3 completed, 1 active not recruiting, 1 not yet recruiting. 1 Phase 1, 1 Phase 2, 1 Phase 3.
| Phase | Trials | Status |
|---|---|---|
| Phase 1 | 1 | Completed 1 |
| Phase 2 | 1 | Completed 1 |
| Phase 3 | 1 | Not yet recruiting 1 |
Follow Primary Hyperoxaluria Type 3: new trials by email Literature Review of Primary Hyperoxaluria Type 3 trials Research Report on Primary Hyperoxaluria Type 3
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 | 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 |
| 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 | |
| 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 |
| 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 |
Hyperoxaluria, Primary (4) · Primary hyperoxaluria type 1 (3) · Primary hyperoxaluria type 2 (3) · 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) · Adenine phosphoribosyltransferase deficiency (1) · Adrenal hyperplasia, congenital, type 5 (1) · Adrenocorticotropic hormone deficiency (1) · Amaurosis congenita of Leber, type 2 (1) · Anemia, Sickle Cell (1) · Angelman Syndrome (1)
Carbohydrate Metabolism, Inborn Errors (5) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (5) · Female Urogenital Diseases (5) · Female Urogenital Diseases and Pregnancy Complications (5) · Genetic Diseases, Inborn (5) · Kidney Diseases (5) · Male Urogenital Diseases (5) · Metabolic Diseases (5) · Metabolism, Inborn Errors (5) · Nutritional and Metabolic Diseases (5) · Urogenital Diseases (5) · Urologic Diseases (5) · Hyperoxaluria (4) · Calcium Metabolism Disorders (2) · Pathological Conditions, Signs and Symptoms (2)
nedosiran (2) · stiripentol (1)
Dicerna Pharmaceuticals, Inc., a Novo Nordisk company (3) · Biocodex (1) · RTI International (1)
United States (4) · Germany (3) · United Kingdom (3) · Canada (2) · Poland (2) · Italy (1) · Japan (1) · Lebanon (1) · Netherlands (1) · Spain (1) · Turkey (Türkiye) (1) · United Arab Emirates (1)
Alexandra Haagensen, MD, MBA (1) · Curt Scharfe, MD, PhD (1) · Sarb Shergill, PhD (1) · Verity Rawson (1)
PMID 31315600, PMID 35142618, PMID 39875734, PMID 40193200, PMID 40601241
2023 (1)