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Hurler Syndrome

Showing 1 - 25 of >10,000
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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Withdrawn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

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 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

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 Trial in Minneapolis (Laronidase)

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

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

    Biomarker for Hurler Disease (BioHurler)

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

      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

      Hurler Syndrome Trial in Minneapolis (IRT Laronidase)

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

      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

      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

      Pediatric Cancer Trial in Manchester (Granulocytes)

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

      Laronidase Enzyme Replacement Therapy With Hematopoietic Stem

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

      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

      (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

      Inherited Metabolic Disorders Who Underwent Hematopoietic Stem

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

      Hurler Syndrome, Cerebral Palsy, Juvenile Rheumatoid Arthritis Trial in Dallas (Growth Hormone, Whole body Protein turnover,

      Withdrawn
      • Hurler Syndrome
      • +4 more
      • Growth Hormone
      • +2 more
      • Dallas, Texas
        Children's Medical Center of Dallas
      Dec 12, 2018

      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

      Mucopolysaccharidosis I, II, and VI Screening inHigh-Risk

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