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Results for “Neuronal Ceroid Lipofuscinosis Type 2 (CLN2)”

15 trials

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Showing 15 of 15 results

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
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
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 applicableActive Not RecruitingNCT04476862
What this trial is testing

Cerliponase Alfa Observational Study in the US

Who this might be right for
Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2
BioMarin Pharmaceutical 35
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
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.
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
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)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
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
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
Not applicableLooking for participantsNCT01873924
What this trial is testing

Clinical and Neuropsychological Investigations in Batten Disease

Who this might be right for
Neuronal Ceroid LipofuscinosisNeuronal Ceroid Lipofuscinosis CLN1Neuronal Ceroid Lipofuscinosis CLN2+7 more
University of Rochester 500
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.
Early research (Phase 1)Study completedNCT01161576
What this trial is testing

Safety Study of a Gene Transfer Vector (Rh.10) for Children With Late Infantile Neuronal Ceroid Lipofuscinosis (LINCL)

Who this might be right for
Batten DiseaseLate-Infantile Neuronal Ceroid Lipofuscinosis
Weill Medical College of Cornell University 12
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