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
- NCT05661643RecruitingPhase 2
Temozolomide for GIST tumors with SDH deficiency
This trial tests how well temozolomide works and how safe it is for people with GIST (a type of stomach/intestine tumor) that is SDH-deficient and has no KIT or PDGFRα mutations. It may help by targeting tumor DNA repair weakness seen in this SDH-deficient subtype.
Seoul, SeoulAges 20 years+ - NCT05672849Recruiting
Testing devices used during fetal surgery for spina bifida
This trial looks at how safe and effective certain surgical devices are when doctors perform a minimally invasive fetal procedure to repair a neural tube defect. If you choose this specific type of fetal surgery, you may be able to join the study to help improve care for future patients.
Houston, TexasAges 18 years+ - NCT05594992Enrolling by invitationPhase 3
Study extension of JR-141 for long-term Hunter syndrome safety
This is a follow-up study for people who already took JR-141 in an earlier trial for Hunter syndrome (MPS II). It checks how well the treatment works over the long term and monitors long-term safety.
Oakland, CaliforniaAges Any age - NCT05626712RecruitingPhase 1/Phase 2
CELZ-201 to preserve insulin production in early type 1 diabetes
This Phase 1/2 study tests whether CELZ-201 is safe and can help the body keep making some of its own insulin in people who were recently diagnosed with type 1 diabetes. You may be eligible if your diabetes started within the past year and your tests show you still have some insulin-making activity.
Miami, FloridaAges 18–35 - NCT05624450RecruitingPhase 3
Test drug tozorakimab for hospitalized viral lung infection needing oxygen
This Phase 3 study tests tozorakimab to see if it helps people hospitalized with a viral lung infection that is causing low oxygen levels. You might qualify if you still need supplemental oxygen when the study starts.
Birmingham, AlabamaAges 18 years+ - NCT05599880RecruitingPhase 2
Bortezomib for adults with hard-to-treat immune low platelets
This Phase 2 trial tests whether bortezomib can safely increase platelet counts in adults with immune thrombocytopenia (ITP) that keeps coming back or doesn’t respond to standard treatments. It’s for people with very low platelets who still need treatment despite prior medicines.
Guri-siAges 19 years+ - NCT05577754RecruitingPhase 2
Trial tests alpelisib for MCAP with PIK3CA mutations
This Phase 2 study tests alpelisib in children and adults with MCAP, a condition caused by a specific PIK3CA gene mutation. The goal is to see if the treatment is effective and safe, while carefully monitoring blood sugar and organ function.
AmiensAges 2–40 - NCT05555589RecruitingPhase 3
RGN-259 eye drops for neurotrophic keratopathy with persistent corneal defects
This Phase 3 trial tests whether a 0.1% RGN-259 eye drop can safely improve a stubborn corneal wound in adults with neurotrophic keratopathy (nerve-related corneal healing problems). You may qualify if you still have an eye surface defect after at least a week of standard treatments, and you meet specific measurements for corneal sensitivity and vision.
Laguna Hills, CaliforniaAges 18 years+ - NCT05562466RecruitingPhase 3
Tested inhaler for children with asthma ages 6–11
This Phase 3 study compares a new inhaler (QMF149: indacaterol plus mometasone) against budesonide to see which works better and is safe for children ages 6 to under 12 with asthma. You may be able to join if your child is already on a stable low-dose steroid inhaler and still has symptoms.
CABA, Buenos AiresAges 6–11 - NCT05583617RecruitingPhase 1/Phase 2
Testing multiple treatments for multiple myeloma after prior therapy
This early-phase study tests how safe and effective several treatment approaches are for people with multiple myeloma who have already had other treatments. Depending on your myeloma features and prior care, a study drug combination may be offered to try to control the disease.
Randwick, New South WalesAges 18 years+ - NCT05588648RecruitingPhase 1/Phase 2
Vactosertib for recurrent or progressive bone cancer
This trial tests a new drug called vactosertib for people with a type of bone cancer called osteosarcoma that has come back or hasn't responded to other treatments. The goal is to see if the drug can help shrink or slow the cancer and to check its safety.
Cleveland, OhioAges 12 years+ - NCT05511363RecruitingPhase 3
Study of a new medicine for psychosis in Alzheimer's disease
This trial tests a new drug called KarXT to see if it can safely reduce hallucinations and delusions in people with Alzheimer's disease. If you or a loved one has Alzheimer's and experiences these symptoms, this study might offer a new treatment option.
Homewood, AlabamaAges 55–90 - NCT05514340RecruitingPhase 2
Test drug for brain injury after low oxygen at birth
This Phase 2 study tests whether sovateltide can improve safety and outcomes for babies whose brains may have been injured by low oxygen around birth. It focuses on babies who show signs of moderate to severe injury soon after delivery, and who receive standard supportive care.
Mangalore, KarnatakaAges Any age - NCT05529927RecruitingPhase 2
Dexamphetamine given long-acting to reduce cocaine use
This Phase 2 study tests whether a long-acting form of dexamphetamine can reduce cocaine use in people with moderate to severe cocaine use disorder. It also checks safety, including heart and mental health side effects, while you attend study visits regularly.
The HagueAges 18 years+ - NCT05541250RecruitingPhase 1
Stem-like nerve cell treatment for severe nerve injuries
This Phase 1 study tests whether adding a patient’s own nerve support cells to a severely injured nerve is safe and helpful. It’s for people with recent major nerve damage in an arm or leg, with strict safety checks.
Miami, FloridaAges 18–65 - NCT05508633Enrolling by invitationPhase 2
Alverine test to lower high pressure in the liver
This trial studies alverine to lower portal hypertension, which is high blood pressure in the liver that can happen with cirrhosis. If it works safely, it may reduce pressure-related complications in people with confirmed liver scarring.
Shanghai, Shanghai MunicipalityAges 18–75 - NCT05462587RecruitingPhase 3
Test drug AVTX-803 for Leukocyte Adhesion Deficiency type II
This Phase 3 trial tests whether AVTX-803 can help prevent or improve infections in people with Leukocyte Adhesion Deficiency (LAD) type II. The study also checks how safe the drug is.
Rochester, MinnesotaAges 6 months–75 years - NCT05477563RecruitingPhase 3
One-time gene therapy for severe sickle cell and transfusion anemia
This trial tests a single dose of gene therapy (CTX001) to improve outcomes for people with severe sickle cell disease or transfusion-dependent beta-thalassemia. It aims to see how well it works and whether it is safe, especially after a planned stem cell transplant process.
New York, New YorkAges 12–35 - NCT05503797RecruitingPhase 2
Study of FORE8394 for cancers with BRAF changes
This trial tests a new drug, FORE8394, for people whose cancers have a change in the BRAF gene. It may be an option if you have a solid tumor or brain tumor that has not responded to other treatments.
Beverly Hills, CaliforniaAges 8 years+ - NCT05468359RecruitingPhase 1/Phase 2
Testing four medicines in children and teens with hard-to-treat tumors
This early-phase trial studies how safe and effective a combination of four medicines is for children and teens with certain solid tumors that have come back or cannot be controlled with standard treatment. It may help by improving tumor control, while also carefully tracking side effects.
Germantown, TennesseeAges Up to 30 years - NCT05466071Recruiting
TAF to prevent hepatitis B transmission during pregnancy
This study tests whether a daily antiviral pill called TAF can reduce the risk of passing hepatitis B to your baby. It is for pregnant women with high levels of the virus who are between 24 and 28 weeks along.
Guangzhou, GuangdongAges 20–40 - NCT05495035RecruitingPhase 1
Olverembatinib plus APG-2575 for children with resistant leukemia
This study tests a new combination of two oral drugs (olverembatinib and APG-2575) for children with a type of leukemia (Ph+ ALL) that has returned or stopped responding to standard treatments. The goal is to see if this combination is safe and effective.
Hefei, AnhuiAges 1–18 - NCT05421858RecruitingPhase 3
Study drug for adults with bloodstream or deep Candida infection
This Phase 3 trial tests fosmanogepix to treat adults with candidemia (Candida in the bloodstream) and/or invasive candidiasis (a deep Candida infection). It looks at whether the medicine is effective and safe, and whether it helps the infection improve when doctors also manage the infection source.
Birmingham, AlabamaAges 18 years+ - NCT05419492RecruitingPhase 1/Phase 2
ETX101 for children with SCN1A Dravet syndrome
This early-phase study tests how safe ETX101 is and whether it can help reduce seizures in young children with Dravet syndrome caused by an SCN1A gene change. It mainly enrolls infants and children with a specific type of seizure history and genetic result.
San Francisco, CaliforniaAges 6 months–17 years
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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.