24 completed, 9 recruiting, 3 unknown, 2 withdrawn. 15 Phase 2, 10 Phase 3, 7 Phase 1, 1 Not applicable.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 15 | Completed 13, Recruiting 2 |
| Phase 3 | 10 | Completed 6, Active not recruiting 1, Recruiting 1, Terminated 1, Withdrawn 1 |
| Phase 1 | 7 | Completed 5, Recruiting 2 |
| Not applicable | 1 | Unknown 1 |
Follow Hyperoxaluria, Primary: new trials by email Literature Review of Hyperoxaluria, Primary trials Research Report on Hyperoxaluria, Primary
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 |
|---|---|---|---|---|---|---|
| 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 | Recruiting | Biocodex | 42 | 2026-09-23 | 2026-11-12 |
| 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 |
| 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 | |
| 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 |
| 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 |
| Evaluation of the Efficacy of Stiripentol (Diacomit) as Monotherapy for the Treatment of Primary Hyperoxaluria stiripentol (Diacomit) - Primary Hyperoxaluria | Phase 2 | Completed | Biocodex | 15 | 2019-05-21 | 2021-03-26 |
| 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 |
| An Extension Study to Evaluate the Long-term Efficacy and Safety of Oxabact in Patients With Primary Hyperoxaluria Oxabact OC5 - Oxalobacter formigenes Strain HC-1 - Hyperoxaluria, Primary | Phase 3 | Terminated | OxThera | 22 | 2019-03-14 | 2021-12-08 |
| 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 |
| Study of ALLN-177 in Patients Aged 12 Years or Older With Enteric or Primary Hyperoxaluria and Hyperoxalemia ALLN-177 - Enteric Hyperoxaluria, Primary Hyperoxaluria | Phase 2 | Completed | Allena Pharmaceuticals | 15 | 2018-07-17 | 2020-12-30 |
| An Extension Study of an Investigational Drug, Lumasiran (ALN-GO1), in Participants With Primary Hyperoxaluria Type 1 Lumasiran - PH1, Primary Hyperoxaluria | Phase 2 | Completed | Alnylam Pharmaceuticals | 20 | 2018-04-04 | 2024-04-25 |
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Hyperoxaluria (21) · Primary hyperoxaluria type 1 (12) · Adenine phosphoribosyltransferase deficiency (6) · Cystinuria (6) · Dent Disease (6) · Kidney Calculi (5) · Primary hyperoxaluria type 2 (5) · Genetic Diseases, Inborn (4) · Kidney Diseases (4) · Urologic Diseases (4) · Cystinosis (3) · Nephrocalcinosis (3) · Diabetes Insipidus, Nephrogenic (2) · Metabolic Diseases (2)
Carbohydrate Metabolism, Inborn Errors (42) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (42) · Female Urogenital Diseases (42) · Female Urogenital Diseases and Pregnancy Complications (42) · Male Urogenital Diseases (42) · Nutritional and Metabolic Diseases (42) · Urogenital Diseases (42) · Metabolic Diseases (40) · Metabolism, Inborn Errors (40) · Genetic Diseases, Inborn (38) · Kidney Diseases (38) · Urologic Diseases (38) · Hyperoxaluria (21) · Genetic Diseases, X-Linked (7) · Pathological Conditions, Signs and Symptoms (7)
lumasiran (6) · nedosiran (4) · Genetic Testing (2) · stiripentol (2) · Angiotensin Receptor Antagonists (1) · Angiotensin-Converting Enzyme Inhibitors (1) · Betaine (1) · Diagnostic Tests, Routine (1) · Hydrogen-Ion Concentration (1) · Hydroxyproline (1) · Lactose (1) · Leucine (1) · Lisinopril (1) · Losartan (1) · Quality of Life (1)
Mayo Clinic (10) · Alnylam Pharmaceuticals (6) · OxThera (6) · Dicerna Pharmaceuticals, Inc., a Novo Nordisk company (5) · Biocodex (2) · YolTech Therapeutics Co., Ltd (2) · Allena Pharmaceuticals (1) · Arbor Biotechnologies (1) · Centre Hospitalier Universitaire de Liege (1) · Cystinose Stiftung (1) · Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico (1) · NYU Langone Health (1) · Novo Nordisk A/S (1) · UK Kidney Association (1) · University Hospital, Strasbourg, France (1)
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David Goldfarb, MD (2) · Dawn Milliner, MD (2) · Gesa Schalk, MD (2) · John C Lieske, M.D. (2) · John C Lieske, MD (2) · Alexandra Haagensen, MD, MBA (1) · Bernd Hoppe, MD PhD (1) · Carla G Monico, M.D. (1) · Daniel Ory, MD (1) · David Clark, MD (1) · David Sas, DO (1) · Dawn Milliner, M.D (1) · Dawn Milliner, M.D. (1)
PMID 40601241, PMID 40193200, PMID 38332115, PMID 15961949, PMID 24385516, PMID 33789010, PMID 33985991, PMID 34906487, PMID 35843439, PMID 35913563, PMID 36007597, PMID 39355649, PMID 39875734, PMID 41343248, PMID 41388232
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