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Rare Diseases clinical trials in North Carolina
6 studies for Rare Diseases are looking for participants at sites in North Carolina.
Cities in North Carolina
Clinical and Genetic Evaluation of Individuals With Undiagnosed Disorders Through the Undiagnosed Diseases Network
- Duke University Health System, Durham
RecruitingObservational studyHealthy volunteers welcome
1 Month – 100 Years · All sexes · Updated Sep 18, 2026 · NCT02450851Elafibranor Pregnancy Surveillance Program: A Study to Evaluate the Safety of Elafibranor During Pregnancy
- Virtual Research Coordination Center, Wilmington
RecruitingObservational study
All ages · Women · Updated Aug 31, 2026 · NCT07556913Odevixibat Pregnancy and Lactation Surveillance Program: A Study to Evaluate the Safety of Odevixibat During Pregnancy and/or Lactation
- Virtual Research Coordination Center Odevixibat (BYLVAY) Pregnancy Surveillance Program, Wilmington
RecruitingObservational study
All ages · Women · Updated Aug 31, 2026 · NCT06258902A Study Evaluating the Real-World Experience of Givinostat in Patients With Duchenne Muscular Dystrophy
- Duke University / Lenox Baker Children's Hospital, Durham
RecruitingObservational study
Ages 6 and older · All sexes · Updated Jul 2, 2026 · NCT07127978Study of S-606001 as an Add-on to Enzyme Replacement Therapy (ERT) in Participants With Late-onset Pompe Disease (LOPD)
- Duke University Medical Center, Durham
RecruitingPhase 2
Ages 18 and older · All sexes · Updated Jun 23, 2026 · NCT07123155The ExTINGUISH Trial of Inebilizumab in NMDAR Encephalitis
- Wake Forest University Health Sciences, Winston-Salem
RecruitingPhase 2
Ages 12 and older · All sexes · Updated Jul 1, 2025 · NCT04372615From ClinicalTrials.gov, data retrieved Oct 1, 2026. Each study sets its own eligibility; the study team decides who can join.