2 completed, 1 active not recruiting. 1 Phase 1.
| Phase | Trials | Status |
|---|---|---|
| Phase 1 | 1 | Active not recruiting 1 |
Follow Sanfilippo Syndrome Type A: new trials by email Literature Review of Sanfilippo Syndrome Type A trials Research Report on Sanfilippo Syndrome Type A
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 |
|---|---|---|---|---|---|---|
| Phase 1 Study of GC1130A in Patients With Sanfilippo Syndrome Type A (MPS IIIA) GC1130A - Sanfilippo Syndrome Type A | Phase 1 | Active not recruiting | GC Biopharma Corp | 9 | 2024-11-21 | 2026-09-08 |
| Neurobehavioral Phenotypes in MPS III Sanfilippo Syndrome Type A, Sanfilippo Syndrome Type B | Completed | University of Minnesota | 30 | 2010-12-01 | 2019-11-01 | |
| A Study of Patients With Sanfilippo Syndrome Type A (MPS IIIA) assessment - Sanfilippo Syndrome Type A | Completed | Shire | 25 | 2010-02-15 | 2021-06-11 |
Mucopolysaccharidosis III (3) · Cognitive Dysfunction (1) · Mucopolysaccharidosis I (1)
Carbohydrate Metabolism, Inborn Errors (3) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (3) · Connective Tissue Diseases (3) · Genetic Diseases, Inborn (3) · Lysosomal Storage Diseases (3) · Metabolic Diseases (3) · Metabolism, Inborn Errors (3) · Mucinoses (3) · Mucopolysaccharidoses (3) · Nutritional and Metabolic Diseases (3) · Skin and Connective Tissue Diseases (3) · Cognition Disorders (1) · Mental Disorders (1) · Neurocognitive Disorders (1)
GC Biopharma Corp (1) · Shire (1) · University of Minnesota (1)
United States (3) · South Korea (1)
Elsa G Shapiro, PhD (1) · Michael Potegal, PhD (1)
PMID 24582005, PMID 25770355, PMID 26787381, PMID 26956723
U54NS065768 (1)