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Sanfilippo

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STUDY
CONDITIONS
INTERVENTIONS
LOCATIONS

Participants With Sanfilippo Syndrome Type IIIC

Not yet recruiting
  • Sanfilippo Syndrome Type C
    • Bron, France
      Hospices Civils De Lyon
    Apr 20, 2023

    Patients With Sanfilippo Disease(s) (MPS3)

    Not yet recruiting
    • Sanfilippo Syndrome
    • MPS3
      • Fairfax, Virginia
        LDRTC
      Jan 20, 2023

      A Natural History Study of Sanfilippo Syndrome Type D

      Recruiting
      • Sanfilippo Syndrome Type D
        • New York, New York
          Columbia University Irving Medical Center
        Jan 11, 2023

        MPS IIIA, Sanfilippo Syndrome, Sanfilippo A Trial in Australia, Spain, United States (ABO-102)

        Active, not recruiting
        • MPS IIIA
        • +3 more
        • ABO-102
        • Columbus, Ohio
        • +4 more
        Nov 8, 2022

        A Study of Patients With Sanfilippo Syndrome Type A (MPS IIIA)

        Completed
        • Sanfilippo Syndrome Type A
        • assessment
        • Minneapolis, Minnesota
          University of Minnesota
        May 19, 2021

        Sanfilippo Syndrome A Trial (assessment)

        Not yet recruiting
        • Sanfilippo Syndrome A
        • assessment
        • (no location specified)
        Jun 6, 2021

        Biomarker for Sanfilippo Type A-B-C-D Disease (BioSanfilippo)

        Active, not recruiting
        • Mental Retardation
        • Developmental Delay
          • Cairo, Egypt
          • +4 more
          May 12, 2021

          Sanfilippo Syndrome Type A (MPS IIIA) Trial in Oakland, Chapel Hill, Ankara (SOBI003)

          Completed
          • Sanfilippo Syndrome Type A (MPS IIIA)
          • Oakland, California
          • +2 more
          Feb 2, 2022

          Mucopolysaccharidosis Type IIIB

          Completed
          • Sanfilippo Syndrome Type B
            • Oakland, California
            • +3 more
            Mar 15, 2021

            Mucopolysaccharidosis Type IIIB, MPS III B Trial in Worldwide (AX 250)

            Active, not recruiting
            • Mucopolysaccharidosis Type IIIB
            • MPS III B
            • AX 250
            • Oakland, California
            • +7 more
            Jan 16, 2023

            MPS III B Trial in Worldwide (AX 250)

            Not yet recruiting
            • MPS III B
            • AX 250
            • Oakland, California
            • +4 more
            Aug 8, 2022

            Sanfilippo Syndrome Type A (MPS IIIA) Trial in Germany, Turkey, United States (SOBI003)

            Completed
            • Sanfilippo Syndrome Type A (MPS IIIA)
            • Oakland, California
            • +3 more
            Nov 18, 2021

            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 Type IIIA Trial in Manchester (Autologous CD34+ cells transduced with a lentiviral vector containing the

            Active, not recruiting
            • Mucopolysaccharidosis Type IIIA
            • Autologous CD34+ cells transduced with a lentiviral vector containing the human SGSH gene
            • Manchester, United Kingdom
              Manchester University NHS Foundation Trust
            Jul 2, 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

            Sanfilippo Syndrome B Trial in Paris (rAAV2/5-hNAGLU)

            Completed
            • Sanfilippo Syndrome B
            • Paris, Le Kremlin-Bicetre Cedex, France
              Hopitaux Universitaires Paris-Sud
            Nov 28, 2019

            Neurobehavioral Phenotypes in MPS III

            Completed
            • Sanfilippo Syndrome Type A
            • +2 more
              • Minneapolis, Minnesota
                University of Minnesota
              Oct 30, 2019

              MPS III B, Mucopolysaccharidosis Type IIIB Trial in Worldwide (AX 250)

              Completed
              • MPS III B
              • Mucopolysaccharidosis Type IIIB
              • AX 250
              • Oakland, California
              • +6 more
              Aug 4, 2020

              Study of Patients With MPS IIIA Treated With ABO-102

              Enrolling by invitation
              • Mucopolysaccharidosis III-A
              • ABO-102
              • Columbus, Ohio
              • +2 more
              Nov 8, 2022

              Mucopolysaccharidosis Type 3 B Trial in Worldwide (rAAV9.CMV.hNAGLU)

              Terminated
              • Mucopolysaccharidosis Type 3 B
              • rAAV9.CMV.hNAGLU
              • Columbus, Ohio
              • +3 more
              Apr 29, 2022

              Study of Patients With MPS IIIB Treated With ABO-101

              Terminated
              • Mucopolysaccharidosis III-B
              • ABO-101
              • Columbus, Ohio
              • +2 more
              May 25, 2022

              Right Ventricle Dysfunction in Critically Ill Patients

              Not yet recruiting
              • Right Ventricular Dysfunction
                • (no location specified)
                Jul 27, 2023

                Adrenoleukodystrophy, Metachromatic Leukodystrophy, Globoid Cell Leukodystrophy Trial in Minneapolis (drug, procedure,

                Completed
                • Adrenoleukodystrophy
                • +8 more
                • Minneapolis, Minnesota
                  Masonic Cancer Center, University of Minnesota
                Jul 10, 2019

                Advanced Soft Tissue Sarcoma Trial in Italy (Standard arm: Trabectedin in monotherapy, Experimental arm: Trabectedin + Olaparib)

                Recruiting
                • Advanced Soft Tissue Sarcoma
                • Standard arm: Trabectedin in monotherapy
                • Experimental arm: Trabectedin + Olaparib
                • Bologna, BO, Italy
                • +13 more
                Oct 29, 2021