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Results for “Neuronal Ceroid Lipofuscinosis CLN3”

33 trials

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Showing 20 of 33 results

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 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 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.
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 RecruitingNCT03285425
What this trial is testing

Natural History of Neuronal Ceroid Lipofuscinosis, Batten's CLN6 Diseae

Who this might be right for
Batten DiseaseCLN6
Emily de los Reyes 30
Testing effectiveness (Phase 2)Not Yet RecruitingNCT07740512
What this trial is testing

Safety, Tolerability, Pharmacokinetics, and Preliminary Efficacy of PLX-200 in Pediatric Patients (Master Protocol)

Who this might be right for
Lysosomal Storage DisordersSandhoff DiseaseKrabbe Disease+2 more
Polaryx Therapeutics, Inc. 24
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.
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.
Testing effectiveness (Phase 2)Study completedNCT01399047
What this trial is testing

Cellcept for Treatment of Juvenile Neuronal Ceroid Lipofuscinosis

Who this might be right for
Juvenile Neuronal Ceroid Lipofuscinosis
University of Rochester 19
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
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
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
Post-approval studies (Phase 4)Study completedNCT00028262
What this trial is testing

Cystagon to Treat Infantile Neuronal Ceroid Lipofuscinosis

Who this might be right for
Infantile Neronal Ceroid Lipofuscinosis
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) 10
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
Large-scale testing (Phase 3)WithdrawnNCT04637282
What this trial is testing

Safety, Tolerability, and Efficacy of PLX-200 in Patients With CLN3

Who this might be right for
Juvenile Neuronal Ceroid Lipofuscinosis
Polaryx Therapeutics, Inc.
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
Testing effectiveness (Phase 2)Active Not RecruitingNCT03770572
What this trial is testing

Gene Therapy for Children With CLN3 Batten Disease

Who this might be right for
CLN3Batten Disease
Neela Therapeutics 7
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
Not applicableEnded earlyNCT04644549
What this trial is testing

Natural History Study of Batten Disease

Who this might be right for
Batten DiseaseNeuronal Ceroid Lipofuscinosis CLN6Neuronal Ceroid Lipofuscinosis CLN3
Emily de los Reyes 10
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
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