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  • A Double-Blind, Randomized, Placebo-Controlled Study to Assess the Safety, Tolerability, and Pharmacodynamics of SYNB8802 in Healthy Volunteers and in Patients with Enteric Hyperoxaluria (Synlogic) Rochester, Minn.

    The primary objective of Part 1 of this study (healthy volunteers) is to evaluate the safety and tolerability of SYNB8802.  The primary objective of Part 2 of this study (patients with Enteric Hyperoxaluria) is to assess the effect of SYNB8802 on UOx amount excreted.

  • A Natural History Study of Patients with Genetically Confirmed Primary Hyperoxaluria Type 3 and, as Applicable per Age, a History of Stone Events (PHYOX-OBX) Rochester, Minn.

    The objective of this study is to collect data on stone formation and the degree of nephrocalcinosis in patients (≥ 2 years of age) with genetically confirmed PH3 and relatively intact renal function and to explore the potential relationship between Uox and new stone formation.

    This is a natural history study of adults, adolescents, and children (≥ 2 years of age) with genetically confirmed primary hyperoxaluria type 3 (PH3) who have a history of stone events during the last 3 years and/or the presence of pre-existing stones detected by renal ultrasound at Screening.

    The relationship between the level of Uox and the incidence of kidney stones and/or nephrocalcinosis in patients with PH3 has not been established. The goal of this study is to record 24-hour Uox levels and the incidence of new stone formation and/or the degree of nephrocalcinosis in patients with PH3 over time.

     

  • A Phase 2 Open-Label Study to Evaluate the Safety and Efficacy of DCR-PHXC in Patients With Primary Hyperoxaluria Type 1 or 2 and Severe Renal Impairment, With or Without Dialysis (204) Rochester, Minn.

    The purpose of this study is to evaluate DCR-PHXC in patients with primary hyperoxaluria type 1 (PH1) or type 2 (PH2) and severe renal impairment, with or without dialysis.

     

  • Biobank Protocol, Rare Diseases Clinical Research Network Rochester, Minn.

    This study is being done to obtain samples from patients with primary hyperoxaluria, cystinuria, adenine phosphoribosyl transferase (APRT) deficiency, and Dent disease, and from their family members, for use in future research.

  • BONAPH1DE, A prospective observational study of patients with primary hyperoxaluria type 1 (PH1) (ALN-GO1-007) Rochester, Minn.

    This is a global, prospective, observational, longitudinal study designed to characterize the long-term safety of lumasiran in PH1 patients in the real-world setting. The data collected in this
    study will also be utilized to further characterize the natural history and real-world clinical management of patients diagnosed with PH1, as well as the long-term effectiveness of lumasiran. Patients will be managed and treated per routine clinical practice. This protocol does not recommend the use of any specific treatments, visits, or procedures. No medication is provided as part of study participation.


    Patients are expected to contribute data for the duration of the study or until study discontinuation (e.g., due to death, withdrawal of consent, loss to follow-up, study termination, enrollment in a clinical trial with an investigational treatment during the study). Study data will be collected at the time of a routine clinical encounter, or by referencing the medical record, and entered into the electronic data capture (EDC) at least once every 12 months using electronic case report forms (eCRFs). If patients attend more than 1 visit per year, sites can enter multiple follow-up visits. In addition to the prospective data collection, a chart review of up to 5 years relative to the time of enrollment in the study will be conducted to collect retrospective data, where available. In cases where diagnosis was more than 5 years prior to enrollment data will also be collected at the time of diagnosis, if available.

  • CT and Urinary Correlates of Renal Stone Precursor Lesions Rochester, Minn.

    We hypothesize that clinical studies to investigate the role of individual proteins in kidney stone pathogenesis have likely been confounded by an unknown variety of underlying renal pathologies. Therefore, we propose to examine urinary protein crystallization inhibitors in patient populations that have been carefully phenotyped relative to renal stone precursor lesions by direct endoscopic visualization. In collaboration with Project #1, our second major goal is to use these accurately phenotyped patients in order to adapt modern dual-energy CT technology to develop a reliable noninvasive technique to accurately and noninvasively determine stone composition and visualize the earliest kidney stone precursor lesions. Our long-term goal is to improve CT technology so that it can be used to allow large-scale clinical protocols of accurately phenotyped, hence, homogeneous, patient populations.

    In a subset we will sample sterile stone, dental plaque, blood and urine samples for detailed microbiome analysis in order to determine the contribution of micro organisms to stone pathogensis.

  • Phase 1-2a Safety, Tolerability, and Pharmacodynamics Controlled Study of NOV-001 in Healthy Volunteers and Patients with Enteric Hyperoxaluria (NOV-001-CL01) Rochester, Minn.

    The purpose of the first stage of this study is to evaluate safety, tolerability, and pharmacodynamics of NOV-001 in adult healthy volunteers.  The purpose of the second stage of this study is to evaluate safety, tolerability, and early effectiveness in patients with enteric hyperoxaluria.

  • Prospective Research Rare Kidney Stones (ProRKS) (ProRKS) Rochester, Minn., Jacksonville, Fla.

    The purpose of this study is to determine the natural history of the hereditary forms of nephrolithiasis and chronic kidney disease (CKD), primary hyperoxaluria (PH), cystinuria, Dent disease and adenine phosphoribosyltransferase deficiency (APRTd) and acquired enteric hyperoxaluria (EH). The investigator will measure blood and urinary markers of inflammation and determine relationship to the disease course. Cross-comparisons among the disorders will allow us to better evaluate mechanisms of renal dysfunction in these disorders.

  • Rare Kidney Stone Consortium Registry for Hereditary Kidney Stone Diseases (RKSC) Rochester, Minn.

    The purpose of this study is to collect medical information from a large number of patients in many areas of the world with primary hyperoxaluria (PH), Dent disease, Cystinuria and APRT deficiency. This information will create a registry that will help us to compare similarities and differences in patients and their symptoms. The more patients we are able to enter into the registry, the more we will be able to understand the Primary Hyperoxalurias,Dent disease, cystinuria and APRT and learn better ways of caring for patients with these diseases.

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