigators hope to understand the fundamental biology of these rare diseases and aim to bank long-term observational data and corresponding biological specimens for researchers to access and further enrich.
Focal Segmental Glomerulosclerosis (FSGS) is a devastating kidney disease which is difficult to treat and carries a poor prognosis, with 50% of affected children progressing to end stage renal disease (ESRD). The purpose of this study is to investigate oral galactose as a benign treatment for FSGS in children. The investigators hypothesize that galactose, a simple milk sugar thought to bind to the protein factor (FSPF) that causes FSGS thereby inactivating it and stopping it from damaging the kidney, resulting in a reduction in glomerular permeability to albumin and decrease in proteinuria in children with nephrotic syndrome secondary to FSGS.
This study will test the safety and effectiveness of a monthly dosing regimen of dexamethasone-a strong steroid medication-to treat patients with focal segmental glomerulosclerosis (FSGS). Patients with this disease have kidney fibrosis (scarring) and proteinuria (excessive excretion of protein in the urine) that, in about half of the patients eventually requires kidney dialysis or transplant. Currently, the most effective treatment for FSGS is high-dose steroids (prednisone) taken daily for 4 to 6 months. However, only about 30 percent of patients respond to this treatment, and it causes serious side effects in many patients. Other drugs, such as cyclosporin and cyclophosphamide, improve proteinuria in even fewer patients (about 10 percent) and also have serious side effects. This study will explore whether a monthly pulse dose of steroids will achieve disease remission with less toxicity. Adults and children with FSGS who: 1) have not received steroid treatment, or 2) could not tolerate daily steroid treatment, or 3) relapsed after conventional steroid treatment may be eligible for this study. Those enrolled will take dexamethasone by mouth for 4 days every 4 weeks for a total of 8 months. Patients will undergo various tests before treatment starts (baseline), during the course of treatment, and in follow-up visits to evaluate the effects of treatment as follows: 1. Review of kidney biopsy, medical evaluation, measurement of total daily urine protein excretion and kidney function, psychiatric testing for depression or other mood disorder 2. Measurements of blood pressure, blood chemistries and urine protein excretion - monthly during treatment 3. Questionnaire about the effects of treatment, if any, on mood and feelings - monthly during treatment 4. Photographs of the face and body (in underwear or shorts and tank top) to evaluate body fat distribution- baseline and 8 months 5. Eye examinations for cataracts and glaucoma - baseline and 8 months 6. Bone density scan (DEXA scan) of the lower spine and hip - baseline, 4 and 12 months 7. Magnetic resonance imaging (MRI) of the hips 8. Psychological evaluation and quality of life evaluation - baseline, 1, 2 and 8 months 9. Blood tests for adrenal gland function - baseline, 4 and 8 months 10. Blood and urine tests - 10, 12, 15, and 18 months Patients who achieve remission (whose urine protein levels decrease to normal) before completing the 8 months of dexamethasone will take one more dose and then stop therapy, but continue with follow-up. Patients who achieve remission but relapse may be offered a second course of treatment.
This study will examine the effectiveness of the drug pirfenidone in treating focal segmental glomerulosclerosis (FSGS). Patients with this disease have kidney fibrosis (scarring) and proteinuria (excessive excretion of protein in the urine). About half of patients with FSGS eventually require kidney dialysis or transplant. Steroids, which are currently used to treat the disease, are effective in only a minority of patients. Other drugs, such as cyclosporin and cyclophosphamide, improve proteinuria in a very small percentage of patients and have serious side effects. Patients with FSGS who wish to participate in this study will undergo pre-study evaluation with blood and urine tests. Patients must be on a stable dose of an ACE inhibitor (a drug that lowers blood pressure and reduces proteinuria) for at list 6 months before starting pirfenidone therapy. (Patients who are not already taking an ACE inhibitor will be started on the drug; those who cannot tolerate ACE inhibitors will be given a different drug.) Patients with elevated cholesterol will take a cholesterol-lowering drug. A diet containing approximately 1 gram of protein per kilogram of body weight per day will be recommended. Patients will take pirfenidone by mouth 3 times a day for 12 months. Blood and urine will be tested once a month, either at NIH or by the patient's local kidney specialist. They will collect two 24-hour urine samples at the beginning of the treatment period, at 2-month intervals throughout the study, and at a 6-month follow-up. Patients will also be asked to give three to five tubes of blood and urine samples for analysis during the study. In animal studies, pirfenidone improved kidney function and proteinuria and reduced kidney scarring in rats with a disease similar to FSGS. In human studies, pirfenidone improved breathing and survival in patients with lung fibrosis.
Focal segmental glomerulosclerosis (FSGS) and minimal change disease are kidney diseases that are associated with increased excretion of protein in the urine. Approximately half of FSGS patients will lose kidney function within 8 years of diagnosis and will require dialysis. The purpose of this study is to determine whether intermittent oral steroid therapy can cause sustained remission of FSGS and MCD. Approximately 70 participants, including adults and children older than age 2, will be enrolled in this study. They will receive 48 doses of oral dexamethasone over a period of 48 weeks. One group will take two daily doses every 2 weeks; the other group will take four daily doses every 4 weeks. Doctors will monitor participants before, during, and after the steroid treatment with extensive exams and testing. At the completion of the study, researchers will evaluate the safety and efficacy of the drug treatment.
Children with frequently relapsing nephrotic syndrome (FRNS) are exposed to prolonged courses of steroids and other immunosuppressant medications. Given the adverse side effect profiles and variable efficacy of these medications, there is an urgent need to identify novel and safe therapies to treat nephrotic syndrome in children. Stimulation of the vagus nerve, which can be activated non invasively by transcutaneous auricular vagus nerve stimulation (taVNS), has immunomodulatory effects mediated by the inflammatory reflex and spleen. taVNS has become a therapy of interest for treating chronic immune mediated illnesses. The aims of the study are (1) To determine the feasibility of protocol implementation and tolerability of taVNS in the treatment of nephrotic syndrome in children (2) To establish proof-of-concept and generate statistical estimates of variance parameters and effect sizes for treatment response outcomes in children with nephrotic syndrome randomized to taVNS therapy compared with sham therapy (3) To investigate the effects of taVNS on inflammatory markers in children with nephrotic syndrome.
Children with steroid resistant nephrotic syndrome (SRNS) are exposed to prolonged courses of immunosuppressant medications. Given the adverse side effect profiles and variable efficacy of these medications, there is an urgent need to identify novel and safe therapies to treat nephrotic syndrome in children. Stimulation of the vagus nerve, which can be activated noninvasively by transcutaneous auricular vagus nerve stimulation (taVNS), has immunomodulatory effects mediated by the inflammatory reflex and spleen. taVNS has become a therapy of interest for treating chronic immune mediated illnesses. The aims of the study are (1) To determine the feasibility of protocol implementation and tolerability of taVNS in the treatment of nephrotic syndrome in children (2) To establish proof-of-concept and generate statistical estimates of variance parameters and effect sizes for treatment response outcomes in children with nephrotic syndrome randomized to taVNS therapy compared with sham therapy (3) To investigate the effects of taVNS on inflammatory markers in children with nephrotic syndrome.
NEPTUNE Match is an additional opportunity offered to NEPTUNE study participants to prospectively recruit and communicate patient-specific clinical trial matching with kidney patients and their physician investigators.
Kidney Disease Biomarkers Summary: This study will identify biomarkers (proteins and other molecules in the blood or urine) that may help scientists predict what kidney disease a patient has and whether a given patient would respond to particular therapies. The study will look for biomarkers in the blood and urine of patients with various kidney diseases and study of the effects of angiotensin converting enzyme inhibitors (ACE inhibitors) and angiotensin receptor blockers (ARB) on biomarkers. Blood and urine from healthy volunteers will be studied for comparison. Healthy people and the following patients may be eligible for this study: adults with diabetic nephropathy 18 years of age and older; children with newly diagnosed clinical idiopathic nephrotic syndrome between 2 and 18 year of age; children and adults with glomerular disease (minimal change disease, focal segmental glomerulosclerosis, or collapsing glomerulopathy). Participants undergo tests and procedures as follows: Glomerular Disease: Adults with glomerular disease provide about four to six blood and urine samples over the course of 6 to 12 months. The samples are collected at the time of regularly scheduled visits for the NIH treatment protocol in which they are participating. Children provide only blood samples. Chronic Kidney Disease: Patients with chronic kidney disease provide a blood and urine sample every 6 months for 3 years or more. Angiotensin Antagonism: Patients with chronic kidney disease who are taking ACE inhibitors or ARBs stop their medicines for 4 weeks, while those who are not taking ACE inhibitors or ARBs begin one of the medicines. In general, patients just starting on the medications continue them after the study is completed, since they are beneficial for chronic kidney disease. * Medication withdrawal group: Patients come to NIH for 2 successive days at the beginning of the study for blood and urine tests (including one 24-hour urine collection) and to receive iothalamate (a chemical used to measure kidney function). Iothalamate is delivered over 24 hours through a needle placed in the abdomen (or elsewhere) via a pump similar to pumps that some diabetics use to deliver insulin. Patients then stop taking their ACE inhibitor or ARB medication. They monitor their blood pressure every day and return to NIH after 1, 2 and 4 weeks for blood tests. During week 4, the iothalamate infusion is repeated, and blood and urine samples are collected as at the beginning of the study. Patients then resume taking their ACE inhibitor or ARB once a day with the dose being increased at 2-week intervals. They come to NIH weekly after 1 week and then every other week for blood tests. Four weeks after reaching the highest FDA-recommended dose of medication tolerated, the iothalamate infusion and blood and urine collections are repeated. * Medication induction group: At the beginning of the study, patients have the iothalamate infusion and blood and urine collections described above and then begin to take either an ACE inhibitor or ARB. The dose is increased after 2 weeks. Patients monitor their blood pressure every day. After being on the highest dose for 4 weeks, patients repeat the iothalamate infusion and blood and urine collections. The study is then complete and they are provided a 2-month supply of medicine to take home. Information is gathered on symptoms, treatments, and results of past laboratory tests of all patients. Healthy volunteers provide blood and urine sample collections every month or every other month for up to four collections to be used for biomarker studies and the screen for common chronic diseases.
This is a pilot study of retinoids for patients with unsatisfactory response to conventional treatment of nephrotic syndrome due to focal segmental glomerulosclerosis or minimal change disease, two renal disorders associated with putatively pathogenic malfunctioning of glomerular podocytes. The hypothesis that retinoids may have reparative effects on these cells is based on previous research showing that retinoids promote the differentiation or redifferentiation of aberrant epithelial cells. Results obtained by 6 months of treatment with retinoids (that have been approved for non-renal indications) will be used as preliminary information upon which to base further testing of these agents in formal clinical trials in refractory cases of these nephrotic syndromes.
The study will comprise primarily a single-ascending dose (SAD) escalation component.
This multi-site, pilot study will assess vitamin D supplementation in children and young adults with Glomerular Disease. .