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Clinical trial condition

Genetic Diseases, X-Linked

Explore clinical trials studying Genetic Diseases, X-Linked. Study availability and eligibility vary by location and protocol.

1,242 public trials 227 recruiting studies
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Genetic Diseases, X-Linked trial results

Recruiting

Functional Capacity in Anderson-Fabry Disease Patients

NCT06884358

Conditions
Anderson-Fabry Disease, Brain Diseases, Brain Diseases, Metabolic, +20 more
Locations
  • Spedali Civili Hospital Brescia, Italy
  • Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico Milan, Italy
  • IRCCS Policlinico San Donato San Donato Milanese, Milan, Italy
  • Fondazione IRCCS San Gerardo dei Tintori Monza, Italy
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Recruiting

A Study of Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of SR604 in Two Participants Groups (Part A: Healthy Participants, and Part B: Participants With Hemophilia A or Hemophilia B or Factor VII Deficiency)

NCT06349473

Conditions
Blood Coagulation Disorders, Blood Coagulation Disorders, Inherited, Coagulation Protein Disorders, +10 more
Locations
  • California Clinical Trials Medical Group (CCTMG) Glendale, California, United States
  • Children's Hospital Los Angeles Los Angeles, California, United States
  • Rush University Medical Center Chicago, Illinois, United States
  • LA Center for Bleeding and Clotting Disorders - Metairie Metairie, Louisiana, United States
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Recruiting

A Study Evaluating the Real-World Experience of Givinostat in Patients With Duchenne Muscular Dystrophy

NCT07127978

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Disease Attributes, Duchene Muscular Dystrophy, +12 more
Locations
  • Arkansas Children's Research Institute Little Rock, Arkansas, United States
  • Children's National Hospital Washington D.C., District of Columbia, United States
  • Ann and Robert H. Lurie Children's Hospital of Chicago Chicago, Illinois, United States
  • Boston Children's Hospital Boston, Massachusetts, United States
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Recruiting

A Study to Evaluate the Tolerability, Safety and Efficacy of GNR-097 Gene Therapy in Pediatric Patients With Duchenne Muscular Dystrophy

NCT07673809

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Duchenne Muscular Dystrophy, Genetic Diseases, Inborn, +8 more
Locations
  • Republican Scientific and Practical Center Mother and Child Minsk, Belarus
  • Russian Children's Clinical Hospital Moscow, Russia
  • National Medical Research Center for Children Moscow, Russia
  • Veltischev Research and Clinical Institute for Pediatrics and Pediatric Surgery of the Pirogov Russian National Research Medical University Moscow, Russia
View Trial Details
Recruiting

A Study of Migalastat in Pediatric Subjects (2 to <12 Yrs) With Fabry Disease and Amenable GLA Variants

NCT06904261

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations
  • Emory Genetics Atlanta, Georgia, United States
  • University of Minnesota Masonic Children's Hospital Minneapolis, Minnesota, United States
  • Atrium Health Levine Children's Hospital Charlotte, North Carolina, United States
  • Cincinnati Children's Hospital Medical Center Cincinnati, Ohio, United States
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Recruiting

A Gene Transfer Therapy Study to Evaluate the Safety of and Expression From Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD) - Non-Ambulatory Cohort

NCT04626674

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Duchenne Muscular Dystrophy, Genetic Diseases, Inborn, +8 more
Locations
  • Arkansas Children's Hospital Little Rock, Arkansas, United States
  • Stanford University Palo Alto, California, United States
  • University of California, Davis Sacramento, California, United States
  • Washington University in St. Louis St Louis, Missouri, United States
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Recruiting

A Trial to Learn How Safe AZD9750 is and How Well it Works in People With Metastatic Prostate Cancer When Given With or Without Other Anticancer Drugs

NCT07336446

Conditions
Bulbo-Spinal Atrophy, X-Linked, Central Nervous System Diseases, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, +20 more
Locations
  • Research Site Duarte, California, United States
  • Research Site San Francisco, California, United States
  • Research Site Tampa, Florida, United States
  • Research Site Boston, Massachusetts, United States
View all 18 locations
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