3 completed, 2 active not recruiting, 2 recruiting, 1 not yet recruiting. 4 Phase 2, 2 Phase 3.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 4 | Completed 3, Recruiting 1 |
| Phase 3 | 2 | Active not recruiting 1, Not yet recruiting 1 |
Follow Primary hyperoxaluria type 2: new trials by email Literature Review of Primary hyperoxaluria type 2 trials Research Report on Primary hyperoxaluria type 2
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 |
|---|---|---|---|---|---|---|
| A Phase 2 Study to Investigate the Efficacy, Safety and Tolerability of Remibrutinib (LOU064) in Adult Patients With Papulopustular Rosacea (PPR) LOU064, Placebo - Papulopustular Rosacea | Phase 2 | Recruiting | Novartis Pharmaceuticals | 100 | 2026-07-30 | 2026-10-15 |
| 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 |
| 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 |
| 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 |
| 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 | |
| Rare Kidney Stone Consortium Biobank Primary Hyperoxaluria, Dent Disease | Recruiting | Mayo Clinic | 2000 | 2013-05-01 | 2026-07-22 | |
| Study of SU011248 in Patients With Advanced Kidney Cancer SU011248 capsule - Carcinoma, Renal Cell | Phase 2 | Completed | Pfizer | 51 | 2005-12-01 | 2010-02-23 |
Primary hyperoxaluria type 1 (6) · Hyperoxaluria, Primary (5) · Adenine phosphoribosyltransferase deficiency (2) · Genetic Diseases, Inborn (2) · Kidney Diseases (2) · Urologic 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) · Adrenal hyperplasia, congenital, type 5 (1) · Adrenocorticotropic hormone deficiency (1)
Female Urogenital Diseases (7) · Female Urogenital Diseases and Pregnancy Complications (7) · Male Urogenital Diseases (7) · Urogenital Diseases (7) · Carbohydrate Metabolism, Inborn Errors (6) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (6) · Metabolic Diseases (6) · Metabolism, Inborn Errors (6) · Nutritional and Metabolic Diseases (6) · Kidney Diseases (5) · Urologic Diseases (5) · Genetic Diseases, Inborn (4) · Hyperoxaluria (4) · Genetic Diseases, X-Linked (2) · Neoplasms (2)
nedosiran (3) · Sunitinib (1) · remibrutinib (1) · stiripentol (1)
Dicerna Pharmaceuticals, Inc., a Novo Nordisk company (2) · Biocodex (1) · Mayo Clinic (1) · Novartis Pharmaceuticals (1) · Novo Nordisk A/S (1) · Pfizer (1) · RTI International (1)
United States (6) · Canada (4) · Germany (4) · Japan (4) · Spain (4) · Italy (3) · Lebanon (3) · United Kingdom (3) · Australia (2) · France (2) · Netherlands (2) · Poland (2) · Turkey (Türkiye) (2) · Bulgaria (1) · China (1)
Curt Scharfe, MD, PhD (1) · John C Lieske, M.D. (1) · Sarb Shergill, PhD (1) · Verity Rawson, MB.CHB (1)
PMID 40193200, PMID 40601241, PMID 31315600, PMID 35142618, PMID 36007597, PMID 39875734
2023 (1), 2025 (1)