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Results for “CLN3 Disease”

44 trials

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Showing 20 of 44 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
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 applicableLooking for participantsNCT03333200
What this trial is testing

Longitudinal Study of Neurodegenerative Disorders

Who this might be right for
MLDKrabbe DiseaseALD+24 more
University of Pittsburgh 1,500
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 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
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 applicableStudy completedNCT05642221
What this trial is testing

Functional Near-Infrared Spectroscopy (fNIRS) Combined With Diffuse Correlation Spectroscopy (DCS) in Neurocognitive Disease as Compared to Healthy Neurotypical Controls

Who this might be right for
Juvenile Neuronal Ceroid LipofuscinosisSmith-Lemli-Opitz SyndromeCreatine Transporter Deficiency+1 more
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) 73
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
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
Early research (Phase 1)Study completedNCT01586455
What this trial is testing

Human Placental-Derived Stem Cell Transplantation

Who this might be right for
Mucopolysaccharidosis IMucopolysaccharidosis VIAdrenoleukodystrophy+13 more
New York Medical College 43
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
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
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 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
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
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)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 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
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