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ADA-SCID

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STUDY
CONDITIONS
INTERVENTIONS
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Patients Previously Treated With Autologous ex Vivo Gene Therapy

Enrolling by invitation
  • Adenosine Deaminase Deficiency
  • Severe Combined Immunodeficiency (SCID)
  • autologous ex vivo gene therapy products based on the EFS LV encoding for the human adenosine deaminase (ADA) gene (EFS-ADA LV)
  • Los Angeles, California
  • +1 more
Mar 31, 2022

Adenosine Deaminase Severe Combined Immune Deficiency Trial (A cryopreserved formulation of autologous mPB CD34+ hematopoietic

Not yet recruiting
  • Adenosine Deaminase Severe Combined Immune Deficiency
  • A cryopreserved formulation of autologous mPB CD34+ hematopoietic stem and progenitor cells transduced ex vivo with the EFS-ADA lentiviral vector encoding the human ADA enzyme
  • (no location specified)
Jun 20, 2022

Severe Combined Immunodeficiency Due to ADA Deficiency Trial in London (Infusion of autologous cryopreserved EFS-ADA LV CD34+

Active, not recruiting
  • Severe Combined Immunodeficiency Due to ADA Deficiency
  • Infusion of autologous cryopreserved EFS-ADA LV CD34+ cells (OTL-101)
  • +2 more
  • London, United Kingdom
    Great Ormond Street Hospital for Children NHS Foundation Trust
Dec 17, 2021

Immunologic Deficiency Syndromes Trial in Milano (Strimvelis)

Enrolling by invitation
  • Immunologic Deficiency Syndromes
  • Strimvelis
  • Milano, Lombardia, Italy
    Orchard Investigational Site
Jun 23, 2021

Severe Combined Immunodeficiency Trial in Los Angeles (ADA gene transfer)

Completed
  • Severe Combined Immunodeficiency
  • ADA gene transfer
  • Los Angeles, California
    University of California, Los Angeles
Apr 21, 2021

Primary Immunodeficiency Diseases

Active, not recruiting
  • Wiskott- Aldrich Syndrome
  • ADA Deficient SCID
    • Bethesda, Maryland
      National Institutes of Health Clinical Center
    Jan 13, 2023

    Adenosine Deaminase Deficiency, Severe Combined Immunodeficiencies (SCID) Trial in London (genetic, other, drug)

    Completed
    • Adenosine Deaminase Deficiency
    • Severe Combined Immunodeficiencies (SCID)
    • Infusion of autologous EFS-ADA LV CD34+ cells
    • +3 more
    • London, United Kingdom
      Great Ormond Street Hospital for Children NHS Foundation Trust
    Aug 20, 2021

    Immunologic Deficiency Syndromes Trial in Jerusalem, Milano (Gene Therapy, Busulfan, PEG-ADA)

    Completed
    • Immunologic Deficiency Syndromes
    • Gene Therapy
    • +2 more
    • Jerusalem, Israel
    • +1 more
    Jul 15, 2020

    Quality of Life and Neuropsychiatric Sequelae in Patients

    Withdrawn
    • ADA-SCID
      • (no location specified)
      Dec 14, 2019

      ADA-SCID, Adenosine Deaminase Deficiency, Severe Combined Immunodeficiency Trial in United States (EZN-2279, Adagen)

      Completed
      • ADA-SCID
      • +2 more
      • EZN-2279
      • Adagen
      • Los Angeles, California
      • +5 more
      Apr 6, 2020

      Adenosine DeAminase Severe Combined ImmunoDeficiency (ADA-SCID) Trial in Shenzhen (TYF-ADA gene-modified autologous stem cells)

      Unknown status
      • Adenosine DeAminase Severe Combined ImmunoDeficiency (ADA-SCID)
      • TYF-ADA gene-modified autologous stem cells
      • Shenzhen, Guangdong, China
        Shenzhen Geno-immune Medical Institute
      Sep 18, 2019

      Natural History Study of SCID Disorders

      Enrolling by invitation
      • Severe Combined Immunodeficiency (SCID)
      • +5 more
        • Birmingham, Alabama
        • +43 more
        Nov 9, 2020

        Patients Treated for SCID (1968-Present)

        Enrolling by invitation
        • SCID
        • +5 more
          • Birmingham, Alabama
          • +35 more
          Nov 9, 2020

          Severe Combined Immunodeficiency Syndrome Trial run by the NHGRI (CD34+ cells transduced with ADA retrovir)

          Completed
          • Severe Combined Immunodeficiency Syndrome
          • CD34+ cells transduced with ADA retrovir
          • Bethesda, Maryland
            National Institutes of Health Clinical Center, 9000 Rockville Pi
          Dec 11, 2019

          Severe Combined Immunodeficiency Due to ADA Deficiency Trial in Milan (STRIMVELIS)

          Completed
          • Severe Combined Immunodeficiency Due to ADA Deficiency
          • Milan, Italy
            Ospedale San Raffaele - Telethon Institute for Gene Therapy (OSR
          Feb 18, 2022

          Investigation of ADA Enzyme Deficiency

          Enrolling by invitation
          • Adenosine Deaminase Deficiency
          • adenosine deaminase test
          • Ankara, Mamak, Turkey
            Ankara Üniversitesi Tıp Fakültesi
          Mar 18, 2022

          Enzyme Deficiency in Pulmonology Clinics (ADA)

          Active, not recruiting
          • Bronchiectasis
          • +2 more
          • adenosine deaminase enzyme deficiency test
          • Ankara, Mamak, Turkey
            Ankara University Medical Faculty
          Mar 18, 2022

          Adenosine Deaminase Enzyme Deficiency Disease in Adult Pulmonary

          Enrolling by invitation
          • Pulmonary Alveolar Proteinosis
          • adenosine deaminase test
          • Istanbul, Maltepe, Turkey
            Istanbul Yedikule Chest Diseases and Thoracic Surgery Training a
          Mar 18, 2022

          Adenosine Deaminase Enzyme Deficiency in Lymphopenia and/or

          Enrolling by invitation
          • Adenosine Deaminase Deficiency
          • ADENOSINE DEAMINASE TEST
          • Konya, Meram, Turkey
            Konya Necmettin Erbakan University
          Mar 18, 2022

          Severe Combined Immunodeficiency Due to RAG1 Deficiency Trial in Leiden (Gene therapy)

          Recruiting
          • Severe Combined Immunodeficiency Due to RAG1 Deficiency
          • Gene therapy
          • Leiden, Netherlands
            Leiden University Medical Center
          Dec 3, 2021

          Retroviral Insertion Site Analysis in Strimvelis Gene Therapy

          Enrolling by invitation
          • Severe Combined Immunodeficiency Due to ADA Deficiency
          • Retroviral Insertion Site Analysis
          • Milan, Italy
            Ospedale San Raffaele - Telethon Institute for Gene Therapy (OSR
          Feb 17, 2022

          Severe Combined Immunodeficiency Trial in Memphis (drug, device, other)

          Recruiting
          • Severe Combined Immunodeficiency
          • Anti-thymocyte globulin (rabbit)
          • +5 more
          • Memphis, Tennessee
            St. Jude Children's Research Hospital
          Oct 6, 2021

          Immune System Diseases Trial (Strimvelis)

          Withdrawn
          • Immune System Diseases
          • Strimvelis
          • (no location specified)
          Jul 13, 2020

          Immune-mediated Diseases

          Recruiting
          • Primary Immunodeficiency
          • +3 more
            • Bethesda, Maryland
              National Institutes of Health Clinical Center
            Jan 10, 2023

            The Role of Bacteria and Genetic Variations in Cystic Fibrosis

            Completed
            • Psuedomas Infection
            • Cystic Fibrosis
              • Bethesda, Maryland
              • +3 more
              Jun 25, 2021