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

Recruiting now Phase 1, PHASE2 Last updated 18 December 2025

What this trial tests

Phase 1, PHASE2 trial testing TTX-381 in Neuronal Ceroid Lipofuscinosis Type 2 in 16 participants. Currently enrolling.

Timeline

17 May 2023

Primary endpoint
30 July 2026

30 July 2031

Quick facts

Lead sponsor	Tern Therapeutics, LLC
Phase	Phase 1, PHASE2
Status	Recruiting now
Study type	INTERVENTIONAL
Allocation	non randomized
Design	sequential
Masking	single
Primary purpose	treatment
Enrollment	16
Start date	17 May 2023
Primary completion	30 July 2026
Estimated completion	30 July 2031
Sites	2 locations across United Kingdom, Germany

Drugs / interventions tested

• TTX-381

Conditions studied

• Neuronal Ceroid Lipofuscinosis Type 2 — all drugs for Neuronal Ceroid Lipofuscinosis Type 2 →

Sponsor

Tern Therapeutics, LLC

Who can join

Adults 12 Months to 84 Months, any sex, with Neuronal Ceroid Lipofuscinosis Type 2. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

This is a first-in-human, open-label, single ascending dose study of TTX-381 for the treatment of ocular manifestations of CLN2 (Batten disease).

Publications & conference data

7 peer-reviewed publications reference this trial (live from Europe PMC):

1. Neuronal Ceroid Lipofuscinosis-Concepts, Classification, and Avenues for Therapy.
   Zhang Y, Du B, Zou M, Peng B, et al · · 2025 · cited 7× · PMID 39925015 · DOI 10.1111/cns.70261
2. Extracellular Vesicles as Tools for Crossing the Blood-Brain Barrier to Treat Lysosomal Storage Diseases.
   Lerussi G, Villagrasa-Araya V, Moltó-Abad M, Del Toro M, et al · · 2025 · cited 7× · PMID 39860010 · DOI 10.3390/life15010070
3. Inherited Retinal Degenerations and Non-Neovascular Age-Related Macular Degeneration: Progress and Unmet Needs.
   Duncan JL, Bowman A, Laster A, Gelfman C, et al · · 2024 · cited 5× · PMID 39688851 · DOI 10.1167/tvst.13.12.28
4. Neuronal ceroid lipofuscinosis: underlying mechanisms and emerging therapeutic targets.
   Ziółkowska EA, Takahashi K, Dickson PI, Sardiello M, et al · · 2025 · cited 4× · PMID 40908342 · DOI 10.1038/s41582-025-01132-4
5. Autosomal Recessive Cerebellar Ataxias: Translating Genes to Therapies.
   Fogel BL, Klopstock T, Lynch DR, Maltecca F, et al · · 2025 · cited 2× · PMID 40464291 · DOI 10.1002/ana.27271
6. Genetic engineering and the eye.
   Murphy R, Martin KR. · · 2025 · cited 2× · PMID 39516652 · DOI 10.1038/s41433-024-03441-2
7. Adeno-associated virus-mediated gene therapy for rare pediatric neurogenetic diseases: Current status and outlook.
   Wang X, Lin X, He H, Peng J. · · 2023 · PMID 38044650 · DOI 10.11817/j.issn.1672-7347.2023.220639