14 completed, 5 active not recruiting, 5 terminated, 3 recruiting. 31 Phase 2, 10 Phase 3, 9 Phase 1.
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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 |
|---|---|---|---|---|---|---|
| Early Double Sequential Defibrillation in Out of Hospital Cardiac Arrest Double sequential defibirllation, Standard defibrillation - Out of Hospital Cardiac Arrest, Ventricular Fibrillation | Phase 2 / Phase 3 | Recruiting | Gabriel Riva | 916 | 2025-09-08 | 2026-02-17 |
| Targeted Therapy With Glycogen Synthase Kinase-3 Inhibition for Arrhythmogenic Cardiomyopathy Tideglusib, Placebo - Arrhythmogenic Cardiomyopathy, Arrhythmogenic Right Ventricular Cardiomyopathy | Phase 2 | Recruiting | Hamilton Health Sciences Corporation | 120 | 2025-03-21 | 2026-03-12 |
| 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 |
| An Extension Study of the Long-Term Safety, Tolerability, and Efficacy of Tividenofusp Alfa (DNL310) in Participants With Mucopolysaccharidosis Type II (MPS II) From Study DNLI-E-0002 or Study DNLI-E-0007 tividenofusp alfa - Mucopolysaccharidosis II | Phase 2 / Phase 3 | Enrolling by invitation | Denali Therapeutics Inc. | 99 | 2023-09-20 | 2026-10-27 |
| 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 |
| Mutation-specific Therapy for the Long QT Syndrome Lumacaftor / Ivacaftor, Orkambi® Oral Tablet - Long QT Syndrome | Phase 2 | Completed | Istituto Auxologico Italiano | 16 | 2021-06-15 | 2025-04-25 |
| 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 |
| 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 |
| The Response To Ajmaline Provocation in Healthy Subjects Ajmaline - Brugada Syndrome, Sudden Death | Phase 2 | Unknown | St George's, University of London | 100 | 2017-10-22 | 2019-11-05 |
| Clinical Study of the LRS ThermoSuit™ System in Post Arrest Patients With Intravenous Infusion of Magnesium Sulfate Cooling with ThermoSuit with Magnesium Sulfate Infusion, Cooling with ThermoSuit with Normal Saline Infusion - Death, Sudden, Cardiac, Coma | Phase 2 | Withdrawn | Life Recovery Systems | 2017-09-01 | 2017-08-25 | |
| 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 |
| Abnormal Ventilatory Response to Carbon Dioxide: a Potential Biomarker for Seizure Induced Respiratory Depression & Modification by SSRI Fluoxetine, Placebo - Epilepsy, SUDEP | Phase 2 | Completed | Rup Kamal Sainju | 30 | 2017-02-16 | 2020-05-04 |
| Study of Intrathecal Idursulfase-IT Administered in Conjunction With Elaprase® in Pediatric Patients With Hunter Syndrome and Early Cognitive Impairment idursulfase-IT, No IT treatment - Hunter Syndrome | Phase 2 / Phase 3 | Completed | Shire | 58 | 2014-03-24 | 2021-06-11 |
| Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT Hydroxyurea, Alemtuzumab, Fludarabine - Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes | Phase 2 | Recruiting | Paul Szabolcs | 100 | 2014-02-04 | 2025-12-15 |
| Vitamin D and Cardiac Autonomic Tone in Hemodialysis Alfacalcidol, Ergocalciferol - Cardiovascular Disease, Sudden Cardiac Death | Phase 2 | Completed | University of Calgary | 56 | 2013-01-01 | 2016-08-31 |
| Study to Develop a Non-invasive Marker for Monitoring Myocardial Fibrosis 99mTc-NC100692 - Hypertrophic Cardiomyopathy, Acute Coronary Syndrome | Phase 2 | Terminated | Ottawa Heart Institute Research Corporation | 120 | 2011-05-01 | 2017-04-24 |
| 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 |
| Coenzyme Q10 in Post-Cardiac Arrest Cerebral Resuscitation Coenzyme Q10, Placebo - Cardiac Arrest, Sudden Cardiac Arrest | Phase 2 | Completed | Beth Israel Deaconess Medical Center | 10 | 2011-02-01 | 2017-07-31 |
Showing 20 of 31. Sign in to see all of them.
Mucopolysaccharidosis II (16) · Sudden Infant Death (16) · Death, Sudden, Cardiac (11) · Lysosomal Storage Diseases (6) · Heart Arrest (3) · Mucopolysaccharidosis I (3) · Tachycardia, Ventricular (3) · Adrenoleukodystrophy (2) · Arrhythmias, Cardiac (2) · Arrhythmogenic Right Ventricular Dysplasia (2) · Leukodystrophy, Globoid Cell (2) · Leukodystrophy, Metachromatic (2) · Mucopolysaccharidoses (2) · Ventricular Fibrillation (2)
Death (31) · Pathologic Processes (31) · Pathological Conditions, Signs and Symptoms (31) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (20) · Nervous System Diseases (17) · Carbohydrate Metabolism, Inborn Errors (16) · Genetic Diseases, Inborn (16) · Infant Death (16) · Metabolism, Inborn Errors (16) · Mucinoses (16) · Neurobehavioral Manifestations (16) · Neurologic Manifestations (16) · Nutritional and Metabolic Diseases (16) · Skin and Connective Tissue Diseases (16)
idursulfase (4) · Ajmaline (1) · Alemtuzumab (1) · Docosahexaenoic Acids (1) · Epinephrine (1) · Ergocalciferols (1) · Fluoxetine (1) · Growth Hormone (1) · Human Growth Hormone (1) · Hydroxyurea (1) · Iduronate Sulfatase (1) · Melphalan (1) · Metoprolol (1) · Prajmaline (1) · Sodium Chloride (1)
Shire (4) · Denali Therapeutics Inc. (3) · Aalborg University Hospital (2) · REGENXBIO Inc. (2) · AO GENERIUM (1) · Beth Israel Deaconess Medical Center (1) · Campus Bio-Medico University (1) · David Haines, MD (1) · Gabriel Riva (1) · Green Cross Corporation (1) · Hamilton Health Sciences Corporation (1) · Istituto Auxologico Italiano (1) · Lantheus Medical Imaging (1) · Lexeo Therapeutics (1) · Life Recovery Systems (1)
United States (19) · Canada (10) · United Kingdom (9) · Brazil (5) · Italy (5) · Spain (5) · France (4) · Germany (4) · Netherlands (4) · Sweden (4) · Argentina (2) · Belgium (2) · Czechia (2) · Denmark (2) · Turkey (Türkiye) (2)
Akil Awad, M.D. Ph.D. (1) · Andreas Claesson, R.N. Ph.D. (1) · Carl Magnusson, R.N., Ph.D. (1) · David E Haines, MD (1) · Dr. Brenda Hemmelgarn, MD, PhD, MN (1) · Dr. Derek Exner, MD, MPH (1) · Dr. Sofia B Ahmed, MD, MMSc (1) · Elijah Behr, MD (1) · Francesco M Serino, MD (1) · Gabriel Riva, M.D. Ph.D. (1) · Giuseppe Rosano, MD (1) · Jason D Roberts, MD MAS (1) · Jeppe H Christensen, MD, DMSci (1)
PMID 21150784, PMID 22929184, PMID 23497636, PMID 23837440, PMID 25098377, PMID 27690095, PMID 32634238, PMID 32750127, PMID 33874971, PMID 35588317, PMID 35961250, PMID 36027721, PMID 36299240, PMID 37915038, PMID 41467650, PMID 42379878
2023 (2), 2004 (1), 2016 (1), 2019 (1)