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Clinical trial condition

Genetic Diseases, Inborn

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

12,553 public trials 2,147 recruiting studies
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Genetic Diseases, Inborn trial results

Recruiting

A Phase 2 Open-label Study to Evaluate the Activity of Etavopivat on Transcranial Doppler Velocities in Pediatric Patients With Sickle Cell Disease Who Are at Increased Risk for Primary Stroke

NCT05953584

Conditions
Anemia, Anemia, Hemolytic, Anemia, Hemolytic, Congenital, +7 more
Locations
  • All India Institute of Medical Sciences (AIIMS), Raipur Raipur, Chhattisgarh, India
  • All India Institute of Medical Sciences-Delhi Delhi, India
  • Nirmal Hospital Pvt. Ltd. Gujarat, India
  • Suretech Hospital and Research Centre Ltd. Maharashtra, India
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Recruiting

Nurse-Led Telehealth for Gout

NCT06971146

Conditions
Adherence, Arthritis, Arthritis, Gouty, +16 more
Locations
  • Aarhus University Hospital Aarhus, Denmark
  • Gødstrup Hospital Herning, Denmark
  • Horsens Hospital Horsens, Denmark
  • Randers Hospital Randers, Denmark
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Recruiting

Pharmacokinetics, Efficacy and Safety of Twice Daily Dosing Regimen of Hydroxycarbamide Dispersible Tablets in Children With Sickle Cell Disease

NCT06578507

Conditions
Anemia, Anemia, Hemolytic, Anemia, Hemolytic, Congenital, +7 more
Locations
  • Centre Hospitalier Intercommunal Créteil Créteil, France
  • GHEF- Site de Marne-la-Vallée Jossigny, France
  • Hôpital Bicêtre Le Kremlin-Bicêtre, France
  • Institut d'Hématologie et d'oncologie pédiatrique - IHOPe Lyon, France
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Recruiting

Tirzepatide in PWS, HO and GNSO

NCT06901245

Conditions
Abnormalities, Multiple, Body Weight, Chromosome Disorders, +29 more
Locations
  • Children's Minnesota Saint Paul, Minnesota, United States
  • Vanderbilt University Medical Center Nashville, Tennessee, United States
  • Seattle Children's Hospital Seattle, Washington, United States
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Recruiting

The European Paediatric Network for Haemophilia Management ( PedNet Registry)

NCT02979119

Conditions
Blood Coagulation Disorders, Blood Coagulation Disorders, Inherited, Coagulation Protein Disorders, +10 more
Locations
  • Universitäts-Klinik für Kinder- und Jugendheilkunde Graz, Austria
  • Medical University of Vienna - Department of Paediatrics Vienna, Austria
  • Service of Pediatric Haematology University Hospital Leuven Leuven, Belgium
  • Division of Hematology/Oncology Hôpital St Justine Montreal, Canada
View all 39 locations
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