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Genetic Disorders clinical trials in North Carolina
9 studies for Genetic Disorders 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 Oct 2, 2026 · NCT02450851Inherited Reproductive Disorders
- NIEHS Clinical Research Unit (CRU), Research Triangle Park
RecruitingObservational study
6 Weeks – 120 Years · All sexes · Updated Jul 28, 2026 · NCT01500447Rett Syndrome Registry
- The University of North Carolina at Chapel Hill, Chapel Hill
RecruitingObservational study
Ages 0 to 99 · All sexes · Updated Jun 30, 2026 · NCT05432349Efficacy, Safety, and Tolerability of Zeleciment Rostudirsen (DYNE-251) Administered Intravenously Every 4 Weeks in Ambulatory Participants With Duchenne Muscular Dystrophy (FORZETTO)
- Rare Disease Research, LLC, Hillsborough
RecruitingPhase 3
Ages 4 to 18 · Men · Updated May 27, 2026 · NCT07608432Helix Research Network
- Cone Health (Gene Connect), Burlington
- WakeMed (PreciselyYou), Raleigh
RecruitingObservational studyHealthy volunteers welcome
Ages 18 and older · All sexes · Updated Mar 23, 2026 · NCT06057181A Study to Assess the Long-term Safety and Clinical Activity of mRNA-3927 in Participants Previously Enrolled in the mRNA-3927-P101 Study
- Duke University Medical System (Duke Health), Durham
RecruitingPhase 1/2
Ages 1 and older · All sexes · Updated Mar 23, 2026 · NCT05130437Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease
- Levine Children's Hospital - Hematology, Charlotte
RecruitingPhase 3
Ages 12 to 35 · All sexes · Updated Mar 23, 2026 · NCT05477563PKP2-ACM Natural History Study
- Duke University, Durham
RecruitingObservational study
Ages 12 and older · All sexes · Updated Feb 27, 2026 · NCT06644742Open-Label Study of mRNA-3927 in Participants With Propionic Acidemia
- Duke University Medical System (Duke Health), Durham
RecruitingPhase 1/2
All ages · All sexes · Updated Jan 22, 2026 · NCT04159103From ClinicalTrials.gov, data retrieved Oct 5, 2026. Each study sets its own eligibility; the study team decides who can join.