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Results for “Neuronal Ceroid Lipofuscinosis CLN2”

23 trials

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Showing 20 of 23 results

Early research (Phase 1)Study completedNCT00151216
What this trial is testing

Safety Study of a Gene Transfer Vector for Children With Late Infantile Neuronal Ceroid Lipofuscinosis

Who this might be right for
Batten DiseaseLate Infantile Neuronal Ceroid Lipofuscinosis
Weill Medical College of Cornell University 10
Not applicableActive Not RecruitingNCT04098211
What this trial is testing

Longitudinal Assessment of Atypical Tripeptidyl Peptidase 1 Enzyme Deficiency Patients

Who this might be right for
Neuronal Ceroid-LipofuscinosesNeuronal Ceroid Lipofuscinosis CLN2Spinocerebellar Ataxia, Autosomal Recessive 7
Children's Hospital of Orange County 5
Testing effectiveness (Phase 2)Active Not RecruitingNCT05152914
What this trial is testing

Intravitreal ERT to Prevent Retinal Disease Progression in Children With CLN2

Who this might be right for
Neuronal Ceroid Lipofuscinosis Type 2
David L Rogers, MD 5
Not applicableStudy completedNCT01035424
What this trial is testing

Genotype-Phenotype Correlations of Late Infantile Neuronal Ceroid Lipofuscinosis

Who this might be right for
Batten DiseaseLate Infantile Neuronal Ceroid Lipofuscinosis
Weill Medical College of Cornell University 48
Testing effectiveness (Phase 2)Study completedNCT01907087
What this trial is testing

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) Disease

Who this might be right for
Jansky-Bielschowsky DiseaseBatten DiseaseLate-Infantile Neuronal Ceroid Lipofuscinosis Type 2+1 more
BioMarin Pharmaceutical 24
Not applicableWithdrawnNCT04480476
What this trial is testing

A Retrospective, Natural History Study in Children With CLN2

Who this might be right for
Late-infantile Neuronal Ceroid Lipofuscinosis Type 2 (CLN2)
REGENXBIO Inc.
Not applicableEnded earlyNCT01698229
What this trial is testing

Collection of Cerebrospinal Fluid in Healthy Children

Who this might be right for
Late Infantile Neuronal Ceroid LipofuscinosisBatten Disease
Weill Medical College of Cornell University 32
Not applicableEnrolling By InvitationNCT03862274
What this trial is testing

Examining Developmental Outcomes of Children Diagnosed With CLN2 Disease

Who this might be right for
Batten DiseaseCLN2Neuronal Ceroid-Lipofuscinoses
Jessica Scherr 30
Early research (Phase 1)Study completedNCT00337636
What this trial is testing

Study of HuCNS-SC Cells in Patients With Infantile or Late Infantile Neuronal Ceroid Lipofuscinosis (NCL)

Who this might be right for
Neuronal Ceroid Lipofuscinosis
StemCells, Inc. 6
Testing effectiveness (Phase 2)Looking for participantsNCT05791864
What this trial is testing

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

Who this might be right for
Neuronal Ceroid Lipofuscinosis Type 2
Tern Therapeutics, LLC 16
Testing effectiveness (Phase 2)Not Yet RecruitingNCT07582484
What this trial is testing

Gene Therapy Trial for CLN6 Batten Disease

Who this might be right for
CLN6Batten DiseaseBatten's Disease+2 more
The Charlotte and Gwenyth Gray Foundation 12
Testing effectiveness (Phase 2)Study completedNCT02678689
What this trial is testing

A Safety, Tolerability, and Efficacy Study of BMN 190 in Pediatric Patients < 18 Years of Age With CLN2 Disease

Who this might be right for
Jansky-Bielschowsky DiseaseBatten DiseaseLate-Infantile Neuronal Ceroid Lipofuscinosis Type 2+2 more
BioMarin Pharmaceutical 14
Early research (Phase 1)WithdrawnNCT01238315
What this trial is testing

Safety and Efficacy Study of HuCNS-SC in Subjects With Neuronal Ceroid Lipofuscinosis

Who this might be right for
Neuronal Ceroid Lipofuscinosis
StemCells, Inc.
Not applicableLooking for participantsNCT04613089
What this trial is testing

Natural History and Longitudinal Clinical Assessments in NCL / Batten Disease, the International DEM-CHILD Database

Who this might be right for
Neuronal Ceroid LipofuscinosisBatten DiseaseCLN1 Disease+12 more
Universitätsklinikum Hamburg-Eppendorf 500
Testing effectiveness (Phase 2)Study completedNCT01414985
What this trial is testing

AAVRh.10 Administered to Children With Late Infantile Neuronal Ceroid Lipofuscinosis

Who this might be right for
Late Infantile Neuronal Ceroid LipofuscinosisBatten Disease
Weill Medical College of Cornell University 8
Testing effectiveness (Phase 2)Study completedNCT02725580
What this trial is testing

Gene Therapy For Children With Variant Late Infantile Neuronal Ceroid Lipofuscinosis 6 (vLINCL6) Disease

Who this might be right for
Variant Late-Infantile Neuronal Ceroid Lipofuscinosis
Emily de los Reyes 13
Not applicableStudy completedNCT00151268
What this trial is testing

Genotype - Phenotype Correlations of LINCL

Who this might be right for
Batten DiseaseLate-Infantile Neuronal Ceroid Lipfuscinosis
Weill Medical College of Cornell University 18
Not applicableStudy completedNCT05687474
What this trial is testing

Baby Detect : Genomic Newborn Screening

Who this might be right for
Congenital Adrenal HyperplasiaFamilial Hyperinsulinemic Hypoglycemia 1Phosphoglucomutase 1 Deficiency+121 more
Centre Hospitalier Universitaire de Liege 6,824
Not applicableWithdrawnNCT04462692
What this trial is testing

An Observational Study in Children With CLN2 Batten Disease

Who this might be right for
Neuronal Ceroid Lipofuscinosis Type 2 (CLN2)
REGENXBIO Inc.
Testing effectiveness (Phase 2)Study completedNCT02485899
What this trial is testing

An Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 190 in Patients With CLN2 Disease

Who this might be right for
Jansky-Bielschowsky DiseaseBatten DiseaseLate-Infantile Neuronal Ceroid Lipofuscinosis Type 2+2 more
BioMarin Pharmaceutical 23
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