Clinical trials · New York, NY
Genetic Diseases, Inborn clinical trials in New York
We’re tracking 3 recruiting genetic diseases, inborn studies with a site in or near New York — including Phase 3 studies, each written for real people, not researchers.
Recruiting studies near New York
- NCT06255782RecruitingPhase 3
Gene therapy study for baby boys with severe OTC deficiency
This trial tests a gene therapy called ECUR-506 for baby boys under 9 months old with a severe form of OTC deficiency. The goal is to see if it can help their bodies process ammonia better, reducing the need for a strict protein-restricted diet and medications.
New York, New YorkAges 1 day–7 months - NCT05477563RecruitingPhase 3
One-time gene therapy for severe sickle cell and transfusion anemia
This trial tests a single dose of gene therapy (CTX001) to improve outcomes for people with severe sickle cell disease or transfusion-dependent beta-thalassemia. It aims to see how well it works and whether it is safe, especially after a planned stem cell transplant process.
New York, New YorkAges 12–35 - NCT04208529Enrolling by invitationPhase 3
Long-term follow-up after receiving CTX001 infusion
This study follows people for a long time after they received CTX001 in an earlier (parent) study. It helps researchers learn about long-term safety and outcomes of the treatment.
New York, New YorkAges 2 years+
Hear when a new Genetic Diseases, Inborn trial opens near New York, NY
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Where these studies are running near New York
Institutions with a site for the recruiting genetic diseases, inborn studies listed above.
- Morgan Stanley Children's Hospital · 2 studies
- Herbert Irving Pavilion - Hematology
- Icahn School of Medicine at Mount Sinai
What taking part in a genetic diseases, inborn study involves
A screening visit first
Before anything else, the study team checks whether you fit — usually a visit with some tests. You can stop at any point, and screening is typically free.
Care at a nearby site
Study visits happen at a clinic or hospital taking part. Many studies cover the cost of the study treatment and related visits, and some reimburse travel.
You stay in control
Taking part is voluntary and you can leave a study at any time, for any reason, without affecting your regular care.
The team decides eligibility
Our fit check is a helpful first read, not a decision. The study team makes the final call after reviewing your health history.
Common questions
- Are there genetic diseases, inborn clinical trials near New York?
- Yes. We're currently tracking 3 recruiting genetic diseases, inborn studies with a site near New York, each rewritten in plain language so you can see what it's testing and who it's for.
- How do I find out if I qualify for a genetic diseases, inborn study near New York?
- Each study lists its eligibility rules — age, diagnosis, prior treatments. On every trial page we explain these in plain language and offer a short, optional pre-screen for a fit read. The study team makes the final decision.
- Do I have to live in New York to take part?
- Not necessarily. These studies have a site near New York, but eligibility is about your health, not your address — some people travel to take part, and a few studies reimburse travel. The study team can tell you what's required.
- Does it cost anything to join a genetic diseases, inborn trial?
- Using Clin2 is always free. Many trials cover the study treatment and related visits; some reimburse travel. The study team explains exactly what's covered before you decide.
Not the right time?
New genetic diseases, inborn studies open near New York regularly. Set up a health profile and we’ll quietly watch for studies that fit you and email you when one opens.