Clinical trials: PH1
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
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
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 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
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, Primary Hyperoxaluria Type 1 (PH1)
A Study of Lumasiran in Infants and Young Children With Primary Hyperoxaluria Type 1
The purpose of this study is to evaluate the efficacy, safety, pharmacokinetics (PK), and pharmacodynamics (PD) of lumasiran in infants and young children with confirmed primary hyperoxaluria type 1 (PH1).
- Trial name
- ILLUMINATE-B (NCT03905694)
- Testing
- Lumasiran
- Where
- 9 sites in United States, France and 3 more
- Volunteers
- 18 people
- Sponsor
- Alnylam Pharmaceuticals
CML (Chronic Myelogenous Leukemia
IIT PH1 KDS-1001 in Patients With CML
This open label, non-randomized, prospective phase I study is designed to evaluate if the addition of natural killer cell therapy (KDS-1001) to tyrosine kinase inhibitors (TKIs) for chronic myeloid leukemia (CML) patients with…
- Trial name
- NCT04808115
- Testing
- KDS-1001
- Sponsor
- Duke University
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, and dose-expansion trial, aiming to assess the safety and tolerability of YOLT-203 in Chinese patients with Primary Hyperoxaluria Type 1 (PH1), and to…
- Trial name
- NCT06511349
- Testing
- YOLT-203
- Where
- China
- Volunteers
- 21 people
- Sponsor
- RenJi Hospital
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, Primary Hyperoxaluria
A Study to Evaluate Lumasiran in Patients With Advanced Primary Hyperoxaluria Type 1
The purpose of this study is to evaluate the efficacy, safety, pharmacokinetics (PK), and pharmacodynamics (PD) of lumasiran in patients with Advanced Primary Hyperoxaluria Type 1 (PH1).
- Trial name
- ILLUMINATE-C (NCT04152200)
- Testing
- Lumasiran
- Where
- 14 sites in United States, Australia and 9 more
- Volunteers
- 21 people
- Sponsor
- Alnylam Pharmaceuticals
Childhood Acute Lymphoblastic Leukemia, Philadelphia Chromosome, Ph^1^, Absent
Treatment Protocol of Children With Philadelphia Chromosome Negative High Risk Acute Lymphoblastic Leukemia
The cure rate for childhood acute lymphoblastic leukemia (ALL) has increased significantly in recent decades and expected cure rates now exceed 85%.
- Conditions
- Childhood Acute Lymphoblastic Leukemia, Philadelphia Chromosome, Ph^1^, Absent, B-cell Childhood Acute Lymphoblastic Leukemia
- Trial name
- NCT01990807
- Testing
- Idarubicin(IDA)
- Where
- China
- Volunteers
- 20 people
- Sponsor
- Chinese Academy of Medical Sciences
Primary Hyperoxaluria Type 1
Proteomics of Primary Hyperoxaluria Type 1
The purpose of this study is to identify unique urine protein markers of Primary Hyperoxaluria type 1 (PH1) compared to healthy controls.
- Trial name
- PH1 (NCT03067142)
- Testing
- Observational
- Volunteers
- 93 people, healthy volunteers welcome
- Sponsor
- Ann & Robert H Lurie Children's Hospital of Chicago
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
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
BONAPH1DE, A Prospective Observational Study of Patients With Primary Hyperoxaluria Type 1 (PH1)
The purpose of this study is to describe the natural history and progression of patients diagnosed with PH1, and to characterize the long-term real-world safety and effectiveness of lumasiran.
- Trial name
- BONAPH1DE (NCT04982393)
- Where
- 30 sites in United States, Belgium and 9 more
- Volunteers
- 207 people
- Sponsor
- Alnylam Pharmaceuticals
Retinal Dystrophy Due to Biallelic ABCA4 Mutations, Stargardt Disease 1
Safety and Preliminary Efficacy of VG801 in Patients With ABCA4 Mutation-associated Retinal Dystrophy (Stargardt Disease)
This is a single-arm, open-label, non-randomized, single dose-escalation, first-in-human (FIH) clinical trial to evaluate the safety and preliminary efficacy of VG801 for treatment of patients with retinal dystrophy (Stargardt…
- Trial name
- NCT07002398
- Testing
- VG801
- Where
- China
- Volunteers
- 18 people
- Sponsor
- VeonGen Therapeutics GmbH
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
Study of YOLT-203 in Children and Adults With Primary Hyperoxaluria Type 1 (PH1)
This study will be conducted to evaluate the efficacy and safety of YOLT-203 in children and adults with Primary Hyperoxaluria Type 1.
- Trial name
- NCT07587021
- Testing
- YOLT-203
- Where
- 4 sites in United States
- Volunteers
- 36 people
- Sponsor
- YolTech Therapeutics Co., Ltd
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
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