Clinical Trials on Genetic Diseases, Inborn

Total 41 results

  • NCT03655678
    Active, not recruiting
    Conditions: Beta-Thalassemia, Thalassemia, Genetic Diseases, Inborn, Hematologic Diseases, Hemoglobinopathies
  • NCT04208529
    Enrolling by invitation
    Conditions: Beta-Thalassemia, Thalassemia, Sickle Cell Disease, Hematologic Diseases, Hemoglobinopathies, Genetic Diseases, Inborn, Sickle Cell Anemia
  • NCT04399694
    Recruiting
    Conditions: Genetic Disease, Inborn Errors of Metabolism, Glycogen Storage Disease, Lysosomal Storage Diseases, Storage Disease
  • NCT05477563
    Recruiting
    Conditions: Beta-Thalassemia, Thalassemia, Hematologic Diseases, Genetic Diseases, Inborn, Hemoglobinopathies, Sickle Cell Anemia, Sickle Cell Disease
  • NCT05126758
    Recruiting
    Conditions: Muscular Dystrophies, Muscular Dystrophy, Duchenne, Muscular Disorders, Atrophic, Muscular Diseases, Neuromuscular Diseases, Genetic Diseases, X-Linked, Genetic Diseases, Inborn, Nervous System Diseases
  • NCT05356195
    Recruiting
    Conditions: Beta-Thalassemia, Thalassemia, Genetic Diseases, Inborn, Hematologic Diseases, Hemoglobinopathies
  • NCT03734588
    Recruiting
    Conditions: Adeno-Associated Virus (AAV), Blood Coagulation Disorder, Blood Coagulation Disorders, Inherited, Coagulation Protein Disorders, Factor VIII (FVIII), Factor VIII (FVIII) Deficiency, Factor VIII (FVIII) Gene, Factor VIII (FVIII) Protein, Genetic Diseases, Inborn, Genetic Diseases, X-Linked, Gene Therapy, Gene Transfer, Hematologic Diseases, Hemorrhagic Disorders, Recombinant, Vector, Inhibitors
  • NCT05479981
    Enrolling by invitation
    Conditions: DM1, Muscular Dystrophies, Myotonic Dystrophy, Myotonic Dystrophy 1, Myotonic Disorders, Muscular Disorders, Atrophic, Muscular Diseases, Musculoskeletal Diseases, Neuromuscular Diseases, Nervous System Diseases, Genetic Diseases, Inborn, Heredodegenerative Disorders, Nervous System, Neurodegenerative Diseases
  • NCT03718234
    Active, not recruiting
    Conditions: Congenital Adrenal Hyperplasia, Hyperplasia, Adrenal Hyperplasia, Congenital Disorders, Adrenocortical Hyperfunction, Disorders of Sex Development, Urogenital Abnormalities, Genetic Diseases, Inborn, Steroid Metabolic Diseases, Inborn, Adrenal Gland Disease, Hydrocortisone
  • NCT03738098
    Completed
    Conditions: Genetic Diseases, Inborn, Genetic Predisposition to Disease
  • NCT03179631
    Active, not recruiting
    Conditions: Muscular Dystrophy, Duchenne, Muscular Dystrophies, Muscular Disorders, Atrophic, Muscular Diseases, Musculoskeletal Disease, Neuromuscular Diseases, Nervous System Diseases, Genetic Diseases, X-Linked, Genetic Diseases, Inborn
  • NCT05290701
    Enrolling by invitation
    Conditions: Congenital Anomalies, Mendelian Disorders, Genetic Diseases, Inborn
  • NCT02921321
    Recruiting
    Conditions: Muscular Dystrophies, Cardiac Fibrosis, Genetic Diseases, Inborn, Musculoskeletal Diseases
  • NCT03609840
    Active, not recruiting
    Conditions: Hematologic Malignancies, Nonmalignant Diseases, Immune Deficiency, Hemoglobinopathies, Genetic Inborn Errors of Metabolism, Fanconi Anemia, Thalassemia, Sickle Cell Disease
  • NCT03876301
    Active, not recruiting
    Conditions: Blood Coagulation Disorder, Blood Coagulation Disorders, Inherited, Coagulation Protein Disorders, Hemophilia A, Genetic Diseases, Inborn, Genetic Diseases, X-Linked, Hematologic Diseases, Hemorrhagic Disorders, Factor VIII Deficiency
  • NCT03548779
    Active, not recruiting
    Conditions: Epilepsy; Seizure, Neuromuscular Diseases, Brain Malformation, Intellectual Disability, Autism Spectrum Disorder, Hypotonia, Inborn Errors of Metabolism, Movement Disorders, Genetic Disease, Development Delay, Chromosome Abnormality, Hearing Loss, Dysmorphic Features, Skeletal Dysplasia, Congenital Abnormality, Microcephaly, Macrocephaly
  • NCT03563066
    Completed
    Conditions: Dermatitis, Atopic, Dermatitis, Eczema, Skin Diseases, Skin Diseases, Genetic, Genetic Diseases, Inborn, Skin Diseases, Eczematous, Hypersensitivity, Hypersensitivity, Immediate, Immune System Diseases
  • NCT05070234
    Not yet recruiting
    Conditions: Small for Gestational Age Infant, Silver-Russell Syndrome, Genetic Diseases, Inborn
  • NCT03609814
    Completed
    Conditions: Hematologic Malignancies, Nonmalignant Diseases, Immunodeficiencies, Hemoglobinopathies, Genetic Inborn Errors of Metabolism, Fanconi's Anemia, Thalassemia, Sickle Cell Disease
  • NCT03609827
    Completed
    Conditions: Hematologic Malignancies, Nonmalignant Diseases, Immunodeficiencies, Hemoglobinopathies, Genetic Inborn Errors of Metabolism, Fanconi's Anemia, Thalassemia, Sickle Cell Disease
  • NCT01316549
    Completed
    Conditions: Hematologic Malignancies, Nonmalignant Diseases, Immunodeficiencies, Hemoglobinopathies, Genetic Inborn Errors of Metabolism, Fanconi Anemia, Thalassemia, Sickle Cell Disease
  • NCT03342495
    Active, not recruiting
    Conditions: Diabetes, Endocrine System Diseases, Gastro-Intestinal Disorder, Neuro-Degenerative Disease, Epilepsy, Autoimmune Diseases, Renal Disease, Cardiac Disease, Metabolic Disease, Genetic Diseases, Inborn, Respiratory Disease, Hematologic Diseases, Autism Spectrum Disorder, Fetal Alcohol Spectrum Disorders, Traumatic Brain Injury, Stroke
  • NCT03627767
    Completed
    Conditions: Dermatitis, Dermatitis, Atopic, Eczema, Skin Diseases, Skin Diseases, Genetic, Genetic Diseases, Inborn, Skin Diseases, Eczematous, Hypersensitivity, Hypersensitivity, Immediate, Immune System Diseases
  • NCT02884063
    Completed
    Conditions: Chromosome Abnormalities, Genetic Diseases, Inborn, Mutation
  • NCT01963650
    Terminated
    Conditions: Lipid Metabolism Disorders, Metachromatic Leukodystrophy (MLD), Nervous System Diseases, Brain Diseases, Central Nervous System Diseases, Demyelinating Diseases, Metabolism, Inborn Errors, Genetic Diseases, Inborn, Sphingolipidoses, Hereditary Central Nervous System Demyelinating Diseases, Metabolic Inborn Brain Diseases, Lysosomal Storage Diseases, Metabolic Diseases, Sulfatidosis