We are particularly interested in the diseases that cause nephrotic syndrome called Focal and Segmental Glomerulosclerosis (FSGS), Minimal Change Disease (MCD), and Membranous Nephropathy (MN). By collecting health information and laboratory samples, our goal is to learn more about diseases and find better ways to prevent and treat people with diseases.…
The purpose of this study is to find out if the study medicine called EDIT-301 will help in the treatment of Severe Sickle Cell Disease (SCD) and how safe it is to be used in people. EDIT-301 (study medicine) is a new investigational therapy which uses the patient's own stem cells, modifies them by CRISPR technology, and infuses them back to the…
The CureGN Bone Study is a prospective study that is assessing the effects of 4-types of kidney disease on the skeleton. In order for us to determine the true effect of kidney disease on bone, we need to determine what healthy bone looks for patients who have the same age, race, sex, and body mass index as the patients with kidney disease. We are looking…
The purpose of this study is to learn about whether the study drug (PF-07209326) is safe and effective in people with sickle cell disease and learn about what the right dose is. We will look at drug levels in the blood, see how fast drug levels rise and fall, and what effects the drug has, all of which are known as the pharmacokinetics (PK) and…
We are testing new experimental drugs such as selinexor in the hopes of finding a treatment that may be effective against tumors that have come back or that have not responded to standard therapy. The goals of this study are: To find the highest safe dose of selinexor that can be given without causing severe side effects; To learn what kind of side effects…
This is a Phase 1 multicenter study of pevonedistat in combination with Temozolomide and Irinotecan in pediatric patients with refractory solid tumors, including CNS tumors and lymphoma. The study treatment is considered experimental because pevonedistat is not approved by the United States (US) Food and Drug Administration (FDA) for treating pediatric…
The MK-8228-002 Study is studying the safety and effectiveness of an investigational medication for preventing cytomegalovirus (CMV) disease in adults with kidney transplants. The MK-8228-002 Study is evaluating an investigational medication (known as MK-8228) versus valganciclovir (VGCV). VGCV is an antiviral medication and is the current standard of care…
The purpose of this study is to examine the effectiveness and tolerability of a study drug called PF-02341066. Additional goals of this study are to learn how the body handles the study drug as well as determine whether or not PF-02341066 is a beneficial treatment for your tumor This study is for children with a recurrent or progressive tumor or lymphoma…
The purpose of this study is to assess the most adequate dose of Acthar Gel (ACTH) in patients withIgA nephropathy. Acthar Gel (ACTH) has been approved by the Food and Drug Administration(FDA) for routine clinical use in the treatment of patients with proteinuria and patients withidiopathic nephrotic syndrome such as IgAN.
The purpose of this study is to assess the safety and effectiveness of a study drug called axitinib on treating tumors that have returned (recurrent) or have not responded to standard therapy (refractory). Children will take axitinib twice a day for 28 days (1 cycle). Children may take study drug for up to 24 cycles (2 years).
The purpose of this study is to evaluate the safety and effectiveness of an investigational medication in patients with focal segmental glomerulosclerosis (FSGS). The goal is to learn whether the investigational medication may change the amount of protein in the urine and decrease proteinuria.
This study is being done to see if giving your child a lower dose of chemotherapy (drug therapy), than has been used historically in bone marrow transplant, to kill his/her own diseased cells before he/she receives a bone marrow transplant, will possibly result in the permanent control of your child's sickle cell disease. Patients will receive busulfan…
Kidney disease patients have a variety of severe bone disorders that result in rapid bone loss and fractures. We are doing this research study to determine relationships between bone quality determined from bone imaging and bone biopsy and by non-invasive measures from blood and skin. We are trying to identify non-invasive markers of bone quality in kidney…
The purpose of this study is to find out more about how the study drug called entrectinib will be used in children, adolescents and young adults with treatment-resistant solid tumor in brain or body. This is a phase 1/1b study, divided into a dose escalation portion (different dose will be tested) and a dose expansion portion (one dose will be tested) in…
This study is for patients with autosomal dominant polycystic kidney disease (ADPKD). The purpose of this study is to test the effectiveness of a study drug called tolvaptan. Tolvaptan continues to be studied in this trial as a possible treatment for ADPKD. ADPKD is a disease that causes kidney cysts (cyst are like fluid-filled balloons), worsening kidney…
The purpose of this study is to find out more about how the study drug called talimogene laherparepvec will be used in children/adolescents/young adults with advanced solid tumors not involving the central nervous system that are available for direct injection. This is a phase 1 study, multicenter, open-label study, that means all subject enrolled in the…
Contrast-Induced Nephropathy (CIN) can occur when patients with pre-existing kidney problems undergo procedures that use iodinated contrast media, such as cardiac catheterizations. RenalGuard Therapy was developed to enable the patient to clear the contrast out of their kidney before it can do significant damage. This study aims to enroll patients with…
This is a phase 1/2 multicenter study of AZD1775 (MK-1775) that will be given in combination with irinotecan. The study treatment is considered experimental because it is not approved by the United States (US) Food and Drug Administration (FDA) for treating children and young adults with solid tumors which have come back or have not responded to standard…
The purpose of this research study is to understand the way Alport Syndrome affects patients kidneys over time. Alport syndrome is a genetic condition that causes damage to your kidneys over time. Currently there is not much information on the disease. Information about how the body is affected by the disease will be collected, along with medical and family…
The goals of this study are to find the highest safe dose of ramucirumab that can be given without causing severe side effects, to learn what kind of side effects ramucirumab can cause, to learn more about the effects of ramucirumab on cells and proteins in the blood and to determine whether ramucirumab is a beneficial treatment for patients with solid…
Focal Segmental Glomerulosclerosis (FSGS) is a disease in which your kidneys allow an excess amount of protein in your urine. Too much protein in the urine (also called proteinuria) is a medical condition called nephrotic syndrome. The purpose of this clinical trial is to evaluate the safety and effectiveness of the study drug, Acthar, in adult patients…
This is a Phase 1 multicenter study of a drug called ABI-009 (nab-Rapamycin) in combination withtemozolomide and irinotecan in children and young adults with solid tumors (including CNS tumors)which have come back or have not responded to standard therapy. The study is considered experimental because ABI-009 (nab-Rapamycin) is not approved by the United…
The purpose of this study is to learn what kind of side effects nivolumab as a single agent and nivolumab in combination with ipilimumab can cause and if these treatments are beneficial for solid tumors. Nivolumab (Dose Level 1) will be given as an intravenous infusion every 14 days of a 28 day cycle. If Dose Level 1 is not tolerable, then it will be…
The purpose of this research is to learn how to help parent youth pairs work together to help youth with sickle cell disease take hydroxyurea every day as a daily habit. This study is for children and teens ages 10-18 with Sickle Cell Anemia taking hydroxyurea, and their parents (or guardians). Many children and teens do not use hydroxyurea every day. Daily…
This study is being carried out by the Childrens Oncology Group (COG) Phase 1 Consortium. COG is an international research group that consists of more than 200 hospitals that treat children with cancer in the United States, Canada, Australia, and Switzerland. The Phase 1 Consortium is the group within COG that consists of 21 hospitals, and participation in…
This research is being done to learn whether organ transplantation from HIV-positive deceased donors is as safe and effective in HIV-positive recipients as transplants from HIV-negative deceased donors. People with end-stage organ disease and HIV infection, and who qualify for organ transplantation, may join this study.
This study is for patients with the following cancers: advanced non-small cell lung cancer, head and neck cancer, melanoma, colon cancer, or bladder cancer. The purpose of this study is to find out more about the effects of AMG 228 in people and on their cancer. AMG 228 is an experimental drug that is being developed to stimulate the bodys immune system to…
A sign of your kidney disease is the presence of too much protein in your urine. The purpose of this study is to evaluate if the investigational study drug abatacept can decrease the amount of protein in your urine and improve your kidney disease beyond the treatment you have received or are already receiving. Abatacept is being used in an investigational…