Gene Therapy Study for Children With CLN5 Batten Disease
← catalyst calendarNCT05228145 · readout ≤ 834 d
Sponsored by Neurogene Inc. (industry) · NGNE — their whole pipeline →.
Phase
Phase 1/2
Status
Active, not recruiting
Study type
Interventional
Design
Non randomized · None · Treatment
Enrollment
6actual
Sites
2
Countries
United Kingdom, United States
Every value on this page is a field the sponsor filed with ClinicalTrials.gov, reproduced. Dates are their own estimates, revised as a study runs, unless the registry marks them actual. sources →
Source: ClinicalTrials.gov, retrieved 2026-08-19
Dates
each axis at the precision it was filed, with the registry's own basis| Date | Filed | Basis | What it is |
|---|---|---|---|
| Start | 2022-01-31 | actual | When the study began enrolling |
| Primary completion | Nov 2028 | estimated | The date the last participant is measured for the primary outcome — the readout window |
| Study completion | Nov 2028 | estimated | The whole study's end, after follow-up |
| First posted | 2022-02-08 | actual | When this record first appeared on the registry |
| Results posted | — | When the sponsor posted results to the registry | |
| Record updated | 2024-08-12 | actual | The sponsor's own last edit to this record — every projection above is as current as this date |
What it studies
Condition
- Neuronal Ceroid Lipofuscinosis CLN5
Intervention
- Genetic: NGN-101
Primary outcomes
what the primary-completion date above is the date OFIncidence of Treatment Emergent Adverse Events (TEAEs)
measured 5 years (multiple visits)
Incidence of Serious Adverse Events (SAEs)
measured 5 years (multiple visits)
Incidence of clinical laboratory abnormalities
measured 5 years (multiple visits)
Incidence of new nerve conduction study (NCS) abnormalities
measured 5 years (multiple visits)
Incidence of new physical and neurologic exam abnormalities
measured 5 years (multiple visits)
Publications
- PMID 37942487 — linked by the registry
- PMID 37614821 — linked by the registry
Linked on the registry record. A “linked by the registry” entry is NLM's own automated PubMed match, not a claim by the sponsor that the paper reports this study.
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