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Hurler-Scheie Syndrome

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Mucopolysaccharidosis Type I (MPS I), Hurler Syndrome, Hurler-Scheie Syndrome Trial in Orange, Philadelphia, Porto Alegre

Active, not recruiting
  • Mucopolysaccharidosis Type I (MPS I)
  • +2 more
  • RGX-111
  • Orange, California
  • +3 more
Jan 9, 2023

Mucopolysaccharidosis I, Hurler's Syndrome, Hurler-Scheie Syndrome Trial in Rome (Aldurazyme (laronidase))

Recruiting
  • Mucopolysaccharidosis I
  • +3 more
  • Aldurazyme (laronidase)
  • Rome, Italy
    Dipartimento di Scienze Pediatriche Medico - Chirurgiche e Neuro
Mar 28, 2022

Mucopolysaccharidosis IH/S, Mucopolysaccharidosis IS Trial in Minneapolis (Autologous Plasmablasts (B cells))

Not yet recruiting
  • Mucopolysaccharidosis IH/S
  • Mucopolysaccharidosis IS
  • Autologous Plasmablasts (B cells)
  • Minneapolis, Minnesota
    University of Minnesota
Jan 20, 2023

Mucopolysaccharidosis Type IH (MPS IH, Hurler Syndrome), Mucopolysaccharidosis Type IH, MPS IH, Hurler Syndrome Trial

Withdrawn
  • Mucopolysaccharidosis Type IH (MPS IH, Hurler Syndrome)
  • +2 more
  • Autologous Plasmablasts
  • (no location specified)
Oct 5, 2022

MPS-IH (Hurler Syndrome) Trial in Worldwide (Experimental: OTL-203, Active Comparator: Allo-HSCT)

Not yet recruiting
  • MPS-IH (Hurler Syndrome)
  • Experimental: OTL-203
  • Active Comparator: Allo-HSCT
  • Oakland, California
  • +6 more
Nov 27, 2023

(LTFU) of Subjects Who Received SB-318, SB-913, or SB-FIX

Active, not recruiting
  • Hemophilia B
  • +2 more
  • SB-318
  • +2 more
  • Oakland, California
  • +4 more
Jan 30, 2023

Biomarker for Hurler Disease (BioHurler)

Active, not recruiting
  • Mucopolysaccharidosis Type I
  • +2 more
    • Cairo, Egypt
    • +4 more
    May 12, 2021

    Penn SICCA Follow-up Study

    Not yet recruiting
    • Dry Eye
    • Sjogren's Syndrome
      • Philadelphia, Pennsylvania
        Scheie Eye Institute
      Jan 7, 2022

      Mucopolysaccharidosis Disorders, Hurler Syndrome, Hunter Syndrome Trial in Minneapolis (Stem Cell Transplantation, IMD

      Recruiting
      • Mucopolysaccharidosis Disorders
      • +28 more
      • Stem Cell Transplantation
      • +7 more
      • Minneapolis, Minnesota
        Masonic Cancer Center, University of Minnesota
      Nov 1, 2022

      Corneal Nerve Structure in Sjogren's

      Completed
      • Sjogren's Syndrome
      • Vitamin D Deficiency
        • Philadelphia, Pennsylvania
          Scheie Eye Institute of the University of Pennsylvania
        Aug 3, 2021

        MPS I, MPS1-S, MPS1-HS Trial in Porto Alegre, London, Salford (SIG-005 (hIDUA Producing Spheres))

        Withdrawn
        • MPS I
        • +2 more
        • SIG-005 (hIDUA Producing Spheres)
        • Porto Alegre, Brazil
        • +2 more
        Dec 16, 2022

        MPS I Trial in Oakland (SB-318)

        Terminated
        Has Results
        • MPS I
        • SB-318
        • Oakland, California
          UCSF Benioff Children's Hospital Oakland
        Jan 6, 2023

        Hurler Syndrome Trial in Minneapolis (Laronidase)

        Completed
        Has Results
        • Hurler Syndrome
        • Minneapolis, Minnesota
          Masonic Cancer Center, University of Minnesota
        Mar 6, 2020

        Dry Eye Syndrome Trial in Italy, United States (Oxervate, Vehicle)

        Active, not recruiting
        • Dry Eye Syndrome
        • Glendale, California
        • +10 more
        Feb 1, 2023

        Bone Marrow Failure Syndrome, Thalassemia, Sickle Cell Disease Trial in Gainesville (Thiotepa--single daily dose,

        Recruiting
        • Bone Marrow Failure Syndrome
        • +15 more
        • Thiotepa--single daily dose
        • Thiotepa--escalated dose
        • Gainesville, Florida
          UF Health Shands Children's Hospital
        Jul 8, 2021

        Mucopolysaccharidosis I, II, and VI Screening inHigh-Risk

        Terminated
        • Mucopolysaccharidosis (MPS)
          • Los Angeles, California
          • +13 more
          Mar 16, 2021

          Dry Eye Syndromes, Sjogren's Syndrome Trial in Philadelphia (TrueTear Intranasal Tear Neurostimulator)

          Completed
          Has Results
          • Dry Eye Syndromes
          • Sjogren's Syndrome
          • TrueTear Intranasal Tear Neurostimulator
          • Philadelphia, Pennsylvania
            Scheie Eye Institute, University of Pennsylvania
          Aug 12, 2020

          Mucopolysaccharidosis IH Trial in Milano (Frozen autologous CD34+ hematopoietic stem and progenitor cells genetically modified

          Active, not recruiting
          • Mucopolysaccharidosis IH
          • Frozen autologous CD34+ hematopoietic stem and progenitor cells genetically modified with the lentiviral vector IDUA LV, encoding for the α-L-iduronidase cDNA, in their final formulation medium.
          • Milano, Italy
            Ospedale San Raffaele
          Aug 6, 2021

          Mucopolysaccharidosis I Trial (AGT-181)

          Completed
          • Mucopolysaccharidosis I
          • (no location specified)
          Sep 21, 2018

          Hurler Syndrome, Inherited Metabolic Disorder, Lysosomal Storage Disorder Trial in Los Angeles, Palo Alto (rivogenlecleucel,

          No longer available
          • Hurler Syndrome
          • +4 more
          • Los Angeles, California
          • +1 more
          Oct 1, 2020

          Mucopolysaccharidosis I, Hurler Syndrome Trial in Minneapolis (Stem Cell Transplant, Laronidase ERT)

          Completed
          Has Results
          • Mucopolysaccharidosis I
          • Hurler Syndrome
          • Stem Cell Transplant
          • Laronidase ERT
          • Minneapolis, Minnesota
            Masonic Cancer Center, University of Minnesota
          Jul 10, 2019

          Ophthalmic Manifestations of Patients With Mucopolysaccharidosis

          Recruiting
          • Ophthalmic Manifestations
            • Assiut, Egypt
              Assiut University
            Feb 22, 2021

            MRS to Determine Neuroinflammation and Oxidative Stress in MPS I

            Completed
            • Mucopolysaccharidosis Type I
              • Minneapolis, Minnesota
                University of Minnesota
              Oct 30, 2019

              Neurobehavioral Phenotypes in MPS III

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

                Hurler Syndrome, Fanconi Anemia, Glanzmann Thrombasthenia Trial in Atlanta (Abatacept)

                Completed
                • Hurler Syndrome
                • +14 more
                • Atlanta, Georgia
                  Children's Healthcare of Atlanta
                Dec 20, 2019