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Clinical trial condition

Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Explore clinical trials studying Congenital, Hereditary, and Neonatal Diseases and Abnormalities. Study availability and eligibility vary by location and protocol.

18,229 public trials 3,117 recruiting studies
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Congenital, Hereditary, and Neonatal Diseases and Abnormalities trial results

Recruiting

A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease (NPC)

NCT07082725

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +20 more
Locations
  • UCSF Children's Hospital and Research Center at Oakland Oakland, California, United States
  • University of Minnesota Medical School Minneapolis, Minnesota, United States
  • Mayo Clinic Children's Center - PIN Rochester, Minnesota, United States
  • Children's Medical Center Dallas Dallas, Texas, United States
View all 34 locations
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Recruiting

A Phase 1b, Open-Label Study of DISC-3405 in Participants With Sickle Cell Disease (SCD)

NCT07187973

Conditions
Anemia, Anemia, Hemolytic, Anemia, Hemolytic, Congenital, +7 more
Locations
  • University of Alabama at Birmingham Birmingham, Alabama, United States
  • Emory University Atlanta, Georgia, United States
  • Augusta University Augusta, Georgia, United States
  • Innovative Hematology - Indiana Hemophilia & Thrombosis Center Indianapolis, Indiana, United States
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Recruiting

Trastuzumab Deruxtecan (DS-8201a) for the Treatment of Recurrent or Refractory Osteosarcoma, Wilms Tumor, and Desmoplastic Small Round Cell Tumor

NCT04616560

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Desmoplastic Small Round Cell Tumor, Female Urogenital Diseases, +25 more
Locations
  • Children's Hospital of Alabama Birmingham, Alabama, United States
  • Children's Hospital Los Angeles Los Angeles, California, United States
  • Children's Hospital of Orange County Orange, California, United States
  • Lucile Packard Children's Hospital Stanford University Palo Alto, California, United States
View all 28 locations
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Recruiting

Effect of Partially Hydrolyzed Formula With Synbiotics on Skin Barrier Function

NCT06943469

Conditions
Atopic Dermatitis (AD), Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dermatitis, +9 more
Locations
  • AZ Sint-Jan Brugge-Oostende AV - Campus Sint-Jan Bruges, Belgium
  • Universitair Ziekenhuis Brussel - Kinderziekenhuis Brussels, Belgium
  • Clinique Ste Elisabeth Namur, Belgium
  • CHU Amiens Picardie Amiens, France
View all 25 locations
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Recruiting

A Study of SGT-212 Gene Therapy in Friedreich's Ataxia

NCT07180355

Conditions
Ataxia, Brain Diseases, Central Nervous System Diseases, +21 more
Locations
  • The University of California, Los Angeles (UCLA) Los Angeles, California, United States
  • The Ohio State University Columbus, Ohio, United States
  • The Children's Hospital of Philadelphia (CHOP) Philadelphia, Pennsylvania, United States
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Recruiting

A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease, GM1 Gangliosidosis or GM2 Gangliosidosis

NCT07054515

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +25 more
Locations
  • UCSF Children's Hospital and Research Center at Oakland Oakland, California, United States
  • University of Minnesota Medical School Minneapolis, Minnesota, United States
  • Mayo Clinic Childrens Center - PIN Rochester, Minnesota, United States
  • Children's Medical Center Dallas Dallas, Texas, United States
View all 38 locations
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Recruiting

Follow-up Study to Monitor the Efficacy and Safety of the Occlutech® mVSD Cases

NCT05329350

Conditions
Cardiovascular Abnormalities, Cardiovascular Diseases, Congenital Abnormalities, +4 more
Locations
  • Children's Health Ireland Dublin, Ireland
  • İzmir Şehir Hastanesi Izmir, Bayraklı, Turkey (Türkiye)
  • Eskişehir Osmangazi Üniversite Hastanesi Eskişehir, Odunpazarı, Turkey (Türkiye)
  • Dicle Üniversitesi Tıp Fakültesi Hastanesi Diyarbakır, Sur, Turkey (Türkiye)
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