Clinical Trials
| NCL Subtype | Sponsor | Intervention | Status | Link to Trial Info |
|---|---|---|---|---|
| CLN1 | JCR Pharmaceuticals | AGT-194 enzyme replacement IV | Investigator-led study – 1 patient treated (Germany). Study no longer active. R&D actively continues. Seeking to initiate collaborative CLN1 natural history study. | |
| CLN1 | Taysha Gene Therapies | TSH-118 Gene therapy (AAV9; CNS) | Ph 1/2 paused (since early 2022) Continuing to explore partnership opportunities Single patient dosed under investigator-initiated study at RUSH, Chicago. Joint Statement to the Global Batten Disease Community. | |
| CLN2 | BioMarin | Cerliponase alfa - enzyme replacement; CNS | FDA Approves BRINEURA® for Children Under 3 Years with CLN2 Disease NCT04476862 Ph 4 -Observational/safety, ongoing (USA) | |
| CLN2 | BioMarin | Cerliponase alfa - Ocular | NCT05152914 Ph 1/2 - Active, not recruiting (USA) | |
| CLN2 | LEXEO Therapeutics | LX1004 Gene therapy (AAVrh10; CNS) | Program no longer active. Lexeo seeks ”strategic partners or alternatives” to continue the program's development. Lexeo Letter to the Batten Community. Joint Statement to the Global Batten Disease Community. | |
| CLN2 | Orphion Therapeutics | ORPH-301 Gene therapy (AAV2; ocular) | Preclinical - No update at this time | |
| CLN2 | REGENXBIO | RGX-181 Gene therapy (AAV9; CNS) | *August 2024 UPDATE* – Tern Therapeutics acquired RGX-181 from REGENXBIO through a global licensing agreement. REGENXBIO Letter to the CLN2 community | |
| CLN2 | REGENXBIO | RGX-381 Gene therapy (AAV9; ocular) | *August 2024 UPDATE* – Tern Therapeutics acquired RGX-181 (or RGX-381) from REGENXBIO through a global licensing agreement. REGENXBIO Letter to the CLN2 community | |
| CLN2 | Tern Therapeutics | TTX-381 Gene therapy (AAV9; ocular) | *August 2024 UPDATE* – Tern Therapeutics acquired RGX-381 from REGENXBIO through a global licensing agreement; now designated "TTX-381" Tern Therapeutics Launches with $15 Million Financing and Pipeline in CLN2 Batten Disease | |
| CLN2 | Tern Therapeutics | TTX-181 Gene therapy (AAV9; CNS) | *August 2024 UPDATE* – Tern Therapeutics acquired RGX-181 from REGENXBIO through a global licensing agreement; now designated "TTX-181" Tern Therapeutics Launches with $15 Million Financing and Pipeline in CLN2 Batten Disease | |
| CLN3 | University of North Carolina (UNC) Hospitals and ForeBatten Foundation | FBF-001 (Zebronkysen); Antisense Oligonucleotide (ASO) | *SEPTEMBER 2024* N-of-2 study announced; currently active. | |
| CLN3 | Amicus Therapeutics | AT-GTX-502 Gene therapy (AAV9; CNS) | NCT03770572 Ph1/2 – active, not recruiting (USA) Program rights and responsibilities returned to Nationwide Children’s Hospital, OH. Joint Statement to the Global Batten Disease Community. | |
| CLN3 | Beyond Batten Disease Foundation/Theranexus | Batten-1 (miglustat) | NCT05174039 Ph 1/2 - active, not recruiting (USA) Ph 3 pediatric study expected to commence 2025 (international sites) *NOVEMBER 2024 UPDATE* Theranexus and BBDF present final Phase I/II Batten-1 trial data at Child Neurology Society Annual Meeting. | |
| CLN3 | Polaryx Therapeutics | PLX-200 (gemfibrozil) | NCT04637282 Ph 3 - not yet recruiting (no movement since Nov 2020) - No update at this time | |
| CLN5 | Neurogene | NGN-101 Gene therapy (AAV9; CNS & ocular) | NCT05228145 Ph 1/2 Active; not recruiting (USA & UK) *AUGUST 2024 UPDATE* Study now fully enrolled (n=6). *NOVEMBER 2024 UPDATE* FDA RMAT Application denied, Neurogene evaluating best path forward for the program. | |
| CLN6 | Amicus Therapeutics | AT-GTX-501 Gene therapy (AAV9; CNS) | Program rights and responsibilities returned to Nationwide Children’s Hospital, OH. Joint Statement to the Global Batten Disease Community. | |
| CLN7 | UT Southwestern/Elpida Therapeutics | Gene therapy (AAV9; CNS) | NCT04737460 Ph1 active; not recruiting (USA) Assessments ongoing; Planning underway to move to Phase 2 (Elpida Therapeutics). |
Natural History Studies
| NCL Subtype | Sponsor | Intervention | Status | Link to Trial Info |
|---|---|---|---|---|
| Natural History Study - all NCL types | University of Rochester, NY | All NCL types | NCT01873924 recruiting international patients – in-person and/or virtual visits | |
| Natural History Study - CLN3 only | National Institutes of Health (NIH) | CLN3; biomarker study | NCT03307304 recruiting international patients – in-person visits (+ virtual visits) | |
| Natural History Study - all NCL types | UKE Hamburg-Eppendorf, Germany | All NCL types | NCT04613089 recruiting international patients – in-person and/or virtual visits |
