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Results for “Batten's Disease”

43 trials

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Showing 20 of 43 results

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
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)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
Large-scale testing (Phase 3)Study completedNCT00176904
What this trial is testing

Stem Cell Transplant for Inborn Errors of Metabolism

Who this might be right for
AdrenoleukodystrophyMetachromatic LeukodystrophyGloboid Cell Leukodystrophy+8 more
Masonic Cancer Center, University of Minnesota 135
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
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)Active Not RecruitingNCT05228145
What this trial is testing

Gene Therapy Study for Children With CLN5 Batten Disease

Who this might be right for
Neuronal Ceroid Lipofuscinosis CLN5
Neurogene Inc. 6
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
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
Large-scale testing (Phase 3)Not Yet RecruitingNCT04637282
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. 39
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
Not applicableStudy completedNCT01966757
What this trial is testing

Neuronal Ceroid Lipofuscinosis and Associated Sleep Abnormalities

Who this might be right for
Neuronal Ceroid LipofuscinosisBatten's Disease
Nationwide Children's Hospital 57
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 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)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
Not applicableLooking for participantsNCT02435940
What this trial is testing

Inherited Retinal Degenerative Disease Registry

Who this might be right for
Eye Diseases HereditaryRetinal DiseaseAchromatopsia+25 more
Foundation Fighting Blindness 20,000
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
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
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