Neuronal Ceroid-Lipofuscinoses clinical trials
The registry lists 49 studies for Neuronal Ceroid-Lipofuscinoses across more than 10 countries. 13 are recruiting or about to start. 37 of them are mainly about Neuronal Ceroid-Lipofuscinoses (how this is decided). Per ClinicalTrials.gov, last updated 2026-11-12.
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Who runs them
Weill Medical College of Cornell University (6) · Emily de los Reyes (4) · BioMarin Pharmaceutical (4) · Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) (3) · IRCCS Fondazione Stella Maris (2) · REGENXBIO Inc. (2) · StemCells, Inc. (2) · University of Rochester (2)
What is being tested here
Drugs under test in Neuronal Ceroid-Lipofuscinoses studies, those with the most open studies first. Medicines only given beside them are not counted. How drugs are counted
Coming up
Studies recruiting or running, from their records. “Main data collection” ends at the primary completion date, the sponsor’s own estimate, which often moves.
All 6
- Dec 2026Longitudinal Assessment of Atypical Tripeptidyl Peptidase 1 Enzyme Deficiency Patients · Children's Hospital of Orange County
- Dec 2026Long-Term Follow Up of CLN6 Batten Disease Subjects Following Gene Transfer · Emily de los Reyes
- Dec 2026Natural History of Neuronal Ceroid Lipofuscinosis, Batten's CLN6 Diseae · Emily de los Reyes
- Dec 2026A Registrational Study of TTX-381 to Treat Vision Loss in CLN2 Disease · Tern Therapeutics, LLC
- Aug 2027Gene Therapy Trial for CLN6 Batten Disease · The Charlotte and Gwenyth Gray Foundation
- Oct 2027UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells · Joanne Kurtzberg, MD
Who these studies take
Of the 11 Neuronal Ceroid-Lipofuscinoses studies recruiting now, 7 (64%) set no upper age limit and 2 (18%) stop at 75 or younger. 10 (91%) take children under 18. 0 (0%) are for women only and 0 (0%) for men only; 2 (18%) take healthy volunteers.
| Age | Women | Men |
|---|---|---|
| 10 | 9 (82%) | 9 (82%) |
| 18 | 10 (91%) | 10 (91%) |
| 30 | 9 (82%) | 9 (82%) |
| 40 | 9 (82%) | 9 (82%) |
| 50 | 9 (82%) | 9 (82%) |
| 65 | 9 (82%) | 9 (82%) |
| 75 | 9 (82%) | 9 (82%) |
| 85 | 9 (82%) | 9 (82%) |
Across all 49 studies, recruiting or not: 51% stop at 75 or younger and 51% at 65; 92% take children; 0% are for women only and 0% for men only; 10% take healthy volunteers. From each study’s registry entry; the criteria on its page decide.
Studies
Recruiting first, then most recently updated. Showing 49 of 49. search them.
- Clinical and Neuropsychological Investigations in Batten DiseaseRecruitingNCT018739241 country
- Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at SanfordRecruitingNCT017931681 country
- A Registrational Study of TTX-381 to Treat Vision Loss in CLN2 DiseaseRecruitingNCT05791864Phase 1/22 countries
- Investigations of Juvenile Neuronal Ceroid LipofuscinosisRecruitingNCT033073041 country
- Caregiving Networks Across Disease Context and the Life CourseRecruitingNCT050079901 country
- UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like CellsRecruitingNCT02254863Phase 11 country
- Inherited Retinal Degenerative Disease RegistryRecruitingNCT024359401 country
- Gene Therapy Trial for CLN6 Batten DiseaseNot yet recruitingNCT07582484Phase 1/21 country
- Italian NCL Registry: a Registry for NCL as an Integration Tool for Future Therapeutic StrategiesRecruitingNCT068448771 country
- Longitudinal Study of Neurodegenerative DisordersRecruitingNCT033332001 country
- Examining Developmental Outcomes of Children Diagnosed With CLN2 DiseaseEnrolling by invitationNCT038622741 country
- NYSCF Scientific Discovery BiobankRecruitingNCT062031061 country
- Natural History and Longitudinal Clinical Assessments in NCL / Batten Disease, the International DEM-CHILD DatabaseRecruitingNCT046130891 country
- Functional Near-Infrared Spectroscopy (fNIRS) Combined With Diffuse Correlation Spectroscopy (DCS) in Neurocognitive Disease as Compared to Healthy Neurotypical ControlsCompletedNCT056422211 country
- Safety, Tolerability, and Efficacy of PLX-200 in Patients With CLN3WithdrawnNCT04637282Phase 3
- Cerliponase Alfa Observational Study in the USActive, not recruitingNCT044768621 country
- Gene Therapy for Children With CLN3 Batten DiseaseActive, not recruitingNCT03770572Phase 1/21 country
- Intravitreal ERT to Prevent Retinal Disease Progression in Children With CLN2Active, not recruitingNCT05152914Phase 1/21 country
- Longitudinal Assessment of Atypical Tripeptidyl Peptidase 1 Enzyme Deficiency PatientsActive, not recruitingNCT040982111 country
- Study for the Treatment for CLN7 DiseaseActive, not recruitingNCT04737460Phase 11 country
- Gene Therapy For Children With Variant Late Infantile Neuronal Ceroid Lipofuscinosis 6 (vLINCL6) DiseaseCompletedNCT02725580Phase 1/21 country
- Long-Term Follow Up of CLN6 Batten Disease Subjects Following Gene TransferActive, not recruitingNCT042732431 country
- An Open-label Safety, Pharmacokinetic, and Efficacy Study of Miglustat for the Treatment of Subjects With Batten Ceroid Lipofuscinosis, Neuronal 3 (CLN3) DiseaseCompletedNCT05174039Phase 1/21 country
- Baby Detect : Genomic Newborn ScreeningCompletedNCT056874741 country
- Natural History Study of Batten DiseaseTerminatedNCT046445491 country
- Natural History of Neuronal Ceroid Lipofuscinosis, Batten's CLN6 DiseaeActive, not recruitingNCT032854251 country
- A Safety, Tolerability, and Efficacy Study of BMN 190 in Pediatric Patients < 18 Years of Age With CLN2 DiseaseCompletedNCT02678689Phase 24 countries
- Gene Therapy Study for Children With CLN5 Batten DiseaseActive, not recruitingNCT05228145Phase 1/22 countries
- Human Placental-Derived Stem Cell TransplantationCompletedNCT01586455Phase 11 country
- An Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 190 in Patients With CLN2 DiseaseCompletedNCT02485899Phase 1/24 countries
- Trehalose in Subjects With Neuronal Ceroid LipofuscinosesCompletedNCT048082971 country
- An Observational Study in Children With CLN2 Batten DiseaseWithdrawnNCT04462692
- A Retrospective, Natural History Study in Children With CLN2WithdrawnNCT04480476
- N-3 Polyunsaturated Fatty Acids Prevent Postoperative Recurrence of Crohn's DiseaseStatus unknownNCT04761952Not applicable
- Safety Study of a Gene Transfer Vector (Rh.10) for Children With Late Infantile Neuronal Ceroid Lipofuscinosis (LINCL)CompletedNCT01161576Phase 11 country
- AAVRh.10 Administered to Children With Late Infantile Neuronal Ceroid LipofuscinosisCompletedNCT01414985Phase 1/21 country
- Genotype-Phenotype Correlations of Late Infantile Neuronal Ceroid LipofuscinosisCompletedNCT010354241 country
- Using NPT to Evaluate Providing PPC as ELNEC-PPC WBT for NursesStatus unknownNCT04461561Not applicable1 country
- Safety Study of a Gene Transfer Vector for Children With Late Infantile Neuronal Ceroid LipofuscinosisCompletedNCT00151216Phase 11 country
- Cellcept for Treatment of Juvenile Neuronal Ceroid LipofuscinosisCompletedNCT01399047Phase 21 country
- A Phase 1/2 Open-Label Dose-Escalation Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Efficacy of Intracerebroventricular BMN 190 in Patients With Late-Infantile Neuronal Ceroid Lipofuscinosis (CLN2) DiseaseCompletedNCT01907087Phase 1/24 countries
- Stem Cell Transplant for Inborn Errors of MetabolismCompletedNCT00176904Phase 2/31 country
- Natural Evolution of AV Conduction Disorders in Patients Implanted With Cardiac PacemakersCompletedNCT0053103711 countries
- Cystagon to Treat Infantile Neuronal Ceroid LipofuscinosisCompletedNCT00028262Phase 41 country
- Genotype - Phenotype Correlations of LINCLCompletedNCT00151268
- Neuronal Ceroid Lipofuscinosis and Associated Sleep AbnormalitiesCompletedNCT019667571 country
- Collection of Cerebrospinal Fluid in Healthy ChildrenTerminatedNCT016982291 country
- Study of HuCNS-SC Cells in Patients With Infantile or Late Infantile Neuronal Ceroid Lipofuscinosis (NCL)CompletedNCT00337636Phase 11 country
- Safety and Efficacy Study of HuCNS-SC in Subjects With Neuronal Ceroid LipofuscinosisWithdrawnNCT01238315Phase 11 country