Clinical Trials

NCL SubtypeSponsorInterventionStatusLink to Trial Info
CLN1JCR PharmaceuticalsAGT-194 enzyme replacement IV
Investigator-led study – 1 patient treated (Germany). Study no longer active. R&D actively continues.
*FEBRUARY 2024 UPDATE* Seeking to initiate collaborative CLN1 natural history study.
CLN1Taysha Gene TherapiesTSH-118 Gene therapy (AAV9; CNS)
Ph 1/2 paused (since early 2022)
Continuing to explore partnership opportunities
*FEBRUARY 2024 UPDATE* Single patient dosed under investigator-initiated study at RUSH, Chicago. Joint Statement to the Global Batten Disease Community.
CLN2BioMarinCerliponase alfa - enzyme replacement; CNS
NCT04476862 Ph 4 -Observational/safety, ongoing (USA)
https://clinicaltrials.gov/study/NCT04476862
CLN2BioMarinCerliponase alfa - Ocular
NCT05152914 Ph 1/2 - *NOVEMBER 2023 UPDATE* Active, not recruiting (USA)
https://clinicaltrials.gov/study/NCT05152914
CLN2LEXEO TherapeuticsLX1004 Gene therapy (AAVrh10; CNS)
*MARCH 2024 UPDATE* Program no longer active.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.
CLN2Orphion TherapeuticsORPH-301 Gene therapy (AAV2; ocular)
Preclinical - No update at this time
CLN2REGENXBIORGX-181 Gene therapy (AAV9; CNS)
Halted
REGENXBIO Letter to the Batten Community
Joint Statement to Our Worldwide Batten Community
*FEBRUARY 2024 UPDATE* RGX continues to actively pursue partnership opportunities for the program. Preliminary data presented at WORLDSymposium.
CLN2REGENXBIORGX-381 Gene therapy (AAV9; ocular)
Halted
REGENXBIO Letter to the Batten Community
Joint Statement to Our Worldwide Batten Community.
*FEBRUARY 2024 UPDATE* RGX continues to actively pursue partnership opportunities for the program. Preliminary data presented at WORLDSymposium.
https://clinicaltrials.gov/study/NCT05791864
CLN3Amicus TherapeuticsAT-GTX-502 Gene therapy (AAV9; CNS)
NCT03770572 Ph1/2 – active, not recruiting (USA)
*FEBRUARY 2024 UPDATE* Program rights and responsibilities returned to Nationwide Children’s Hospital, OH. Joint Statement to the Global Batten Disease Community.
https://clinicaltrials.gov/study/NCT03770572
CLN3Beyond Batten Disease Foundation/TheranexusBatten-1 (miglustat)
NCT05174039 Ph 1/2 - active, not recruiting (USA)
Phase I/II study 12-month preliminary data announced, Sept. 29, 2023
Ph 3 pediatric study expected to commence 2024
(international sites)
*FEBRUARY 2024 UPDATE* Theranexus announces unanticipated delays. Options are currently being considered, updated timeline TBA soon.
https://clinicaltrials.gov/study/NCT05174039
CLN3Polaryx TherapeuticsPLX-200 (gemfibrozil)
NCT04637282 Ph 3 - not yet recruiting
(no movement since Nov 2020) - No update at this time
https://clinicaltrials.gov/study/NCT04637282
CLN5NeurogeneNGN-101 Gene therapy (AAV9; CNS & ocular)
NCT05228145 Ph 1/2 actively enrolling (USA & UK)
*JANUARY 2024 UPDATE* Cohorts 1 & 2 fully enrolled. Final higher dose cohort now enrolling.
Read the press release here.
https://clinicaltrials.gov/study/NCT05228145
CLN6Amicus TherapeuticsAT-GTX-501 Gene therapy (AAV9; CNS)
*FEBRUARY 2024 UPDATE* Program rights and responsibilities returned to Nationwide Children’s Hospital, OH. Joint Statement to the Global Batten Disease Community.
CLN7UT Southwestern/Elpida TherapeuticsGene therapy (AAV9; CNS)
NCT04737460 Ph1 active; not recruiting (USA)
Assessments ongoing;
*JANUARY 2024 UPDATE* Planning underway to move to Phase 2 (Elpida Therapeutics).
https://clinicaltrials.gov/study/NCT04737460
CLN7NeurogenePreclinical + natural history study
Discontinued

Natural History Studies

NCL SubtypeSponsorInterventionStatusLink to Trial Info
Natural History Study - all NCL typesUniversity of Rochester, NYAll NCL typesNCT01873924 recruiting international patients – in-person and/or virtual visits
https://clinicaltrials.gov/study/NCT01873924
Natural History Study - CLN3 onlyNational Institutes of Health (NIH)CLN3; biomarker studyNCT03307304 recruiting international patients – in-person visits (+ virtual visits)
https://clinicaltrials.gov/study/NCT03307304
Natural History Study - all NCL typesUKE Hamburg-Eppendorf, GermanyAll NCL typesNCT04613089 recruiting international patients – in-person and/or virtual visits
https://clinicaltrials.gov/study/NCT04613089