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Results for “Batten Disease”

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Showing 20 of 0 results

Not applicableEnded earlyNCT04974307
What this trial is testing

Optic-to-Audio Device in a Pediatric Cohort With CLN3-related Conditions or Low Vision

Who this might be right for
CLN3-related DisordersNon-CLN3 Related Low Vision
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) 11
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
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
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
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)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)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 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 applicableUnknownNCT04461561
What this trial is testing

Using NPT to Evaluate Providing PPC as ELNEC-PPC WBT for Nurses

Who this might be right for
CancerCardiac AnomalyCystic Fibrosis+4 more
Altoosi University College 172
Not applicableStudy completedNCT03822650
What this trial is testing

A Natural History Study of Neuronal Ceroid Lipofuscinosis Type 5 (CLN5)

Who this might be right for
Ceroid Lipofuscinosis, Neuronal 5
Neurogene Inc. 37
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
Early research (Phase 1)Active Not RecruitingNCT04737460
What this trial is testing

Study for the Treatment for CLN7 Disease

Who this might be right for
CLN7
Benjamin Greenberg 4
Not applicableStudy completedNCT04808297
What this trial is testing

Trehalose in Subjects With Neuronal Ceroid Lipofuscinoses

Who this might be right for
Neuronal Ceroid-Lipofuscinoses
IRCCS Fondazione Stella Maris 8
Not applicableLooking for participantsNCT03307304
What this trial is testing

Investigations of Juvenile Neuronal Ceroid Lipofuscinosis

Who this might be right for
Juvenile Neuronal Ceroid Lipofuscinosis (CLN3)Batten Disease
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) 300
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)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
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 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
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 participantsNCT06844877
What this trial is testing

Italian NCL Registry: a Registry for NCL as an Integration Tool for Future Therapeutic Strategies

Who this might be right for
Neuronal Ceroid Lipofuscinosis
IRCCS Fondazione Stella Maris 50
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