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Batten

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Neuronal Ceroid Lipofuscinosis, Batten's CLN6 Diseae

Recruiting
  • Batten Disease
  • CLN6
  • Natural history
  • Columbus, Ohio
    Nationwide Children's Hospital
Oct 20, 2022

Natural History Study of Batten Disease

Terminated
  • Batten Disease
  • +2 more
    • Rochester, New York
      University of Rochester Medical Center
    Apr 1, 2022

    CLN3, Batten Disease Trial in Columbus (AT-GTX-502)

    Active, not recruiting
    • CLN3
    • Batten Disease
    • AT-GTX-502
    • Columbus, Ohio
      Nationwide Children's Hospital
    Jul 18, 2022

    Nasal Obstruction, Septal Defect, Allergic Rhinitis Trial in Poplar Bluff, New York, Staten Island (Batten batten graft,

    Recruiting
    • Nasal Obstruction
    • +4 more
    • Batten batten graft
    • +2 more
    • Poplar Bluff, Missouri
    • +2 more
    Mar 11, 2022

    CLN6 Batten Disease Subjects Following Gene Transfer

    Active, not recruiting
    • CLN6
    • Batten Disease
    • AT-GTX-501
    • Columbus, Ohio
      Nationwide Children's Hosptial
    Dec 17, 2021

    Investigations of Juvenile Neuronal Ceroid Lipofuscinosis

    Recruiting
    • Juvenile Neuronal Ceroid Lipofuscinosis (CLN3)
    • Batten Disease
      • Bethesda, Maryland
        National Institutes of Health Clinical Center
      Jan 31, 2023

      Neuronal Ceroid Lipofuscinosis CLN5 Trial in Rochester (NGN-101)

      Recruiting
      • Neuronal Ceroid Lipofuscinosis CLN5
      • NGN-101
      • Rochester, New York
      • +1 more
      Jan 19, 2023

      Caregiving Networks Across Disease Context and the Life Course

      Recruiting
      • Inherited Metabolic Disorders
      • +4 more
        • Bethesda, Maryland
          National Institutes of Health Clinical Center
        Aug 3, 2022

        Neuronal Ceroid Lipofuscinosis, Batten Disease, CLN1 Disease Trial in Hamburg (Natural History)

        Recruiting
        • Neuronal Ceroid Lipofuscinosis
        • +14 more
        • Natural History
        • Hamburg, Germany
          University Medical Center Hamburg-Eppendorf
        Oct 22, 2021

        Batten Disease Trial in Houston (Miglustat 100Mg Oral Capsule)

        Recruiting
        • Batten Disease
        • Miglustat 100Mg Oral Capsule
        • Houston, Texas
          Texas Children Hospital
        May 17, 2022

        Jansky-Bielschowsky Disease, Batten Disease, Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2 Trial in Worldwide (BMN 190,

        Completed
        Has Results
        • Jansky-Bielschowsky Disease
        • +4 more
        • BMN 190
        • Intracerebroventricular (ICV) access device
        • Columbus, Ohio
        • +3 more
        Jul 28, 2022

        Adrenoleukodystrophy, Batten Disease, Mucopolysaccharidosis II Trial in Durham (DUOC-01)

        Recruiting
        • Adrenoleukodystrophy
        • +11 more
        • DUOC-01
        • Durham, North Carolina
          Duke University Medical Center
        Oct 24, 2022

        Children Diagnosed With CLN2 Disease

        Recruiting
        • Batten Disease
        • +2 more
        • CLN2 Treatment
        • Columbus, Ohio
          Nationwide Children's Hospital
        Aug 2, 2021

        Mucopolysaccharidosis I, Mucopolysaccharidosis VI, Adrenoleukodystrophy Trial in Denver, Valhalla, Salt Lake City (Human

        Completed
        • Mucopolysaccharidosis I
        • +15 more
        • Human Placental Derived Stem Cell
        • Denver, Colorado
        • +2 more
        Oct 24, 2022

        The Natural History of Metachromatic Leukodystrophy

        Recruiting
        • MLD
        • +26 more
        • Palliative Care
        • Hematopoetic Stem Cell Transplantation
        • Pittsburgh, Pennsylvania
          The Program for the Study of Neurodevelopment in Rare Disorders,
        Nov 2, 2021

        Batten Disease, Late-Infantile Neuronal Ceroid Lipofuscinosis Trial in New York (AAVrh.10CUhCLN2 vector 9.0x10^11 genome copies,

        Completed
        • Batten Disease
        • Late-Infantile Neuronal Ceroid Lipofuscinosis
        • AAVrh.10CUhCLN2 vector 9.0x10^11 genome copies
        • AAVrh.10CUhCLN2 vector 2.85x10^11 genome copies
        • New York, New York
          Weill Cornell Medical College
        Jan 31, 2021

        Late Infantile Neuronal Ceroid Lipofuscinosis, Batten Disease Trial in New York (AAVrh.10CUCLN2)

        Completed
        • Late Infantile Neuronal Ceroid Lipofuscinosis
        • Batten Disease
        • AAVrh.10CUCLN2
        • New York, New York
          Weill Cornell Medical College
        Aug 28, 2020

        Neuronal Ceroid Lipofuscinosis Type 2 Trial in London (RGX-381)

        Not yet recruiting
        • Neuronal Ceroid Lipofuscinosis Type 2
        • RGX-381
        • London, United Kingdom
          Greater Ormond Street Hospital
        Mar 17, 2023

        Late Infantile Neuronal Ceroid Lipofuscinosis

        Completed
        • Batten Disease
        • Late Infantile Neuronal Ceroid Lipofuscinosis
          • New York, New York
            Weill Cornell Medicine
          Jul 28, 2020

          Batten Disease, Late Infantile Neuronal Ceroid Lipofuscinosis Trial in New York (AAV2CUhCLN2 (3x10^12 particle units))

          Completed
          • Batten Disease
          • Late Infantile Neuronal Ceroid Lipofuscinosis
          • AAV2CUhCLN2 (3x10^12 particle units)
          • New York, New York
            New York Presbyterian Hospital - Weill Medical College of Cornel
          Jul 22, 2020

          Late Infantile Variant CLN5 And CLN7 Disease

          Recruiting
          • Ceroid Lipofuscinosis, Neuronal 5
          • Ceroid Lipofuscinosis, Neuronal 7
            • Rochester, New York
              University of Rochester Medical Center
            Mar 1, 2022

            Trehalose in Subjects With Neuronal Ceroid Lipofuscinoses

            Completed
            • Neuronal Ceroid-Lipofuscinoses
              • Pisa, PI, Italy
                IRCCS Fondazione Stella Maris
              Apr 1, 2022

              Variant Late-Infantile Neuronal Ceroid Lipofuscinosis Trial in Columbus (AT-GTX-501)

              Completed
              • Variant Late-Infantile Neuronal Ceroid Lipofuscinosis
              • AT-GTX-501
              • Columbus, Ohio
                Nationwide Children's Hosptial
              Jan 21, 2022

              CLN7 Trial in Dallas (AAV9/CLN7)

              Recruiting
              • CLN7
              • AAV9/CLN7
              • Dallas, Texas
                Children' Health
              May 16, 2022

              COVID-19 Trial in Seattle (Rehabilitation-focused program)

              Recruiting
              • COVID-19
              • Rehabilitation-focused program
              • Seattle, Washington
                VA Puget Sound Health Care System Seattle Division, Seattle, WA
              Dec 2, 2021