Trials / Conditions / Mucopolysaccharidosis Type IIIA
Mucopolysaccharidosis Type IIIA
4 registered clinical trials studyying Mucopolysaccharidosis Type IIIA.
| Status | Trial | Sponsor | Phase |
|---|---|---|---|
| Active Not Recruiting | Study of DNL126 in Pediatric Participants With Mucopolysaccharidosis Type IIIA (Sanfilippo Syndrome Type A) NCT06181136 | Denali Therapeutics Inc. | Phase 1 / Phase 2 |
| Active Not Recruiting | Gene Therapy with Modified Autologous Hematopoietic Stem Cells for Patients with Mucopolysaccharidosis Type II NCT04201405 | University of Manchester | Phase 1 / Phase 2 |
| Unknown | Study of AAVrh10-h.SGSH Gene Therapy in Patients With Mucopolysaccharidosis Type IIIA (MPS IIIA) NCT03612869 | LYSOGENE | Phase 2 / Phase 3 |
| Completed | Natural History Studies of Mucopolysaccharidosis III NCT02037880 | Nationwide Children's Hospital | — |