Clinical trials
Mutation clinical trials
Below are recruiting mutation clinical trials, each written for real people, not researchers. We’re tracking 714 recruiting studies, each written for real people, not researchers, below.
Recruiting studies
- NCT07241169RecruitingEarly Phase 1
Gene therapy trial for ABCA4-related retinal disease
This trial tests a new gene therapy called ZVS106e for people with inherited retinal degeneration caused by changes in both copies of the ABCA4 gene. The therapy aims to slow or stop vision loss, and the study will check if it is safe and if it works.
Beijing, Beijing MunicipalityAges 8 years+ - NCT07242963Recruiting
Study of Shh medulloblastoma with U1 mutation
This study looks at people with a certain type of brain tumor called sonic hedgehog medulloblastoma that has come back or not responded to treatment. Researchers want to learn more about these tumors that have a specific genetic change (U1 mutation), which may help find better treatments in the future.
Houston, TexasAges 3–50 - NCT07231068RecruitingPhase 1/Phase 2
Dositinib tablets for advanced lung cancer with EGFR mutation
This trial tests a new targeted therapy, Dositinib, for people with advanced non-small cell lung cancer that has an EGFR mutation and has stopped responding to earlier EGFR-targeted drugs. It aims to find the best dose and see how well it shrinks tumors.
BeijingAges 18–75 - NCT07230691Recruiting
Study of amivantamab for advanced EGFR-mutated lung cancer
This study looks at how well a treatment called amivantamab works in people with a specific type of advanced lung cancer (EGFR-mutated non-small cell lung cancer). It aims to track real-world outcomes in patients who are starting amivantamab for the first time.
GrazAges 18 years+ - NCT07222215RecruitingPhase 2
Capacitabine with or without elacestrant for ER+ breast cancer
This trial tests if adding a new oral targeted therapy (elacestrant) to standard chemotherapy (capecitabine) works better than chemotherapy alone for people with a specific type of advanced breast cancer that is hormone-sensitive (ER-positive) and has stopped responding to hormone therapy.
Boston, MassachusettsAges 18 years+ - NCT07226297Enrolling by invitationPhase 1/Phase 2
Personalized treatment for GARS1-related CMT2D
This trial tests a personalized medicine designed for a single person with a specific genetic mutation (GARS1) that causes Charcot-Marie-Tooth disease type 2D. The treatment is an antisense oligonucleotide, a custom-made molecule that targets the faulty gene.
Houston, TexasAges 13 years+ - NCT07221851RecruitingPhase 3
Weekly vs daily growth hormone for short stature in children
This trial tests a weekly growth hormone injection (lonapegsomatropin) compared to the standard daily growth hormone shot in children with short stature due to certain conditions. It aims to see if the weekly shot works as well or better and is safe.
Palo Alto, CaliforniaAges 2–17 - NCT07220772RecruitingPhase 3
A drug for severe obesity caused by leptin gene mutations
This trial tests a new drug called mibavademab for people with severe obesity caused by changes in the leptin (LEP) gene. It aims to see if the drug can help with weight loss in children, teens, and adults who have this rare genetic condition.
UlmAges 2 years+ - NCT07218887RecruitingPhase 1/Phase 2
Gene therapy for BAG3-related heart failure
This study tests an experimental gene therapy called ALXN2350 for people with dilated cardiomyopathy caused by a specific genetic change in the BAG3 gene. It aims to see if the therapy can improve heart function and slow disease progression.
Birmingham, AlabamaAges 18–70 - NCT07215416RecruitingPhase 1/Phase 2
Gene therapy for ataxia-telangiectasia with specific ATM mutation
This trial tests a new genetic medicine designed for people with A-T who have a specific ATM gene change. It aims to see if the treatment is safe and if it can improve symptoms.
Boston, MassachusettsAges birth–17 years - NCT07213557RecruitingPhase 2
Simvastatin with immunotherapy for ARID1A-mutated stomach cancer
This trial tests whether adding the cholesterol drug simvastatin to standard immunotherapy and chemotherapy can help people with a specific type of gastroesophageal cancer (ARID1A mutated, HER2 negative). It is for patients who have not had prior treatment for advanced disease.
Naples, ItaliaAges 18 years+ - NCT06096974RecruitingPhase 1
Study of YL-17231 for advanced solid tumors with RAS mutations
This trial tests a new drug called YL-17231 in people with advanced solid tumors that have specific changes (mutations) in the KRAS, HRAS, or NRAS genes. It is for patients whose cancer has stopped responding to or cannot tolerate standard treatments.
Cincinnati, OhioAges 18 years+ - NCT07207070RecruitingPhase 3
New drug combination for PIK3CA-mutated breast cancer
This trial tests a new combination of two drugs (JS105 plus dalpiciclib and fulvestrant) against a standard combination (dalpiciclib plus fulvestrant) for people whose breast cancer has a PIK3CA mutation. The goal is to see if adding JS105 helps control the cancer longer.
Beijing, Beijing MunicipalityAges 18–75 - NCT07211958RecruitingPhase 3
Revumenib plus chemo for newly diagnosed AML with NPM1 mutation
This trial tests a new drug, revumenib, combined with strong chemotherapy for people with a specific type of AML (NPM1 mutation). The goal is to see if adding revumenib helps treat the leukemia better than chemo alone.
Goodyear, ArizonaAges 12 years+ - NCT07207278Recruiting
Genetics and immune study of lung signet ring cell cancer
This research study is looking at the unique genetic makeup and immune features of a rare type of lung cancer called signet ring cell carcinoma. By studying tumor samples and medical data, researchers hope to find better ways to understand and eventually treat this cancer.
Hangzhou, ZhejiangAges 18–85 - NCT07204392Recruiting
Genetic study for early-onset or familial MPN
This study explores whether certain inherited gene changes make people more likely to develop myeloproliferative neoplasms (MPNs). It is looking at patients who were diagnosed at a young age or have a family history of blood cancers, to better understand the genetic causes of these conditions.
Pavia, LombardyAges 18 years+ - NCT07207395RecruitingPhase 2
Study of JBI-802 with or without pembrolizumab for STK11-mutated lung cancer
This trial tests a new oral drug, JBI-802, alone or with pembrolizumab, for people with advanced non-small cell lung cancer that has a specific change in the STK11 gene. The goal is to see if this treatment can help shrink tumors or slow their growth.
Cincinnati, OhioAges 18 years+ - NCT07198074RecruitingPhase 3
Testing Avastin added to chemo plus Keytruda for certain endometrial cancer
This study tests adding Avastin (bevacizumab) to the standard combination of chemotherapy (carboplatin and paclitaxel) plus immunotherapy (Keytruda) for advanced or recurrent endometrial cancer with a specific genetic makeup (pMMR and TP53 mutation). The goal is to see if adding Avastin helps shrink tumors or slow their growth.
Birmingham, AlabamaAges 18 years+ - NCT07195695RecruitingPhase 3
Zongertinib after surgery for HER2-mutated lung cancer
This trial tests a new pill, zongertinib, against standard of care for people with early-stage (II–IIIB) non-small cell lung cancer that has a specific HER2 mutation in the tyrosine kinase domain. The goal is to see if zongertinib can prevent the cancer from coming back after complete surgical removal.
Fresno, CaliforniaAges 18 years+ - NCT07199127Recruiting
Studying duodenal polyps in FAP
This study reviews duodenal polyps in people with FAP to improve how they are classified. It uses existing medical records from endoscopies (scope tests) to help doctors better understand and manage polyp growth.
Milan, MIAges 18 years+ - NCT07198841RecruitingPhase 2
Studying IBI351 plus Cetuximab beta for a type of lung cancer
This trial tests a combination of two targeted drugs, IBI351 and Cetuximab beta, for people with advanced non-small cell lung cancer that has a specific KRAS G12C mutation and hasn't been treated yet. The goal is to see if this combination can shrink tumors or slow cancer growth.
Guangzhou, GuangdongAges 18–75 - NCT07201519RecruitingPhase 2
Chemo infusion for liver cancer using a hybrid care model
This trial tests a special type of chemotherapy (hepatic artery infusion) for people with cancers that have spread to the liver, such as colorectal cancer or bile duct cancer (intrahepatic cholangiocarcinoma). It studies whether delivering this treatment partly at a community center and partly at a university hospital is safe and feasible.
Lexington, KentuckyAges 18–99 - NCT06945705RecruitingPhase 2
Targeted therapy for EGFR-mutant lung cancer with brain spread
This trial tests a combination of two targeted pills (furmonertinib and anlotinib) as a first treatment for people with advanced EGFR-mutant non-small cell lung cancer that has spread to the brain. It aims to see if this combination works better than the standard approach for controlling the cancer.
ZhengzhouAges 18 years+ - NCT07188740RecruitingPhase 1
Metformin to target leukemia-related mutation during remission
This trial tests if metformin can reduce a specific mutation (DNMT3A R882) in people with acute leukemia who are in remission. The goal is to see if it helps prevent relapse.
Tianjin, Tianjin MunicipalityAges 14 years+
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Common questions
- Are there clinical trials for mutation?
- Yes. Clin2 currently lists 714 recruiting mutation 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 mutation 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 mutation 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.