2 completed, 2 recruiting, 2 terminated, 1 active not recruiting. 2 Phase 1, 2 Phase 3, 1 Phase 2, 1 Phase 4.
| Phase | Trials | Status |
|---|---|---|
| Phase 1 | 2 | Recruiting 1, Terminated 1 |
| Phase 3 | 2 | Active not recruiting 1, Completed 1 |
| Phase 2 | 1 | Terminated 1 |
| Phase 4 | 1 | Completed 1 |
Follow MPS II: new trials by email Literature Review of MPS II trials Research Report on MPS II
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 |
| MPS II Immunophenotyping MPS II, Mucopolysaccharidosis II | Terminated | Duke University | 6 | 2022-04-01 | 2024-09-19 | |
| 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 |
| 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 |
| 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 |
| Longitudinal Study of Neurodegenerative Disorders Palliative Care, Hematopoetic Stem Cell Transplantation - MLD, Krabbe Disease | Recruiting | University of Pittsburgh | 1500 | 2012-01-11 | 2026-02-09 | |
| Safety and Clinical Outcomes in Hunter Syndrome Patients 5 Years of Age and Younger Receiving Idursulfase Therapy Idursulfase - Hunter Syndrome, Mucopolysaccharidosis II | Phase 4 | Completed | Shire | 28 | 2007-12-31 | 2021-06-08 |
Sudden Infant Death (7) · Mucopolysaccharidosis II (5) · Gaucher Disease (2) · Lysosomal Storage Diseases (2) · Mucopolysaccharidosis IV (2) · Hypermethioninemia (1) · Language (1) · Leukodystrophy, Globoid Cell (1) · Leukoencephalopathies (1) · Mucopolysaccharidosis III (1) · Mucopolysaccharidosis VI (1) · Mucopolysaccharidosis VII (1) · Multiple Sulfatase Deficiency Disease (1) · Neuronal Ceroid-Lipofuscinoses (1) · Niemann-Pick Diseases (1)
Carbohydrate Metabolism, Inborn Errors (7) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (7) · Connective Tissue Diseases (7) · Death (7) · Death, Sudden (7) · Genetic Diseases, Inborn (7) · Infant Death (7) · Metabolic Diseases (7) · Metabolism, Inborn Errors (7) · Mucinoses (7) · Mucopolysaccharidoses (7) · Nervous System Diseases (7) · Nutritional and Metabolic Diseases (7) · Pathologic Processes (7) · Pathological Conditions, Signs and Symptoms (7)
idursulfase (2) · GAA protein, human (1) · GALNS protein, human (1) · Iduronidase (1) · Palliative Care (1) · Sebelipase alfa (1) · galsulfase (1) · vestronidase alfa (1)
Duke University (1) · GC Biopharma Corp (1) · REGENXBIO Inc. (1) · Sangamo Therapeutics (1) · Shire (1) · University of California, San Francisco (1) · University of Pittsburgh (1)
United States (5) · Brazil (1) · Poland (1) · South Korea (1) · Taiwan (1)
Alexander Schramm (1) · Priya Kishnani, MD (1) · Tippi MacKenzie, MD (1)
PMID 15324318, PMID 19948615, PMID 21637107, PMID 23510062, PMID 25902842, PMID 28374935, PMID 28657663, PMID 30337566, PMID 32102934, PMID 36299240, PMID 37955580, PMID 39891377, PMID 42421116, PMID 9918480