Clinical trials: Primary Hyperoxaluria
- Condition Primary Hyperoxaluria
- Funder type Industry (pharma, biotech, devices)
- Age group Children (under 18)
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
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
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
Primary Hyperoxaluria
Evaluation of the Efficacy of Stiripentol (Diacomit) as Monotherapy for the Treatment of Primary Hyperoxaluria
Evaluation of the efficacy of stiripentol (Diacomit) as monotherapy for the treatment of primary hyperoxaluria. Pilot clinical study, open, prospective and multicenter.
- Trial name
- NCT03819647
- Testing
- stiripentol (Diacomit)
- Where
- 3 sites in France
- Volunteers
- 15 people
- Sponsor
- Biocodex
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 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
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
A Study to Evaluate the Efficacy and Safety of Oxabact in Patients With Primary Hyperoxaluria
This study will evaluate the efficacy and safety of OC5 in patients with PH.
- Trial name
- NCT03116685
- Testing
- Oxabact OC5 - Oxalobacter formigenes HC-1
- Where
- 10 sites in United States, Belgium and 5 more
- Volunteers
- 25 people
- Sponsor
- OxThera
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
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
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
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 3
Natural History of Patients With PH3 and a History of Stone Events
This is a natural history study of adults, adolescents, and children (starting at birth) with genetically confirmed primary hyperoxaluria type 3 (PH3) who have a history of stone events during the last 3 years and/or the presence…
- Trial name
- PHYOX-OBX (NCT04542590)
- Where
- 6 sites in United States, Canada and 3 more
- Volunteers
- 7 people
- Sponsor
- Dicerna Pharmaceuticals, Inc., a Novo Nordisk company
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 (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)
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