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Pyruvate Metabolism, Inborn Errors

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STUDY
CONDITIONS
INTERVENTIONS
LOCATIONS

Induced Pluripotent Stem Cells for Development of Novel Drug

Recruiting
  • Inborn Errors of Metabolism
  • Skin biopsy
  • Withdraw of blood
  • Lahore, Pakistan
    Children Hospital and Institute of Child Health
May 14, 2021

Carbohydrate Intolerance Trial in Springfield (Low-FODMAP diet group (LFD), Low Sugar diet group (LAS))

Recruiting
  • Carbohydrate Intolerance
  • Low-FODMAP diet group (LFD)
  • Low Sugar diet group (LAS)
  • Springfield, Oregon
    Oregon Health and Science University
Sep 21, 2023

COVID-19 and Hereditary Metabolic Diseases

Recruiting
  • Covid19
  • Metabolism, Inborn Errors
    • Lille, France
      Hop Claude Huriez Chu Lille
    Feb 28, 2022

    Medium-chain Acyl-CoA Dehydrogenase Deficiency Trial in Pittsburgh (Triheptanoin)

    Recruiting
    • Medium-chain Acyl-CoA Dehydrogenase Deficiency
    • Pittsburgh, Pennsylvania
      UPMC Children's Hospital of Pittsburgh
    Sep 27, 2023

    Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS) Trial (Triheptanoin)

    Available
    • Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS)
    • (no location specified)
    Nov 22, 2022

    A Non-Interventional Post-Authorization Study of Carbaglu for

    Enrolling by invitation
    • Hyperammonemia
    • +2 more
    • carglumic acid
    • Indianapolis, Indiana
      Riley Children's Hospital
    Apr 24, 2023

    Medium-chain Acyl-CoA Dehydrogenase Deficiency Trial in Pittsburgh (Sodium phenylbutyrate)

    Recruiting
    • Medium-chain Acyl-CoA Dehydrogenase Deficiency
    • Sodium phenylbutyrate
    • Pittsburgh, Pennsylvania
      UPMC Children's Hospital of Pittsburgh
    Oct 4, 2023

    Mitochondrial Diseases, Pearson Syndrome Trial in Ramat Gan (MNV-201)

    Recruiting
    • Mitochondrial Diseases
    • Pearson Syndrome
    • MNV-201
    • Ramat Gan, Israel
      Sheba Medical Center
    Aug 24, 2023

    Thiotepa and TEPA Drug Exposure in Pediatric Hematopoietic Stem

    Active, not recruiting
    • Hematologic Malignancies
    • +7 more
    • San Francisco, California
      University of California, San Francisco
    Feb 28, 2022

    Urea Cycle Disorder, Inborn Errors of Metabolism Trial in Philadelphia (UCD Anamix Infant)

    Completed
    • Urea Cycle Disorder
    • Inborn Errors of Metabolism
    • UCD Anamix Infant
    • Philadelphia, Pennsylvania
      Children's hospital of Philadelphia
    Dec 8, 2021

    Methylmalonic Acidemia (MMA) Trial run by the Selecta Biosciences, Inc. (SEL-302)

    Recruiting
    • Methylmalonic Acidemia (MMA)
    • Bethesda, Maryland
      National Human Genome Research Institute, National Institutes of
    Mar 9, 2023

    Patients Who Received hLB-001 Gene Therapy

    Recruiting
    • Methylmalonic Acidemia
    • Nashville, Tennessee
    • +1 more
    Aug 17, 2022

    Pyruvate Kinase Deficiency, Congenital Non-Spherocytic Hemolytic Anemia Trial in Worldwide

    Completed
    • Pyruvate Kinase Deficiency
    • Congenital Non-Spherocytic Hemolytic Anemia
      • Phoenix, Arizona
      • +29 more
      May 20, 2020

      Cardiac Manifestation and Inherited Metabolic Diseases

      Recruiting
      • Metabolism, Inborn Errors
        • Lausanne, Vaud, Switzerland
          Lausanne University Hospital
        Aug 7, 2021

        Leukemia, Inborn Errors of Metabolism, Bone Marrow Failure Syndromes Trial in Philadelphia (Transplant of stem cells with

        Recruiting
        • Leukemia
        • +4 more
        • Transplant of stem cells with CD3+/CD19+ depletion (CliniMACs)
        • Philadelphia, Pennsylvania
          Children's Hospital of Philadelphia
        Nov 2, 2021

        Methylmalonic Acidemia, Propionic Acidemia Trial in San Francisco, Remote (Registry)

        Recruiting
        • Methylmalonic Acidemia
        • Propionic Acidemia
        • Registry
        • San Francisco, California
        • +2 more
        Jun 27, 2022

        Melphalan Drug Exposure in Pediatric Hematopoietic Stem Cell

        Completed
        • Hematologic Malignancies
        • +7 more
        • San Francisco, California
          University of California, San Francisco
        Sep 27, 2021

        Fludarabine Drug Exposure in Pediatric Bone Marrow

        Completed
        • Hematologic Malignancies
        • +7 more
        • San Francisco, California
          University of California, San Francisco
        Sep 27, 2021

        GLUT1 Deficiency Syndrome, Glucose Transporter Type 1 Deficiency Syndrome, Glucose Transporter type1 (GLUT-1) Deficiency Trial

        Recruiting
        • GLUT1 Deficiency Syndrome
        • +3 more
        • No intervention
        • Dallas, Texas
          UT Southwestern Medical Center
        Feb 1, 2022

        Clofarabine and Fludarabine Drug Exposure in Pediatric Bone

        Completed
        • Hematologic Malignancies
        • +7 more
        • San Francisco, California
          University of California, San Francisco
        Sep 27, 2021

        Methylmalonic Acidemia Trial in United States (hLB-001)

        Recruiting
        • Methylmalonic Acidemia
        • hLB-001
        • Atlanta, Georgia
        • +3 more
        Aug 16, 2022

        Measurements in Inborn Errors of Metabolism

        Completed
        • Inborn Errors of Metabolism
        • +7 more
        • Survey
        • Zurich, Switzerland
          University Children's Hospital Zurich
        Nov 3, 2020

        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

        Congenital Sucrase-Isomaltase Deficiency, CSID, Sucrase Isomaltase Deficiency Trial in Buffalo, Oklahoma City (Sucraid

        Recruiting
        • Congenital Sucrase-Isomaltase Deficiency
        • +2 more
        • Sucraid (sacrosidase) Oral Solution 8500 IU/mL
        • Buffalo, New York
        • +1 more
        Aug 16, 2022

        Carbaglu® for Treatment of MMA and PA in Adults and Pediatrics

        Recruiting
        • Hyperammonemia
        • +2 more
        • Carglumic Acid
        • Washington, District of Columbia
        • +1 more
        Jun 30, 2022