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Dominant - ClinicalTrials.gov

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Last Updated: 05 June 2022

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Natural History of Autosomal Dominant Hearing Loss

"Hereditary hearing loss is one of the most common sensory disorders affecting newborns. " Hearing aids and cochlear implantation are two of the primary treatment options for patients with hereditary hearing loss. Many studies have shown the effectiveness of using gene therapy to enhance hearing in mouse models of human hereditary hearing loss in mouse models. Since patients with DFNA often experience hearing loss later in childhood or even adulthood, the most common method for gene therapy is nonsyndromic autosomal dominant sensorineural hearing loss. We intend to determine the effectiveness of genome editing on cultured primary or immortalized fibroblasts obtained from DFNA patients in this report. The natural history of DFNA will also be chronicled by these patients and their families. This will give DFNA baseline data for DFNA, which will be useful in interpreting the findings of forthcoming clinical trials of inner ear gene therapy.

Source link: https://clinicaltrials.gov/ct2/show/NCT04501081


FDG PET to Assess Therapeutic Response in Patients With Bone-Dominant Metastatic Breast Cancer, FEATURE

"Evaluate the efficacy of fludeoxyglucose F-18 -positron emission tomography response parameters as a binary predictor of progression-free survival in patients with bone-dominant metastatic breast cancer treated with systemic therapy. " Evaluate the ability of FDG-PET/CT updated PERCIST guidelines to reliably predict PFS in patients with BD MBC. Evaluate the ability of FDG-PET/CT modified PERCIST tools to predict time to skeletal related events and overall survival in patients with BD MBC. Patients with BD MBC are unable to determine PFS, time to SRE, and OS using FDG-PET/CT metrics. Evaluation of disease progression by FDG-PET/CT to detect disease progression by the detection of new lesions that were not detected by standard CT and bone scans. Patients are FDG intravenously and undergoing PET/CT scan over 15-30 minutes at baseline and 12 weeks after commencing with standard systemic therapy in the absence of unacceptable outcomes.

Source link: https://clinicaltrials.gov/ct2/show/NCT04316117


Time Restricted Feeding in Overweight and Obese Adults With Autosomal Dominant Polycystic Kidney Disease

"Mounting evidence shows that a metabolic defect exists in autosomal dominant polycystic kidney disease," the disease's founders speculated. " In the Halt Progression of Polycystic Kidney Disease Study A, the researchers found that obesity and obesity were strong predictors of more rapid kidney growth. The investigators have novel preliminary results using magnetic resonance images obtained from a small number of participants from HALT-PKD Study A's abdominal adiposity is an independent predictor of kidney development and kidney function decline. The investigators are currently undergoing a continuing behavioral weight loss pilot study in adults with ADPKD who are overweight/obese. In a rodent model of ADPKD, which includes kidney: body mass and mammalian target of rapamycin production, it may be an alternative and simpler to adhere to a diet plan to slow ADPKD progression. Aim: Determine the possibility of TRF without energy intake limitations in adults with ADPKD and overweight/obesity. The study will determine adherence to TRF by determining the percentage of participants meeting the goal of eating within an 8-hour TRF window, measured objectively with a photographic food record, and verified with continuous glucose monitoring results. ".

Source link: https://clinicaltrials.gov/ct2/show/NCT04534985


A Prospective First-In-Human Study to Evaluate the Safety and Tolerability of QR-1123 in Subjects With Autosomal Dominant Retinitis Pigmentosa (adRP) Due to the P23H Mutation in the RHO Gene

"It is hypothesized that the reduction of mutant P23H mRNA will minimize the deleterious effects of the dominant-negative protein's deleterious properties and may result in increased production of WT rhodopsin protein in photoreceptors. " In the single dose cohorts study, QR-1123 will be administered in an open label fashion. In a double masked fashion, the repeat dose cohorts participants will be randomly assigned to either a unilateral IVT injection of QR-1123 every 3 months or a unilateral sham treatment every 3 months.

Source link: https://clinicaltrials.gov/ct2/show/NCT04123626


Phase II Study of Nivolumab (Anti-PD1), Tadalafil and Oral Vancomycin in Patients With Refractory Primary Hepatocellular Carcinoma or Liver Dominant Metastatic Cancer From Colorectal or Pancreatic Cancers

"Background: Patients with liver cancer, including hepatocellular carcinoma and advanced liver cancers, are limited, and ignore the fact of known genetic and genetic variability in these diseases. Nivolumab is a complete human monoclonal immunoglobulin G4 antibody that is specific for the human programmed death-1 cell surface membrane receptor. Tadalafil is a phosphodiesterase type 5 inhibitor with the FDA approved for the treatment of pulmonary arterial hypertension, benign prostatic hyperplasia, and erectile dysfunction with a relatively stable clinical profile. Multiple malignancies and tumor cell lines have been evaluated for their direct anticancer properties, for their efficacy as chemosensitizers, and for cancer chemoprevention. The aim of the study is to determine whether immune checkpoint inhibition in advanced liver cancer can increase the immunomodulatory activity induced by PDE5 inhibitor and ororal vancomycin. Object: : To determine the greatest overall response in patients with refractory primary HCC or pancreatic adenocarcinoma, please refer to Response Criteria. Proposed Study: The current research is based on a phase II review of combined nivolumab, oral vancomycin, and tadalafil therapy in patients with HCC or liver-dominant cancers of colorectal or pancreatic cancers. Patients will be seen in the Clinical Center on a monthly basis, with disease status assessment every 8 weeks after the start of study therapy. ".

Source link: https://clinicaltrials.gov/ct2/show/NCT03785210

* Please keep in mind that all text is summarized by machine, we do not bear any responsibility, and you should always check original source before taking any actions

* Please keep in mind that all text is summarized by machine, we do not bear any responsibility, and you should always check original source before taking any actions