42 completed, 10 recruiting, 5 unknown, 4 terminated. 21 Phase 2, 13 Phase 1, 13 Phase 3, 7 Not applicable, 2 Phase 4.
Follow Hyperoxaluria: new trials by email Literature Review of Hyperoxaluria trials Research Report on Hyperoxaluria
Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.
| Trial | Phase | Status | Sponsor | Enrollment | Start | Updated |
|---|---|---|---|---|---|---|
| Study of YOLT-203 in Children and Adults With Primary Hyperoxaluria Type 1 (PH1) YOLT-203, Placebo - Primary Hyperoxaluria Type 1, PH1 | Phase 2 | Recruiting | YolTech Therapeutics Co., Ltd | 36 | 2026-09-16 | 2026-10-09 |
| Validation Testing for Plasma Oxalate Levels in the Biochemical Laboratory at the Galilee Medical Center, in Collaboration With the Biochemistry Laboratory at CHARITE Hospital in Berlin, and Testing the Relationship Between Oxalate Levels and Vitamin C Levels in Plasma This is an non-interventional study - Hyperoxaluria | Not yet recruiting | Western Galilee Hospital-Nahariya | 50 | 2026-08-01 | 2026-05-07 | |
| Phase I Exploratory Study of YOLT-203 in Patients With Primary Hyperoxaluria Type 1 (PH1) YOLT-203 - Primary Hyperoxaluria Type 1, PH1 | Phase 1 | Recruiting | YolTech Therapeutics Co., Ltd | 9 | 2026-08-01 | 2026-08-20 |
| Phase 1/2 Study of ABO-101 in Primary Hyperoxaluria Type 1 (redePHine) ABO-101 - Primary Hyperoxaluria Type 1 (PH1) | Phase 1 / Phase 2 | Recruiting | Arbor Biotechnologies | 23 | 2025-06-16 | 2026-10-19 |
| Oxalate Excretion Profile in Patients With a Heterozygous Mutation of the AGXT (Alanine-glyoxylate Aminotransferase) Gene Lithiasis assessment - Hyperoxaluria (Disorder) | Not applicable | Completed | Hospices Civils de Lyon | 25 | 2024-12-11 | 2026-05-06 |
| Evaluation of the Efficacy and Safety of Stiripentol in Patients 6 Years and Older With Primary Hyperoxaluria Type 1, 2 or 3 Stiripentol Oral Capsule, Placebo Oral Capsule, Urine samples collect - Primary Hyperoxaluria Type 1, Primary Hyperoxaluria Type 2 | Phase 3 | Not yet recruiting | Biocodex | 42 | 2024-08-01 | 2024-06-18 |
| A Novel Approach for Reducing Hyperoxaluria and Kidney Stone Risk. Tenapanor, Placebo - Hyperoxaluria | Phase 4 | Completed | University of Texas Southwestern Medical Center | 18 | 2024-07-01 | 2026-10-07 |
| Gut Oxalate Absorption in Calcium Oxalate Stone Disease Low-oxalate diet, High-oxalate diet, soluble oxalate gut absorption test - Kidney Stone, Kidney Calculi | Not applicable | Recruiting | University of Alabama at Birmingham | 80 | 2024-04-17 | 2026-10-13 |
| O. Formigenes Colonization in Calcium Oxalate Kidney Stone Disease Low oxalate fixed diets pre-colonization, Moderately high oxalate fixed diets pre-colonization, Colonization with Oxalobacter formigenes - Kidney Stone, Kidney Calculi | Not applicable | Recruiting | University of Alabama at Birmingham | 40 | 2024-04-17 | 2026-10-13 |
| Dapagliflozin and Hydrochlorothiazide in Recurring Kidney Stone Patients Dapagliflozin, Hydrochlorothiazide - Urolithiasis, Hyperoxaluria | Phase 4 | Unknown | Medical University of Vienna | 32 | 2024-03-04 | 2023-10-23 |
| A Study of BMN 255 in Participants With Non-Alcoholic Fatty Liver Disease And Hyperoxaluria BMN 255, Placebo - Hyperoxaluria, Nonalcoholic Fatty Liver Disease | Phase 1 | Withdrawn | BioMarin Pharmaceutical | 2023-09-26 | 2024-06-11 | |
| Baby Detect : Genomic Newborn Screening Congenital Adrenal Hyperplasia, Familial Hyperinsulinemic Hypoglycemia 1 | Completed | Centre Hospitalier Universitaire de Liege | 6824 | 2022-09-01 | 2025-08-12 | |
| Follow-up of Pediatric Patients Affected by Primary Hyperoxaluria Type 1 (PH1) Treated With Lumasiran Hyperoxaluria, Primary | Enrolling by invitation | Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico | 30 | 2022-07-06 | 2026-10-14 | |
| Genetic Newborn Screening for Cystinosis and Primary Hyperoxaluria Diagnostic test - Cystinosis, Primary Hyperoxaluria | Not applicable | Unknown | Cystinose Stiftung | 200000 | 2022-03-15 | 2023-05-06 |
| Nedosiran in Pediatric Patients From Birth to 11 Years of Age With PH and Relatively Intact Renal Function nedosiran - Primary Hyperoxaluria, Primary Hyperoxaluria Type 1 | Phase 2 | Completed | Dicerna Pharmaceuticals, Inc., a Novo Nordisk company | 27 | 2022-02-22 | 2026-04-16 |
| Natural History of Patients With PH3 and a History of Stone Events Primary Hyperoxaluria Type 3 | Completed | Dicerna Pharmaceuticals, Inc., a Novo Nordisk company | 7 | 2021-09-09 | 2024-12-02 | |
| Safety, Tolerability, and Pharmacodynamics of NOV-001 in Adult Subjects NOV-001, NB1000S, NB2000P - Healthy Volunteers, Enteric Hyperoxaluria | Phase 1 / Phase 2 | Terminated | Novome Biotechnologies Inc | 153 | 2021-06-02 | 2023-05-17 |
| Phenotyping of Primary Hyperoxaluria Primary Hyperoxaluria | Unknown | University Hospital, Strasbourg, France | 186 | 2021-01-22 | 2021-11-04 | |
| Study to Evaluate Safety, Tolerability, PK and PD of DCR-PHXC in PH Type 3 Patients DCR-PHXC, Sterile Normal Saline (0.9% NaCl) - Primary Hyperoxaluria Type 3 | Phase 1 | Completed | Dicerna Pharmaceuticals, Inc., a Novo Nordisk company | 6 | 2020-09-14 | 2024-09-19 |
| Plasma Oxalate in Patient With Short Bowel Blood sampling, Urine sampling, Pulse Wave Velocity (PWV) measurement - Short Bowel Syndrome, Hyperoxaluria | Completed | Hospices Civils de Lyon | 47 | 2020-02-27 | 2022-07-21 |
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Hyperoxaluria, Primary (42) · Kidney Calculi (17) · Primary hyperoxaluria type 1 (13) · Kidney Diseases (9) · Nephrolithiasis (9) · Urologic Diseases (9) · Urolithiasis (7) · Adenine phosphoribosyltransferase deficiency (6) · Cystinuria (6) · Dent Disease (6) · Genetic Diseases, Inborn (5) · Primary hyperoxaluria type 2 (5) · Cystinosis (3) · Metabolic Diseases (3)
Female Urogenital Diseases (69) · Female Urogenital Diseases and Pregnancy Complications (69) · Male Urogenital Diseases (69) · Urogenital Diseases (69) · Kidney Diseases (60) · Urologic Diseases (60) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (43) · Nutritional and Metabolic Diseases (43) · Carbohydrate Metabolism, Inborn Errors (42) · Metabolic Diseases (40) · Metabolism, Inborn Errors (40) · Genetic Diseases, Inborn (38) · Pathological Conditions, Signs and Symptoms (23) · Pathological Conditions, Anatomical (18)
oxalate decarboxylase (7) · lumasiran (6) · reloxaliase (6) · nedosiran (4) · Genetic Testing (2) · stiripentol (2) · Angiotensin Receptor Antagonists (1) · Angiotensin-Converting Enzyme Inhibitors (1) · Betaine (1) · Blood Specimen Collection (1) · Curcumin (1) · Diagnostic Tests, Routine (1) · Hydrochlorothiazide (1) · Hydrogen-Ion Concentration (1) · Hydroxyproline (1)
Mayo Clinic (13) · Allena Pharmaceuticals (7) · Alnylam Pharmaceuticals (6) · Dicerna Pharmaceuticals, Inc., a Novo Nordisk company (6) · OxThera (6) · Aga Khan University (2) · Biocodex (2) · Hospices Civils de Lyon (2) · London Health Sciences Centre Research Institute OR Lawson Research Institute of St. Joseph's (2) · University of Alabama at Birmingham (2) · VA New York Harbor Healthcare System (2) · YolTech Therapeutics Co., Ltd (2) · Arbor Biotechnologies (1) · BioMarin Pharmaceutical (1) · Centre Hospitalier Universitaire de Liege (1)
United States (42) · Germany (22) · United Kingdom (18) · France (17) · Netherlands (10) · Canada (9) · Israel (7) · Italy (7) · Spain (7) · Belgium (6) · Lebanon (4) · Australia (3) · Iceland (3) · Japan (3) · Poland (3)
Annamaria Kausz, MD MS (3) · John C Lieske, MD (3) · David Clark, MD (2) · David Goldfarb, MD (2) · Dawn Milliner, MD (2) · Gesa Schalk, MD (2) · John C Lieske, M.D. (2) · John Lieske, MD (2) · Sonia Fargue, PhD (2) · Alexandra Haagensen, MD, MBA (1) · Annamaria Kausz, MD, MS (1) · Bernd Hoppe, MD PhD (1) · Carla G Monico, M.D. (1) · Christian Tielemans, MD, PhD (1)
PMID 40601241, PMID 40193200, PMID 38332115, PMID 15961949, PMID 20736987, PMID 23944302, PMID 24385516, PMID 30783888, PMID 33789010, PMID 33985991, PMID 34906487, PMID 35843439, PMID 35913563, PMID 36007597, PMID 38319254, PMID 39355649, PMID 39875734, PMID 41343248, PMID 41388232, PMID 8411612
U54DK083908 (5), 2018 (3), 2023 (2), R01DK073354 (2), R01DK137784 (2), 1UL1TR003163 (1), 2015 (1), 2016 (1), 2017 (1), 2019 (1), P50DK083007 (1), R21AT002534 (1), UL1RR024150 (1)