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
- NCT06544798Enrolling by invitationPhase 2
Long-term follow-up of gene therapy for dry mouth
This study follows patients who previously received gene therapy for dry mouth caused by radiation. It aims to track long-term safety and any lasting benefits.
Gilbert, ArizonaAges 18 years+ - NCT05921162Enrolling by invitation
Long-term follow-up after a vision gene eye injection
This study checks how people who previously received a gene therapy eye injection (vMCO-I) are doing over the long term. It may help researchers understand long-lasting effects and safety.
Cuttack, OdishaAges 18 years+ - NCT04833907Enrolling by invitationPhase 1/Phase 2
Gene therapy study for typical Canavan disease in children
This early-phase trial tests a gene therapy medicine (delivered by a one-time infusion) for children with typical Canavan disease. It aims to improve safety and help evaluate whether the treatment may benefit children over time.
Dayton, OhioAges 3 months–5 years - NCT04628585Enrolling by invitation
Follow-up for people with sickle cell treated with gene therapy
This study is a long-term follow-up for people who already received gene therapy for sickle cell disease in a specific sponsor’s clinical trial. It helps researchers watch for long-term safety and outcomes over time.
Birmingham, AlabamaAges 2–53 - NCT04266093Enrolling by invitation
Gene therapy follow-up for adults previously treated at NCI
This study is a follow-up check for people who were previously enrolled in certain NCI gene therapy treatment studies. It helps the research team monitor long-term effects and outcomes after treatment.
Bethesda, MarylandAges 18–120 - NCT06008392Recruiting
Mayo Clinic cancer genetics research study
This study collects blood, saliva, tumor tissue, or hair samples to look for inherited and tumor-related gene changes that may explain your cancer, guide prevention, or help find better treatments. You can join if you are a Mayo Clinic patient with cancer and your doctor has ordered genetic testing.
Scottsdale, ArizonaAges 18 years+ - NCT06379789RecruitingPhase 1/Phase 2
Gene therapy for severe hemophilia B
This trial tests a new gene therapy (REGV131-LNP1265) that aims to let your body produce its own factor IX, reducing or stopping the need for regular infusions. It is for children and adults with severe hemophilia B who are already using factor IX replacement therapy.
Los Angeles, CaliforniaAges 2 years+ - NCT04626674RecruitingPhase 1
Gene therapy safety study for non-ambulatory Duchenne
This Phase 1 trial studies a one-time gene therapy treatment (SRP-9001) to see if it is safe and whether it can produce the needed gene activity in people with Duchenne muscular dystrophy (DMD) who are not walking. It may help researchers understand whether this approach could be useful for DMD patients, especially in later stages.
Little Rock, ArkansasAges 2 years+ - NCT06465537Recruiting
Gene editing therapy for glaucoma with MYOC mutation
This trial is testing a one-time gene-editing treatment for people with glaucoma caused by a MYOC gene mutation. The goal is to lower eye pressure and possibly reduce the need for eye drops.
Beijing, Beijing MunicipalityAges 18–65 - NCT06112327Enrolling by invitation
Long-term follow-up for a gene editing heart therapy
This study follows people who already received an experimental gene editing treatment for cardiovascular disease in a prior Verve trial. The goal is to monitor their health and any long-term effects over time.
AucklandAges 18 years+ - NCT05724004Recruiting
Alectinib then targeted radiation for ALK-positive lung cancer
This trial tests giving alectinib first, then adding targeted radiation to remaining spots of cancer in people with ALK-rearranged advanced non-small cell lung cancer. It aims to control the cancer after an initial response and help keep disease from progressing.
StockholmAges 20 years+ - NCT06357754Recruiting
Testing tumor samples after BMS cell therapy and a second cancer
This trial offers testing of tumor samples for people who developed a new, different cancer after receiving a Bristol-Myers Squibb gene-modified cell therapy. The goal is to understand the genetics of that new cancer and see if it relates to the prior treatment.
Portland, OregonAges Any age - NCT07143812RecruitingPhase 1
Stem cell gene therapy for newly diagnosed glioblastoma
This trial tests a new stem cell-based gene therapy for people with newly diagnosed glioblastoma who have already received standard treatment. The goal is to see if it is safe and potentially effective.
Seongnam, KyunggidoAges 19–70 - NCT06405295Enrolling by invitation
Intensive adjuvant therapy for BRCA-related triple-negative breast cancer
This trial tests an intensive add-on treatment for people with triple-negative breast cancer and a BRCA mutation who are at high risk of recurrence after surgery and standard treatments. The goal is to see if extra therapy helps prevent the cancer from coming back.
Beijing, Beijing MunicipalityAges 18–75 - NCT07680803RecruitingPhase 2
Gene therapy for transfusion-dependent beta-thalassemia
This trial tests a one-time gene therapy using your own stem cells to help you produce healthy red blood cells and reduce or eliminate the need for regular blood transfusions. It is for people with transfusion-dependent beta-thalassemia who are between 3 and 35 years old.
Rome, LazioAges 3–35 - NCT06478251RecruitingPhase 1
Engineered immune cells for KRAS-mutated advanced solid tumors
This trial tests a new type of cell therapy that uses your own immune cells, specially trained to attack cancer cells with a KRAS G12V or G12D mutation. It may be an option if you have advanced solid tumors that no longer respond to standard treatments.
Hangzhou, ZhejiangAges 18–75 - NCT06484556RecruitingPhase 1
T cell therapy for KRAS-mutated advanced solid tumors
This trial tests a personalized T cell therapy designed to target cancer cells with specific KRAS mutations (G12V or G12D). It may be an option for people with advanced solid tumors who have exhausted standard treatments.
Wuhan, HubeiAges 18–75 - NCT06484790RecruitingPhase 1
Engineered T cell therapy for KRAS-mutated advanced solid tumors
This trial tests a personalized T cell therapy designed to target specific KRAS mutations (G12V or G12D) in people with advanced solid tumors who have no standard treatment options left. The treatment involves collecting your own immune cells, engineering them to recognize and attack cancer cells with these mutations, and then infusing them back into you.
Tianjin, Tianjin MunicipalityAges 18–75 - NCT07665021RecruitingPhase 1/Phase 2
Gene therapy for children with severe bone disease
This trial tests a gene therapy that uses a child's own stem cells to treat autosomal recessive osteopetrosis (a genetic bone disease). It aims to improve bone health and reduce complications like fractures and vision loss.
Milan, ItalyAges 4 weeks–2 years - NCT03435796RecruitingPhase 2/Phase 3
Follow-up for people treated with gene-modified T cells
This study is a long-term check-in for people who previously received gene-modified T-cell infusions in a related Celgene/Juno/BMS trial. It helps researchers track safety and long-term effects over time.
Birmingham, AlabamaAges Any age - NCT06824155RecruitingEarly Phase 1
Radioactive drug for HER2-positive advanced cancers
This trial tests a radioactive drug (lutetium-177) that targets the HER2 protein, which is found on some cancer cells. It's for people with advanced, HER2-positive or HER2-low solid tumors that haven't responded to other treatments.
Kingswood, New South WalesAges 18 years+ - NCT05818319Recruiting
Tracking kidney urine effects after a short pause in CF therapy
This study checks how a common cystic fibrosis (CF) medicine affects urine markers after briefly stopping it and then restarting. The goal is to better understand whether the treatment shift changes kidney-related signals in people with CF.
Aarhus C, Central JutlandAges 18 years+ - NCT06775470Recruiting
How BRCA gene variants affect ovarian cancer treatment results
This study looks at how different BRCA gene changes affect how well ovarian cancer responds to treatment and the long-term outlook. If you were diagnosed with ovarian cancer and had BRCA testing at Bologna's S.Orsola hospital, your data could help researchers better understand treatment outcomes.
BolognaAges Any age - NCT06868836RecruitingPhase 2
Study of a new antibody combination for advanced lung cancer after targeted therapy
This trial tests a new drug (JS207) that targets both PD-1 and VEGF pathways, given alone or with other treatments, for people with advanced non-small cell lung cancer that has a specific gene change and has stopped responding to targeted therapy (TKI). The goal is to see if this combination can help shrink tumors or slow cancer growth.
Guangzhou, GuangdongAges 18–75
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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.