RecruitingPhase 1/2

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

Germany, United Kingdom16 participantsStarted 2023-05-17

Plain-language summary

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).

Age range12 Months – 84 Months

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Ocular and overall AE and SAEs through Day 360

Timeframe: 360 days

NCT IDNCT05791864

SponsorTern Therapeutics, LLC

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2026-07-30

Tern Therapeutics Patient Advocacy