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Orphan Disease

Showing 1 - 25 of >10,000
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Completed
  • Eczema/Atopic Dermatitis
  • Cancer
    • Boston, Massachusetts
      Boston Children's Hopsital
    May 6, 2022

    CDKL5 Deficiency Disorder (CDD) Trial in Philadelphia (Survey Instrument)

    Recruiting
    • CDKL5 Deficiency Disorder (CDD)
    • Survey Instrument
    • Philadelphia, Pennsylvania
      University of Pennsylvania Orphan Disease Center
    Sep 24, 2021

    STXBP1 Encephalopathy With Epilepsy, SLC6A1 Neurodevelopmental Disorder Trial in Aurora, New York (Glycerol Phenylbutyrate 1100

    Active, not recruiting
    • STXBP1 Encephalopathy With Epilepsy, SLC6A1 Neurodevelopmental Disorder
    • Glycerol Phenylbutyrate 1100 MG/ML [Ravicti]
    • Aurora, Colorado
    • +1 more
    Jan 24, 2023

    Autoimmune Inner Ear Disease Trial in New Hyde Park (Anakinra 100Mg/0.67Ml Inj Syringe (Period 1), Placebo injection (Period 1),

    Recruiting
    • Autoimmune Inner Ear Disease
    • Anakinra 100Mg/0.67Ml Inj Syringe (Period 1)
    • +3 more
    • New Hyde Park, New York
      Northwell Health, Hearing& Speech Center
    Oct 18, 2022

    Kawasaki Disease Trial in Le Kremlin-Bicêtre (ANAKINRA, Intravenous immunoglobulin)

    Not yet recruiting
    • Kawasaki Disease
    • Le Kremlin-Bicêtre, Val De Marne, France
      CHU de Bicêtre
    Jun 17, 2022

    GVHD, Malignancy Trial in Canada, United States (Abatacept, )

    Active, not recruiting
    Has Results
    • Graft vs Host Disease
    • Malignancy
    • Los Angeles, California
    • +13 more
    Jan 9, 2023

    Alpha-1 Antitrypsin Deficiency (AATD), Pi*ZZ, Pi*SZ, Pi*Null, Another Rare Phenotype/Genotype Known to be Associated With Either

    Recruiting
    • Alpha-1 Antitrypsin Deficiency (AATD)
    • +2 more
    • Alvelestat (MPH966)
    • Placebo
    • Birmingham, Alabama
    • +8 more
    Aug 31, 2021

    Duchenne Muscular Dystrophy Trial in Hyogo, Tokyo (DS-5141b)

    Completed
    • Duchenne Muscular Dystrophy
    • Hyogo, Kobe-shi, Japan
    • +1 more
    Dec 14, 2020

    COVID-19 Trial in India, United Kingdom (C21, Placebo)

    Completed
    Has Results
    • COVID-19
    • Ahmadabad, Gujarat, India
    • +8 more
    Jun 21, 2021

    Fatty Acid Oxidation Disorder Trial in Moscow (Lipano formula)

    Completed
    • Fatty Acid Oxidation Disorder
    • Lipano formula
    • Moscow, Russian Federation
      Russian Children's Hospital Center for Orphan Diseases
    May 21, 2020

    Oral Health Status and Diet History Analysis Among Orphan House

    Unknown status
    • Gingival Disease Due to Bacteria
      • (no location specified)
      Nov 18, 2019

      Vascular Disease Discovery Protocol

      Recruiting
      • Vascular Dysfunction
      • +2 more
        • Bethesda, Maryland
          National Institutes of Health Clinical Center
        Jan 25, 2023

        Pseudomyxoma Peritonei, Peritoneal Cancer, Mucinous Adenocarcinoma Trial (Bromelain, Acetylcysteine, Interventional radiology

        Not yet recruiting
        • Pseudomyxoma Peritonei
        • +3 more
        • (no location specified)
        Feb 28, 2022

        IFN? and Inflammatory Mediators in Hemophagocytic

        Completed
        • Hemophagocytic Lymphohistiocytoses
        • Blood Draws Data Collection
        • Boston, Massachusetts
          Brigham and Women's Hospital
        Sep 14, 2023

        Gene Discovery Core, The Manton Center

        Recruiting
        • Undiagnosed Conditions
        • +2 more
          • Boston, Massachusetts
            Boston Children's Hospital
          Aug 9, 2021

          Hematopoietic and Lymphoid Cell Tumor, Malignant Solid Tumor, Metastatic Lung Non-Small Cell Carcinoma Trial in Seattle

          Terminated
          Has Results
          • Hematopoietic and Lymphoid Cell Neoplasm
          • +12 more
          • Laboratory Biomarker Analysis
          • ROR1 CAR-specific Autologous T-Lymphocytes
          • Seattle, Washington
            Fred Hutch/University of Washington Cancer Consortium
          Aug 11, 2022

          Genetic Traits and Linkages in Families

          Recruiting
          • Hidradenitis Suppurativa
            • Chicago, Illinois
              University of Chicago Medicine
            Jan 25, 2023

            Mortality of Rare Diseases in Taiwan With Real-world Evidence

            Completed
            • Rare Diseases
            • +5 more
            • longitudinal observational study
            • Tainan, Taiwan
              Department of Family Medicine, National Cheng Kung Univ Hosp
            May 9, 2022

            Evaluate the Use and Effectiveness of Doptelet® in ITP

            Recruiting
            • Immune Thrombocytopenia
            • ITP
            • Zagreb, Croatia
            • +59 more
            Jan 12, 2023

            Macrophage Activation Syndrome, Secondary Hemophagocytic Lymphohistiocytosis, Still Disease Trial in Worldwide (Emapalumab)

            Recruiting
            • Macrophage Activation Syndrome
            • +6 more
            • Birmingham, Alabama
            • +41 more
            Jan 3, 2023

            Resilience in Pulmonary Arterial Hypertension and Genetically

            Recruiting
            • Idiopathic Pulmonary Arterial Hypertension
            • +3 more
              • Nashville, Tennessee
                Vanderbilt University Medical Center
              Dec 9, 2022

              LGMD Trial (GenPHSat injection (Safety), GenPHSat injection (Efficacy), Muscle Biopsy (Safety))

              Not yet recruiting
              • LGMD
              • GenPHSat injection (Safety)
              • +2 more
              • (no location specified)
              Oct 20, 2022

              Still's Disease, Juvenile Onset, Still's Disease, Adult-Onset Trial in Japan (Anakinra, Placebo)

              Recruiting
              • Still's Disease, Juvenile Onset
              • Still's Disease, Adult-Onset
              • Chiba-shi, Chiba, Japan
              • +17 more
              Apr 3, 2023

              Orphan Drugs for Inherited Metabolic Diseases

              Recruiting
              • Inborn Errors of Metabolism
                • Lausanne, Vaud, Switzerland
                  Lausanne University Hospital
                Apr 19, 2023

                Kawasaki Disease, Children Trial in Le Kremlin-Bicêtre (Anakinra)

                Completed
                • Kawasaki Disease
                • Children
                • Le Kremlin-Bicêtre, France
                  AP-HP,Bicêtre Hospital
                Jul 1, 2019