Not applicableLooking for participantsNCT03333200What this trial is testingLongitudinal Study of Neurodegenerative DisordersWho this might be right forMLDKrabbe DiseaseALD+24 more University of Pittsburgh 1,500
Early research (Phase 1)Active Not RecruitingNCT04737460What this trial is testingStudy for the Treatment for CLN7 DiseaseWho this might be right forCLN7 Benjamin Greenberg 4
Not applicableWithdrawnNCT04480476What this trial is testingA Retrospective, Natural History Study in Children With CLN2Who this might be right forLate-infantile Neuronal Ceroid Lipofuscinosis Type 2 (CLN2) REGENXBIO Inc.
Not applicableLooking for participantsNCT05007990What this trial is testingCaregiving Networks Across Disease Context and the Life CourseWho this might be right forInherited Metabolic DisordersUndiagnosed DiseasesBatten's Disease+2 more National Human Genome Research Institute (NHGRI) 2,800
Post-approval studies (Phase 4)Study completedNCT00028262What this trial is testingCystagon to Treat Infantile Neuronal Ceroid LipofuscinosisWho this might be right forInfantile Neronal Ceroid Lipofuscinosis Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) 10
Not applicableActive Not RecruitingNCT04476862What this trial is testingCerliponase Alfa Observational Study in the USWho this might be right forLate-Infantile Neuronal Ceroid Lipofuscinosis Type 2 BioMarin Pharmaceutical 35
Testing effectiveness (Phase 2)Study completedNCT02725580What this trial is testingGene Therapy For Children With Variant Late Infantile Neuronal Ceroid Lipofuscinosis 6 (vLINCL6) DiseaseWho this might be right forVariant Late-Infantile Neuronal Ceroid Lipofuscinosis Emily de los Reyes 13
Not applicableLooking for participantsNCT01873924What this trial is testingClinical and Neuropsychological Investigations in Batten DiseaseWho this might be right forNeuronal Ceroid LipofuscinosisNeuronal Ceroid Lipofuscinosis CLN1Neuronal Ceroid Lipofuscinosis CLN2+7 more University of Rochester 500
Not applicableUnknownNCT04461561What this trial is testingUsing NPT to Evaluate Providing PPC as ELNEC-PPC WBT for NursesWho this might be right forCancerCardiac AnomalyCystic Fibrosis+4 more Altoosi University College 172
Not applicableLooking for participantsNCT04613089What this trial is testingNatural History and Longitudinal Clinical Assessments in NCL / Batten Disease, the International DEM-CHILD DatabaseWho this might be right forNeuronal Ceroid LipofuscinosisBatten DiseaseCLN1 Disease+12 more Universitätsklinikum Hamburg-Eppendorf 500
Not applicableEnded earlyNCT01698229What this trial is testingCollection of Cerebrospinal Fluid in Healthy ChildrenWho this might be right forLate Infantile Neuronal Ceroid LipofuscinosisBatten Disease Weill Medical College of Cornell University 32
Early research (Phase 1)Study completedNCT00337636What this trial is testingStudy of HuCNS-SC Cells in Patients With Infantile or Late Infantile Neuronal Ceroid Lipofuscinosis (NCL)Who this might be right forNeuronal Ceroid Lipofuscinosis StemCells, Inc. 6
Not applicableLooking for participantsNCT06203106What this trial is testingNYSCF Scientific Discovery BiobankWho this might be right forALSAmyotrophic Lateral SclerosisAlzheimer Disease+26 more New York Stem Cell Foundation Research Institute 10,000
Not applicableEnded earlyNCT04974307What this trial is testingOptic-to-Audio Device in a Pediatric Cohort With CLN3-related Conditions or Low VisionWho this might be right forCLN3-related DisordersNon-CLN3 Related Low Vision Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) 11
Testing effectiveness (Phase 2)Study completedNCT02485899What this trial is testingAn Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 190 in Patients With CLN2 DiseaseWho this might be right forJansky-Bielschowsky DiseaseBatten DiseaseLate-Infantile Neuronal Ceroid Lipofuscinosis Type 2+2 more BioMarin Pharmaceutical 23
Large-scale testing (Phase 3)Not Yet RecruitingNCT04637282What this trial is testingSafety, Tolerability, and Efficacy of PLX-200 in Patients With CLN3Who this might be right forJuvenile Neuronal Ceroid Lipofuscinosis Polaryx Therapeutics, Inc. 39
Testing effectiveness (Phase 2)Study completedNCT01907087What this trial is testingA 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) DiseaseWho this might be right forJansky-Bielschowsky DiseaseBatten DiseaseLate-Infantile Neuronal Ceroid Lipofuscinosis Type 2+1 more BioMarin Pharmaceutical 24
Large-scale testing (Phase 3)Study completedNCT00176904What this trial is testingStem Cell Transplant for Inborn Errors of MetabolismWho this might be right forAdrenoleukodystrophyMetachromatic LeukodystrophyGloboid Cell Leukodystrophy+8 more Masonic Cancer Center, University of Minnesota 135
Not applicableStudy completedNCT00151268What this trial is testingGenotype - Phenotype Correlations of LINCLWho this might be right forBatten DiseaseLate-Infantile Neuronal Ceroid Lipfuscinosis Weill Medical College of Cornell University 18
Not applicableEnrolling By InvitationNCT03862274What this trial is testingExamining Developmental Outcomes of Children Diagnosed With CLN2 DiseaseWho this might be right forBatten DiseaseCLN2Neuronal Ceroid-Lipofuscinoses Jessica Scherr 30