Drugs / SAF-301
Trials 2
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 1/2 | NCT02053064 | May 2013 → May 2017 | mucopolysaccharidosis type 3A | LYSOGENE | Completed | No outcome recorded |
| Phase 1/2 | NCT01474343 | Aug 2011 → May 2013 | mucopolysaccharidosis type 3A | LYSOGENE | Completed | No outcome recorded |
Evidence & citations 5 cited values
Every value below carries the sentence it was read from. 2 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | SAF-301 | ClinicalTrials.gov intervention name — accepted as the source's own label NCT01474343 ↗ |
| Modality | Gene therapy (AAV / viral vector) | “Adeno-associated Viral Vector Serotype 10 Carrying the Human SGSH and SUMF1 cDNAs” NCT01474343 ↗ |
| Route | Other | “direct injection of the investigational medicinal product SAF-301 to both sides of the brain” NCT01474343 ↗ |
| Target | SGSH | “carrying the human SGSH and SUMF1 cDNAs” NCT01474343 ↗ |
| Target | SUMF1 | “carrying the human SGSH and SUMF1 cDNAs” NCT01474343 ↗ |