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Ceroid Storage Disease - 21 Studies Found

Status Study
Withdrawn Study Name: Safety and Efficacy Study of HuCNS-SC in Subjects With Neuronal Ceroid Lipofuscinosis
Condition: Neuronal Ceroid Lipofuscinosis
Date: 2010-11-05
Interventions: Biological: HuCNS-SC Surgery to implant human CNS stem cells
Completed Study Name: Study of HuCNS-SC Cells in Patients With Infantile or Late Infantile Neuronal Ceroid Lipofuscinosis (NCL)
Condition: Neuronal Ceroid Lipofuscinosis
Date: 2006-06-13
Interventions:
  • Procedure: Surgery to implant human CNS stem cells (HuCNS-SC)

Completed Study Name: Cellcept for Treatment of Juvenile Neuronal Ceroid Lipofuscinosis
Condition: Juvenile Neuronal Ceroid Lipofuscinosis
Date: 2011-07-05
Interventions:
  • Drug: Mycophenolate mofetil Th
Completed Study Name: Neuronal Ceroid Lipofuscinosis and Associated Sleep Abnormalities
Condition:
  • Neuronal Ceroid Lipofuscinosis
  • Batten's Disease

Date: 2013-09-25
Active, not recruiting Study Name: Safety Study of a Gene Transfer Vector for Children With Late Infantile Neuronal Ceroid Lipofuscinosis
Condition:
  • Batten Disease
  • Late Infantile Neuronal Ceroid Lipofuscinosis Date: 2005-09-06
    Interventions: Genetic: AAV2CUhCLN2 (3x10^12 particle units) gene transfer; one-time administration via neuro surgery p
Recruiting Study Name: Genotype-Phenotype Correlations of Late Infantile Neuronal Ceroid Lipofuscinosis
Condition:
  • Batten Disease
  • Late Infantile Neuronal Ceroid Lipofuscinosis Date: 2009-12-16
Recruiting Study Name: Safety Study of a Gene Transfer Vector (Rh.10) for Children With Late Infantile Neuronal Ceroid Lipofuscinosis
Condition:
  • Batten Disease
  • Late-Infantile Neuronal Ceroid Lipofuscinosis Date: 2010-03-22
    Interventions:
    • Genetic: AAVrh.10CUhCLN2 vector 9.0x10^11 genome copies

Completed Study Name: Cystagon to Treat Infantile Neuronal Ceroid Lipofuscinosis
Condition: Infantile Neronal Ceroid Lipofuscinosis
Date: 2001-12-17
Interventions: Drug: Cystagon
Completed Study Name: 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
Condition:
  • Jansky-Bielschowsky Disease
  • Batten Disease
  • Late-Infantile
    Date: 2013-07-19
    Interventions: Biological: BMN 190 30-300 mg ICV infusion administered every other week for at least 48 weeks.
Recruiting Study Name: AAVRh.10 Administered to Children With Late Infantile Neuronal Ceroid Lipofuscinosis
Condition:
  • Late Infantile Neuronal Ceroid Lipofuscinosis
  • Batten Disease Date: 2010-12-13
    Interventions: Biological: AAVrh.10CUCLN2 There will be 2 dose cohorts in this study. Group A consists of 2 subjects wh
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