8 completed, 7 recruiting, 4 active not recruiting, 1 not yet recruiting. 6 Phase 2, 5 Phase 3, 4 Phase 1, 2 Early Phase 1, 1 Not applicable.
Follow Primary hyperoxaluria type 1: new trials by email Literature Review of Primary hyperoxaluria type 1 trials Research Report on Primary hyperoxaluria type 1
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 |
| 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 |
| Clinical Exploration Study of YOLT-203 in the Treatment of Type 1 Primary Hyperoxaluria (PH1) YOLT-203 - Type 1 Primary Hyperoxaluria | Early Phase 1 | Active not recruiting | Guangzhou Women and Children's Medical Center | 2 | 2025-02-27 | 2025-03-24 |
| 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 |
| Clinical Exploration Study of YOLT-203 in the Treatment of Type 1 Primary Hyperoxaluria (PH1) YOLT-203 - Type 1 Primary Hyperoxaluria | Early Phase 1 | Recruiting | RenJi Hospital | 21 | 2024-07-15 | 2025-05-16 |
| Retrospective and Prospective Follow-up of Patients With Primary Hyperoxaluria Type 1 Treated With Lumasiran in France. Oxaluria evolution. - Patients With PH1 Treated With Lumasiran in France | Recruiting | Hospices Civils de Lyon | 100 | 2023-01-01 | 2024-01-26 | |
| 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 |
| BONAPH1DE, A Prospective Observational Study of Patients With Primary Hyperoxaluria Type 1 (PH1) Primary Hyperoxaluria Type 1 | Active not recruiting | Alnylam Pharmaceuticals | 207 | 2021-12-13 | 2026-10-06 | |
| Safety & Efficacy of DCR-PHXC in Patients With PH1 and ESRD DCR-PHXC - Primary Hyperoxaluria Type 1, End Stage Renal Disease | Phase 2 | Recruiting | Dicerna Pharmaceuticals, Inc., a Novo Nordisk company | 28 | 2021-04-15 | 2025-12-24 |
| A Study to Evaluate Lumasiran in Patients With Advanced Primary Hyperoxaluria Type 1 Lumasiran - Primary Hyperoxaluria Type 1, Primary Hyperoxaluria | Phase 3 | Completed | Alnylam Pharmaceuticals | 21 | 2020-01-21 | 2025-07-18 |
| A Study to Evaluate DCR-PHXC in Children and Adults With Primary Hyperoxaluria Type 1 and Primary Hyperoxaluria Type 2 DCR-PHXC, Sterile Normal Saline (0.9% NaCl) - Primary Hyperoxaluria Type 1 (PH1), Primary Hyperoxaluria Type 2 (PH2) | Phase 2 | Completed | Novo Nordisk A/S | 35 | 2019-10-28 | 2024-05-22 |
| Long Term Extension Study in Patients With Primary Hyperoxaluria DCR-PHXC - Primary Hyperoxaluria Type 1 (PH1), Primary Hyperoxaluria Type 2 (PH2) | Phase 3 | Active not recruiting | Dicerna Pharmaceuticals, Inc., a Novo Nordisk company | 75 | 2019-07-09 | 2026-10-08 |
| A Study of Lumasiran in Infants and Young Children With Primary Hyperoxaluria Type 1 Lumasiran - Primary Hyperoxaluria, Primary Hyperoxaluria Type 1 (PH1) | Phase 3 | Completed | Alnylam Pharmaceuticals | 18 | 2019-04-22 | 2025-02-14 |
| A Study to Evaluate Lumasiran in Children and Adults With Primary Hyperoxaluria Type 1 Placebo, Lumasiran - Primary Hyperoxaluria Type 1 (PH1) | Phase 3 | Completed | Alnylam Pharmaceuticals | 39 | 2018-11-27 | 2024-08-12 |
| Early Check: Expanded Screening in Newborns Confirmatory Testing - Spinal Muscular Atrophy, Fragile X Syndrome | Active not recruiting | RTI International | 30000 | 2018-10-15 | 2026-07-01 | |
| Proteomics of Primary Hyperoxaluria Type 1 Observational - Primary Hyperoxaluria Type 1 | Completed | Ann & Robert H Lurie Children's Hospital of Chicago | 93 | 2017-01-05 | 2019-06-12 | |
| A Study of DCR-PH1 in Patients With Primary Hyperoxaluria Type 1 (PH1) DCR-PH1, Placebo - Primary Hyperoxaluria Type 1 | Phase 1 | Terminated | Dicerna Pharmaceuticals, Inc., a Novo Nordisk company | 41 | 2016-05-13 | 2024-07-11 |
| Study of Lumasiran in Healthy Adults and Patients With Primary Hyperoxaluria Type 1 Lumasiran, Placebo - Primary Hyperoxaluria Type 1 (PH1) | Phase 1 / Phase 2 | Completed | Alnylam Pharmaceuticals | 52 | 2016-03-08 | 2020-01-30 |
| IDENTIFICATION OF A MULTI-ANALYTE PROFILE FOR PRIMARY HYPEROXALURIA AND COMPARISON WITH HEALTHY SIBLINGS AND IDIOPATHIC HYPERCALCIURIA Urines samples, Blood sample - Primary Hyperoxaluria Type 1, Idiopathic Hypercalciuria | Not applicable | Completed | Hospices Civils de Lyon | 5 | 2013-05-01 | 2016-07-13 |
Showing 20 of 22. Sign in to see all of them.
Hyperoxaluria, Primary (12) · Hyperoxaluria (6) · Primary hyperoxaluria type 2 (6) · Genetic Diseases, Inborn (3) · Kidney Calculi (3) · Urologic Diseases (3) · Adenine phosphoribosyltransferase deficiency (2) · Kidney Diseases (2) · 3-Hydroxy-3-Methylglutaryl-CoA Lyase Deficiency (1) · 3-Hydroxyacyl-CoA Dehydrogenase Deficiency (1) · 3-methylcrotonyl CoA carboxylase 1 deficiency (1) · 3-methylcrotonyl CoA carboxylase 2 deficiency (1) · Acidemia, isovaleric (1) · Acrodermatitis enteropathica (1)
Female Urogenital Diseases (15) · Female Urogenital Diseases and Pregnancy Complications (15) · Male Urogenital Diseases (15) · Urogenital Diseases (15) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (14) · Nutritional and Metabolic Diseases (14) · Carbohydrate Metabolism, Inborn Errors (13) · Kidney Diseases (13) · Metabolic Diseases (13) · Metabolism, Inborn Errors (13) · Urologic Diseases (12) · Genetic Diseases, Inborn (11) · Hyperoxaluria (7) · Pathological Conditions, Signs and Symptoms (5) · Calculi (3)
nedosiran (5) · lumasiran (4) · Blood Specimen Collection (1) · Watchful Waiting (1) · stiripentol (1)
Alnylam Pharmaceuticals (5) · Dicerna Pharmaceuticals, Inc., a Novo Nordisk company (5) · Hospices Civils de Lyon (2) · YolTech Therapeutics Co., Ltd (2) · Ann & Robert H Lurie Children's Hospital of Chicago (1) · Arbor Biotechnologies (1) · Biocodex (1) · Guangzhou Women and Children's Medical Center (1) · Mayo Clinic (1) · Novo Nordisk A/S (1) · RTI International (1) · RenJi Hospital (1)
United States (12) · France (11) · Germany (10) · United Kingdom (9) · Netherlands (7) · Israel (6) · Italy (6) · Lebanon (5) · Spain (5) · Canada (4) · United Arab Emirates (4) · Australia (3) · China (3) · Japan (3) · Turkey (Türkiye) (3)
Craig B Langman, MD (1) · Curt Scharfe, MD, PhD (1) · Daniel Ory, MD (1) · John C Lieske, M.D. (1) · Sarb Shergill, PhD (1) · Tracy McGregor, MD, MSCI (1) · Verity Rawson, MB.CHB (1) · Wenhao Zhou, PhD, MD (1) · Wensu Yu (1)
PMID 40193200, PMID 40601241, PMID 38332115, PMID 31315600, PMID 33789010, PMID 33985991, PMID 34906487, PMID 35142618, PMID 35843439, PMID 35913563, PMID 36007597, PMID 39355649, PMID 39875734, PMID 41343248, PMID 41388232
2018 (2), 2023 (2), 2015 (1), 2019 (1), U54DK083908 (1)