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Neuronal Ceroid-Lipofuscinoses

Showing 1 - 25 of 884
STUDY
CONDITIONS
INTERVENTIONS
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LAST UPDATED

Trehalose in Subjects With Neuronal Ceroid Lipofuscinoses

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

    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

      Atypical Tripeptidyl Peptidase 1 Enzyme Deficiency Patients

      Active, not recruiting
      • Neuronal Ceroid-Lipofuscinoses
      • +2 more
        • Orange, California
          Children's Hospital of Orange County
        Jul 18, 2022

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

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

        Neuronal Ceroid Lipofuscinosis, Batten's CLN6 Diseae

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

        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

        Natural History Study of Batten Disease

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

          Functional Near-Infrared Spectroscopy Combined With Diffuse

          Recruiting
          • Juvenile Neuronal Ceroid Lipofuscinosis
          • +3 more
            • Bethesda, Maryland
              National Institutes of Health Clinical Center
            Jan 30, 2023

            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

            Juvenile Neuronal Ceroid Lipofuscinosis Trial (PLX-200, Placebo)

            Not yet recruiting
            • Juvenile Neuronal Ceroid Lipofuscinosis
            • (no location specified)
            Mar 31, 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

            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

              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

                A Retrospective, Natural History Study in Children With CLN2

                Withdrawn
                • Late-infantile Neuronal Ceroid Lipofuscinosis Type 2 (CLN2)
                  • (no location specified)
                  Oct 25, 2021

                  Neuronal Ceroid Lipofuscinosis Type 2 Trial in Columbus (Cerliponase Alfa)

                  Enrolling by invitation
                  • Neuronal Ceroid Lipofuscinosis Type 2
                  • Cerliponase Alfa
                  • Columbus, Ohio
                    Nationwide Children's Hospital
                  Nov 29, 2021

                  Children Diagnosed With CLN2 Disease

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

                  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

                  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

                  Cerliponase Alfa Observational Study in the US

                  Recruiting
                  • Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2
                  • Cerliponase Alfa
                  • Administration Kit
                  • Little Rock, Arkansas
                  • +13 more
                  Jun 2, 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

                  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

                  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

                  ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn

                  Enrolling by invitation
                  • Acid Sphingomyelinase Deficiency
                  • +13 more
                  • Confirmatory Testing
                  • Bronx, New York
                  • +9 more
                  May 9, 2022