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Genetic Disorders clinical trials in New York
7 studies for Genetic Disorders are looking for participants at sites in New York.
Cities in New York
Long-term Follow-up (LTFU) Study of Participants in Any iECURE Protocol Using an Investigational Product (IP)
- Icahn School of Medicine at Mount Sinai, New York
RecruitingObservational study
7 Months – 15 Months · All sexes · Updated Aug 31, 2026 · NCT06805695Safety of Anumigilimab (CSL324) in Adults With Sickle Cell Disease (SCD)
- Jacobi Medical Center, The Bronx
RecruitingPhase 2
Ages 18 and older · All sexes · Updated Aug 5, 2026 · NCT07224360Helix Research Network
- Rochester Regional Health (GenoWell), Rochester
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
- Icahn School of Medicine at Mount Sinai, New York
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
- New York Presbyterian Hospital - Morgan Stanley Children's Hospital, New York
RecruitingPhase 3
Ages 12 to 35 · All sexes · Updated Mar 23, 2026 · NCT05477563Open-Label Study of mRNA-3927 in Participants With Propionic Acidemia
- Icahn School of Medicine at Mount Sinai - Clinical Research Unit, New York
RecruitingPhase 1/2
All ages · All sexes · Updated Jan 22, 2026 · NCT04159103STXBP1 and SYNGAP1 Related Disorders Natural History Study
- Weill Cornell Medicine, New York
RecruitingObservational study
All ages · All sexes · Updated Oct 29, 2025 · NCT06555965From ClinicalTrials.gov, data retrieved Oct 5, 2026. Each study sets its own eligibility; the study team decides who can join.