Example: Heart Attack

Clinical Trials on Genetic Diseases, X-Linked

Total 1082 results

    • NCT04157595
      Enrolling by invitation
      Mackenzie's Mission: The Australian Reproductive Carrier Screening Project
      Conditions: X-Linked Genetic Diseases; Autosomal Recessive Disorder
    • NCT00359411
      Completed
      Genetic Studies of X-linked Lymphoproliferative Disease
      Conditions: X-Linked Lymphoproliferative Disease; Lymphoproliferative Disease; Genetic Diseases, X-Linked
    • NCT00100230
      Completed
      DHA and X-Linked Retinitis Pigmentosa
      Conditions: Retinitis Pigmentosa; X-linked Genetic Diseases
    • NCT00001405
      Recruiting
      Use of G-CSF to Obtain Blood Cell Precursors
      Conditions: Granuloma; Granulomatous Disease, Chronic; Leukocyte Disease; Genetic Disease, X-Linked; Genetic Disease, Inborn
    • NCT03512314
      Recruiting
      Therapeutic Use of Tadekinig Alfa in NLRC4 Mutation and XIAP Deficiency as Open Label Extension
      Conditions: XIAP Deficiency; NLRC4-MAS
    • NCT03113760
      Recruiting
      Therapeutic Use of Tadekinig Alfa in NLRC4 Mutation and XIAP Deficiency
      Conditions: NLRC4-MAS; XIAP Deficiency
    • NCT03321604
      Recruiting
      Kidney Information Network for Disease Research and Education
      Conditions: ESRD; Fabry Disease; Rare Diseases; Kidney Diseases; End Stage Renal Disease; Genetic Diseases, X-Linked
    • NCT03275714
      Completed
      Evaluation of an App for Smartphones for People With a Bipolar Affective Disorder
      Conditions: Bipolar Affective Disorder
    • NCT00397605
      Withdrawn
      Cannabinoids in Bipolar Affective Disorder
      Conditions: Bipolar Affective Disorder
    • NCT03862950
      Recruiting
      A Trial of Metformin in Individuals With Fragile X Syndrome (Met)
      Conditions: Fragile X Syndrome; Fragile X Mental Retardation Syndrome; Mental Retardation, X-Linked; Genetic Diseases, X-Linked; Trinucleotide Repeat Expansion; Fra(X) Syndrome; Intellectual Disability; FXS; Neurobehavioral Manifestations; Sex Chromosome Disorders
    • NCT03479476
      Recruiting
      A Trial of Metformin in Individuals With Fragile X Syndrome
      Conditions: Fragile X Syndrome; Fragile X Mental Retardation Syndrome; Mental Retardation, X Linked; Genetic Diseases, X-Linked; Trinucleotide Repeat Expansion; Fra(X) Syndrome; Intellectual Disability; FXS; Neurobehavioral Manifestations; Sex Chromosome Disorders
    • NCT03876301
      Active, not recruiting
      Lead-in Study to Collect Prospective Efficacy and Safety Data of Current FVIII Prophylaxis Replacement Therapy in Adult Hemophilia A Participants
      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
    • NCT03734588
      Active, not recruiting
      Dose-finding Study of SPK-8016 Gene Therapy in Patients With Hemophilia A to Support Evaluation in Individuals With FVIII Inhibitors
      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
    • NCT03406780
      Completed
      A Study of CAP-1002 in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy
      Conditions: Muscular Dystrophies; Muscular Dystrophy, Duchenne; Muscular Disorders, Atrophic; Muscular Diseases; Neuromuscular Diseases; Nervous System Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn
    • NCT03179631
      Recruiting
      Long-Term Outcomes of Ataluren in Duchenne Muscular Dystrophy
      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
    • NCT02090959
      Terminated
      An Extension Study of Ataluren (PTC124) in Participants With Nonsense Mutation Dystrophinopathy
      Conditions: Muscular Dystrophy, Duchenne; Muscular Dystrophies; Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn
    • NCT01826487
      Completed
      Phase 3 Study of Ataluren in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)
      Conditions: Muscular Dystrophy, Duchenne; Muscular Dystrophies; Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn
    • NCT03019458
      Completed
      MINGO Supplemental Trial in X-linked Dystonia-Parkinsonism Patients
      Conditions: X-Linked Dystonia Parkinsonism
    • NCT04168697
      Not yet recruiting
      Effect of Behavioral Intervention on Cannabinoid Receptors in BAD
      Conditions: Bipolar Affective Disorder, Currently in Remission; Bipolar Disorder I; Bipolar Disorder II
    • NCT03249857
      Completed
      Study of Sensorimotor Compatibility Effects in Bipolar Affective Disorder.
      Conditions: Bipolar Affective Disorder
    • NCT00474357
      Unknown status
      Myths About Bipolar Affective Disorder: The Role of Structured Group Psychoeducation Therapy
      Conditions: Bipolar Affective Disorder
    • NCT01108068
      Completed
      Trial of Lithium Carbonate for Treatment of Osteoporosis-pseudoglioma Syndrome
      Conditions: Osteoporosis Pseudoglioma
    • NCT02642653
      Completed
      Combining Lovastatin and a Parent-Implemented Language Intervention for Fragile X Syndrome
      Conditions: Fragile X Syndrome; Genetic Diseases
    • NCT02484560
      Unknown status
      Efficacy of Stem Cell Therapy in Ambulatory and Non-ambulatory Children With Duchenne Muscular Dystrophy - Phase 1-2
      Conditions: Duchenne Muscular Dystrophy
    • NCT04539340
      Completed
      A Multi-cohort Study of the Tolerance, Safety, and Pharmacokinetics of GNR-055 in Healthy Volunteers
      Conditions: Mucopolysaccharidosis Type II; Metabolic Diseases
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