Clinical trials: Primary Hyperoxaluria Type 1 (PH1)
Primary Hyperoxaluria Type 1, Primary Hyperoxaluria Type 2
Evaluation of the Efficacy and Safety of Stiripentol in Patients 6 Years and Older With Primary Hyperoxaluria Type 1, 2 or 3
Evaluation of the efficacy and safety of stiripentol in patients 6 years and older with primary hyperoxaluria type 1, 2 or 3.
- Conditions
- Primary Hyperoxaluria Type 1, Primary Hyperoxaluria Type 2, Primary Hyperoxaluria Type 3
- Trial name
- CRYSTAL (NCT06465472)
- Testing
- Stiripentol Oral Capsule, Urine samples collect, Blood samples collect
- Volunteers
- 42 people
- Sponsor
- Biocodex
Primary Hyperoxaluria Type 1, Idiopathic Hypercalciuria
IDENTIFICATION OF A MULTI-ANALYTE PROFILE FOR PRIMARY HYPEROXALURIA AND COMPARISON WITH HEALTHY SIBLINGS AND IDIOPATHIC HYPERCALCIURIA
The aim of this study is to know the difference between protein profiles (multi-analyte profile) of PH1 patients, idiopathic hypercalciuria (IH) patients and PH1 patients 'siblings.
- Trial name
- PH1 (NCT02830009)
- Testing
- Urines samples, Blood sample
- Where
- France
- Volunteers
- 5 people, healthy volunteers welcome
- Sponsor
- Hospices Civils de Lyon
Primary Hyperoxaluria Type 1, End Stage Renal Disease
Safety & Efficacy of DCR-PHXC in Patients With PH1 and ESRD
The aim of this study is to evaluate DCR-PHXC in participants with PH1 and severe renal impairment, with or without dialysis.
- Trial name
- PHYOX7 (NCT04580420)
- Testing
- DCR-PHXC
- Where
- 18 sites in United States, France and 8 more
- Volunteers
- 28 people
- Sponsor
- Dicerna Pharmaceuticals, Inc., a Novo Nordisk company
Hyperoxaluria, Primary
Follow-up of Pediatric Patients Affected by Primary Hyperoxaluria Type 1 (PH1) Treated With Lumasiran
The primary objective of the study is to confirm, in pediatric patients with type 1 hyperoxaluria treated with Lumasiran, the reduction in urinary oxalate excretion and plasma oxalate levels-already described at six months-on a…
- Trial name
- LUMA-KIDS (NCT07874074)
- Where
- Italy
- Volunteers
- 30 people
- Sponsor
- Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico
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, Primary Hyperoxaluria Type 1
Nedosiran in Pediatric Patients From Birth to 11 Years of Age With PH and Relatively Intact Renal Function
The aim of this study is to evaluate nedosiran in participants 11 years of age and younger who have Primary Hyperoxaluria with relatively intact renal function.
- Conditions
- Primary Hyperoxaluria, Primary Hyperoxaluria Type 1, Primary Hyperoxaluria Type 2, Primary Hyperoxaluria Type 3
- Trial name
- PHYOX8 (NCT05001269)
- Testing
- nedosiran
- Where
- 13 sites in United States, Canada and 9 more
- Volunteers
- 27 people
- Sponsor
- Dicerna Pharmaceuticals, Inc., a Novo Nordisk company
Primary Hyperoxaluria Type 1 (PH1)
A Study to Evaluate Lumasiran in Children and Adults With Primary Hyperoxaluria Type 1
The purpose of this study is to evaluate the efficacy and safety of lumasiran in children and adults with primary hyperoxaluria type 1 (PH1).
- Trial name
- ILLUMINATE-A (NCT03681184)
- Testing
- Lumasiran
- Where
- 17 sites in United States, France and 6 more
- Volunteers
- 39 people
- Sponsor
- Alnylam Pharmaceuticals
Primary Hyperoxaluria Type 1, PH1
Phase I Exploratory Study of YOLT-203 in Patients With Primary Hyperoxaluria Type 1 (PH1)
This is a single-arm, open-label, single-dose dose-escalation study. It aims to evaluate the safety and tolerability of YOLT-203 in Chinese patients with Primary Hyperoxaluria Type 1 (PH1), and to preliminarily assess the effect…
- Trial name
- NCT07776626
- Testing
- YOLT-203
- Where
- China
- Volunteers
- 9 people
- Sponsor
- YolTech Therapeutics Co., Ltd
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 Type 1 (PH1)
Study of Lumasiran in Healthy Adults and Patients With Primary Hyperoxaluria Type 1
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of single-ascending doses (SAD) and multiple-ascending doses (MAD) of lumasiran in healthy adult volunteers and subjects…
- Trial name
- NCT02706886
- Testing
- Lumasiran
- Where
- 9 sites in France, Germany and 3 more
- Volunteers
- 52 people, healthy volunteers welcome
- Sponsor
- Alnylam Pharmaceuticals
Primary Hyperoxaluria, Dent Disease
Rare Kidney Stone Consortium Patient Registry
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.
- Conditions
- Primary Hyperoxaluria, Dent Disease, Cystinuria, APRT Deficiency
- Trial name
- RKSC (NCT00588562)
- Where
- 4 sites in United States, Iceland
- Volunteers
- 730 people
- Sponsor
- Mayo Clinic
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
PH1, Primary Hyperoxaluria
An Extension Study of an Investigational Drug, Lumasiran (ALN-GO1), in Participants With Primary Hyperoxaluria Type 1
The purpose of this study is to evaluate the long-term safety and tolerability of lumasiran in participants with Primary Hyperoxaluria Type 1.
- Conditions
- PH1, Primary Hyperoxaluria, RNAi Therapeutic, siRNA
- Trial name
- NCT03350451
- Testing
- Lumasiran
- Where
- 9 sites in France, Germany and 3 more
- Volunteers
- 20 people
- Sponsor
- Alnylam Pharmaceuticals
Primary Hyperoxaluria Type 1 (PH1)
Phase 1/2 Study of ABO-101 in Primary Hyperoxaluria Type 1 (redePHine)
The goal of the redePHine study is to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of ABO-101 in participants with primary hyperoxaluria type 1 (PH1). The trial will consist of 2 Study Periods.
- Trial name
- NCT06839235
- Testing
- ABO-101
- Where
- 7 sites in United States, France and 3 more
- Volunteers
- 23 people
- Sponsor
- Arbor Biotechnologies
Primary Hyperoxaluria Type 1 (PH1), Primary Hyperoxaluria Type 2 (PH2)
Long Term Extension Study in Patients With Primary Hyperoxaluria
The proposed study is designed to provide patients previously enrolled in Phase 1 and 2 studies of DCR-PHXC and their siblings (<18 years old) long-term access to DCR-PHXC, and to evaluate the long-term safety and efficacy of…
- Conditions
- Primary Hyperoxaluria Type 1 (PH1), Primary Hyperoxaluria Type 2 (PH2), Kidney Diseases, Urologic Diseases
- Trial name
- PHYOX3 (NCT04042402)
- Testing
- DCR-PHXC
- Where
- 23 sites in United States, Australia and 11 more
- Volunteers
- 75 people
- Sponsor
- Dicerna Pharmaceuticals, Inc., a Novo Nordisk company
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
Primary Hyperoxaluria, Dent Disease
Rare Kidney Stone Consortium Biobank
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.
- Conditions
- Primary Hyperoxaluria, Dent Disease, APRT Deficiency, Cystinuria
- Trial name
- NCT02026388
- Where
- United States
- Volunteers
- 2,000 people
- Sponsor
- Mayo Clinic
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
Spinal Muscular Atrophy, Fragile X Syndrome
Early Check: Expanded Screening in Newborns
Early Check provides voluntary screening of newborns for a selected panel of conditions. The study has three main objectives: 1) develop and implement an approach to identify affected infants, 2) address the impact on infants and…
- Conditions
- Spinal Muscular Atrophy, Fragile X Syndrome, Fragile X - Premutation, Duchenne Muscular Dystrophy
- Trial name
- NCT03655223
- Testing
- Confirmatory Testing
- Where
- United States
- Volunteers
- 30,000 people, healthy volunteers welcome
- Sponsor
- RTI International
Primary Hyperoxaluria Type 1 (PH1), Primary Hyperoxaluria Type 2 (PH2)
A Study to Evaluate DCR-PHXC in Children and Adults With Primary Hyperoxaluria Type 1 and Primary Hyperoxaluria Type 2
The purpose of this study is to evaluate the efficacy and safety of DCR-PHXC in Children and Adults with Primary Hyperoxaluria Type 1 (PH1) and Primary Hyperoxaluria Type 2 (PH2)
- Conditions
- Primary Hyperoxaluria Type 1 (PH1), Primary Hyperoxaluria Type 2 (PH2), Kidney Diseases, Urologic Diseases
- Trial name
- PHYOX2 (NCT03847909)
- Testing
- DCR-PHXC, Sterile Normal Saline (0.9% NaCl)
- Where
- 28 sites in United States, Australia and 13 more
- Volunteers
- 35 people
- Sponsor
- Novo Nordisk A/S