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Hereditary Central Nervous System Demyelinating Diseases - 20 Studies Found

Estado Estudiar
Completed Nombre del estudio: Aclarubicin for the Treatment of Retinal Vasculopathy With Cerebral Leukodystrophy
Condición:
  • Vasculopathy, Retinal, With Cerebral Leukodystrophy
  • Cerebroretinal Vasculopathy, Hereditary<
    Fecha: 2016-03-02
    Intervenciones: Drug: aclarubicin Aclarubicin (3 mg/m²) will be administered intravenously through a central venous
Terminated Nombre del estudio: Natural History Study of Children With Metachromatic Leukodystrophy
Condición:
  • Lipid Metabolism Disorders
  • Metachromatic Leukodystrophy (MLD)
  • Biological: Recombinant human arylsulfatase A

Completed Nombre del estudio: Intracerebral Gene Therapy for Children With Early Onset Forms of Metachromatic Leukodystrophy
Condición: Metachromatic Leukodystrophy
Fecha: 2013-01-28
Intervenciones: Genetic: intracerebral administration of AAVrh.10cuARSA
Active, not recruiting Nombre del estudio: Gene Therapy for Metachromatic Leukodystrophy
Condición: Lysosomal Storage Disease
Fecha: 2012-03-16
Intervenciones: Genetic: Autologous CD34+ stem cells transduced with ARSA encoding lentiviral vector Autologous hematopo
Completed Nombre del estudio: Multicenter Study of HGT-1110 Administered Intrathecally in Children With Metachromatic Leukodystrophy (MLD)
Condición: Metachromatic Leukodystrophy (MLD)
Fecha: 2011-12-14
Intervenciones: Biological: Recombinant human arylsulfatase A 6 patients treated with HGT-1110 EOW by IT injection
Completed Nombre del estudio: Long-Term Follow-Up Study of Human Stem Cells Transplanted in Subjects With Connatal Pelizaeus-Merzbacher Disease (PMD)
Condición:
  • Pelizaeus-Merzbacher Disease
  • PMD

Fecha: 2011-07-06
Intervenciones: Biological: HuCNS-SC transplant in the lead-in phase Long-term safety follow-up study
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