Clinical Research Directory

Phase 2a Study of VX-407 in Participants With ADPKD Who Have a Subset of PKD1 Gene Variants (AGLOW)

The purpose of the study is to evaluate the effect of VX-407 on height-adjusted total kidney volume (htTKV), safety, tolerability, and pharmacokinetics (PK) of VX-407.

Metabolic Impacts of Ren-Nu: A Dietary Program for Polycystic Kidney Disease

This is a 16 week pilot study of the impact of a nutritionist led ketogenic diet (Ren-Nu) supplemented with the medical food KetoCitra on autosomal dominant polycystic kidney disease.

Phase 1 Study to Evaluate the Safety and Tolerability of Intravenously Administered PYC-003

This is a Phase 1, First-in-Human study to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD) and immunogenicity of PYC-003 in healthy adult participants and adult participants with confirmed PKD1 mutation-associated Autosomal Dominant Polycystic Kidney Disease (ADPKD) There are 3 parts in this study, i.e. Part A, Part B and Part C

Polycystic Kidney Disease 1 (PKD1) Gene Variant Groups in Autosomal Dominant Polycystic Kidney Disease

The purpose of this study is to estimate the prevalence, demographic, and clinical characteristics of PKD1/2 gene variant groups in the ADPKD population.

Di-PKD: A Pilot Trial of Dietary Intervention in Patients With Autosomal Dominant Polycystic Kidney Disease

The goal of this interventional study is to evaluate the impact of a specific diet (low salt, limited caffeine, high potassium, low phosphate, limited protein, limited carbohydrate intake, adequate water intake) on changes in serum and urinary biomarkers, total kidney volume (TKV), and cyst progression in subjects with autosomal dominant polycystic kidney disease (ADPKD). Researchers will compare the results of subjects in the study diet (interventional) arm to the results of subjects in the regular diet (control) arm.

Robotic vs Open Nephrectomy for ADPKD

The goal of this observational prospective cohort study is to evaluate the feasibility, safety, and postoperative outcomes of robotic-assisted unilateral native nephrectomy using the Medtronic Hugo™ RAS system compared with the open surgical approach in patients with autosomal dominant polycystic kidney disease (ADPKD) The main question(s) it aims to answer are: Is robotic-assisted unilateral nephrectomy with the Hugo™ RAS system feasible and safe in patients with ADPKD? Does robotic-assisted nephrectomy provide comparable or improved postoperative outcomes (e.g., complications, transfusion rates, length of hospital stay) compared with open nephrectomy? Researchers will compare robotic-assisted unilateral nephrectomy (Hugo™ RAS) with open unilateral nephrectomy to see if the robotic approach results in similar morbidity with shorter hospital stay, despite longer operative times. Participants are adult patients with ADPKD undergoing unilateral native nephrectomy at a single tertiary transplant center, most of whom have end-stage renal disease and are candidates for or recipients of kidney transplantation. Both male and female patients were included; patients undergoing bilateral nephrectomy were excluded.

Drug-Drug Interaction of JMKX003142 With Amiodarone and Febuxostat in Healthy Subjects

This is a open-label, non-randomized, multiple oral dosings, three-period, drug-drug interaction study of JMKX003142 tablet, Amiodarone Hydrochloride Tablets and Febuxostat Tablets in Chinese healthy adult subjects

Simulated and Synthetic Health Data: Improving Clinical Research on Rare Diseases. A Real-World Data Simulation of Autosomal Dominant Polycystic Kidney Disease (ADPKD) Trials. A Retrospective, Observational Study

This is a no-profit, retrospective observational study involving real-world data (RWD), retrieved from ADPKD-related electronic health records stored at Mario Negri Institute IRCCS. RWD will be used to generate simulated and synthetic datasets, using AI tools. RWD and generated data (GD) will be used to conduct three virtual RCTs, which main outcome is change in Total Kidney Volume (TKV). Statistical tests will be performed to assess quality and privacy preservation of GD compared with RWD. GD will be also evaluated in exploratory sample size estimations.

Study of Tamibarotene in Patients With ADPKD

Clinical trial of tamibarotene in patients with Autosomal Dominant Polycystic Kidney Disease

Artificial Intelligence-based Image Processing Methods to Advance the Characterization of Polycystic Kidney Disease

The primary aim of this observational exploratory study is to develop AI-based image processing methods to advance the characterization of Polycystic Kidney Disease using medical images and associated clinical data, including: 1. AI-based fully automatic segmentation techniques for the accurate identification of kidneys, liver, and cysts, with a focus on AI interpretability and robustness; 2. advanced AI-based image processing techniques allowing to identify new imaging biomarkers, including through the use of radiomics, to characterize ADPKD tissue microstructure and therefore stage the disease and monitor and predict disease progression and response to therapy; 3. multiparametric models including image-based radiomic features alongside clinical and laboratory data to stratify ADPKD patients and predict ADPKD progression over time. The study will also have the secondary aim of validating the novel techniques against gold standard (manual) methods, when available.