10 completed, 2 active not recruiting, 2 recruiting, 2 terminated. 17 Phase 1, 9 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 1 | 17 | Completed 10, Active not recruiting 2, Recruiting 2, Terminated 2, Withdrawn 1 |
| Phase 2 | 9 | Completed 5, Active not recruiting 2, Terminated 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 Phase 1, Dose Escalation Trial of RP-A601 in Subjects With PKP2 Variant-Mediated Arrhythmogenic Cardiomyopathy (PKP2-ACM) RP-A601 - PKP2 Arrhythmogenic Cardiomyopathy (PKP2-ACM) | Phase 1 | Recruiting | Rocket Pharmaceuticals Inc. | 9 | 2023-08-29 | 2026-04-16 |
| Safety and Efficacy of HMI-203 in ERT-Treated Adults With MPS II Genetic HMI-203 - Mucopolysaccharidosis II | Phase 1 | Withdrawn | Homology Medicines, Inc | 2022-09-08 | 2023-08-28 | |
| PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders) Aldurazyme (laronidase) - MPS I, MPS II | Phase 1 | Recruiting | University of California, San Francisco | 10 | 2021-07-01 | 2026-03-17 |
| RGX-121 Gene Therapy in Children 5 Years of Age and Over With MPS II (Hunter Syndrome) RGX-121 - Mucopolysaccharidosis Type II (MPS II) | Phase 1 / Phase 2 | Completed | REGENXBIO Inc. | 6 | 2021-03-11 | 2025-01-28 |
| 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 |
| Ascending Dose Study of Genome Editing by the Zinc Finger Nuclease (ZFN) Therapeutic SB-913 in Subjects With MPS II SB-913 - Mucopolysaccharidosis II, MPS II | Phase 1 / Phase 2 | Terminated | Sangamo Therapeutics | 9 | 2017-05-11 | 2022-10-25 |
| Study to Evaluate the Effect of Eleclazine on QT, Safety, and Tolerability in Participants With Long QT2 Syndrome Eleclazine, Placebo - LQT2 Syndrome | Phase 1 | Completed | Gilead Sciences | 13 | 2015-07-20 | 2020-12-30 |
| Safety and Dose Ranging Study of Insulin Receptor MoAb-IDS Fusion Protein in Patients With Hunter Syndrome AGT-182 - Mucopolysaccharidosis II | Phase 1 | Completed | ArmaGen, Inc | 6 | 2015-04-01 | 2018-09-18 |
| The Effects of Enzymes and Flavonoids on Inflammation and Coagulation After Marathon Wobenzym plus, PL 1 - Inflammation, Death, Sudden, Cardiac | Phase 1 | Completed | Technical University of Munich | 160 | 2013-08-01 | 2015-06-30 |
| Phase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic Disorders hematopoietic stem cell infusion - Hurler Syndrome (MPS I), Hurler-Scheie Syndrome | Phase 1 / Phase 2 | Terminated | Talaris Therapeutics Inc. | 3 | 2011-04-01 | 2023-04-12 |
| An Extension Study of HGT-HIT-045 Evaluating Long-Term Safety and Clinical Outcomes of Idursulfase-IT in Conjunction With Elaprase in Pediatric Participants With Hunter Syndrome and Cognitive Impairment Idursulfase-IT, Elaprase - Hunter Syndrome | Phase 1 / Phase 2 | Completed | Takeda | 15 | 2010-08-01 | 2025-08-06 |
| To Evaluate the Safety and Efficacy of GC1111 (Recombinant Human Iduronate-2-sulfatase) in Hunter Syndrome Patients GC1111_0.5mg/kg, GC1111_1.0mg/kg, Elaprase_0.5mg/kg - Mucopolysaccharidosis II | Phase 1 / Phase 2 | Completed | Green Cross Corporation | 31 | 2010-05-01 | 2012-02-14 |
| A Safety and Dose Ranging Study of Idursulfase (Intrathecal) Administration Via an Intrathecal Drug Delivery Device in Pediatric Patients With Hunter Syndrome Who Have Central Nervous System Involvement and Are Receiving Treatment With Elaprase® Control, Idursulfase IT (1 mg), Idursulfase IT (10 mg) - Hunter Syndrome | Phase 1 / Phase 2 | Completed | Shire | 16 | 2009-11-18 | 2021-06-28 |
| Intravenous n-3 Fatty Acids and Ventricular Tachycardia in Patients With Implantable Cardioverter Defibrillator (ICD)-Pacemaker Omegaven, Isotonic saline - Ventricular Tachycardia, Sudden Cardiac Death | Phase 1 / Phase 2 | Completed | Aalborg University Hospital | 8 | 2007-10-01 | 2015-04-16 |
| Automatic External Defibrillation Monitoring in Cardiac Arrest Defibrillation of pulseless VT/VF by AECD - Death, Sudden, Cardiac, Ventricular Fibrillation | Phase 1 | Completed | Foundation for Atlanta Veterans Education and Research, Inc. | 192 | 2006-10-01 | 2015-07-15 |
| Nurses Helping Sudden Cardiac Arrest Survivors Recover After Getting An Implantable Cardioverter Defibrillator (ICD) Nursing Telephone Support Protocol, Usual Care - Sudden Cardiac Arrest | Phase 1 | Completed | National Institute of Nursing Research (NINR) | 150 | 1999-09-01 | 2008-09-10 |
Sudden Infant Death (10) · Mucopolysaccharidosis II (9) · Death, Sudden, Cardiac (7) · Arrhythmogenic Right Ventricular Dysplasia (2) · Heart Arrest (2) · Lysosomal Storage Diseases (2) · Tachycardia, Ventricular (2) · Adrenoleukodystrophy (1) · Blood Coagulation Disorders (1) · Cardiomyopathies (1) · Cardiovascular Diseases (1) · Death (1) · Gaucher Disease (1) · Heart Diseases (1)
Pathological Conditions, Signs and Symptoms (17) · Death (16) · Pathologic Processes (16) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (12) · Carbohydrate Metabolism, Inborn Errors (10) · Connective Tissue Diseases (10) · Genetic Diseases, Inborn (10) · Infant Death (10) · Metabolic Diseases (10) · Metabolism, Inborn Errors (10) · Mucinoses (10) · Nervous System Diseases (10) · Nutritional and Metabolic Diseases (10) · Skin and Connective Tissue Diseases (10)
idursulfase (2) · GAA protein, human (1) · GALNS protein, human (1) · Iduronidase (1) · Sebelipase alfa (1) · Sodium Chloride (1) · Stem Cell Transplantation (1) · Wobenzym (1) · eleclazine (1) · fish oil triglycerides (1) · galsulfase (1) · vestronidase alfa (1)
Aalborg University Hospital (1) · ArmaGen, Inc (1) · Denali Therapeutics Inc. (1) · Foundation for Atlanta Veterans Education and Research, Inc. (1) · Gilead Sciences (1) · Green Cross Corporation (1) · Homology Medicines, Inc (1) · Lexeo Therapeutics (1) · National Institute of Nursing Research (NINR) (1) · REGENXBIO Inc. (1) · Rocket Pharmaceuticals Inc. (1) · Sangamo Therapeutics (1) · Shire (1) · Takeda (1) · Talaris Therapeutics Inc. (1)
United States (14) · Canada (4) · United Kingdom (3) · Germany (2) · Denmark (1) · Netherlands (1) · Philippines (1) · South Korea (1)
A. Maziar Zafari, M.D., Ph.D (1) · Barry Greenberg, MD (1) · Cynthia Dougherty, ARNP, PhD (1) · Johannes Scherr, Dr. med. (1) · Patrice Rioux, MD PhD (1) · Sam Lu, MD (1) · Suzanne T Ildstad, MD (1) · Tippi MacKenzie, MD (1) · Trine Madsen, MD (1)
PMID 15324318, PMID 18547438, PMID 19948615, PMID 21637107, PMID 23497636, PMID 23510062, PMID 24559067, PMID 26093876, PMID 28374935, PMID 28657663, PMID 29370708, PMID 30337566, PMID 32102934, PMID 35588317, PMID 36299240, PMID 37955580, PMID 37979797, PMID 39891377, PMID 41467650, PMID 9918480
2019 (1), 2023 (1), R01NR004766 (1)