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Hurler's

Showing 1 - 25 of 62
STUDY
CONDITIONS
INTERVENTIONS
LOCATIONS
LAST UPDATED

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Withdrawn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

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

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

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 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 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

Biomarker for Hurler Disease (BioHurler)

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

    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

    Hurler Syndrome Trial in Minneapolis (Laronidase)

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

    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

    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

    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

      (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

      Long Term Follow-Up for RGX-111

      Enrolling by invitation
      • Mucopolysaccharidosis I
      • No Intervention
      • Orange, California
      • +2 more
      Oct 25, 2023

      Mucopolysaccharidosis I, Mucopolysaccharidosis II, Mucopolysaccharidosis IV A Trial in San Francisco (There is no intervention)

      Recruiting
      • Mucopolysaccharidosis I
      • +7 more
      • There is no intervention
      • San Francisco, California
        University of California San Francisco
      Nov 9, 2022

      Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT With RIC

      Recruiting
      • Primary Immunodeficiency (PID)
      • +4 more
      • data collection
      • Pittsburgh, Pennsylvania
        UPMC Children's Hospital of Pittsburgh
      Feb 9, 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 (IRT Laronidase)

      Completed
      • Hurler Syndrome
      • IRT Laronidase
      • Minneapolis, Minnesota
        University of Minnesota, Fairview
      Nov 30, 2018

      Pediatric Cancer Trial in Manchester (Granulocytes)

      Recruiting
      • Pediatric Cancer
      • Granulocytes
      • Manchester, United Kingdom
        Royal Manchester Childrens Hospital, MFT
      Jun 17, 2022

      Hematologic Malignancies, Inborn Errors of Metabolism Disorders, Immune Deficiencies Trial in Durham (CliniMACS CD34 Reagent

      Available
      • Hematologic Malignancies
      • +2 more
      • CliniMACS CD34 Reagent System
      • Durham, North Carolina
        Duke University Medical Center
      Nov 12, 2021

      Inherited Metabolic Disorders Who Underwent Hematopoietic Stem

      Terminated
      • Inherited Metabolic Disorders (IMD)
      • Safety and efficacy assessments
      • Minneapolis, Minnesota
        University of Minnesota
      Mar 10, 2021

      Mucopolysaccharidosis I, II, and VI Screening inHigh-Risk

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

        Laronidase Enzyme Replacement Therapy With Hematopoietic Stem

        Terminated
        • Mucopolysaccharidosis Type IH
        • +2 more
        • Minneapolis, Minnesota
          Masonic Cancer Center, University of Minnesota
        Sep 25, 2018