Clinical trials
Efficacy and Safety clinical trials
Below are recruiting efficacy and safety clinical trials, each written for real people, not researchers. We’re tracking 2,733 recruiting studies, each written for real people, not researchers, below.
Recruiting studies
- NCT07516444Recruiting
Tricuspid valve replacement using a new catheter device
This trial tests a new device that replaces a leaky tricuspid valve (a heart valve) without open-heart surgery. If your tricuspid valve isn't working well and medicines haven't helped enough, this procedure might improve your heart function and symptoms.
Minneapolis, MinnesotaAges 18 years+ - NCT07517133RecruitingPhase 3
Mitapivat for Children With Non-Transfusion-Dependent Thalassemia
This study tests a medicine called mitapivat in children ages 1–17 with thalassemia who don't need frequent blood transfusions. The medicine may help their bodies make more healthy red blood cells and reduce anemia symptoms.
Palo Alto, CaliforniaAges 1–17 - NCT07514923RecruitingEarly Phase 1
ALT001 safety study for cerebellar multiple system atrophy
This early-stage trial tests a new biologic treatment called ALT001 in people with multiple system atrophy-cerebellar type (MSA-C), a rare neurological condition affecting balance and coordination. The study focuses on whether ALT001 is safe and whether it shows early signs of helping slow disease progression.
Beijing, Beijing MunicipalityAges 30–75 - NCT07506863RecruitingPhase 3
Mitapivat for children with transfusion-dependent thalassemia
This study tests a study pill called mitapivat for children aged 1 to 17 with transfusion-dependent thalassemia. The goal is to see if mitapivat can reduce the need for blood transfusions and improve blood counts.
Atlanta, GeorgiaAges 1–17 - NCT07495215RecruitingPhase 1/Phase 2
Personalized mRNA vaccine plus immunotherapy for liver cancer after surgery
This trial tests a personalized mRNA vaccine (iNeo-Vac-R01) combined with a PD-1 inhibitor to prevent liver cancer from coming back after surgery. It is for people with early-stage liver cancer who have a high risk of recurrence.
Hangzhou, ZhejiangAges 18–75 - NCT07491458RecruitingPhase 2
Study of HU6 versus placebo for adults with MASH
This trial tests if HU6 is safe and effective for people with MASH, a type of fatty liver disease. You will either receive HU6 or a placebo to see how it affects your liver health.
Chandler, ArizonaAges 30 years+ - NCT07476183RecruitingPhase 1
Testing APR-2020 for RPS19-deficient Diamond-Blackfan Anemia
This trial is testing a new treatment called APR-2020 for children and young adults with a specific type of Diamond-Blackfan Anemia (caused by a change in the RPS19 gene). The goal is to see if this treatment is safe, tolerable, and can help reduce the need for blood transfusions.
Stanford, CaliforniaAges 2–25 - NCT06780410RecruitingPhase 3
Study of bempedoic acid for lowering cholesterol
This trial tests a drug called bempedoic acid to see if it can safely help lower cholesterol in adults. It may be an option if you have high cholesterol and need a different treatment.
BeijingAges 18 years+ - NCT07468708RecruitingEarly Phase 1
Testing a new imaging agent for prostate cancer
This trial tests a new radioactive tracer called ⁶⁸Ga-FC516 to see how well it works for imaging prostate cancer. It may help doctors find and monitor prostate cancer more accurately.
Guangzhou, GuangdongAges 18 years+ - NCT07455734RecruitingPhase 1/Phase 2
Testing BNT3214 for advanced solid tumors
This trial tests an experimental drug, BNT3214, in people with advanced solid tumors that have stopped responding to standard treatments. The goal is to see if BNT3214 is safe and helps shrink tumors.
ClaytonAges 18 years+ - NCT07459283RecruitingEarly Phase 1
Study of 177Lu-INN805 for advanced solid tumors
This trial tests a new treatment called 177Lu-INN805 for people with advanced solid tumors that have not responded to standard therapies. It aims to see if the drug is safe, where it goes in the body, and whether it can shrink tumors.
Wuxi, JiangsuAges 18–75 - NCT06228924RecruitingPhase 1
Gene therapy for ARVC with PKP2 mutation
This trial tests a new gene therapy called TN-401 for people with ARVC caused by a specific PKP2 gene mutation. The goal is to see if the treatment is safe and can help reduce dangerous heart rhythms.
San Francisco, CaliforniaAges 18–65 - NCT07429240RecruitingPhase 1/Phase 2
Gene editing trial for Duchenne muscular dystrophy (ages 2-7)
This trial tests a new gene editing treatment called PBGENE-DMD for boys with Duchenne muscular dystrophy (DMD) whose genetic mutation falls within a specific range of the dystrophin gene (exons 45-55). The goal is to see if it is safe and can help improve muscle function.
Little Rock, ArkansasAges 2–7 - NCT07429721RecruitingEarly Phase 1
Testing QI-019A for multiple myeloma that came back
This trial tests a new drug called QI-019A for people whose multiple myeloma has returned or stopped responding to at least two prior treatments. The goal is to see if it is safe and if it can help control the disease.
Wuhan, HubeiAges 18 years+ - NCT06820424RecruitingEarly Phase 1
CDH17-targeted CAR-T cells for advanced solid tumors
This trial tests a new type of immunotherapy that uses your own modified immune cells (CAR-T cells) to find and attack cancer cells that carry a specific marker called CDH17. It is for adults with advanced solid tumors that have CDH17 and who have run out of standard treatment options.
Kunming, YunnanAges 18–75 - NCT07413042RecruitingPhase 1
Radiation drug combined with immunotherapy for extensive-stage small cell lung cancer
This trial tests a combination of a radioactive drug ([225Ac]Ac-DOTATATE) and an immunotherapy drug (Tislelizumab) as maintenance treatment for people with extensive-stage small cell lung cancer (ES-SCLC) whose tumors have a specific protein (SSTR). If you have completed first-line treatment and your cancer shows SSTR on a PET scan, this study may be an option.
Beijing, Beijing MunicipalityAges 18–80 - NCT07413562Recruiting
Nerve stimulation for memory problems after brain injury
This trial tests whether stimulating a nerve in the wrist (median nerve) can help improve thinking and memory problems in people who have had a recent traumatic brain injury. It is for adults with moderate injuries who have trouble with memory and thinking.
Beijing, Beijing MunicipalityAges 18–64 - NCT06162338Recruiting
Study of LY-M001 for Gaucher Disease Type I
This study tests an injection called LY-M001 for adults with Gaucher disease type I. The goal is to see if it can safely help replace the missing enzyme and improve symptoms.
Hanzhou, ZhejiangAges 18–60 - NCT07387068RecruitingPhase 1
Testing GEN1079 for advanced solid tumors
This trial tests an experimental drug called GEN1079 for people with advanced solid tumors that have spread or cannot be removed. The goal is to see if it's safe and if it helps shrink tumors.
New Haven, ConnecticutAges 18 years+ - NCT05989958RecruitingPhase 1/Phase 2
HepaCure for acute-on-chronic liver failure
This trial tests a new treatment called HepaCure for people with acute-on-chronic liver failure (a sudden worsening of liver function in someone with long-term liver disease). The goal is to see if it is safe and can help improve liver function.
Beijing, Beijing MunicipalityAges 18–65 - NCT07368803RecruitingPhase 1/Phase 2
Personalized mRNA vaccine for bile duct cancer after surgery
This trial tests a personalized mRNA vaccine (iNeo-Vac-R01) made from your tumor's unique markers to help prevent cancer from coming back after surgery for bile duct or other digestive cancers. It may be an option if standard treatments have stopped working or caused side effects.
Hangzhou, ZhejiangAges 18–75 - NCT07357051RecruitingPhase 3
Flu treatment study for children ages 2 to 12
This trial tests a new oral medicine (JKN2301) for children with the flu. It aims to see if the medicine is safe and works well to treat flu symptoms like fever and cough.
Guangzhou, GuangdongAges 2–11 - NCT07293351RecruitingPhase 1/Phase 2
Pumitamig alone or with other drugs for advanced kidney cancer
This study tests a new drug called pumitamig, given alone or with other treatments, for people with advanced kidney cancer that has spread or cannot be removed. It aims to see if the drug is safe and if it helps shrink tumors.
Palo Alto, CaliforniaAges 18 years+ - NCT07282847RecruitingPhase 1/Phase 2
Gene therapy for adults with Pompe disease
This study tests a one-time gene therapy (AB-1009) for adults with late-onset Pompe disease. It aims to see if the treatment is safe and improves muscle and breathing function.
Phoenix, ArizonaAges 18–65
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Common questions
- Are there clinical trials for efficacy and safety?
- Yes. Clin2 currently lists 2,733 recruiting efficacy and safety 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 efficacy and safety 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 efficacy and safety 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.