Ocular manifestations of Neuronal Ceroid Lipofuscinosis (CLN) Type 2
Conditions
Brief summary
Ocular and overall AEs and SAEs through Day 360
Detailed description
Change from baseline in area of EZ loss as measured by SD-OCT at Day 180, Change from baseline in area of ellipsoid zone (EZ) loss as measured by SD-OCT at Day 360, Change from baseline in central subfield PR layer thickness as measured by SD-OCT at Day 180, Change from baseline in central subfield photoreceptor (PR) layer thickness as measured by SD-OCT at Day 360, Measurement of TTX-381 TPP1 in aqueous humor at Day 90, Measurement of TTX-381 TPP1 in aqueous humor at Day 360, TTX-381 detected by qPCR in urine and tears, Anti-AAV9 antibodies in serum, ATPA in serum and CSF
Interventions
None listed
Sponsors
Tern Therapeutics LLC
Eligibility
Sex/Gender
All
Age
0 Years to 17 Years
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Ocular and overall AEs and SAEs through Day 360 | — |
Secondary
| Measure | Time frame |
|---|---|
| Change from baseline in area of EZ loss as measured by SD-OCT at Day 180, Change from baseline in area of ellipsoid zone (EZ) loss as measured by SD-OCT at Day 360, Change from baseline in central subfield PR layer thickness as measured by SD-OCT at Day 180, Change from baseline in central subfield photoreceptor (PR) layer thickness as measured by SD-OCT at Day 360, Measurement of TTX-381 TPP1 in aqueous humor at Day 90, Measurement of TTX-381 TPP1 in aqueous humor at Day 360, TTX-381 detected by qPCR in urine and tears, Anti-AAV9 antibodies in serum, ATPA in serum and CSF | — |
Countries
Germany
Outcome results
None listed