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21 studies found for:    "congenital neuronal ceroid lipofuscinosis" OR "Neuronal Ceroid-Lipofuscinoses"
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Rank Status Study
1 Completed Neuronal Ceroid Lipofuscinosis and Associated Sleep Abnormalities
Conditions: Neuronal Ceroid Lipofuscinosis;   Batten's Disease
Intervention:
2 Active, not recruiting Safety Study of a Gene Transfer Vector for Children With Late Infantile Neuronal Ceroid Lipofuscinosis
Conditions: Batten Disease;   Late Infantile Neuronal Ceroid Lipofuscinosis
Intervention: Genetic: AAV2CUhCLN2
3 Completed 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
Conditions: Jansky-Bielschowsky Disease;   Batten Disease;   Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2;   CLN2 Disease
Intervention: Biological: BMN 190
4 Recruiting Safety Study of a Gene Transfer Vector (Rh.10) for Children With Late Infantile Neuronal Ceroid Lipofuscinosis
Conditions: Batten Disease;   Late-Infantile Neuronal Ceroid Lipofuscinosis
Interventions: Genetic: AAVrh.10CUhCLN2 vector 9.0x10^11 genome copies;   Genetic: AAVrh.10CUhCLN2 vector 2.85x10^11 genome copies
5 Completed Study of HuCNS-SC Cells in Patients With Infantile or Late Infantile Neuronal Ceroid Lipofuscinosis (NCL)
Condition: Neuronal Ceroid Lipofuscinosis
Interventions: Procedure: Surgery to implant human CNS stem cells (HuCNS-SC);   Drug: Medication to suppress the immune system
6 Withdrawn Safety and Efficacy Study of HuCNS-SC in Subjects With Neuronal Ceroid Lipofuscinosis
Condition: Neuronal Ceroid Lipofuscinosis
Intervention: Biological: HuCNS-SC
7 Completed Cellcept for Treatment of Juvenile Neuronal Ceroid Lipofuscinosis
Condition: Juvenile Neuronal Ceroid Lipofuscinosis
Interventions: Drug: Mycophenolate mofetil;   Drug: Liquid Placebo
8 Recruiting Clinical and Neuropsychological Investigations in Batten Disease
Condition: Neuronal Ceroid Lipofuscinosis
Intervention:
9 Recruiting AAVRh.10 Administered to Children With Late Infantile Neuronal Ceroid Lipofuscinosis
Conditions: Late Infantile Neuronal Ceroid Lipofuscinosis;   Batten Disease
Intervention: Biological: AAVrh.10CUCLN2
10 Recruiting Genotype-Phenotype Correlations of Late Infantile Neuronal Ceroid Lipofuscinosis
Conditions: Batten Disease;   Late Infantile Neuronal Lipofuscinosis
Intervention:
11 Terminated Collection of Cerebrospinal Fluid in Healthy Children
Conditions: Late Infantile Neuronal Ceroid Lipofuscinosis;   Batten Disease
Intervention: Other: Group 1
12 Active, not recruiting A Multicenter, Multinational, Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 190 in Patients With CLN2 Disease
Conditions: Jansky-Bielschowsky Disease;   Batten Disease;   Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2;   CLN2 Disease;   CLN2 Disorder
Intervention: Biological: BMN 190
13 Completed
Has Results
Cystagon to Treat Infantile Neuronal Ceroid Lipofuscinosis
Condition: Infantile Neronal Ceroid Lipofuscinosis
Intervention: Drug: Cystagon
14 Recruiting Batten CLN6 Gene Therapy
Conditions: Batten Disease;   CLN6
Intervention: Drug: scAVV9.CB.CLN6
15 Completed Genotype - Phenotype Correlations of LINCL
Conditions: Batten Disease;   Late-Infantile Neuronal Ceroid Lipfuscinosis
Intervention:
16 Enrolling by invitation A Safety, Tolerability, and Efficacy Study of Intracerebroventricular BMN 190 in Patients With CLN2 Disease
Conditions: Jansky-Bielschowsky Disease;   Batten Disease;   Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2;   CLN2 Disease;   CLN2 Disorder
Intervention: Drug: BMN190 recombinant human tripeptidyl peptidase-1 (rhTPP1)
17 Recruiting UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells
Conditions: Adrenoleukodystrophy;   Batten Disease;   Mucopolysaccharidosis II;   Leukodystrophy, Globoid Cell;   Leukodystrophy, Metachromatic;   Neimann Pick Disease;   Pelizaeus-Merzbacher Disease;   Sandhoff Disease;   Tay-Sachs Disease;   Brain Diseases, Metabolic, Inborn
Intervention: Biological: DUOC-01
18 Recruiting Inherited Retinal Degenerative Disease Registry
Conditions: Eye Diseases Hereditary;   Retinal Disease;   Achromatopsia;   Bardet-Biedl Syndrome;   Bassen-Kornzweig Syndrome;   Batten Disease;   Best Disease;   Choroidal Dystrophy;   Choroideremia;   Cone Dystrophy;   Cone-Rod Dystrophy;   Congenital Stationary Night Blindness;   Enhanced S-Cone Syndrome;   Fundus Albipunctatus;   Goldmann-Favre Syndrome;   Gyrate Atrophy;   Juvenile Macular Degeneration;   Kearns-Sayre Syndrome;   Leber Congenital Amaurosis;   Refsum Syndrome;   Retinitis Pigmentosa;   Retinitis Punctata Albescens;   Retinoschisis;   Rod-Cone Dystrophy;   Rod Dystrophy;   Rod Monochromacy;   Stargardt Disease;   Usher Syndrome
Intervention:
19 Recruiting Human Placental-Derived Stem Cell Transplantation
Conditions: Mucopolysaccharidosis I;   Mucopolysaccharidosis VI;   Adrenoleukodystrophy;   Niemann-Pick Disease;   Metachromatic Leukodystrophy;   Wolman Disease;   Krabbe's Disease;   Gaucher's Disease;   Fucosidosis;   Batten Disease;   Severe Aplastic Anemia;   Diamond-Blackfan Anemia;   Amegakaryocytic Thrombocytopenia;   Myelodysplastic Syndrome;   Acute Myelogenous Leukemia;   Acute Lymphocytic Leukemia
Intervention: Drug: Human Placental Derived Stem Cell
20 Completed
Has Results
Stem Cell Transplant for Inborn Errors of Metabolism
Conditions: Adrenoleukodystrophy;   Metachromatic Leukodystrophy;   Globoid Cell Leukodystrophy;   Gaucher's Disease;   Fucosidosis;   Wolman Disease;   Niemann-Pick Disease;   Batten Disease;   GM1 Gangliosidosis;   Tay Sachs Disease;   Sandhoff Disease
Interventions: Procedure: Stem Cell Transplant;   Drug: Busulfan, Cyclophosphamide, Antithymocyte Globulin

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Indicates status has not been verified in more than two years