21 completed, 4 unknown, 2 active not recruiting, 2 recruiting. 32 Phase 1, 18 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 1 | 32 | Completed 21, Unknown 4, Active not recruiting 2, Recruiting 2, Terminated 2, Withdrawn 1 |
| Phase 2 | 18 | Completed 12, Active not recruiting 2, Terminated 2, Unknown 2 |
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 |
|---|---|---|---|---|---|---|
| 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 |
| Effects of Dexmedetomidine in Patients With Agitated Delirium in Palliative Care Dexmedetomidine Hydrochloride - Hyperactive Delirium, Delirium of Mixed Origin | Phase 1 / Phase 2 | Unknown | Bruyère Health Research Institute. | 50 | 2024-01-30 | 2025-04-15 |
| 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Safety and Efficacy of HMI-203 in ERT-Treated Adults With MPS II Genetic HMI-203 - Mucopolysaccharidosis II | Phase 1 | Withdrawn | Homology Medicines, Inc | 2022-09-08 | 2023-08-28 | |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 |
| Bilateral Orthotopic Lung Transplant - Bone Marrow Transplant CD3/CD19 neg allogeneic BMT - Primary Immunodeficiency, PID | Phase 1 / Phase 2 | Completed | National Institute of Allergy and Infectious Diseases (NIAID) | 5 | 2017-12-01 | 2025-07-22 |
| 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 |
| Safety and Efficacy of Autologous Umbilical Cord Blood Mononuclear Cells Transfusion in Neonates Autologous Umbilical Cord Blood Mononuclear Cells Therapy, 0.9% Sodium Chloride - Safety Issues, Effect of Drugs | Phase 1 | Unknown | Guangdong Women and Children Hospital | 200 | 2017-02-10 | 2017-04-10 |
| Non-randomized, Open-labeled, Interventional, Single Group, Proof of Concept Study With Multi-modality Approach in Cases of Brain Death Due to Traumatic Brain Injury Having Diffuse Axonal Injury BQ-A Peptide Extract, Mesenchymal Stem Cells, Transcranial Laser Therapy - Brain Death | Phase 1 | Completed | Bioquark Inc. | 20 | 2016-07-01 | 2020-10-01 |
| 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 |
| Is the WHO Recommended Dose of Pralidoxime Effective in the Treatment of Organophosphorus Poisoning? Pralidoxime - Deaths | Phase 1 | Completed | Sheri Kashmir Institute of Medical Sciences | 100 | 2012-04-01 | 2014-01-20 |
| 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 |
| Ex-Vivo Lung Perfusion to Increase the Number of Organs for Transplantation EVLP Group - Disorder Related to Lung Transplantation, Brain Death | Phase 1 | Completed | Policlinico Hospital | 7 | 2011-01-01 | 2013-10-23 |
| 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 |
Showing 20 of 32. Sign in to see all of them.
Sudden Infant Death (10) · Mucopolysaccharidosis II (9) · Death, Sudden, Cardiac (7) · Asphyxia (3) · Brain Death (3) · Perinatal Death (3) · Arrhythmogenic Right Ventricular Dysplasia (2) · Heart Arrest (2) · Hypoxia-Ischemia, Brain (2) · Lysosomal Storage Diseases (2) · Pre-Eclampsia (2) · Tachycardia, Ventricular (2) · Abruptio Placentae (1) · Adrenoleukodystrophy (1)
Pathological Conditions, Signs and Symptoms (32) · Pathologic Processes (31) · Nervous System Diseases (17) · Death, Sudden (16) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (13) · Neurobehavioral Manifestations (13) · Neurologic Manifestations (13) · Genetic Diseases, Inborn (11) · Carbohydrate Metabolism, Inborn Errors (10) · Connective Tissue Diseases (10) · Infant Death (10) · Metabolic Diseases (10) · Metabolism, Inborn Errors (10) · Mucinoses (10)
Sodium Chloride (2) · idursulfase (2) · Dexmedetomidine (1) · Dinoprostone (1) · Erythropoietin (1) · GAA protein, human (1) · GALNS protein, human (1) · Iduronidase (1) · Misoprostol (1) · Oximes (1) · Palliative Care (1) · Sebelipase alfa (1) · Stem Cell Transplantation (1) · Thyroxine (1) · Wobenzym (1)
University of California, San Francisco (2) · Zhengzhou University (2) · Aalborg University Hospital (1) · ArmaGen, Inc (1) · Bandim Health Project (1) · Bioquark Inc. (1) · Bruyère Health Research Institute. (1) · Denali Therapeutics Inc. (1) · Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) (1) · Foundation for Atlanta Veterans Education and Research, Inc. (1) · Gilead Sciences (1) · Green Cross Corporation (1) · Guangdong Women and Children Hospital (1) · Homology Medicines, Inc (1) · Hormozgan University of Medical Sciences (1)
United States (16) · Canada (6) · United Kingdom (4) · China (3) · Germany (3) · India (2) · Denmark (1) · Egypt (1) · Guinea-Bissau (1) · Iran (1) · Italy (1) · Netherlands (1) · Philippines (1) · South Korea (1)
Changlian Zhu, MD, PhD (2) · A. Maziar Zafari, M.D., Ph.D (1) · Barry Greenberg, MD (1) · Cynthia Dougherty, ARNP, PhD (1) · Franco Valenza, MD (1) · Himanshu Bansal, MD (1) · Irene J Higginson, BMBS FRCP FFPHM PhD (1) · James Downar, MDCM, MSc (1) · Jie Yang, Phd (1) · Johannes Scherr, Dr. med. (1) · Michael D Sharpe, MD FRCPC (1)
PMID 15324318, PMID 16015266, PMID 16553654, PMID 17058754, PMID 18507817, PMID 18547438, PMID 18805742, PMID 19280394, PMID 19833477, PMID 19948615, PMID 20809942, PMID 20955600, PMID 21190486, PMID 21637107, PMID 21978993, PMID 23497636, PMID 23510062, PMID 23673863, PMID 23884915, PMID 24559067, PMID 24628890, PMID 26093876, PMID 28374935, PMID 28657663, PMID 29370708, PMID 30337566, PMID 32102934, PMID 32300579, PMID 35588317, PMID 36299240, PMID 37955580, PMID 37979797, PMID 39891377, PMID 41467650, PMID 9918480
2019 (1), 2023 (1), R01NR004766 (1), U01AI125050 (1)