Clinical Trials

The purpose of this study is to determine the efficacy and safety of ALLN-177 in patients with enteric hyperoxaluria.

The purpose of this study is to assess the burden that patients and their caregivers face to due primary hyperoxaluria (PH).

The purpose of the study is to determine if the investigational medicine, lumasiran, is able to reduce the production of oxalate in the liver and to evaluate the safety of lumasiran and how the body responds to it.

This study will evaluate the efficacy and safety of OC5 in patients with PH.

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.

The purpose of this study is to determine if Oxalobacter formigenes is effective at lowering plasma oxalate levels in patients with primary hyperoxaluria who are on dialysis.

This study aims to evaluate the safety and tolerability of a single dose of DCR-PHXC in patients with PH3 and to characterize the plasma PK of a single dose of DCR-PHXC in patients with PH3. It also aims to assess the efficacy of a single dose of DCR-PHXC in reducing oxalate burden in patients with PH3. 

The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of single- and multiple-ascending doses of ALN-GO1 in healthy adult volunteers and subjects with PH1.

Specific mutations relating to hyperoxaluria will be determined via DNA analysis by the Mayo RKSC research staff.

The purpose of this research study is to determine if the frequent use of polyethylene glycol (MiraLAX) may cause hyperoxaluria - an abnormally high level of oxalate in the urine.

The purpose of this study is to evaluate the safety, effectiveness, and pharmacokinetics (PK, how a drug is metabolized by the body) of Nedosiran in neonates, infants, and young children (birth to 11 years of age) with primary hyperoxaluria (type 1 [PH1] and type 2 [PH2]), and relatively intact renal function based upon eGFR and serum creatinine.

The purpose of this study is to evaluate the effectiveness, safety, pharmacokinetics (PK), and pharmacodynamics (PD) of lumasiran in patients with Advanced Primary Hyperoxaluria Type 1 (PH1).

The purpose of this study is to better understand your PH1 condition and how it has affected your body from the time of diagnosis until present. This study is being done for research purposes only.

Evaluate the efficacy and safety of ALLN-177 in reducing plasma and urinary oxalate levels in adult and pediatric patients with enteric hyperoxaluria and hyperoxalemia or primary hyperoxaluria.

The purpose of this study is to evaluate the long-term effectiveness and safety of DCR-PHXC in children and adults with Primary Hyperoxaluria Type 1 (PH1) and Primary Hyperoxaluria Type 2 (PH2) that have completed a previous DCR-PHXC clinical trial.

The primary purpose of this study is to identify women with Primary Hyperoxaluria (PH) who have had pregnancies from a large, international registry and describe the maternal and fetal complications of pregnancies in this cohort, as well as compare outcomes of pregnancies before and after organ transplantation in women with PH.

The purpose of this study is to evaluate the effectiveness and safety of Lumasiran in Children and Adults with Primary Hyperoxaluria Type 1 (PH1).

Primary hyperoxaluria is an inborn error of metabolism that results in marked overproduction of oxalate by the liver. The excess oxalate causes kidney failure and can cause severe systemic disease due to oxalate deposition in multiple body tissues.

Metabolic pathways that lead to oxalate are poorly understood, but recent evidence suggests that hydroxyproline may play a role. Sources of hydroxyproline include the diet and bone turnover. If hydroxyproline can be confirmed as a signficant factor in primary hyperoxaluria, diet modification might be of value in reducing the severity of disease.

This protocol, in which hydroxyproline labelled with a cold isotope is infused ...

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.

This study will help us determine whether certain genetic mutations, more than others, are a cause of more severe disease in Primary Hyperoxaluria.

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.

The purpose of this study is to evaluate the safety, tolerability, and effectiveness of different doses of ALLN 177 for reducing urinary oxalate excretion in patients with secondary hyperoxaluria and recurrent kidney stones.

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.

The purpose of this study is to determine the effectiveness, durability, and long-term safety of Reloxaliase in patients with Enteric Hyperoxaluria.

Evaluate the safety, tolerability, and efficacy of 28 days of treatment with ALLN-177 for reducing urinary oxalate excretion in patients with secondary hyperoxaluria and kidney stones.

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 ...

This study aims to evaluate the mechanisms leading to hyperoxaluria and increased risk of kidney stone formation after bariatric surgery.

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.