6 active not recruiting. 6 Phase 3, 3 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 3 | 6 | Active not recruiting 6 |
| Phase 2 | 3 | Active not recruiting 3 |
Follow Death: new trials by email Literature Review of Death trials Research Report on Death
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 |
|---|---|---|---|---|---|---|
| RGX-121-3102 Gene Therapy in Participants With MPS II (Hunter Syndrome) RGX-121-3102 - MPS II, Hunter Syndrome (MPS II) | Phase 3 | Active not recruiting | REGENXBIO Inc. | 2 | 2025-11-25 | 2026-06-04 |
| The Effect of Retatrutide Once Weekly on Cardiovascular Outcomes and Kidney Outcomes in Adults Living With Obesity (TRIUMPH-Outcomes) Retatrutide, Placebo - Atherosclerotic Cardiovascular Disease (ASCVD), Chronic Kidney Disease (CKD) | Phase 3 | Active not recruiting | Eli Lilly and Company | 10000 | 2024-04-30 | 2026-10-08 |
| The RENAL LIFECYCLE Trial: A RCT to Assess the Effect of Dapagliflozin on Renal and Cardiovascular Outcomes in Patients With Severe CKD Dapagliflozin 10 mg/day (oral), Placebo - Kidney Disease, Chronic, Renal Transplant Failure | Phase 3 | Active not recruiting | University Medical Center Groningen | 1750 | 2022-11-08 | 2026-10-08 |
| A Study to Determine the Efficacy and Safety of Tividenofusp Alfa (DNL310) vs Idursulfase in Pediatric and Young Adult Participants With Neuronopathic (nMPS II) or Non-Neuronopathic Mucopolysaccharidosis Type II (nnMPS II) tividenofusp alfa, idursulfase - Mucopolysaccharidosis II | Phase 2 / Phase 3 | Active not recruiting | Denali Therapeutics Inc. | 65 | 2022-07-21 | 2026-09-09 |
| A Multi-cohort Study of Safety, Efficacy, PK and PD of GNR-055 in Patients With Mucopolysaccharidosis Type II GNR-055 1.0-2.0-3.0 mg/kg, GNR-055 2.0 mg/kg, GNR-055 3.0 mg/kg - Mucopolysaccharidosis Type II, Metabolic Diseases | Phase 2 / Phase 3 | Active not recruiting | AO GENERIUM | 32 | 2021-11-30 | 2026-09-21 |
| CAMPSIITE™ RGX-121 Gene Therapy in Subjects With MPS II (Hunter Syndrome) RGX-121 - Mucopolysaccharidosis Type II (MPS II) | Phase 2 / Phase 3 | Active not recruiting | REGENXBIO Inc. | 48 | 2018-09-27 | 2025-01-28 |
Mucopolysaccharidosis II (4) · Sudden Infant Death (4) · Lysosomal Storage Diseases (2) · Renal Insufficiency, Chronic (2) · Atherosclerosis (1) · Cardiovascular Diseases (1) · Cerebrovascular Disorders (1) · Cognition Disorders (1) · Cognitive Dysfunction (1) · Connective Tissue Diseases (1) · Coronary Artery Disease (1) · Diabetes Mellitus, Type 2 (1) · Genetic Diseases, Inborn (1) · Genetic Diseases, X-Linked (1)
Pathologic Processes (6) · Pathological Conditions, Signs and Symptoms (6) · Nutritional and Metabolic Diseases (5) · Carbohydrate Metabolism, Inborn Errors (4) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (4) · Death, Sudden (4) · Infant Death (4) · Metabolic Diseases (4) · Metabolism, Inborn Errors (4) · Mucinoses (4) · Mucopolysaccharidoses (4) · Nervous System Diseases (4) · Skin and Connective Tissue Diseases (4) · Connective Tissue Diseases (3)
dapagliflozin (1) · idursulfase (1) · retatrutide (1)
REGENXBIO Inc. (2) · AO GENERIUM (1) · Denali Therapeutics Inc. (1) · Eli Lilly and Company (1) · University Medical Center Groningen (1)
United States (4) · Belgium (3) · Brazil (3) · Germany (3) · Netherlands (3) · Spain (3) · Argentina (2) · Australia (2) · Canada (2) · Czechia (2) · France (2) · Italy (2) · Turkey (Türkiye) (2) · United Kingdom (2) · Austria (1)
Alexander Schramm (1) · Jose Alcantara Rodriguez, PharmD (1) · Oksana A. Markova, MD, MSc (1) · Ron Gansevoort (1)
PMID 37170711, PMID 39928546, PMID 41160422, PMID 41315742, PMID 41551843, PMID 41667648
2023 (2)