Actively 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
Led by Tern Therapeutics, LLC · Updated on 2025-12-18
16
Participants Needed
2
Research Sites
428 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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).
CONDITIONS
Official Title
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
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Has biallelic CLN2 mutations
- Has decreased leukocyte TPP1 activity
- Has clinical signs or symptoms consistent with CLN2 disease or an older sibling with confirmed CLN2 diagnosis
- Currently receives biweekly intracerebroventricular enzyme replacement therapy with cerliponase alfa
- Meets baseline disease condition based on age and central retinal thickness (CRT) as assessed by SD-OCT and confirmed by CRC
- Willing to adhere to protocol and 5-year visit schedule
- Sexually active females of childbearing potential or fertile males must use medically accepted contraception from screening until 6 weeks after treatment
- Previously administered TTX-381 and met eligibility criteria at that time, with trial recommendation by IDMC
You will not qualify if you...
- Any ocular or systemic condition preventing treatment or evaluation (eg, lens or corneal opacities, glaucoma, amblyopia, retinal abnormalities)
- Difference in CRT between eyes greater than 10 micrometers
- Prior severe hypersensitivity reaction to cerliponase alfa
- Contraindications to cerliponase alfa administration, including device issues
- Prior participation in a gene therapy study, except with approval for compassionate use cases
- Prior participation in ocular clinical trials except limited prior cerliponase alfa injections with washout
- Prior intraocular injections other than specified exceptions
- Participation in nonocular investigational drug studies within 6 months except for cerliponase alfa
- Ocular surgery within 6 months except subretinal TTX-381 administration
- Prior bone marrow transplant
- Use of specified medications within 30 days prior to treatment
- Known sensitivity or contraindications to peri-operative medications
- Contraindications to systemic immunosuppression
- Severe kidney or liver insufficiency
- Mutations in other CLN genes or inherited retinal disease genes
- Contraindications to intraocular surgery
- Positive pregnancy test at screening for females of childbearing potential
- Any condition preventing follow-up or making participant unsuitable
- Positive COVID-19 PCR test within 4 weeks or persistent COVID-19 symptoms
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 2 locations
1
University Medical Center Hamburg-Eppendorf (UKE)- Childrens Hospital
Hamburg, Germany
Actively Recruiting
2
Greater Ormond Street Hospital
London, United Kingdom, Wc1N 3JH
Actively Recruiting
Research Team
T
Tern Therapeutics Patient Advocacy
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
SINGLE
Allocation
NON_RANDOMIZED
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
3
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