Clinical trials: Primary Hyperoxaluria Type 1
Primary Hyperoxaluria Type 1 (PH1)
Treatment of Primary Hyperoxaluria Type 1 With Nedosiran
The purpose of this program is to provide participants with access to an investigational drug, nedosiran, for treatment of primary hyperoxaluria type 1 (PH1).
- Trial name
- NCT05993416
- Testing
- Nedosiran
- Sponsor
- Dicerna Pharmaceuticals, Inc., a Novo Nordisk company
Primary Hyperoxaluria
Phenotyping of Primary Hyperoxaluria
985 / 5000 Résultats de traduction Primary hyperoxaluria is a rare autosomal recessive disease with an estimated prevalence of around 1 to 3 cases per million population.
- Trial name
- PHENO-HOPLA (NCT05107830)
- Where
- France
- Volunteers
- 186 people
- Sponsor
- University Hospital, Strasbourg, France
Cystinosis, Primary Hyperoxaluria
Genetic Newborn Screening for Cystinosis and Primary Hyperoxaluria
In Germany parents of newborns are offered newborn screening (NBS) for 17 congenital diseases as a standard benefit of statutory health insurance. NBS in Germany is voluntary. Cystinosis and hyperoxaluria are very rare diseases.
- Trial name
- GENESIS (NCT05843851)
- Testing
- Diagnostic test
- Where
- Germany
- Volunteers
- 200,000 people
- Sponsor
- Cystinose Stiftung
Hyperoxaluria
Renal Protective Effect of ACEI and ARB in Primary Hyperoxaluria
This study will test the effectiveness of two medications: ACEI (angiotensin converting enzyme inhibitor)and ARB (angiotensin receptor blocker) in reducing the renal injury induced by hyperoxaluria in patients with Primary…
- Trial name
- NCT00280215
- Testing
- ACEI / Angiotensin converting enzyme inhibitor, ARB /Angiotensin Receptor Blocker
- Sponsor
- Mayo Clinic
Oxalate, Primary Hyperoxaluria, Microbiome
Descriptive Analysis of Gut Microbiome Alterations in Hyperoxaluric Patients
To characterize the microbiome in 4 groups of subjects (primary hyperoxaluria type I (PH1), idiopathic CaOx stone, enteric hyperoxaluria (EH) and healthy participants) by comparing the number of species and diversity of the…
- Trial name
- NCT02794649
- Where
- United States
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- VA New York Harbor Healthcare System
Papulopustular Rosacea
A Phase 2 Study to Investigate the Efficacy, Safety and Tolerability of Remibrutinib (LOU064) in Adult Patients With Papulopustular Rosacea (PPR)
This Phase 2 study aims to evaluate whether Bruton's tyrosine kinase (BTK) inhibition with remibrutinib can produce a clinically meaningful reduction in inflammatory lesions in adults with moderate-to-severe papulopustular…
- Trial name
- NCT07681271
- Testing
- LOU064
- Where
- 12 sites in United States, Bulgaria and 6 more
- Volunteers
- 100 people
- Sponsor
- Novartis Pharmaceuticals
Primary Hyperoxaluria
Study of DCR-PHXC-101 in Normal Healthy Volunteers and Patients With Primary Hyperoxaluria
This is a double-blind, placebo-controlled, dose escalation trial of DCR-PHXC in Healthy Volunteers (HVs) and patients with Primary Hyperoxaluria (PH).
- Trial name
- NCT03392896
- Testing
- DCR-PHXC
- Where
- 7 sites in United States, France and 3 more
- Volunteers
- 43 people, healthy volunteers welcome
- Sponsor
- Dicerna Pharmaceuticals, Inc., a Novo Nordisk company
Primary Hyperoxaluria, Cystinuria
Health-related Quality of Life in Rare Kidney Stone
Assessment of Health-related Quality of Life in Rare Kidney Stone Formers in the Rare Kidney Stone Consortium
- Conditions
- Primary Hyperoxaluria, Cystinuria, Adenine Phosphoribosyl Transferase Deficiency, Dent Disease
- Trial name
- NCT02124395
- Testing
- Health-related Quality of Life in Rare Kidney Stone Questionnaire
- Where
- 3 sites in United States, Iceland
- Volunteers
- 365 people
- Sponsor
- NYU Langone Health
Type 1 Primary Hyperoxaluria
Clinical Exploration Study of YOLT-203 in the Treatment of Type 1 Primary Hyperoxaluria (PH1)
This study is a single-arm, open-label, single-dose, dose-escalation trial, aiming to evaluate the safety and tolerability of YOLT-203 in the Chinese population with type 1 primary hyperoxaluria (PH1); and to preliminarily assess…
- Trial name
- PH1 (NCT06892301)
- Testing
- YOLT-203
- Where
- China
- Volunteers
- 2 people
- Sponsor
- Guangzhou Women and Children's Medical Center
Primary Hyperoxaluria
Primary Hyperoxaluria Mutation Genotyping
This study will help us determine whether certain genetic mutations, more than others, are a cause of more severe disease in Primary Hyperoxaluria.
- Trial name
- NCT00589225
- Testing
- Genetic Analysis
- Where
- United States
- Volunteers
- 902 people, healthy volunteers welcome
- Sponsor
- Mayo Clinic
Primary Hyperoxaluria, Nephrocalcinosis
International Registry for Primary Hyperoxaluria
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.
- Conditions
- Primary Hyperoxaluria, Nephrocalcinosis, Kidney Stones
- Trial name
- NCT00875823
- Where
- 4 sites in United States
- Sponsor
- Mayo Clinic
Primary Hyperoxaluria
Phase 2/3 Oxabact Study
The purpose of this study is to determine if Oxalobacter formigenes is effective at lowering urinary oxalate levels in patients with primary hyperoxaluria.
- Trial name
- NCT01037231
- Testing
- Oxalobacter formigenes
- Where
- 3 sites in United States, Germany and 1 more
- Volunteers
- 36 people
- Sponsor
- OxThera
Primary Hyperoxaluria Type 3
Study to Evaluate Safety, Tolerability, PK and PD of DCR-PHXC in PH Type 3 Patients
The DCR-PHXC-104 study is designed to assess the safety, tolerability, and pharmacological parameters of a single dose of DCR-PHXC in Primary Hyperoxaluria Type 3 (PH3).
- Trial name
- PHYOX4 (NCT04555486)
- Testing
- DCR-PHXC, Sterile Normal Saline (0.9% NaCl)
- Where
- 6 sites in United States, Germany and 2 more
- Volunteers
- 6 people
- Sponsor
- Dicerna Pharmaceuticals, Inc., a Novo Nordisk company
Primary Hyperoxaluria
Expanded Access Protocol to Provide Lumasiran to Patients With Primary Hyperoxaluria Type 1
The purpose of this study is to provide expanded access to lumasiran for adults and pediatric patients with Primary Hyperoxaluria Type 1 (PH1),
- Trial name
- NCT04125472
- Testing
- Lumasiran
- Sponsor
- Alnylam Pharmaceuticals
Primary Hyperoxaluria Type 1
A Study of DCR-PH1 in Patients With Primary Hyperoxaluria Type 1 (PH1)
A phase 1 study of DCR-PH1 in patients with primary hyperoxaluria type 1 (PH1) to determine the safety, tolerability, pharmacokinetic (PK) and pharmacodynamics (PD) effects of DCR-PH1 administered via-intravenous infusion (IV)
- Trial name
- NCT02795325
- Testing
- DCR-PH1
- Where
- 2 sites in Germany, Netherlands
- Volunteers
- 41 people
- Sponsor
- Dicerna Pharmaceuticals, Inc., a Novo Nordisk company
Enteric Hyperoxaluria, Primary Hyperoxaluria
Study of ALLN-177 in Patients Aged 12 Years or Older With Enteric or Primary Hyperoxaluria and Hyperoxalemia
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
- Conditions
- Enteric Hyperoxaluria, Primary Hyperoxaluria, Hyperoxalemia
- Trial name
- NCT03391804
- Testing
- ALLN-177
- Where
- 9 sites in United States, Germany and 1 more
- Volunteers
- 15 people
- Sponsor
- Allena Pharmaceuticals
Hyperoxaluria, Cystinuria
Prospective Research Rare Kidney Stones (ProRKS)
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…
- Conditions
- Hyperoxaluria, Cystinuria, Dent Disease, Lowe Syndrome
- Trial name
- ProRKS (NCT02780297)
- Where
- 11 sites in United States, Canada and 2 more
- Volunteers
- 220 people
- Sponsor
- Mayo Clinic
Primary Hyperoxaluria
Study to Evaluate the Efficacy and Safety of OxabactTM on Reduction of Urinary Oxalate in Primary Hyperoxaluria Patients
The main purpose of this study is to determine if Oxalobacter formigenes is effective at lowering urinary oxalate levels in patients with primary hyperoxaluria.
- Trial name
- PHOENIX (NCT00638703)
- Testing
- Oxalobacter formigenes
- Where
- United States
- Volunteers
- 43 people
- Sponsor
- OxThera
Primary Hyperoxaluria Type I
Trial on Treatment of Patients With Primary Hyperoxaluria Type I With Pyridoxal-phosphate
In this study the investigators will prospectively analyze the reduction of urinary oxalate excretion under the treatment with PLP in dosages of 5mg/kg/day up to 20 mg/kg/day and serum level response relationship with PLP as an…
- Trial name
- PHOX-B6-Pilot (NCT01281878)
- Testing
- Vitamin B 6
- Where
- Germany
- Volunteers
- 12 people
- Sponsor
- University of Cologne
Rare Kidney Stone Diseases
Monogenic Kidney Stone - Genetic Testing
This study will attempt to identify the specific gene (coded in the DNA) and changes (mutations) within that gene that are the cause of monogenic kidney stone disease.
- Trial name
- NCT03305835
- Where
- United States
- Volunteers
- 6,000 people
- Sponsor
- Mayo Clinic