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Genetic Disorders clinical trials in New York

8 studies for Genetic Disorders are looking for participants at sites in New York.

Cities in New York

A Phase 3 Evaluation of Sutacimig in Glanzmann Thrombasthenia

  • New York-Presbyterian - Weill Cornell Medical Center, New York
RecruitingPhase 3
Ages 12 to 70 · All sexes · Updated Nov 13, 2026 · NCT07935356

Helix Research Network

  • Rochester Regional Health (GenoWell), Rochester
RecruitingObservational studyHealthy volunteers welcome
Ages 18 and older · All sexes · Updated Nov 12, 2026 · NCT06057181

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 Nov 5, 2026 · NCT06805695

Safety of Anumigilimab (CSL324) in Adults With Sickle Cell Disease (SCD)

  • NYC Health + Hospitals/Queens, Jamaica
  • Columbia University Medical Center, New York
  • Jacobi Medical Center, The Bronx
RecruitingPhase 2
Ages 18 and older · All sexes · Updated Nov 3, 2026 · NCT07224360

A 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 · NCT05130437

Evaluation 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 · NCT05477563

Open-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 · NCT04159103

STXBP1 and SYNGAP1 Related Disorders Natural History Study

  • Weill Cornell Medicine, New York
RecruitingObservational study
All ages · All sexes · Updated Oct 29, 2025 · NCT06555965

From ClinicalTrials.gov, data retrieved Nov 15, 2026. Each study sets its own eligibility; the study team decides who can join.