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Study identifier: NCT05791864 Synced from ClinicalTrials.gov · July 29, 2026
● Recruiting

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

Condition: Neuronal Ceroid Lipofuscinosis Type 2  ·  Sponsor: Tern Therapeutics, LLC

PhasePhase 1/Phase 2
Planned participants16
Who can joinAll sexes, 12 Months to 84 Months
Healthy volunteersNo

About this study

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).

This description comes directly from the study's public registry record.

Talk to the study team

Tern Therapeutics Patient Advocacy  ·  202-644-8488  ·  patientadvocacy@terntx.com

Always discuss trial participation with your own doctor first.

Locations (2)

University Medical Center Hamburg-Eppendorf (UKE)- Childrens HospitalHamburg, GermanyRecruiting
Greater Ormond Street HospitalLondon, United KingdomRecruiting

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Source record: clinicaltrials.gov/study/NCT05791864