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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.

12,597 public trials 2,159 recruiting studies
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Genetic Diseases, Inborn trial results

Active, not recruiting

A Clinical Evaluation of Non-Invasive Vagus Nerve Stimulation for Temper Outbursts in People With PWS

NCT06144645

Conditions
Abnormalities, Multiple, Chromosome Disorders, Congenital Abnormalities, +13 more
Locations
  • Stanford University Palo Alto, California, United States
  • Rady Children's Hospital San Diego San Diego, California, United States
  • Children's Hospital Colorado Aurora, Colorado, United States
  • Emory University Atlanta, Georgia, United States
View all 18 locations
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Active, not recruiting

9-ING-41 in Patients With Advanced Cancers

NCT03678883

Conditions
Acute T Cell Leukemia Lymphoma, Bone Cancer, Bone Diseases, +90 more
Locations
  • Mayo Clinic Phoenix, Arizona, United States
  • Arizona Oncology Associates Tucson, Arizona, United States
  • The University of Arizona Cancer Center Tucson, Arizona, United States
  • University of California Irvine Health Orange, California, United States
View all 66 locations
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Active, not recruiting

A Study of LY3200882 in Participants With Solid Tumors

NCT02937272

Conditions
Bone Diseases, Bone Diseases, Developmental, Camurati-Engelmann Syndrome, +5 more
Locations
  • University of Louisville Louisville, Kentucky, United States
  • Weill Cornell Medical College New York, United States
  • University of Texas MD Anderson Cancer Center Houston, Texas, United States
  • Calvary Mater Newcastle Newcastle, New South Wales, Australia
View all 18 locations
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Active, not recruiting

A Study to Evaluate the Safety and Efficacy of Zilganersen (ION373) in Patients With Alexander Disease (AxD)

NCT04849741

Conditions
Alexander Disease, Brain Diseases, Brain Diseases, Metabolic, +13 more
Locations
  • Lucile Packard Children's Hospital Stanford Palo Alto, California, United States
  • Children's Hospital of Atlanta Atlanta, Georgia, United States
  • Massachusetts General Hospital Boston, Massachusetts, United States
  • Children's Hospital of Philadelphia Philadelphia, Pennsylvania, United States
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Active, not recruiting

Roll-over Study to Collect and Assess Long-term Safety of Everolimus in Patients With TSC and Refractory Seizures Who Have Completed the EXIST-3 Study [CRAD001M2304] and Who Are Benefitting From Continued Treatment

NCT02962414

Conditions
Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Genetic Diseases, Inborn, +17 more
Locations
  • TGen APNNA Phoenix, Arizona, United States
  • David Geffen School of Medicine at UCLA Los Angeles, California, United States
  • UCSF Benioff Children s Hospital Oakland, California, United States
  • Rady Children s Hospital San Diego, California, United States
View all 68 locations
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Active, not recruiting

Astroscape: A Study of Radiprodil on Safety, Tolerability, Pharmacokinetics, and Effect on Seizures and Behavioral Symptoms in Patients With TSC or FCD Type II

NCT06392009

Conditions
Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Focal Cortical Dysplasia, +14 more
Locations
  • Queensland Children Hospital South Brisbane, Australia
  • University Hospitals Leuven, Pediatric Neurology Leuven, Belgium
  • Alberta Children's Hospital Calgary, Canada
  • The Hospital for Sick Children (Sick Kids) Toronto, Canada
View all 18 locations
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