The purpose of this study is to learn how well a new mobile app helps families manage their child's nephrotic syndrome. We will be asking what parents think of the new app and how we can improve the design of the app. We will look for 60 parents to participate in the study, from 4 children's kidney clinics in the United States. The study will last 12 months. Participants will be asked to check their child's urine protein levels at home daily and answer surveys. If participants are randomly assigned to the mobile app study arm, they will be asked to use the app. The risks associated with participating in this study include loss of privacy and breach of confidentiality. There are no anticipated benefits to your child from participating in the study, but we hope that in the future the app will help families manage nephrotic syndrome. The alternative is to not participate in this study. Participants will be compensated for their participation in the study.
This research study is being done to determine whether it is safe to give a kidney transplant from a donor with hepatitis C to a patient without hepatitis C. This study is looking for participants in need of a kidney transplant who are willing to accept a kidney from a patient with hepatitis C.
Patients between the ages of 12 months and 11 years who are undergoing ureteral reimplantation surgery will be randomized to receive intrathecal morphine, or bilateral quadratrus lumborum block. We will compare the effect that intrathecal morphine and quadratus lumborum blocks have on the duration of pain control as demonstrated by charted pain scores and morphine equivalents in the first 48 hours. This study will also assess the side effects of each intervention such as nausea and vomiting, and itching.
This randomized, double-blind, placebo-controlled Phase 2/3 adaptive study will first evaluate two doses of VX-147 in subjects with APOL1-mediated proteinuric kidney disease for 12 weeks to select a dose for Phase 3 and subsequently evaluate the efficacy and safety of the single, selected dose in the Phase 3 portion of the study.
The study comprises three periods: screening, treatment, and follow-up. The initial treatment period for Phase 2 is 12 weeks. Subjects enrolled for Phase 2 and Phase 3 who receive the non-selected dose will switch to the Phase 3 dose in a blinded manner after the Phase 3 dose is determined. The study will complete when 187 composite clinical outcome events are accrued among subjects in Phase 2 and Phase 3 who received placebo or the Phase 3 dose and when all enrolled subjects have at least 2 years of eGFR data.
This research study aims to learn more about children and adolescents who have a shiga toxin-producing E. coli (STEC) infection. E. coli is a type of bacteria found in the intestines. Although most types of E. coli are harmless, some produce toxins that can make children sick. This study will assess what type of treatment is best for this infection.
This study is for patients that have already received standard treatments for their cancer and their cancer has gotten worse or returned after their last treatment. The purpose of this study is to learn about the effects of the study drug XL092 when given alone, in combination with atezolizumab, and in combination with avelumab by testing its safety, the ability of your body to accept the drug(s), to measure the drug(s) and/or its break-down products levels in your blood, and how your body reacts to the drug(s). This research study will be the first time XL092 alone or in combination with atezolizumab will be given to people. The study drugs in this research have not been approved by the United States Food and Drug Administration (FDA). There are two parts in this study, a Dose Escalation part and a Cohort Expansion part. The first part of the study was the Dose Escalation phase, when different participants were given different doses of the study drug until the safest, most effective dose was found; this part of the study has been completed. The study is now enrolling to the Cohort Expansion part of this study, where the dose that has been determined to be safe will be given to more participants with different types of cancer. The Dose Escalation part of this study has stopped and the safe dose has been found. This study is now enrolling to the Cohort Expansion part of this study, where the safe dose of the study drug found in the Dose Escalation part will be given to more participants with different types of cancer. Participants will either be assigned to the treatment group that will receive XL092 alone or the treatment group that XL092 in combination with atezolizumab. Which treatment group they are assigned depends on the type of cancer they have. TXL092 is in tablet form taken by mouth. Avelumab will be given as an intravenous (IV) infusion once every 2 weeks at the study site. Atezolizumab will be given as an intravenous (IV) infusion once every 3 weeks at the study site. Total study duration is expected to be about 6 months but participants could be in the study for up to 2 years.
This research is studying the use of a drug called Humira in a small number of patients with Focal Segmental Glomerulosclerosis (FSGS) or treatment resistant Minimal Change Disease (TR-MCD). Specifically, some patients with FSGS and MCD have been shown in research studies to have overactive tumor necrosis factor (TNF) mechanisms in their kidney tissue. In these patients, high levels of urine markers called MCP1 and TIMP1 have been seen.
In this study, patients with FSGS or MCD with high levels of MCP1 and TIMP1 will be given a medication to block TNF, called Humira, to see if it reduces the urine levels of MCP1 and TIMP1. Subjects' health-related information, blood, and urine will be collected for this research study. This study may help us to develop individualized treatment options for future patients with TNF-driven FSGS or MCD.
Some of the more common risks with taking Humira include: infections, injection site reactions, headache and rash. This study may offer some benefit to subjects now by possibly reducing the amount of protein in their urine. The study may also benefit other patients in the future based on the knowledge gained. Alternatives to joining this study include treatment with steroids or other immunosuppressive medications.
A prospective parallel cohort study generating two groups of participants will be performed in NEPTUNE. The two groups are: (1) Cohort A which includes the FSGS/MCD Cohort; and the MN Cohort, both incipient and prevalent biopsied patients; and (2) Cohort B – a non-biopsy, treatment-naïve, pediatric cohort less than 19 years of age, cNEPTUNE. The sample size for the combined FSGS/MCD and MN Cohorts is a minimum of 800 participants, with a minimum of 375 new patients recruited under Protocol V5.0. The sample size for the second group, cNEPTUNE, will be a minimum of 200 participants. Participants will be recruited into each subgroup concurrently. All participants who meet the inclusion criteria at the participating centers will be enrolled if the participants or their legally authorized representative(s) provide comprehensive written informed consent. A recruit-to-replace strategy will be employed throughout the enrollment phase. Cohort A study visits including screening/eligibility, baseline, biopsy, and follow-up visits, and SMS texting. Study visits for Cohort B, cNEPTUNE, including screening/eligibility, baseline, follow-up visits, and SMS texting.