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
- NCT03608631RecruitingPhase 1/Phase 2
Exosome treatment for KRAS-mutated metastatic pancreatic cancer
This Phase 1/2 study tests an investigational treatment made from “exosomes” for people with metastatic pancreatic cancer that has a specific KRAS mutation (KRAS G12D). It aims to see if this treatment can control the cancer and is safe for patients whose cancer has grown or stayed the same after prior chemotherapy.
Houston, TexasAges 18 years+ - NCT03667417Recruiting
Study of people with BRCA1/BRCA2 cancer gene changes
This study follows people who carry a harmful BRCA1 or BRCA2 gene mutation, whether or not they currently have breast or ovarian cancer. The goal is to better understand cancer risk over time and how being a carrier may affect screening and care.
MarseilleAges 18 years+ - NCT03471260RecruitingPhase 1/Phase 2
Ivosidenib and venetoclax for IDH1-mutated leukemia or MDS
This Phase 1/2 study tests whether the medicines ivosidenib and venetoclax (with or without azacitidine) can help people with certain “IDH1-mutated” blood cancers like AML or high-risk MDS. It’s meant for patients whose disease has come back, doesn’t respond, or who can’t safely get standard chemotherapy.
Boston, MassachusettsAges 18 years+ - NCT03383575RecruitingPhase 2
Azacitidine plus enasidenib for IDH2-mutant MDS
This trial tests whether combining azacitidine with enasidenib helps people with myelodysplastic syndrome (MDS) that has an IDH2 mutation. It’s designed for people who are either new to “hypomethylating” treatment or whose MDS returned/worsened after it.
Baltimore, MarylandAges 12 years+ - NCT03478761Recruiting
Registry for people with CYP24A1 vitamin D–related kidney stone risk
This is a research registry for people who have a confirmed CYP24A1 genetic change tied to high calcium and kidney stone problems. It helps researchers better understand who is affected and may guide future studies or care approaches.
Rochester, MinnesotaAges Any age - NCT03190941RecruitingPhase 1/Phase 2
T-cell therapy for HLA-A11:01 and KRAS G12V tumors
This trial tests a custom immune-cell treatment made from your blood that targets the KRAS G12V change in certain cancers. It is for people with advanced cancer who have already tried standard treatments, and it may help your immune system recognize and attack tumor cells.
Bethesda, MarylandAges 18–72 - NCT03161808Recruiting
CF rare mutation blood and tissue sample collection study
This study collects cells from people with cystic fibrosis (CF) caused by rare CFTR gene changes. The goal is to build a cell bank for future research, which may help scientists develop better tests and treatments for rare CF types.
Birmingham, AlabamaAges 17 years+ - NCT03031249Enrolling by invitationPhase 1/Phase 2
Testing ATO plus ATRA for NPM1-mutated AML after remission
This trial studies whether a combination medicine (ATO plus ATRA) is safe and works better in people with a specific type of AML (acute myeloid leukemia) caused by an NPM1 mutation. You would join only after your leukemia has reached remission, meaning the active leukemia has cleared after initial treatment.
Tianjin, Tianjin MunicipalityAges 14–55 - NCT02775461Recruiting
Registry for people at high risk for pancreatic cancer
This study is a registry (a way to track health over time) for people with a strong family history or certain inherited genetic risks for pancreatic cancer. It may help doctors learn how best to monitor and protect high-risk people, and to see who benefits most from follow-up.
New York, New YorkAges 18 years+ - NCT02727803RecruitingPhase 2
Personalized natural killer cell therapy for blood cancers
This trial tests a customized immune-cell treatment (NK cells) for certain blood cancers to try to control or eliminate cancer cells. You may qualify if your cancer has come back, did not fully respond, or has higher risk features.
Houston, TexasAges 15–80 - NCT02302742Recruiting
Registry for triple-negative breast cancer and inherited mutation carriers
This registry studies people with triple-negative breast cancer or inherited “HBOC” gene mutations (like BRCA, PALB2, PTEN, TP53). It collects information to better understand risk, outcomes, and how these cancers behave—helping future research and care.
Hays, KansasAges Any age - NCT02523014RecruitingPhase 2
Drug combo for meningioma that keeps growing despite treatment
This Phase 2 trial tests several targeted medicines (given together or by specific assignment) for meningiomas inside the brain that are progressing or not fully removed after surgery. It may help slow the tumor’s growth and improve symptoms by blocking tumor growth pathways.
Birmingham, AlabamaAges 18 years+ - NCT02432092Recruiting
Genetic testing for heart muscle disease in children and families
This study looks at genetic (DNA) changes that may cause cardiomyopathy, a disease where the heart muscle is weakened. It may help families understand possible causes and inform future care.
Indianapolis, IndianaAges Any age - NCT01861106RecruitingPhase 2
Bone marrow transplant for GATA2 mutations
This trial tests a bone marrow transplant (called allogeneic hematopoietic stem cell transplant) as a treatment for people with GATA2 mutations or MonoMAC. The goal is to replace faulty immune cells with healthy donor cells, which may reduce infections and improve immune function.
Bethesda, MarylandAges 6–70 - NCT01443468Recruiting
Study of Li-Fraumeni syndrome family cancer risk
This study collects medical and genetic information to better understand how often cancer develops in people with Li-Fraumeni syndrome and related family risks. It may help families and doctors plan earlier awareness and follow-up over a person’s lifetime.
Bethesda, MarylandAges Any age - NCT01689584Recruiting
Study of genetic variants in cancer families
This study looks at how a specific DNA genetic change (variant) runs within cancer families. It may help researchers better understand cancer risk and improve genetic testing and interpretation.
Bastia, CorsicaAges 18 years+ - NCT01668186Recruiting
Study of how peroxisome disorders change over time
This study follows people with peroxisome biogenesis disorders (PBD) or closely related enzyme/protein defects to learn how symptoms and health changes over time. It may help researchers better understand the condition and plan future treatments.
Montreal, QuebecAges Any age - NCT01060371Recruiting
Study of genetic causes of spinocerebellar ataxia
This study follows people with certain types of spinocerebellar ataxia (a group of conditions that cause progressive movement problems) to understand how the disease changes over time and how genes might influence it. It may help researchers learn more about these conditions and support future treatments.
Los Angeles, CaliforniaAges 6 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.