15 studies found for:    "infantile neuronal ceroid lipofuscinosis" OR "Santavuori-Haltia disease" OR "Neuronal Ceroid-Lipofuscinoses"
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Rank Status Study
1 Recruiting 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
2 Recruiting AAVRh.10 Administered to Children With Late Infantile Neuronal Ceroid Lipofuscinosis With Uncommon Genotypes or Moderate/Severe Impairment
Conditions: Late Infantile Neuronal Ceroid Lipofuscinosis;   Batten Disease
Intervention: Biological: AAVrh.10CUCLN2
3 Enrolling by invitation Collection of Cerebrospinal Fluid in Healthy Children
Conditions: Late Infantile Neuronal Ceroid Lipofuscinosis;   Batten Disease
Intervention:
4 Recruiting Genotype-Phenotype Correlations of Late Infantile Neuronal Ceroid Lipofuscinosis
Conditions: Batten Disease;   Late Infantile Neuronal Lipofuscinosis
Intervention:
5 Active, not recruiting Safety Study of a Gene Transfer Vector for Children With Late Infantile Neuronal Ceroid Lipofuscinosis
Conditions: Batten Disease;   Late Infantile Neuronal Lipofuscinosis
Interventions: Genetic: AAV2CUhCLN2;   Procedure: Neurological surgery
6 Recruiting Safety Study of a Gene Transfer Vector (Rh.10) for Children With Late Infantile Neuronal Ceroid Lipofuscinosis
Conditions: Batten Disease;   Late Infantile Neuronal Lipofuscinosis
Intervention: Genetic: AAVrh.10CUhCLN2 vector
7 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
8 Withdrawn Safety and Efficacy Study of HuCNS-SC in Subjects With Neuronal Ceroid Lipofuscinosis
Condition: Neuronal Ceroid Lipofuscinosis
Intervention: Biological: HuCNS-SC
9 Completed Cystagon to Treat Infantile Neuronal Ceroid Lipofuscinosis
Condition: Infantile Neronal Ceroid Lipofuscinosis
Intervention: Drug: Cystagon
10 Recruiting Neuronal Ceroid Lipofuscinosis and Associated Sleep Abnormalities
Conditions: Neuronal Ceroid Lipofuscinosis;   Batten's Disease
Intervention:
11 Completed Genotype - Phenotype Correlations of LINCL
Conditions: Batten Disease;   Late Infantile Neuronal Lipofuscinosis
Intervention:
12 Recruiting Cellcept for Treatment of Juvenile Neuronal Ceroid Lipofuscinosis
Condition: Juvenile Neuronal Ceroid Lipofuscinosis
Interventions: Drug: Mycophenolate mofetil;   Drug: Liquid Placebo
13 Recruiting Clinical and Neuropsychological Investigations in Batten Disease
Condition: Neuronal Ceroid Lipofuscinosis
Intervention:
14 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
15 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

Indicates status has not been verified in more than two years