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
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 · NCT05791864 · Registry updated 2025-12-18Clinical trials
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| Trial | Conditions | Status | Phase | Updated | More info | NCT ID |
|---|---|---|---|---|---|---|
| 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 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Neuronal Ceroid-Lipofuscinoses, Nutritional and Metabolic Diseases, hereditary disease, inborn errors of metabolism, inherited neurodegenerative disorder, lysosomal lipid storage disorder, metabolic disease, nervous system disorder, neurodegenerative disease, neuronal ceroid lipofuscinosis 2 | Recruiting | Phase 1 / Phase 2 | 2025-12-18 | NCT05791864 |
1 results · Page 1 of 1
Organizations
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| Organizations | Linked trials |
|---|---|
| Tern Therapeutics, LLC | 1 |
2 results · Page 1 of 1
Research sites
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| Research sites | City | Country | Linked trials |
|---|---|---|---|
| Greater Ormond Street Hospital | London | United Kingdom | 1 |
| University Medical Center Hamburg-Eppendorf (UKE)- Childrens Hospital | Hamburg | Germany | 1 |