Clinical trials
Gene Therapy clinical trials
Below are recruiting gene therapy clinical trials, each written for real people, not researchers. We’re tracking 210 recruiting studies, each written for real people, not researchers, below.
Recruiting studies
- NCT07148089RecruitingPhase 1
Gene therapy study for CPVT heart condition
This trial tests a new gene therapy called SGT-501 for people with a genetic heart condition called CPVT, which can cause dangerous fast heartbeats. The goal is to see if the treatment can reduce these episodes.
Boston, MassachusettsAges 7 years+ - NCT07180355RecruitingPhase 1
Gene therapy trial for Friedreich's ataxia
This trial tests a gene therapy called SGT-212 for people with Friedreich's ataxia. It aims to help treat the condition by delivering a working copy of the gene.
Los Angeles, CaliforniaAges 18–40 - NCT07223944RecruitingPhase 3
Gene therapy trial for Gaucher disease type 1
This trial tests a gene therapy called FLT201 for adults with Gaucher disease type 1. It aims to provide a long-term treatment option for people who have been stable on enzyme replacement therapy (ERT) or substrate reduction therapy (SRT) for at least two years.
Los Angeles, CaliforniaAges 18 years+ - NCT07290062RecruitingPhase 1
Gene therapy injection for ALS safety study
This trial tests a single injection of a gene therapy called INS1202 into the spinal fluid. It is for people with ALS, including those with a specific genetic form (SOD1 mutation). The goal is to see if it is safe and how it affects the body.
La Jolla, CaliforniaAges 18–79 - NCT07629115Recruiting
Long-term follow-up study for severe Parkinson's gene therapy
This study follows patients who previously received IPS101A, an experimental gene therapy treatment for severe Parkinson's disease. Researchers want to check how you're doing over time and gather information about the treatment's long-term effects.
SeoulAges 50–80 - NCT07346144RecruitingPhase 1/Phase 2
Gene therapy study for high-grade glioma
This trial tests a new gene therapy given directly into the brain tumor. It's for people with high-grade glioma, either newly diagnosed or after one recurrence, to see if it can help control the tumor.
Columbus, OhioAges 18–70 - NCT07364357RecruitingPhase 1
Study of CREPT-618 for advanced liver cancer
This phase 1 trial tests a new drug called CREPT-618 for people with advanced liver cancer that hasn't responded to standard treatments. The drug is designed to target cancer cells with specific markers (CREPT and ASGPR), so only patients whose tumors have these markers can join.
BeijingAges 18–75 - NCT01166009Recruiting
CIBMTR research database for transplant and bone marrow injury patients
This study collects health and treatment information in a large research database. It may help researchers better understand outcomes after blood stem cell transplants or bone marrow injury from treatment like radiation.
Minneapolis, MinnesotaAges Any age - NCT03217032RecruitingPhase 1
Gene therapy trial for hemophilia A using FVIII
This Phase 1 study tests a gene therapy designed to help the body make factor VIII (FVIII), which is missing in hemophilia A. It may help reduce bleeding and the need for FVIII infusions, but it is an early-safety trial.
Shenzhen, GuangdongAges 2–65 - NCT03217617RecruitingPhase 1/Phase 2
Gene therapy by vein for SCID-X1 immune deficiency
This trial tests an experimental gene therapy given through a vein to help children with SCID-X1 make needed immune cells. It may help reduce severe infections when standard options like a matched donor are not available.
Shenzhen, GuangdongAges 1 month–1 year - NCT02315599Enrolling by invitation
Follow-up for delayed side effects after gene-cell therapy
This study checks people for delayed side effects after receiving gene-based cell therapy from the NIH. It helps doctors learn what problems may happen later and how to monitor them safely.
Bethesda, MarylandAges 1–99 - NCT02473757Enrolling by invitation
Follow-up program for adults previously treated with CAR T
This is a follow-up study for people age 18 or older who previously took part in a CAR T-cell gene therapy trial. It helps researchers check your health over time after that treatment and supports long-term safety and outcomes tracking.
Bethesda, MarylandAges 18 years+ - NCT07401121RecruitingPhase 1
Study of CTx1000 for people with ALS
This trial tests a new drug, CTx1000, for people with ALS. It aims to see if the drug is safe and tolerable, and may offer a new treatment option.
Sydney, New South WalesAges 18–80 - NCT07673809RecruitingPhase 1/Phase 2
Gene therapy trial for Duchenne muscular dystrophy in young boys
This trial tests a gene therapy called GNR-097 for boys with Duchenne muscular dystrophy (DMD). The therapy aims to help improve muscle function, and the study will check how safe and effective it is.
MinskAges 4–9 - NCT06285643RecruitingPhase 2
Gene therapy for moderate Parkinson's disease
This trial tests a gene therapy (AAV2-GDNF) to see if it can slow or improve symptoms of Parkinson's disease. It is for people who have had moderate Parkinson's for 4 to 10 years and still respond to levodopa therapy.
Phoenix, ArizonaAges 45–75 - NCT07643844RecruitingPhase 1
Gene therapy for propionic acidemia in infants
This trial tests a new gene therapy treatment designed to help the body process certain amino acids better in infants with propionic acidemia. The therapy uses a modified virus to deliver a working copy of a faulty gene, potentially reducing dangerous acid buildups and hospital visits.
Rochester, MinnesotaAges 6 months–2 years - NCT07503613Enrolling by invitation
Long-term follow-up for AAVAnc80 gene therapy
This study follows people who already received the experimental AAVAnc80-antiVEGF gene therapy for vestibular schwannoma. It monitors long-term safety and effects of the treatment.
Dallas, TexasAges 18 years+ - NCT07264166Enrolling by invitation
Gene therapy trial for cerebral palsy and HIE
This trial tests a gene therapy given as a nose spray for people with cerebral palsy or HIE. It aims to see if the treatment is safe and helps improve movement over two years.
Monterrey, Nuevo LeónAges 2–65 - NCT07159581Enrolling by invitation
Gene therapy for Wilson disease study with PET scans
This trial uses a special PET scan with a copper tracer to see how well gene therapy is working in people with Wilson disease. It may help doctors understand if the treatment is helping your body handle copper properly.
Aarhus, Aarhus NAges 18 years+ - NCT07050160Enrolling by invitation
Long-term follow-up after gene therapy for arrhythmogenic cardiomyopathy
This study follows participants who have already received the gene therapy LX2020 for a genetic heart condition called arrhythmogenic cardiomyopathy. It tracks their long-term safety and health outcomes, with no new treatment given.
Ann Arbor, MichiganAges 18–67 - NCT07023965Enrolling by invitation
Long-term follow-up study for gene therapy in critical limb ischemia
This study follows up with people who previously took part in a clinical trial testing an experimental gene therapy for critical limb ischemia (severe leg pain or sores due to blocked arteries). It aims to see how well the treatment works over time. If you were in that earlier trial and received at least one dose, joining this follow-up could help researchers learn more about the therapy's lasting effects.
Beijing, Beijing MunicipalityAges 20–85 - NCT06885424Enrolling by invitation
Long-term follow-up for A2 Bio gene therapy patients
This study follows up with people who previously received a gene therapy product from A2 Biotherapeutics in an earlier trial. The goal is to monitor their health over time and check for any long-term effects.
Gilbert, ArizonaAges 18 years+ - NCT06851767Enrolling by invitationPhase 1/Phase 2
Gene therapy for X-linked severe combined immunodeficiency
This trial tests a gene therapy that uses base-edited stem cells to treat X-SCID, a genetic immune disorder. It may help improve immune function and reduce infections in patients who still have low immune cells or severe infections despite previous treatments.
Bethesda, MarylandAges 3–99 - NCT06664541Enrolling by invitation
Gene therapy decisions and quality of life
This study looks at how people with blood diseases and their families make decisions about gene therapy, and how it affects their quality of life. It involves interviews or surveys to understand your experience.
Washington D.C., District of ColumbiaAges 18 years+
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Common questions
- Are there clinical trials for gene therapy?
- Yes. Clin2 currently lists 210 recruiting gene therapy studies from the U.S. registry, each rewritten for real people, not researchers, so you can see what it’s testing and who it’s for.
- How do I know if I qualify for a gene therapy trial?
- Each study lists its eligibility criteria — rules about age, diagnosis, and prior treatments. On every Clin2 trial page we explain these in words written for real people and offer a short, optional pre-screen for a fit read. The study team makes the final decision.
- Does it cost anything to join a gene therapy trial?
- Using Clin2 is always free. Many trials cover the cost of the study treatment and related visits; some reimburse travel. The study team explains exactly what’s covered before you decide.
Related conditions
Clin2 helps you find and understand clinical trials and does not provide medical advice. Study data comes from ClinicalTrials.gov. Talk with your doctor about whether a specific trial is right for you.