Status | Study |
Recruiting |
Study Name: Human Placental-Derived Stem Cell Transplantation Condition: Mucopolysaccharidosis I Mucopolysaccharidosis VI Adrenoleu Date: 2012-04-25 Interventions: Drug: Human Placental Derived Stem Cell Infusions of thawed HPDSC to be given following UCB infusion. |
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 |
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 |
Withdrawn |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 |
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 |
Recruiting |
Study Name: Genotype-Phenotype Correlations of Late Infantile Neuronal Ceroid Lipofuscinosis Condition: Batten Disease Late Infantile Neuronal Ceroid Lipofuscinosis Date: 2009-12-16 |
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: Stem Cell Transplant for Inborn Errors of Metabolism Condition: Adrenoleukodystrophy Metachromatic Leukodystrophy Globoid Date: 2005-09-12 Interventions: Procedure: Stem Cell Transplant |
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 |
Completed |
Study Name: Genotype - Phenotype Correlations of LINCL Condition: Batten Disease Late-Infantile Neuronal Ceroid Lipfuscinosis Date: 2005-09-06 |