3 recruiting, 2 terminated, 1 completed, 1 unknown. 3 Phase 1, 2 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 1 | 3 | Recruiting 1, Terminated 1, Withdrawn 1 |
| Phase 2 | 2 | Terminated 1, Withdrawn 1 |
Follow MPS I: new trials by email Literature Review of MPS I trials Research Report on MPS I
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 |
|---|---|---|---|---|---|---|
| Cardiac Structure and Function in MPS MPS I, MPS IV A | Recruiting | Children's Hospital of Orange County | 240 | 2025-09-01 | 2026-10-21 | |
| Safety and Efficacy of Encapsulated Allogeneic MPS-1 Therapy SIG-005 (hIDUA Producing Spheres) - MPS I, MPS1-S | Phase 1 / Phase 2 | Withdrawn | Sigilon Therapeutics, Inc. | 2021-11-15 | 2023-04-11 | |
| The Intensively Follow-up Examinations for Asymptomatic MPS I Infants in Taiwan MPS I - MPS I | Unknown | Mackay Memorial Hospital | 20 | 2021-09-15 | 2022-03-17 | |
| 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 |
| Ascending Dose Study of Genome Editing by the Zinc Finger Nuclease (ZFN) Therapeutic SB-318 in Subjects With MPS I SB-318 - MPS I | Phase 1 / Phase 2 | Terminated | Sangamo Therapeutics | 3 | 2017-05-24 | 2023-01-26 |
| Ultrasound Findings of Finger, Wrist and Knee Joints in Mucopolysaccharidosis MPS I, MPS IV | Completed | Children's Hospital of Eastern Ontario | 18 | 2013-09-01 | 2018-01-24 | |
| Laronidase (Aldurazyme TM) Enzyme Replacement Therapy With Hematopoietic Stem Cell Transplant for Hurler Syndrome Laronidase - Mucopolysaccharidosis Type IH, MPS I | Terminated | Masonic Cancer Center, University of Minnesota | 20 | 2012-03-28 | 2018-09-27 | |
| 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 |
Mucopolysaccharidosis IV (4) · Mucopolysaccharidosis I (3) · Gaucher Disease (2) · Sudden Infant Death (2) · Hypermethioninemia (1) · Language (1) · Leukodystrophy, Globoid Cell (1) · Leukoencephalopathies (1) · Lysosomal Storage Diseases (1) · Mucopolysaccharidoses (1) · Mucopolysaccharidosis III (1) · Mucopolysaccharidosis VI (1) · Mucopolysaccharidosis VII (1) · Multiple Sulfatase Deficiency Disease (1) · Neuronal Ceroid-Lipofuscinoses (1)
Carbohydrate Metabolism, Inborn Errors (7) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (7) · Connective Tissue Diseases (7) · Genetic Diseases, Inborn (7) · Metabolic Diseases (7) · Metabolism, Inborn Errors (7) · Mucinoses (7) · Nutritional and Metabolic Diseases (7) · Skin and Connective Tissue Diseases (7) · Lysosomal Storage Diseases (6) · Mucopolysaccharidoses (6) · Brain Diseases (2) · Brain Diseases, Metabolic (2) · Brain Diseases, Metabolic, Inborn (2) · Central Nervous System Diseases (2)
Iduronidase (2) · GAA protein, human (1) · GALNS protein, human (1) · Palliative Care (1) · Sebelipase alfa (1) · galsulfase (1) · idursulfase (1) · vestronidase alfa (1)
Children's Hospital of Eastern Ontario (1) · Children's Hospital of Orange County (1) · Mackay Memorial Hospital (1) · Masonic Cancer Center, University of Minnesota (1) · Sangamo Therapeutics (1) · Sigilon Therapeutics, Inc. (1) · University of California, San Francisco (1) · University of Pittsburgh (1)
United States (5) · Brazil (1) · Canada (1) · Taiwan (1) · United Kingdom (1)
Johannes Roth, MD (1) · Paul Orchard, M.D. (1) · Tippi MacKenzie, MD (1)
PMID 15324318, PMID 19948615, PMID 21637107, PMID 23510062, PMID 28374935, PMID 28657663, PMID 30337566, PMID 32102934, PMID 32278382, PMID 36299240, PMID 37955580, PMID 39891377, PMID 9918480
R01HL173371 (1)