Clinical trials: CLN6
Variant Late-Infantile Neuronal Ceroid Lipofuscinosis
Gene Therapy For Children With Variant Late Infantile Neuronal Ceroid Lipofuscinosis 6 (vLINCL6) Disease
This is a phase 1/2, open-label, single dose study to evaluate the safety and efficacy of AT-GTX-501 delivered intrathecally into the lumbar spinal cord region of participants with mild to moderate variant late infantile neuronal…
- Trial name
- NCT02725580
- Testing
- AT-GTX-501
- Where
- United States
- Volunteers
- 13 people
- Sponsor
- Emily de los Reyes