7 completed, 4 active not recruiting, 3 terminated, 1 enrolling by invitation. 16 Phase 2, 8 Phase 3, 7 Phase 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 |
|---|---|---|---|---|---|---|
| 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 | 2026-11-09 |
| 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 |
| 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 |
| 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 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 |
| 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 |
| 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 |
| 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 (16) · Lysosomal Storage Diseases (6) · Mucopolysaccharidosis I (3) · Adrenoleukodystrophy (2) · Leukodystrophy, Globoid Cell (2) · Leukodystrophy, Metachromatic (2) · Mucopolysaccharidoses (2) · alpha-Mannosidosis (2) · Anemia, Diamond-Blackfan (1) · Anemia, Sickle Cell (1) · Arthritis, Juvenile (1) · Autoimmune Lymphoproliferative Syndrome (1) · Chediak-Higashi Syndrome (1) · Cognition Disorders (1)
Carbohydrate Metabolism, Inborn Errors (16) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (16) · Death (16) · Death, Sudden (16) · Infant Death (16) · Metabolism, Inborn Errors (16) · Mucinoses (16) · Nutritional and Metabolic Diseases (16) · Pathologic Processes (16) · Pathological Conditions, Signs and Symptoms (16) · Skin and Connective Tissue Diseases (16) · Connective Tissue Diseases (15) · Genetic Diseases, Inborn (15) · Genetic Diseases, X-Linked (15) · Heredodegenerative Disorders, Nervous System (15)
idursulfase (4) · Alemtuzumab (1) · Growth Hormone (1) · Human Growth Hormone (1) · Hydroxyurea (1) · Iduronate Sulfatase (1) · Melphalan (1) · Stem Cell Transplantation (1) · Thiotepa (1) · fludarabine (1) · fludarabine phosphate (1)
Shire (4) · Denali Therapeutics Inc. (3) · REGENXBIO Inc. (2) · AO GENERIUM (1) · Green Cross Corporation (1) · Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center (1) · Paul Szabolcs (1) · Sangamo Therapeutics (1) · Takeda (1) · Talaris Therapeutics Inc. (1)
United States (14) · United Kingdom (8) · Canada (7) · Brazil (5) · France (4) · Germany (4) · Spain (4) · Italy (3) · Netherlands (3) · Sweden (3) · Argentina (2) · Belgium (2) · Czechia (2) · Turkey (Türkiye) (2) · Australia (1)
Jose Alcantara Rodriguez, PharmD (1) · Lynda E Polgreen, M.D. (1) · Oksana A. Markova, MD, MSc (1) · Paul Szabolcs, MD (1) · Sam Lu, MD (1) · Suzanne T Ildstad, MD (1)
PMID 21150784, PMID 22929184, PMID 23497636, PMID 23837440, PMID 32634238, PMID 32750127, PMID 33874971, PMID 35588317, PMID 35961250, PMID 36027721, PMID 36299240, PMID 37915038, PMID 41467650
2023 (2), 2004 (1), 2019 (1)