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orphan drug

Showing 1 - 25 of >10,000
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Fanconi Anemia Complementation Group A Trial in Madrid (RP-L102)

Recruiting
  • Fanconi Anemia Complementation Group A
  • RP-L102
  • Madrid, Spain
    Hospital Infantil Universitario Niño Jesús (HIUNJ)
Oct 16, 2020

Orphan Drugs for Inherited Metabolic Diseases

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

    Evaluate the Use and Effectiveness of Doptelet® in ITP

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

    Advanced Cancer Trial in New Orleans, Detroit (4-Demethyl-4-cholesteryloxycarbonylpenclomedine)

    Completed
    • Advanced Cancer
    • New Orleans, Louisiana
    • +2 more
    Nov 3, 2022

    SARS-CoV 2, COVID, ARDS Trial (ZYESAMI™ (aviptadil acetate), Placebo, Nebulized administration of ZYESAMI™ or Placebo)

    Withdrawn
    • SARS-CoV 2
    • +5 more
    • ZYESAMI™ (aviptadil acetate)
    • +2 more
    • (no location specified)
    Feb 2, 2023

    Epidermolysis Bullosa Trial in Chicago (APR-TD011)

    Not yet recruiting
    • Epidermolysis Bullosa
    • APR-TD011
    • Chicago, Illinois
      Ann & Robert H. Lurie Children's Hospital of Chicago
    Sep 6, 2022

    Primary Hemophagocytic Lymphohistiocytosis Trial in China (Emapalumab-Lzsg 5 MG/ML [Gamifant])

    Recruiting
    • Primary Hemophagocytic Lymphohistiocytosis
    • Emapalumab-Lzsg 5 MG/ML [Gamifant]
    • Shanghai, Fudan, China
    • +6 more
    Feb 23, 2023

    Real-world Effectiveness and Usage of Alprolix in Haemophilia B

    Completed
    • Haemophilia B
    • Bordeaux, France
    • +17 more
    Jan 11, 2023

    SARS-CoV 2, COVID, ARDS Trial in United States (ZYESAMI™ (aviptadil acetate), Placebo, Nebulized administration of ZYESAMI™ or

    Terminated
    • SARS-CoV 2
    • +5 more
    • ZYESAMI™ (aviptadil acetate)
    • +2 more
    • Fullerton, California
    • +12 more
    Feb 2, 2023

    rFVIIIFc (Elocta®) ITI Chart Review in Haemophilia A

    Completed
    • Hemophilia A With Inhibitor
    • rFVIIIFc
    • Paris, France
    • +17 more
    Jan 11, 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

    Non Severe Aplastic Anemia, Untreated Trial in China (Herombopag)

    Recruiting
    • Non Severe Aplastic Anemia
    • Untreated
    • Tangshan, Hebei, China
    • +3 more
    Dec 14, 2022

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

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

    Real-world Usage and Effectiveness of Elocta and Alprolix in

    Completed
    • Haemophilia A
    • Haemophilia B
    • Berlin, Germany
    • +21 more
    May 3, 2022

    Evaluate Effectiveness of Elocta Compared to Conventional Factor

    Completed
    • Hemophilia A
    • efmoroctocog alfa
    • Factor VIII
    • Innsbruck, Austria
    • +35 more
    Mar 30, 2022

    Sickle Cell Disease, Pain Trial in Bronx (Immune Globulin Intravenous, Normal saline)

    Recruiting
    • Sickle Cell Disease
    • Pain
    • Immune Globulin Intravenous
    • Normal saline
    • Bronx, New York
      Montefiore Medical Center
    Jul 25, 2022

    Idiopathic Pulmonary Fibrosis Trial in Hialeah Gardens (20mM sodium pyruvate nasal spray, Saline Placebo control nasal spray)

    Recruiting
    • Idiopathic Pulmonary Fibrosis
    • 20mM sodium pyruvate nasal spray
    • Saline Placebo control nasal spray
    • Hialeah Gardens, Florida
      Family First Medical Research Center
    Sep 16, 2023

    Hereditary Hemorrhagic Telangiectasia, HHT, Rendu Osler Weber Disease Trial in Madrid (Registry)

    Recruiting
    • Hereditary Hemorrhagic Telangiectasia
    • +2 more
    • Registry
    • Madrid, Spain
      Hospital Universitario Ramón y Cajal
    Sep 30, 2022

    Tuberous Sclerosis Complex, Epilepsy Trial in United States (Sirolimus, Placebo)

    Recruiting
    • Tuberous Sclerosis Complex
    • Epilepsy
    • Birmingham, Alabama
    • +7 more
    Sep 2, 2022

    Advanced Cancer, Brain Metastases Trial in New Orleans (DM-CHOC-PEN)

    Completed
    • Advanced Cancer
    • Brain Metastases
    • New Orleans, Louisiana
      Tulane University Medical Center
    Nov 3, 2022

    GM2 Gangliosidosis, Supportive Care Trial in Kashan, Mashhad, Tehran (Miglustat)

    Recruiting
    • GM2 Gangliosidosis
    • Supportive Care
    • Kashan, Isfahan, Iran, Islamic Republic of
    • +2 more
    Oct 23, 2022

    Fanconi Anemia Trial in Barcelona, Madrid (procedure, biological, other, drug)

    Unknown status
    • Fanconi Anemia
    • IV administration of Genetically Engineered Hematopoietic Stem/Progenitors Cells (HSPCs)
    • +5 more
    • Barcelona, Spain
    • +1 more
    Nov 23, 2020

    Amputation, Surgical Trial in Pittsburgh (K-Socket-Harness)

    Not yet recruiting
    • Amputation, Surgical
    • K-Socket-Harness
    • Pittsburgh, Pennsylvania
      VA Pittsburgh Healthcare System University Drive Division, Pitts
    Aug 4, 2023

    Transfusion-dependent Thalassemia Trial in Dhaka (Thalidomide Capsules, Placebo)

    Recruiting
    • Transfusion-dependent Thalassemia
    • Thalidomide Capsules
    • Placebo
    • Dhaka, Bangladesh
      Bangabandhu Sheikh Mujib Medical University
    Oct 18, 2023

    Sarcoma Trial in Seoul (Durvalumab, pazopanib)

    Recruiting
    • Sarcoma
    • Durvalumab, pazopanib
    • Seoul, Korea, Republic of
      Severance Hospital, Yonsei University Health System
    Apr 21, 2021