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Genetic Disorders clinical trials in New York City, NY

5 studies for Genetic Disorders are looking for participants at sites in and around New York City (within about 30 miles). Open a study to see who can join and how to contact the study team.

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Long-term Follow-up (LTFU) Study of Participants in Any iECURE Protocol Using an Investigational Product (IP)

RecruitingObservational study
  • Icahn School of Medicine at Mount Sinai, New York
7 Months – 15 Months · All sexes · Updated Aug 31, 2026 · NCT06805695

Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease

RecruitingPhase 3
  • New York Presbyterian Hospital - Morgan Stanley Children's Hospital, New York
Ages 12 to 35 · All sexes · Updated Mar 23, 2026 · NCT05477563

A Study to Assess the Long-term Safety and Clinical Activity of mRNA-3927 in Participants Previously Enrolled in the mRNA-3927-P101 Study

RecruitingPhase 1/2
  • Icahn School of Medicine at Mount Sinai, New York
Ages 1 and older · All sexes · Updated Mar 23, 2026 · NCT05130437

Open-Label Study of mRNA-3927 in Participants With Propionic Acidemia

RecruitingPhase 1/2
  • Icahn School of Medicine at Mount Sinai - Clinical Research Unit, New York
All ages · All sexes · Updated Jan 22, 2026 · NCT04159103

STXBP1 and SYNGAP1 Related Disorders Natural History Study

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

About Genetic Disorders

A mutation in a person's genes can cause a medical condition called a genetic disorder. Learn about the types and how they are detected.

More about Genetic Disorders on MedlinePlus

Genetic Disorders trials in other cities

Other studies in New York City

From ClinicalTrials.gov, data retrieved Oct 6, 2026. Each study sets its own eligibility; the study team decides who can join.