5 active not recruiting. 5 Phase 2, 3 Phase 3, 2 Phase 1.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 5 | Active not recruiting 5 |
| Phase 3 | 3 | Active not recruiting 3 |
| Phase 1 | 2 | Active not recruiting 2 |
Follow Death, Sudden: new trials by email Literature Review of Death, Sudden trials Research Report on Death, Sudden
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 |
|---|---|---|---|---|---|---|
| Gene Therapy for ACM Due to a PKP2 Pathogenic Variant LX2020 - Arrhythmogenic Cardiomyopathy, PKP2-ACM | Phase 1 / Phase 2 | Active not recruiting | Lexeo Therapeutics | 10 | 2024-02-29 | 2025-11-12 |
| 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 |
| A Study of Tividenofusp Alfa (DNL310) in Pediatric Participants With Hunter Syndrome tividenofusp alfa - Mucopolysaccharidosis II | Phase 1 / Phase 2 | Active not recruiting | Denali Therapeutics Inc. | 47 | 2020-07-16 | 2025-08-07 |
| 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) · Arrhythmogenic Right Ventricular Dysplasia (1) · Cardiomyopathies (1) · Cognition Disorders (1) · Cognitive Dysfunction (1) · Connective Tissue Diseases (1) · Death, Sudden, Cardiac (1) · Genetic Diseases, Inborn (1) · Genetic Diseases, X-Linked (1) · Heart Arrest (1) · Heredodegenerative Disorders, Nervous System (1) · Intellectual Disability (1) · Mental Disorders (1)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (5) · Death (5) · Pathologic Processes (5) · Pathological Conditions, Signs and Symptoms (5) · Carbohydrate Metabolism, Inborn Errors (4) · Infant Death (4) · Metabolism, Inborn Errors (4) · Mucinoses (4) · Mucopolysaccharidoses (4) · Nutritional and Metabolic Diseases (4) · Skin and Connective Tissue Diseases (4) · Connective Tissue Diseases (3) · Genetic Diseases, Inborn (3) · Genetic Diseases, X-Linked (3)
idursulfase (1)
Denali Therapeutics Inc. (2) · AO GENERIUM (1) · Lexeo Therapeutics (1) · REGENXBIO Inc. (1)
United States (4) · Brazil (2) · Canada (2) · Netherlands (2) · United Kingdom (2) · Argentina (1) · Belgium (1) · Czechia (1) · France (1) · Germany (1) · Italy (1) · Russia (1) · Spain (1) · Sweden (1) · Turkey (Türkiye) (1)
Jose Alcantara Rodriguez, PharmD (1) · Oksana A. Markova, MD, MSc (1) · Sam Lu, MD (1)
2019 (1), 2023 (1)