Clinical trials
Gene Therapy clinical trials
Below are recruiting gene therapy clinical trials, each written for real people, not researchers. We’re tracking 191 recruiting studies, each written for real people, not researchers, below.
Recruiting studies
- NCT05878860RecruitingPhase 3
Gene therapy for X-linked retinoschisis
This trial tests a gene therapy called ATSN-201 for people with X-linked retinoschisis (XLRS), a condition that affects the retina and can cause vision loss. The therapy aims to fix the faulty gene that causes the condition, potentially halting vision loss and improving vision. Participants receive a one-time injection into the eye, and the study monitors safety and effectiveness.
Phoenix, ArizonaAges 5 years+ - NCT05861986RecruitingPhase 4
Risdiplam for very young children after gene therapy for SMA
This trial studies whether risdiplam, given early after gene therapy, is safe and helps children with spinal muscular atrophy (SMA) keep improving or stay stable. It focuses on children under 2 years old who already received gene therapy and need to meet timing and health requirements.
Little Rock, ArkansasAges 3 months–2 years - NCT05861999RecruitingPhase 4
Risdiplam for young children with SMA after gene therapy plateau
This trial studies whether risdiplam is safe and helpful for children under 2 with spinal muscular atrophy (SMA) whose function has leveled off or declined after gene therapy. It may be an option if you saw benefits from gene therapy but then noticed a stop or setback in key abilities.
Little Rock, ArkansasAges 3 months–2 years - NCT05686798RecruitingPhase 1
Gene therapy plus radiation for recurring brain tumor
This early-phase study tests a “suicide gene” therapy delivered by a virus along with radiation for adults whose high-grade brain tumor has come back or grown again. The goal is to see if the treatment is safe and can help control the tumor.
Detroit, MichiganAges 18 years+ - NCT04998396RecruitingPhase 1/Phase 2
Gene therapy trial for Canavan disease in young children
This Phase 1/2 trial tests an AAV9 gene therapy to treat Canavan disease. It aims to improve disease-related findings in children whose genetic test confirms ASPA gene changes.
Oakland, CaliforniaAges Up to 2.5 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 - NCT04416178Recruiting
Survey and interviews for families affected by sickle cell disease
This study collects feedback through surveys, interviews, and focus groups from people affected by sickle cell disease. Your input helps the research team improve how genomic and gene-therapy information and needs are understood for patients and caregivers.
Memphis, TennesseeAges 13 years+ - 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 - NCT03596086RecruitingPhase 1/Phase 2
Study drug plus radiation and chemo for recurrent brain cancer
This Phase 1/2 study tests whether combining an HSV-tk “gene switch” treatment, valacyclovir tablets, focused radiation (SBRT), and chemotherapy can help people with recurrent glioblastoma or anaplastic astrocytoma. It may help shrink tumors or slow growth after standard treatments have already been tried.
Houston, TexasAges 18 years+ - NCT03603405RecruitingPhase 1/Phase 2
Test vaccine and radiation with chemotherapy for new brain tumor
This trial studies a gene-based treatment (HSV-tk) given with radiation and chemotherapy for people newly diagnosed with glioblastoma or anaplastic astrocytoma. It may help the new brain tumor respond better to standard treatments, though risks and benefits are still being tested.
Houston, TexasAges 18 years+ - NCT03538899RecruitingPhase 1/Phase 2
Gene therapy for children with an Artemis SCID immune problem
This early-phase study tests an “autologous” gene therapy, meaning they use the child’s own cells, adjusted with a working gene, to help restore immune function. It is for babies and children with Artemis-deficient SCID, aiming to improve T-cell immunity and reduce the need for lifelong infection risk management.
San Francisco, CaliforniaAges 2 months+ - 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+ - 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 - 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.
Phoenix, ArizonaAges 2 years+ - 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+ - NCT04626674RecruitingPhase 1
Gene therapy for non-ambulatory Duchenne muscular dystrophy
This study tests a gene therapy that aims to help muscles produce a protein they are missing. It is for people with Duchenne muscular dystrophy who are no longer able to walk, and it may help slow the progression of the disease.
Little Rock, ArkansasAges 2 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+ - NCT03541928RecruitingPhase 2
Gene therapy with standard treatment for high-risk prostate cancer
This trial combines gene therapy with hormone therapy, radiation, and possibly surgery to treat high-risk prostate cancer. It aims to improve outcomes by adding gene therapy to standard treatments and will require additional biopsies.
Houston, TexasAges 18 years+ - 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 - 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 - 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
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Common questions
- Are there clinical trials for gene therapy?
- Yes. Clin2 currently lists 191 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.