# A First-in-Human, Open-Label, Dose-Escalation Study to Evaluate the Safety and Tolerability of Gene Therapy With TTX-381 for the Ocular Manifestations Associated With Neuronal Ceroid Lipofuscinosis Type 2 (CLN2) Disease

> **NCT05791864** · PHASE1,PHASE2 · RECRUITING · sponsor: **Tern Therapeutics, LLC** · enrollment: 16 (estimated)

## Conditions studied

- Neuronal Ceroid Lipofuscinosis Type 2

## Interventions

- **GENETIC:** TTX-381

## Key facts

- **NCT ID:** NCT05791864
- **Lead sponsor:** Tern Therapeutics, LLC
- **Sponsor class:** INDUSTRY
- **Phase:** PHASE1,PHASE2
- **Study type:** INTERVENTIONAL
- **Status:** RECRUITING
- **Start date:** 2023-05-17
- **Primary completion:** 2026-07-30
- **Final completion:** 2031-07-30
- **Target enrollment:** 16 (ESTIMATED)
- **Last updated:** 2025-12-18


## Primary source

ClinicalTrials.gov registry: https://clinicaltrials.gov/study/NCT05791864

## Citation

> US National Library of Medicine, ClinicalTrials.gov registration NCT05791864, "A First-in-Human, Open-Label, Dose-Escalation Study to Evaluate the Safety and Tolerability of Gene Therapy With TTX-381 for the Ocular Manifestations Associated With Neuronal Ceroid Lipofuscinosis Type 2 (CLN2) Disease". Retrieved via AI Analytics 2026-06-26 from https://api.ai-analytics.org/clinical/NCT05791864. Licensed CC0.

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*[Clinical trials dataset](/datasets/clinical-trials) · CC0 1.0*