6 completed, 4 active not recruiting, 2 terminated, 1 enrolling by invitation. 13 Phase 3, 8 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 3 | 13 | Completed 6, Active not recruiting 4, Terminated 2, Enrolling by invitation 1 |
| Phase 2 | 8 | Active not recruiting 3, Completed 3, Enrolling by invitation 1, Terminated 1 |
Follow Sudden Infant Death: new trials by email Literature Review of Sudden Infant Death trials Research Report on Sudden Infant 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 |
| 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 |
| 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 |
| An Extension Study to Assess the Long-term Safety and Efficacy of Hunterase (Idursulfase Beta) Hunterase - MPS II, Hunter Syndrome (MPS II) | Phase 3 | Completed | GC Biopharma Corp | 30 | 2018-05-23 | 2026-01-15 |
| A Study of GC1111 in Hunter Syndrom Patients GC1111, Comparator - Hunter Syndrome | Phase 3 | Completed | Green Cross Corporation | 32 | 2017-03-09 | 2024-04-16 |
| 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 |
| Clinical Trial of Growth Hormone in MPS I, II, and VI Somatropin (DNA origin) - Mucopolysaccharidosis I, Mucopolysaccharidosis II | Phase 2 / Phase 3 | Terminated | Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center | 2 | 2008-11-01 | 2018-09-05 |
| ALD-101 Adjuvant Therapy of Unrelated Umbilical Cord Blood Transfusion (UCBT) in Patients With Inherited Metabolic Diseases ALD-101 - Inherited Metabolic Diseases, Lysosomal Storage Disorders | Phase 3 | Terminated | Aldagen | 40 | 2008-03-01 | 2014-07-08 |
| Influence of Pacifiers on Breastfeeding Duration offer a pacifier - Sudden Infant Death | Phase 3 | Completed | Fundacion para la Salud Materno Infantil | 1021 | 2006-03-01 | 2008-09-19 |
| Extension of Study TKT024 Evaluating Long-Term Safety and Clinical Outcomes in MPS II Patients Receiving Idursulfase Idursulfase - Hunter Syndrome, Mucopolysaccharidosis II (MPS II) | Phase 2 / Phase 3 | Completed | Shire | 94 | 2004-09-13 | 2021-06-10 |
| Iduronate-2-sulfatase Enzyme Replacement Therapy in Mucopolysaccharidosis II (MPS II) Iduronate-2-sulfatase enzyme replacement therapy, iduronate-2-sulfatase enzyme replacement therapy, Placebo - Mucopolysaccharidosis II | Phase 2 / Phase 3 | Completed | Shire | 96 | 2003-09-18 | 2021-06-10 |
Mucopolysaccharidosis II (12) · Lysosomal Storage Diseases (5) · Mucopolysaccharidosis I (2) · Mucopolysaccharidosis VI (2) · Adrenoleukodystrophy (1) · Breast Feeding (1) · Canavan Disease (1) · Cognition Disorders (1) · Cognitive Dysfunction (1) · Connective Tissue Diseases (1) · Dwarfism (1) · Fucosidosis (1) · Gangliosidosis, GM1 (1) · Genetic Diseases, Inborn (1)
Death (13) · Death, Sudden (13) · Infant Death (13) · Pathologic Processes (13) · Pathological Conditions, Signs and Symptoms (13) · Carbohydrate Metabolism, Inborn Errors (12) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (12) · Mucinoses (12) · Nutritional and Metabolic Diseases (12) · Skin and Connective Tissue Diseases (12) · Connective Tissue Diseases (11) · Genetic Diseases, Inborn (11) · Genetic Diseases, X-Linked (11) · Heredodegenerative Disorders, Nervous System (11) · Intellectual Disability (11)
idursulfase (3) · Growth Hormone (1) · Human Growth Hormone (1) · Iduronate Sulfatase (1)
Shire (3) · Denali Therapeutics Inc. (2) · REGENXBIO Inc. (2) · AO GENERIUM (1) · Aldagen (1) · Fundacion para la Salud Materno Infantil (1) · GC Biopharma Corp (1) · Green Cross Corporation (1) · Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center (1)
United States (9) · Brazil (5) · United Kingdom (5) · Canada (4) · France (4) · Germany (4) · Spain (4) · Argentina (3) · Italy (3) · Sweden (3) · Belgium (2) · Czechia (2) · Netherlands (2) · South Korea (2) · Turkey (Türkiye) (2)
Alejandro Jenik, M.D. (1) · Alexander Schramm (1) · DongKyu Jin, M.D., Ph.D. (1) · James Hinson, MD (1) · Joanne Kurtzberg, MD (1) · Jose Alcantara Rodriguez, PharmD (1) · Lynda E Polgreen, M.D. (1) · Oksana A. Markova, MD, MSc (1)
PMID 21150784, PMID 22929184, PMID 23837440, PMID 33874971, PMID 35961250, PMID 36027721, PMID 37915038, PMID 42421116
2004 (1), 2023 (1)