Disease by etiologic mechanism

This early-stage trial tests whether transplanting lab-grown dopamine-producing cells into the brains of people with Parkinson's disease is safe and might help replace the cells lost to the disease. About 7 adults with Parkinson's who have not responded well to standard medicatio…

This early-stage trial is testing an investigational therapy called B019, which uses a patient's own immune cells engineered to recognize and attack leukemia cells. The study focuses on people with B-cell acute lymphoblastic leukemia that has returned or not responded to standard…

This Phase III trial is testing whether a single infusion of AZD0120, a CAR-T therapy that targets two proteins on myeloma cells, can keep the cancer at bay better than continuous standard treatment. It enrolls adults newly diagnosed with multiple myeloma who are not eligible for…

This study tests an experimental flu vaccine called BPL-1357, given as a shot or nasal spray, to see if it is safe and can protect against multiple flu types. About 129 healthy adults aged 18-55 will receive the vaccine or a placebo, then be exposed to a flu virus in a controlled…

This early-stage trial will test two experimental mRNA vaccines designed to teach the immune system to make antibodies that target HIV. About 42 adults with well-controlled HIV will receive the vaccines and then temporarily pause their regular HIV medication under close monitorin…

This study tests an experimental vaccine called RSVpreF in adults to see if it is safe and triggers a strong immune response against respiratory syncytial virus (RSV). Around 540 adults in India will receive either the vaccine or a placebo. Researchers will monitor for side effec…

This early-stage trial is testing a new COVID-19 booster vaccine called OCU500, which is given as a nasal spray or inhaled mist instead of a shot. The study involves 80 healthy adults aged 18-64 who have already been vaccinated against COVID-19. Researchers are checking if the va…

This study tests whether adding the immunotherapy drug nivolumab to standard chemotherapy (gemcitabine and cisplatin) before radiation therapy is safe and effective for children and young adults (up to age 21) with newly diagnosed nasopharyngeal carcinoma. The goal is to see if t…

This study tests an experimental drug called ATV-1601 in people with moderate to severe HHT, a condition that causes frequent nosebleeds and anemia. The trial has two parts: first, participants receive either the drug or a placebo to check safety and effectiveness; then, all elig…

This study tests a new medicine, PF-08634404, in adults with advanced bladder cancer that has spread. Some participants receive the drug alone, while others get it combined with another cancer drug. The goal is to check safety, how well it works, and how it affects the body. Abou…

This trial is testing whether combining two immunotherapy drugs, cemiplimab and dupilumab, can safely and effectively shrink tumors in people with early-stage, operable non-small cell lung cancer. Participants receive both drugs before their scheduled surgery, and researchers wil…

This phase III trial is testing whether a precise, high-dose radiation technique called stereotactic radiosurgery (SRS) is better at preserving memory and thinking than the current standard of whole brain radiation that avoids the hippocampus (the brain's memory center) plus a dr…

This study tests whether fecal microbiota transplantation (FMT) can reduce gut-related side effects from antibiotics in 40 adults with B-cell lymphoma who are receiving standard CAR-T cell therapy. Participants will receive FMT alongside their cancer treatment to see if it improv…

This study tests a new way to deliver radiation therapy for people with diffuse glioma, a type of brain tumor. Instead of giving the same dose throughout treatment, doctors will use MRI and PET scans taken during treatment to adjust the radiation dose to target resistant areas an…

This phase 3 trial is testing whether a new drug, nemtabrutinib, combined with venetoclax works better than the current standard of venetoclax plus rituximab for people with relapsed or refractory chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL). About 735 p…

This study tests a drug called SIL-8301 (senicapoc) in 105 adults with sickle cell disease who have low hemoglobin but few pain crises. Participants take the drug or a placebo daily for 24 weeks, along with their usual hydroxyurea. The main goal is to see if the drug raises hemog…

This study tests whether a blood test that looks for cancer DNA (ctDNA) can find breast cancer returning earlier than usual scans or exams. About 976 people with HR-positive/HER2-negative early breast cancer who had surgery will give blood samples over time. If the test shows sig…

This program offers daraxonrasib to adults with metastatic pancreatic cancer that has worsened after prior treatment and who have no other good options. It is for patients who cannot join ongoing clinical trials. The goal is to provide access to this experimental drug when no sta…

This early-phase study tests a combination of two drugs—losartan (a blood pressure medicine) and sunitinib (a cancer drug)—in people aged 10 and older with osteosarcoma (a type of bone cancer) that has returned or not responded to treatment. The main goal is to find the safest do…

This phase 2 study tests an oral drug called nemtabrutinib in about 490 people with blood cancers like chronic lymphocytic leukemia, non-Hodgkin lymphoma, and Waldenstrom's macroglobulinemia. The goal is to see if the drug can shrink tumors and control the cancer, while also chec…

This Phase 2 trial tests the drug alisertib in 120 people with extensive stage small cell lung cancer that has worsened after standard treatments like chemotherapy and immunotherapy. The goal is to see if alisertib can shrink tumors or slow the disease, and to identify which pati…

This Phase 3 trial is testing whether a new medicine called PF-08634404, combined with standard chemotherapy, works better than the current treatment Avastin plus chemo for people whose colorectal cancer has spread. About 800 participants who have not had prior treatment for meta…

This study tests an oral drug called nizubaglustat in children and teens aged 4 and older with Niemann-Pick type C disease, a rare genetic disorder that affects movement and thinking. Participants will take the drug or a placebo for 18 months to see if it slows the disease and im…

This study tests a new medicine called PF-08634404, given alone or with chemotherapy, for adults with early or locally advanced non-small cell lung cancer. The trial has three parts: before surgery, after surgery if cancer remains, or after chemoradiation. Researchers will check …

This phase II trial is testing whether newer targeted drugs (trastuzumab emtansine and trastuzumab deruxtecan) work better than standard chemotherapy plus trastuzumab for people with HER2-positive or HER2-low salivary gland cancer that has come back, spread, or cannot be removed.…

This phase 2 trial tests whether adding the drug nemtabrutinib to a CAR T-cell therapy called brexu-cel can help people with mantle cell lymphoma that has come back or not responded to treatment. Nemtabrutinib blocks a protein that helps cancer cells grow, while brexu-cel uses a …

This study tests two experimental drugs, PF-08653945 and PF-08653944, given alone or together, for weight loss in adults with overweight or obesity. About 872 participants will receive either the drugs or a placebo injection. The main goal is to see how much weight they lose over…

This early-phase trial tests giving radiation therapy before surgery to people whose adrenal cancer has come back. The goal is to find a safe radiation dose and see if it helps shrink tumors before removal. About 32 adults with resectable recurrent ACC will receive daily radiatio…

This early-phase study tests a new drug called HLD-0117 in about 33 women with a type of advanced breast cancer (ER-positive) that has spread and stopped responding to standard therapies. The main goals are to check the drug's safety and find the right dose, while also seeing if …

This study tests whether adding an experimental drug called PDS01ADC to standard chemotherapy (docetaxel) can help men with prostate cancer that has spread to other parts of the body. About 86 men with either castration-sensitive or castration-resistant metastatic prostate cancer…

This study tests whether adjusting cancer drug doses for people with the Duffy null blood trait can reduce treatment delays and dangerous fevers. About 90 participants with multiple myeloma or triple-negative breast cancer will receive standard chemotherapy with modified dosing g…

This Phase 3 trial tests whether adding the targeted drug calderasib to the immunotherapy pembrolizumab helps people with a specific KRAS G12C-mutated lung cancer live longer without their cancer growing. About 675 participants with advanced non-squamous non-small cell lung cance…

This study tests whether a less invasive surgery called sentinel lymph node biopsy can replace standard neck dissection for early-stage oral cavity cancer. About 686 patients will be randomly assigned to one of the two procedures. The goal is to see if the gentler approach mainta…

This study tests whether adding enasidenib to standard treatment (ASTX727) improves outcomes for people with a high-risk form of myelodysplastic syndrome (MDS) that has a specific gene mutation (IDH2). About 54 adults who have not had prior cancer therapy will be randomly assigne…

This Phase 3 trial tests ZL-1310, a drug that delivers chemotherapy directly to cancer cells, in people with small cell lung cancer that has returned after initial treatment. About 480 participants will receive either ZL-1310 or one of three standard treatments. The study aims to…

This early-stage study tests a new drug called AO-252 in people with advanced solid tumors, including those that have spread to the brain. The main goals are to check safety, find the best dose, and see if the drug can shrink tumors or stop them from growing. About 86 adults whos…

This early-stage study tests a new medicine called PF-08046033 in adults with advanced lung, esophageal, or skin cancers that have spread or can't be removed. The main goal is to check safety and find the right dose. Researchers will also look for early signs that the drug may he…

This phase 3 trial tests whether the drug vorasidenib can delay tumor progression in people with IDH-mutant grade 2 or 3 astrocytoma after they finish initial chemoradiotherapy. About 468 participants will receive either vorasidenib or a placebo daily. The main goal is to see if …

This study tests a program called CO-LEAD that trains doctors to use effective communication strategies and a patient survey to identify barriers to taking lupus medications. Researchers will enroll 480 patients and their clinicians at two academic centers. The goal is to see if …

This study tests a new drug called AI-081 in people with advanced solid tumors that have spread or cannot be removed by surgery. The drug is a bispecific antibody that targets two proteins (PD-1 and VEGF) involved in cancer growth and immune evasion. The trial has two phases: fir…

This early-stage study tests a new treatment called C.001 for people with retinal diseases that cause vision loss, such as geographic atrophy and Stargardt disease. The treatment is given as a single injection into the eye. The main goal is to check if it is safe, and researchers…

This study looks at women with breast cancer that has spread to lymph nodes under the arm, found before surgery. Normally, doctors remove many lymph nodes, which can cause arm swelling and pain. The trial tests if removing only the first few affected nodes is just as effective fo…

This Phase 2 trial tests whether combining pembrolizumab (an immunotherapy) with aldesleukin (IL-2) can shrink tumors in people with advanced melanoma or kidney cancer that hasn't responded to prior treatments. About 78 adults will receive two cycles of the combo in the hospital,…

This study tests a new drug called elacestrant against standard hormone therapy in people with a certain type of early breast cancer (ER+/HER2-) that has spread to lymph nodes and has a high chance of returning. About 4,220 participants will be randomly assigned to receive either…

This study tests whether taking probiotic supplements can improve the gut and vaginal microbiome in women with advanced or recurrent ovarian cancer who are receiving platinum-based chemotherapy. About 161 participants will be randomly assigned to receive either probiotics or a pl…

This phase 3 trial will test whether adding the drug quizartinib to the standard two-drug combination (venetoclax and azacitidine) helps adults with newly diagnosed acute myeloid leukemia (AML) live longer. The study includes 376 people aged 18 and older who cannot receive intens…

This study tests whether patients can safely stop the drug tacrolimus earlier than usual after a stem cell transplant. Tacrolimus is given to prevent graft-versus-host disease (GVHD), but newer methods have lowered GVHD risk. The trial will enroll 50 people with blood cancers and…

This phase 3 trial tests whether adding a precise, high-dose radiation treatment (SBRT) to standard chemoradiation and immunotherapy improves survival for people with locally advanced non-small cell lung cancer that cannot be surgically removed. About 474 participants will be ran…

This phase I trial is testing whether giving a single, high dose of radiation (radiosurgery) before surgery can help control high grade glioma, a fast-growing brain tumor. About 28 adults with newly diagnosed or previously biopsied high grade glioma will receive radiosurgery foll…

This early-stage study tests whether a single dose of an experimental drug called IAP086 is safe in 30 adults with HIV whose virus is already well-controlled by standard antiretroviral therapy. Participants receive one intravenous infusion and are monitored closely for 28 days. T…

This phase 3 trial is testing whether a combination of two immunotherapy drugs (nivolumab and relatlimab) plus chemotherapy works better than the current standard (pembrolizumab plus chemotherapy) for people with advanced non-squamous lung cancer whose tumors have PD-L1 levels of…

This early-stage trial tests a drug called fostamatinib in 30 adults who have developed donor-specific antibodies after a lung transplant. These antibodies can attack the new lung and raise the risk of death. Participants take fostamatinib or a placebo for 8 weeks, along with the…

This study tests whether adding a new targeted medicine (vepugratinib) to two standard cancer drugs can help people with advanced bladder cancer that has a specific genetic change (FGFR3). About 450 adults whose cancer has spread or cannot be removed by surgery will take part. Th…

This early study tests a new device that uses heat to remove abnormal cells in the esophagus of people with Barrett's esophagus, a condition that can lead to cancer. The study has two parts: first, a small group of people scheduled for esophagus removal surgery will help find the…

This study tests whether a newer drug called pirtobrutinib works better than the current standard drug ibrutinib for people with chronic lymphocytic leukemia or small lymphocytic lymphoma. About 737 adults who need treatment for their cancer will be randomly assigned to receive o…

This early-stage trial explores whether adding two experimental capsules, ND-12 and H-6, to standard mesalazine treatment can better control chronic inflammatory diarrhea in adults, including those with active ulcerative colitis. Participants will take the combination or a placeb…

This study tests a new oral drug, VS-7375, alone or with another drug (cetuximab), in 180 people with metastatic pancreatic cancer that has a specific genetic change called KRAS G12D. The goal is to see if the drug shrinks tumors and is safe. Participants must have this mutation …

This phase 2 trial is testing a drug called calderasib (MK-1084), given alone or with cetuximab, in 150 people with advanced solid tumors that have a specific KRAS G12C mutation. The goal is to see if the cancer shrinks or disappears and to check for side effects. Participants mu…

This study tests a new drug called BMS-986340, alone or with other cancer treatments, in people with advanced solid tumors like lung, colon, or breast cancer. The goal is to find safe doses and check for side effects. About 1,100 adults with tumors that have worsened after standa…

This phase II trial tests whether adding enasidenib to standard ASTX727 and venetoclax improves remission rates in older adults (or younger unfit patients) with newly diagnosed IDH2-mutated acute myeloid leukemia (AML). About 93 participants will be randomly assigned to receive e…

This Phase 2 trial tests a personalized cell therapy called Descartes-08 for people with dermatomyositis or antisynthetase syndrome, two rare autoimmune diseases that cause muscle weakness and inflammation. The therapy uses a patient's own immune cells, modified to target and red…

This study tests a procedure called mucous fistula refeeding in 120 infants who have a temporary bowel opening (enterostomy). The idea is to take the baby's own stool and put it back into the lower bowel to help it get ready to work again. The goal is to see if this helps babies …

This early-stage study tests a new oral drug, APR-1051, in about 90 adults with advanced solid tumors that have not responded to other treatments. The main goals are to check the drug's safety and find the right dose. Researchers will also look for early signs that the drug might…

This phase III trial tests whether giving a higher dose of radiation can help people with locally advanced pancreatic cancer live longer. Participants must have already had 4-6 months of chemotherapy. The study compares higher-dose radiation to standard care (more chemo, observat…

This early-phase trial is exploring whether a radioactive drug called [211At]MABG, given in several smaller doses, is safe and tolerable for children with neuroblastoma that has relapsed or not responded to treatment. The study will include about 10 participants aged 1 year and o…

This phase 2 trial tests whether the drug nivolumab can slow or stop the growth of Epstein-Barr virus (EBV)-positive lymphoproliferative disorders and non-Hodgkin lymphomas. About 40 people aged 12 and older whose cancers have not responded to standard therapy will receive nivolu…

This phase 3 trial tests whether adding the targeted pill calderasib (MK-1084) to the immunotherapy durvalumab helps people with a specific KRAS G12C-mutated lung cancer live longer without their cancer growing. About 310 participants who have finished chemotherapy and radiation …

This study tests whether a combination of two drugs (ZEN003694 and talazoparib) can shrink tumors in people with advanced solid cancers that have stopped responding to standard treatments. About 88 adults will take part. Researchers will also look at participants' genes to see if…

This early-phase study tests gilteritinib, a drug already approved for a type of leukemia, in about 40 adults with ALK-positive non-small cell lung cancer whose tumors no longer respond to standard ALK inhibitors. Participants take gilteritinib tablets daily in 28-day cycles. The…

This study tests the safety and effectiveness of targeted therapies or immunotherapies in people with metastatic colorectal cancer (cancer that has spread). Participants are assigned to a treatment based on specific biomarkers found in their tumor. The goal is to see if these per…

This phase 3 trial is testing a drug called fitusiran in about 85 boys aged 1 to under 12 with severe hemophilia A or B, including those with inhibitors that make standard treatments less effective. The drug is given as a regular shot under the skin to help prevent bleeding episo…

This phase III trial tests whether proton craniospinal irradiation (pCSI) can improve survival compared to standard involved-field radiation therapy (IFRT) for patients with breast or non-small cell lung cancer that has spread to the lining of the brain and spinal cord (leptomeni…

This Phase 2 trial tests whether adding a special immune cell infusion (GKL-006) to a standard procedure that blocks blood flow to tumors (TACE) can shrink liver cancers that cannot be removed by surgery. About 48 adults with unresectable hepatocellular carcinoma will receive the…

This early-phase trial is testing a new cell therapy called CD83 CAR T cells in 26 adults with acute myeloid leukemia (AML) that has returned or not responded to treatment. The therapy uses a patient's own immune cells, genetically modified to recognize and attack a protein (CD83…

This study tests a new medicine called PF-08634404 given with chemotherapy for adults with extensive-stage small cell lung cancer, a fast-growing cancer that has spread. The trial has two parts: first checking safety, then comparing the new combo to an approved treatment (atezoli…

This phase 2 trial tests whether a patient's own white blood cells, genetically modified in a lab to recognize and attack their specific tumor mutations, can shrink metastatic cancers. Participants include people with several advanced solid tumors or multiple myeloma who have exh…

This study looks at the long-term safety of a drug called zigakibart for adults with IgA nephropathy, a kidney disease. It is for people who have already finished a previous zigakibart study. Researchers will monitor side effects and check kidney function over 96 weeks.

This early-stage trial tests a modified herpes virus (C5252) injected directly into the brain tumor cavity of 36 adults with recurrent high-grade glioma who have run out of standard options. The goal is to see if the virus is safe and can help control the disease. Participants wi…

This study is for adolescents and young adults (ages not specified) with newly diagnosed Philadelphia chromosome-negative acute lymphoblastic leukemia (ALL). Researchers want to see if a specific combination of chemotherapy drugs plus the immunotherapy blinatumomab helps more pat…

This study tests whether a continuous glucose monitor (CGM) can help people with glioblastoma, a severe brain cancer, manage their blood sugar levels and potentially live longer. High blood sugar during treatment is linked to worse outcomes, often caused by steroids. The CGM devi…

This phase 3 trial is testing a new drug called palazestrant (OP-1250) combined with ribociclib against the standard letrozole plus ribociclib for people with ER+, HER2- advanced breast cancer who haven't had prior treatment. About 1,000 participants will be randomly assigned to …

This early-stage trial is evaluating an investigational drug called ACT201, given as an injection, to see if it is safe and tolerable. The study first tests single and multiple doses in healthy volunteers, then moves to people with chronic hepatitis B whose virus is already suppr…

This early-phase trial tests whether adding a menin inhibitor (SNDX-5613) to standard chemotherapy can better control acute myeloid leukemia (AML) in people with certain gene changes (NPM1 or MLL). About 38 newly diagnosed adults will receive the combination to find the safest do…

This phase 3 trial tests whether a personalized mRNA cancer vaccine (intismeran) alone or combined with an immunotherapy drug (pembrolizumab) can prevent non-small cell lung cancer from coming back after complete surgical removal. About 876 people with high-risk stage I lung canc…

This study tests two different drug combinations for younger adults with a specific type of acute myeloid leukemia (CBF-AML). One group gets standard chemotherapy plus a targeted antibody, while the other gets the same chemotherapy plus a newer targeted pill. The goal is to see w…

This study tests whether omega-3 fatty acid supplements from fish oil, combined with a healthy diet, can reduce disease activity in adults with dermatomyositis, a rare autoimmune disease causing muscle weakness and skin rashes. About 300 participants will take either fish oil cap…

This trial tests whether two approved drugs, dabrafenib and trametinib, can treat rare cancers that have a specific genetic change called BRAF V600. These drugs are already used for melanoma and lung cancer, but researchers want to see if they help people with other cancer types.…

This phase 3 trial tests two injectable drugs, pozelimab and cemdisiran, alone or together, to slow the growth of geographic atrophy—an advanced form of dry age-related macular degeneration that causes central vision loss. About 975 participants will receive either the drugs or a…

This early-phase trial tests a new drug called metarrestin in people with advanced solid tumors that have spread (metastasized). The goal is to find a safe dose and see if it can shrink tumors. The study includes adults with pancreatic, breast, or other solid tumors, and children…

This early-stage trial is testing an investigational drug called GIM-122 in adults with advanced solid tumors that have not responded to standard treatments, including PD-1/PD-L1 inhibitors. GIM-122 is a special antibody designed to fight cancer in two ways. The study aims to fin…

This study tests a once-monthly injection called berobenatide to help adults with overweight or obesity lose weight. About 954 participants will receive either the drug or a placebo for 20 months. The goal is to see if the drug is safe and effective for weight loss, including in …

This study looks at whether the diabetes drug pioglitazone can improve how the liver processes energy in people with nonalcoholic fatty liver disease (NAFLD) and type 2 diabetes. Researchers will measure liver mitochondrial function using special tracers before and after 16 weeks…

This study tests the drug nirogacestat in 50 premenopausal women with desmoid tumors (a type of non-cancerous but aggressive growth). The main goal is to see if the drug affects ovarian function, such as menstrual periods and hormone levels. Participants take nirogacestat orally,…

This early-stage trial is testing a new drug called DNL952 in 32 adults with late-onset Pompe disease. The main goal is to check if the drug is safe and how the body processes it. Participants will receive the drug through an IV infusion at different doses.

This phase 2 trial tests a drug called FG-3246, which is designed to seek out and attack prostate cancer cells that have a specific marker (CD46) on their surface. The study will enroll 75 men with metastatic castration-resistant prostate cancer that has worsened after hormone th…

This study tests whether adding the drug dinutuximab to standard chemotherapy and other treatments helps children with high-risk neuroblastoma live longer without their cancer returning. About 478 children and young adults up to age 30 will be randomly assigned to receive either …

This Phase III trial tests whether giving radiation in three smaller doses (FSRS) works better than a single large dose (SRS) for cancer that has spread to the brain. About 269 adults with lung, breast, melanoma, kidney, or gastrointestinal cancers will be randomly assigned to on…

This study follows 138 adults in Bulgaria and Slovenia with HER2-low advanced breast cancer who are taking trastuzumab deruxtecan (T-DXd), a targeted therapy. Researchers will track how long patients go before needing a different treatment or dying. The goal is to see how well T-…

This phase 2 trial tests whether adding the immunotherapy drug cemiplimab to standard chemotherapy before surgery helps people with sinonasal squamous cell carcinoma. About 108 participants will be randomly assigned to receive either chemo alone or chemo plus cemiplimab before th…

This phase 2 trial is testing a new treatment called FT819 for people with lupus nephritis, a serious kidney complication of lupus. FT819 is made from donor immune cells that are engineered to target and destroy faulty B cells. The study will enroll 53 participants and aims to se…

This study tests two experimental drugs, gocatamig and ifinatamab deruxtecan, alone or with standard chemotherapy and immunotherapy, for people with extensive-stage small cell lung cancer. The goal is to see if the combination is safe and can shrink tumors. About 170 participants…

This early-stage trial tests a new mRNA cancer vaccine called CryptiVax-1001 in 50 women with advanced ovarian cancer. The vaccine is designed to train the immune system to recognize and attack cancer cells, potentially delaying or preventing the cancer from coming back. The main…

This study tests whether a new drug combination (BMS-986489) works better than the current standard (durvalumab) as follow-up treatment after chemoradiation for limited-stage small cell lung cancer. About 250 adults whose cancer hasn't spread widely will be randomly assigned to o…

This study tests a new treatment called AZD0120 for people with multiple myeloma that has come back or stopped responding to at least three prior therapies. AZD0120 is a personalized cell therapy that reprograms a patient's own immune cells to attack two targets on myeloma cells …

This study tests whether a combination of three drugs (cyclophosphamide, bortezomib, and maraviroc) can safely prevent graft-versus-host disease after a half-matched bone marrow transplant in people living with HIV who also have a blood cancer. The trial involves 265 participants…

This study tests personalized radiotherapy approaches for people with lung cancer, brain metastases, or other solid tumors. It aims to see if adapting radiation based on how a patient responds is safe and effective. About 45 adults will participate, receiving either extra radiati…

This early-phase trial is testing a combination of two drugs—teclistamab and iberdomide—in people with multiple myeloma that has come back or stopped responding to treatment. The goal is to find the safest and most effective dose of iberdomide when used with teclistamab. About 26…

This phase 1 trial investigates a new treatment for advanced ovarian cancer that has stopped responding to platinum chemotherapy. The therapy uses a patient's own immune cells, called T cells, which are genetically modified in a lab to recognize and attack a protein called TAG72 …

This phase 3 trial is testing whether adding immunotherapy drugs (brentuximab vedotin and nivolumab) to standard chemotherapy (with or without radiation) improves survival for people aged 5 to 60 with newly diagnosed stage I or II classical Hodgkin lymphoma. About 1,875 participa…

This early-stage trial tests a new drug called M7437 in people with advanced solid tumors that have not responded to standard treatments. M7437 is an antibody-drug conjugate designed to deliver chemotherapy directly to cancer cells that have a specific marker (Ly6E). The study ai…

This early-phase trial is testing a new drug called BL-M07D1 in people with advanced HER2-positive or low-expression breast cancer and other solid tumors that have not responded to standard treatments. The study has two parts: first, finding the safest dose, and then testing that…

This phase 3 trial will compare two drug combinations for people newly diagnosed with multiple myeloma who are not eligible for or refuse a stem cell transplant. About 500 participants will receive either belantamab mafodotin or daratumumab, both combined with standard drugs (bor…

This study tests an oral drug called pacritinib in people aged 12 and older with myelodysplastic syndromes (MDS) or related blood cancers (MDS/MPN). The goal is to find the best dose and see if it can shrink or control the disease. Participants take capsules twice daily for up to…

This early-stage trial tests a new drug called PPM, which are tiny particles loaded with the chemotherapy drug paclitaxel and coated with a molecule (PLZ4) that helps them target bladder cancer cells. The drug is given directly into the bladder through a catheter once a week for …

This phase 2 trial tests an experimental drug called vamifeport in 84 adults with HFE-related hereditary hemochromatosis, a genetic condition causing iron buildup. The drug aims to reduce liver iron levels, potentially offering an alternative to regular blood removal. Participant…

This trial asks whether removing a wider set of lymph nodes (D3) during left colon cancer surgery improves 5-year survival compared to the standard removal (D2). It enrolls about 1,381 people with adenocarcinoma located between the splenic flexure and rectosigmoid junction. Resea…

This trial compares two ways of feeding very preterm infants (born before 32 weeks and under 1500 grams). One group receives a short period of small 'trophic' feeds for one day before advancing to full feeds, while the other receives trophic feeds for three days. The goal is to s…

This study compares two treatments for people with advanced head and neck cancer who cannot use the standard drug cisplatin. About 454 participants will receive either cetuximab (a targeted antibody) or a chemotherapy combo (carboplatin + paclitaxel) alongside precise radiation t…

This trial tests whether an internet-based form of cognitive behavior therapy (CBT) can reduce symptoms of common mental disorders like anxiety, depression, and stress-related conditions in adults seeking primary care. About 500 participants will be randomly assigned to either th…

This phase II trial tests whether adding precise, high-dose radiation (SABR) to the main kidney tumor improves outcomes for people with metastatic kidney cancer that cannot be surgically removed. Participants receive standard immunotherapy drugs (avelumab, axitinib, or cabozantin…

This phase 1 trial tests the safety and best dose of an oral drug called ASTX727 (a combination of cedazuridine and decitabine) along with filgrastim in children with high-risk acute myeloid leukemia (AML) that has returned or not responded to treatment. All participants have alr…

Cystinosis is a rare inherited disease that causes kidney failure and poor growth due to cystine buildup in cells. This study follows 330 patients taking cysteamine, a drug that lowers cystine levels, to track long-term effects, detect new complications, and support genetic resea…

This study tests a gene therapy called ETX101 for children with Dravet syndrome, a severe form of epilepsy caused by a genetic change. The treatment uses a harmless virus to deliver a working copy of the gene to help control seizures. The trial includes infants and children up to…

This early-phase trial is testing a gene therapy called SGT-212 for Friedreich's ataxia, a rare genetic disease that damages the nervous system. Ten adults with the condition will receive the therapy through an injection into the brain and a vein. The main goal is to see if it is…

This study tests a drug called enpatoran for people with lupus who have active skin rashes or lesions. About 202 participants will take either enpatoran or a placebo twice daily for 24 weeks, alongside their usual treatments. The goal is to see if the drug can significantly impro…

This study is testing whether adding the immunotherapy drug dostarlimab to standard chemotherapy and radiation can help women with a rare and aggressive type of cervical cancer called small cell neuroendocrine carcinoma. About 45 women with early to advanced stages of the disease…

This study compares standard chemotherapy (gemcitabine and oxaliplatin) alone versus the same drugs plus a liver-directed chemotherapy (HAI FUDR/dexamethasone) in people with bile duct cancer that has spread within the liver but cannot be removed surgically. About 164 adults with…

This phase 3 trial tests whether injecting the drug cemiplimab directly into early-stage skin cancer tumors can work as well as surgery. About 369 people with small tumors on the head, neck, hand, or shin will be randomly assigned to get either the injections or standard surgery.…

This phase 2 trial tests whether a combination of enfortumab vedotin and pembrolizumab can help people with muscle-invasive bladder cancer avoid bladder removal. About 240 adults who are candidates for surgery will receive the drugs intravenously. The main goals are to see how ma…

This early-stage trial is testing an experimental drug called SIM0505 in adults with advanced solid tumors that have not responded to standard treatments. The study aims to find a safe dose and check for any early signs that the drug shrinks tumors. About 414 participants will re…

This clinical trial is testing whether adding the immunotherapy drug nivolumab to standard chemotherapy (paclitaxel and ramucirumab) can help people with advanced stomach or esophageal cancer live longer. The study includes 224 adults whose cancer has a specific marker (PD-L1 CPS…

This study looks at two ways to protect the heart and metabolism in men with prostate cancer who are on hormone therapy (ADT). ADT can increase the risk of heart problems. The study compares time-restricted eating (a form of intermittent fasting) and two GLP-1 drugs (semaglutide …

This early-stage study is testing an experimental oral drug, VVD-133214, in people with advanced solid tumors that have specific genetic features (MSI or dMMR). The drug works by blocking a protein that helps these cancers grow. The study will check safety and how well the drug w…

This early-stage study tests a new treatment called UB-VV111 given with rapamycin for people with certain types of blood cancer (large B-cell lymphoma or chronic lymphocytic leukemia) that have come back or stopped responding to other treatments. The main goal is to check the saf…

This study tests a device called PRIMA, which is implanted under the retina to help people with inherited retinal diseases like Stargardt disease and retinitis pigmentosa see better. The trial will enroll 5 adults with severe central vision loss and measure improvements in visual…

This early-phase trial is testing a new drug, FOG-001, in about 595 people with advanced or metastatic solid tumors, including colorectal, prostate, and liver cancers. The drug is given alone or with other cancer treatments to see if it is safe and shrinks tumors. The study is cu…

This early-phase trial tests a drug called XER-001 (a form of amifostine given through a tube into the small intestine) combined with precise radiation therapy for people with locally advanced pancreatic cancer that cannot be removed by surgery. The study will enroll 36 participa…

This trial asks whether giving COPD medication through a nebulizer works better than a dry powder inhaler. It enrolls adults over 40 with COPD symptoms and significant air trapping. Participants receive either a nebulizer or inhaler treatment, and researchers compare changes in l…

This study tests a new medicine called ersodetug for people whose tumors cause dangerously low blood sugar that standard treatments can't control. About 16 adults with insulin-producing tumors will receive the drug alongside their usual care. The main goal is to see if ersodetug …

This phase 3 trial is testing whether the immunotherapy drug durvalumab, given after surgery, can prevent early-stage non-small cell lung cancer from coming back. About 306 participants who have had their tumor completely removed will be randomly assigned to receive durvalumab or…

This phase 3 trial tests whether a new drug called ABP 938 works as well as the approved treatment Eylea HD for wet age-related macular degeneration, a leading cause of vision loss. About 304 people aged 50 and older with untreated wet AMD will receive injections of either drug i…

This phase 3 trial is testing a daily pill called giredestrant in 300 people with ER+/HER2- early breast cancer who had to stop standard aromatase inhibitors due to side effects. The study will track how well patients stick with the treatment, their quality of life, and any side …

This study is exploring whether giving radiotherapy at the same time as immunotherapy and chemotherapy, versus giving it after, works better as a first treatment for extensive-stage small cell lung cancer. The trial will enroll about 60 adults with this advanced cancer. Participa…

This study compares a new drug called Rina-S to standard chemotherapy (paclitaxel or doxorubicin) in people with endometrial cancer that has returned or worsened after platinum-based chemo and immunotherapy. About 660 participants will be randomly assigned to receive either Rina-…

This phase 2 trial tests whether injecting the experimental drug INT230-6 directly into the tumor before standard chemotherapy and immunotherapy can improve outcomes for people with early triple-negative breast cancer (TNBC). About 61 participants will be randomly assigned to rec…

This study tests whether a non-invasive brain stimulation technique called rTMS, given before cognitive-behavioral therapy, can help adults with cocaine use disorder quit. Thirty participants will receive either real or sham rTMS daily for 3 weeks, followed by 12 weeks of weekly …

This study tests whether a stem cell transplant from a healthy donor can treat GATA2 deficiency, a rare genetic condition that weakens the immune system and raises the risk of leukemia. Participants aged 6 to 70 with GATA2 mutations will receive chemotherapy or radiation before t…

This study tests a drug called tovorafenib (DAY101) in children and young adults (ages 6 months to 22 years) with Langerhans cell histiocytosis that has returned or not responded to treatment. The drug works by blocking enzymes that help cancer cells grow. Researchers will check …

This study compares an experimental drug (PF-08046054) to standard chemotherapy (docetaxel) in about 680 adults with advanced PD-L1 positive non-small cell lung cancer that has worsened after prior treatments. Participants are randomly assigned to receive one of the two drugs by …

This early-stage study is testing whether combining two drugs—teclistamab and mezigdomide—is safe and effective for people with multiple myeloma that has come back or stopped responding to other treatments. About 18 participants will receive the drugs in a specific sequence. The …

This study tests several new drug combinations given before and after surgery for stomach or esophageal cancer. About 100 people with resectable tumors will receive these treatments to see if they are safe and can shrink or eliminate the cancer. The goal is to improve outcomes by…

This study compares two surgical approaches for Fuchs endothelial dystrophy, a condition that clouds the cornea. One is a full corneal transplant (DMEK), the other is a simpler procedure that removes only the diseased cells (DSO) plus Ripasudil eye drops to help healing. 60 parti…

This phase 3 trial is for people with advanced liver cancer that has spread to nearby blood vessels. It compares standard immunotherapy drugs (atezolizumab and bevacizumab) alone versus the same drugs plus targeted radiation (SBRT). The goal is to see if adding radiation helps pa…

This early-stage study tests a new drug, PF-07994525, in about 120 adults with advanced multiple myeloma that has come back or not responded to treatment. The main goals are to check the drug's safety, find the right dose, and see if it can shrink tumors. The study has two parts:…

This study tests whether a new drug called pumitamig, combined with chemotherapy, works better than the current standard treatment (bevacizumab plus chemo) for people with advanced colorectal cancer that cannot be removed by surgery. About 990 adults who have not yet been treated…

This study tests a new medicine called fosmanogepix for adults with serious mold infections that are hard to treat. About 234 people will join, receiving either the new drug or standard care. The main goal is to see if fewer people die by day 42. The study is currently recruiting…

This phase 2 trial is testing whether adjusting the dose of the drug belinostat based on a person's genetics can make treatment safer and more effective for high-grade neuroendocrine carcinoma. About 60 adults will receive belinostat along with two other chemotherapy drugs, cispl…

This study tests whether giving a personalized dose of the chemotherapy drug fludarabine before CAR T-cell therapy can improve outcomes for children and young adults with relapsed or hard-to-treat B-cell acute lymphoblastic leukemia. About 130 participants will be randomly assign…

This study tests a new drug called TUB-040 for people with ovarian cancer that no longer responds to platinum chemotherapy, or with a type of lung cancer that has come back. TUB-040 is an antibody-drug conjugate, meaning it delivers a powerful chemotherapy directly to cancer cell…

This study tests whether a new experimental drug called PF-08634404, given with chemotherapy, works better than the current standard treatment (pembrolizumab plus chemo) for adults with advanced non-small cell lung cancer. About 1,410 participants will be randomly assigned to rec…

This study investigates whether a mobile app called reSET can help men who have sex with men, are living with HIV, and have a stimulant use disorder reduce their drug use and keep their HIV under control. Participants use the app for 12 weeks, completing modules on topics like ma…

This early study tests baricitinib, a drug already approved for other immune conditions, in 20 people with Job syndrome who also have lupus-like disease or eczema. Participants take the pill daily for 6 months. The main goal is to see if it is safe and tolerable, while also check…

This study tests an experimental drug called SHR-4610 in 258 adults with advanced solid tumors that have stopped responding to standard treatments or have no standard options. The trial has two phases: first, finding the safest dose, and second, checking how well the drug shrinks…

This Phase 3 trial tests whether adding an experimental mRNA vaccine (V940) to standard immunotherapy (Keytruda) can prevent non-small cell lung cancer from coming back after surgery. About 868 people with stage II-IIIB lung cancer who have had their tumors completely removed wil…

This study tests a new drug called PF-08634404, given alone or with another drug (ipilimumab), in adults with advanced liver cancer that cannot be surgically removed. The goal is to check safety, side effects, and whether the drug shrinks tumors. About 138 participants will recei…

This study tests a drug called BPC2001 to prevent acute graft-versus-host disease (aGVHD), a serious complication where donor immune cells attack the patient's body after a stem cell transplant. Fifty adults with blood cancers like leukemia will receive six weekly doses of BPC200…

This study asks whether a dose-dense chemotherapy combination called CMF (cyclophosphamide, methotrexate, and fluorouracil) can be a practical alternative to the standard AC regimen (doxorubicin and cyclophosphamide) for people aged 70 and older with early-stage breast cancer. Th…

This study uses botulinum toxin (Botox) injections to treat movement disorders such as dystonia, tremor, and spasticity. Up to 2,000 participants aged 2 and older will receive tailored injections, with doctors trained in proper techniques. The goal is to manage symptoms and build…

This trial tests whether treating hepatitis C in primary care clinics, with non-specialist doctors, works as well as the standard specialist care in Brazil. It also evaluates the use of rapid tests and self-testing to find more people with the infection. The goal is to see if a s…

This study tests a new experimental drug, PF-08634404, combined with chemotherapy in people with advanced stomach, gastroesophageal junction, or esophageal cancer that has not been treated before. The trial has two parts: first checking safety and response, then comparing the new…

This study tests whether taking oral paclitaxel plus fruquintinib can help people with advanced stomach or gastroesophageal junction cancer after their first treatment stops working. About 150 adults will receive either the new drug combo or standard chemotherapy chosen by their …

This study tests whether combining three drugs—pemigatinib, atezolizumab, and bevacizumab—can help control advanced bile duct cancer that has a specific genetic change (FGFR2 fusion). About 25 adults whose cancer has not responded to other treatments will take part. The main goal…

This Phase 3 trial is testing whether adding an experimental drug called calderasib (MK-1084) to standard immunotherapy (pembrolizumab) can keep non-small cell lung cancer from returning after surgery. The study enrolls 400 people with a specific KRAS G12C mutation whose cancer w…

This study tests a new exercise approach for people with COPD, a lung disease that makes breathing hard. Researchers will compare standard pulmonary rehab with a version that uses a blood flow restriction cuff during low-intensity aerobic exercise. The goal is to see if this meth…

This study tests whether adding the experimental drug obrixtamig to standard chemotherapy and immunotherapy helps people with advanced small cell lung cancer live longer. About 670 adults with extensive-stage disease will be randomly assigned to receive either the new combination…

This study tests a new drug called PF-08052667 for adults with a type of bladder cancer that hasn't spread into the muscle layer but is likely to return. The drug is given directly into the bladder, sometimes with other medicines like BCG or sasanlimab. The trial has three parts …

This study tests a drug called AGA2115 in 80 adults with osteogenesis imperfecta (brittle bone disease) types I, III, or IV. Participants receive either the drug or a placebo as a shot under the skin. The main goal is to see if the drug increases bone density in the spine after 1…

This study tests an experimental drug called AOC 1044 for boys with Duchenne muscular dystrophy (DMD) who have a specific genetic change (exon 44 skipping). About 70 boys aged 7 to 16 who can still walk will receive either the drug or a placebo. The main goal is to see if the dru…

This trial asks whether giving children a low-dose ketamine infusion after heart surgery can protect their brains from damage. Children aged 1 to 60 months undergoing surgery for certain congenital heart defects will receive either ketamine at one of two doses or no ketamine. Res…

This study tests whether a new drug called SUL-238 can improve energy production in brain cells of people with early, untreated Parkinson's disease. Forty-five participants will take either a low or high dose of SUL-238 or a placebo daily for 28 days. Researchers will use a speci…

This phase 3 trial is testing whether adding the targeted drugs calderasib and cetuximab to standard chemotherapy can help people with a specific gene mutation (KRAS G12C) in advanced colorectal cancer live longer without their cancer growing. About 477 participants will receive …

This study tests a combination of up to 4 experimental drugs (retifanlimab, TriAdeno vaccine, N-803, and SX-682) in people with metastatic colorectal cancer that has spread and not responded to prior treatments. The goal is to see if the combination can shrink tumors and improve …

This study tests whether a new treatment called Bria-IMT, given with an immunotherapy drug, helps people with advanced metastatic breast cancer live longer compared to standard chemotherapy. About 404 adults whose cancer has not responded to other treatments will participate. The…

This early-phase trial tests a personalized treatment for people with blood cancers like leukemia. Doctors take a patient's own T cells, modify them in the lab to recognize unique cancer mutations, and infuse them back after a short course of chemotherapy. The goal is to see if t…

This study tests a new medicine called PF-08634404 given with chemotherapy for adults with transformed small cell lung cancer (T-SCLC), a rare and aggressive cancer that develops from another lung cancer type. About 40 participants will receive the drug combo through IV infusions…

This trial tests whether wrapping the nerves around the prostate with a special tissue allograft during surgery can reduce erectile dysfunction and incontinence. 25 men with localized prostate cancer will receive the wrap during nerve-sparing prostate removal. The study focuses o…

This study tests a new drug called YH42946 in people with advanced solid tumors that have certain gene changes (HER2 or EGFR). The main goals are to check safety and see if the drug shrinks tumors. About 161 adults who have run out of standard options will take part.

This study tests whether mobile health technology, like smartphone apps and activity trackers, can help overweight or obese African American women increase their physical activity. Participants will wear a wrist device to track steps and sleep, use a study app, and may receive co…

This trial tests a new drug called TUB-030 in adults with advanced solid tumors, including head and neck, lung, and breast cancers. The goal is to find the safest and most effective dose. Participants receive the drug every three weeks and are monitored for side effects and tumor…

This phase 2 trial is testing whether adding evorpacept to standard trastuzumab and chemotherapy can shrink tumors in people with metastatic HER2-positive breast cancer who have already tried the drug Enhertu. About 120 participants will receive the combination intravenously. The…

This Phase 3 trial is testing whether adding the drug zanzalintinib to belzutifan helps people with advanced kidney cancer live longer and keep the cancer from growing. About 758 participants who have already tried standard treatments will receive either the combination or belzut…

This phase 3 trial tests whether less intense drug combinations can control relapsed or refractory acute myeloid leukemia (AML) as well as high-dose chemotherapy. About 339 adults whose AML has returned or not responded to initial treatment will be randomly assigned to either a l…

This phase 2 trial is testing a type of radiation called craniospinal irradiation that avoids the hippocampus, a brain area key for memory. It is for people with breast or lung cancer that has spread to the fluid and lining around the brain and spinal cord. The goal is to see if …

This early-stage trial is testing a drug called orellanine, derived from a mushroom, in 75 people with advanced kidney cancer who are also on dialysis. The drug is given intravenously to see if it is safe and can shrink tumors. All participants have end-stage kidney disease and h…

This phase II trial is testing whether combining two immunotherapy drugs, nivolumab and ipilimumab, works better than nivolumab alone for women with recurrent endometrial cancer that has a specific DNA repair defect (dMMR). About 81 participants will receive either the combinatio…

This study tests a procedure called transarterial infusion chemotherapy (TAIC) for people with colorectal cancer that cannot be removed by surgery. Instead of giving chemo through a vein, TAIC delivers it directly to the tumor's blood supply, which may boost effectiveness and red…

This study tests whether a new drug, sacituzumab tirumotecan (Sac-TMT), given alone or with pembrolizumab, helps people with advanced triple-negative breast cancer live longer or keep their cancer from growing. About 1,000 adults whose cancer has spread and who have not had prior…

This study tests whether a combination of two drugs (avutometinib and defactinib) works better than standard treatments for people with recurrent low-grade serous ovarian cancer. About 270 participants whose cancer returned after platinum-based therapy will be randomly assigned t…

This study tests whether adding the experimental drug zilovertamab vedotin to standard chemotherapy helps people with diffuse large B-cell lymphoma that has come back or not responded to treatment. About 290 adults will be randomly assigned to receive either the new combination o…

This Phase 2 trial tests the drug asciminib in 50 adults newly diagnosed with chronic myeloid leukemia (CML). The goal is to see if asciminib can control the disease and to check for side effects. Participants take the drug by mouth, and researchers monitor their health closely.

This study tests a new drug called raludotatug deruxtecan (R-DXd) in people whose high-grade serous ovarian, peritoneal, or fallopian tube cancer has returned after platinum chemotherapy. R-DXd is an antibody-drug conjugate that seeks out cancer cells and delivers a toxic payload…

This study tests a combination of two drugs, venetoclax and azacitidine, in about 100 adults in India with newly diagnosed acute myeloid leukemia (AML) who are not healthy enough for standard intensive chemotherapy. The goal is to see if the combo can put the cancer into remissio…

This Phase 3 trial tests whether DYNE-101 can improve muscle function and daily life in 150 adults with myotonic dystrophy type 1 (DM1). Participants receive either the drug or a placebo by IV every few weeks for 48 weeks. The study measures how quickly people can stand from a ch…

This study tests whether adding pivekimab sunirine to the standard drugs venetoclax and azacitidine helps adults with newly diagnosed acute myeloid leukemia (AML) who are not healthy enough for intensive chemotherapy. About 660 participants will be randomly assigned to receive ei…

This trial tests whether a structured six-month lifestyle modification program can improve penile blood flow and erectile function in men with metabolic syndrome-associated erectile dysfunction. Participants receive dietary counseling, exercise guidance, weight management, smokin…

This early-phase study tests a new drug called SV001 in 53 healthy Chinese adults to see if it is safe and how the body processes it. Participants receive a single dose of SV001 or a placebo. The goal is to check for side effects and measure drug levels in the blood. This is a fi…

This trial compares two treatment approaches for men with aggressive, metastatic prostate cancer that has stopped responding to hormone therapy and docetaxel. One approach uses a combination of two chemotherapy drugs (cabazitaxel and carboplatin), while the other uses a radioacti…

This Phase 3 trial is testing a new weekly injection called enicepatide for weight loss in 1600 people who are obese or overweight and also have type 2 diabetes. The drug works by mimicking natural hormones that help control appetite and blood sugar. Participants will receive eit…

This study tests an experimental drug called DONQ52 in people with active celiac disease who still have gut damage and symptoms even after following a gluten-free diet for at least a year. About 92 adults will receive either the drug or a placebo to see if it can heal the small i…

This study tests an experimental drug called telisotuzumab adizutecan combined with standard chemotherapy in people whose colorectal cancer has spread. The goal is to see if the combination shrinks tumors and is safe. About 390 adults will take part at hospitals worldwide.

This phase 2 study tests an investigational drug called telisotuzumab adizutecan in about 100 people aged 12 and older with advanced solid tumors that have a specific genetic change (MET amplification). The drug is given through an IV and aims to shrink tumors or stop them from g…

This early-stage study is testing an experimental drug called pivekimab sunirine in about 18 children whose acute myeloid leukemia (AML) has come back or not responded to standard treatments. The main goals are to check the drug's safety and how it moves through the body. Partici…

This study tests whether psilocybin, a psychedelic substance, given with psychological support is safe and helps reduce symptoms of anorexia nervosa in young adults aged 16 to 35. Participants receive two doses of psilocybin (25 mg) four weeks apart, plus therapy before, during, …

This phase 3 trial tests whether a new drug, sacituzumab tirumotecan, given with or without bevacizumab, can help people with platinum-sensitive recurrent ovarian cancer live longer without their cancer worsening. About 770 participants who have already completed two rounds of ch…

This study tests whether combining two drugs, atezolizumab and selinexor, can shrink tumors in people with a rare cancer called alveolar soft part sarcoma (ASPS). About 27 adults with advanced or spreading ASPS will receive the combination. The goal is to see if this approach wor…

This trial is testing whether a lower weekly dose of the corticosteroid prednisolone (5 mg/kg per week) can be as effective as the standard higher dose (10 mg/kg per week) in preserving motor function in young boys with Duchenne muscular dystrophy (DMD). The study will enroll boy…

This study tests whether adjusting common over-the-counter medications can make immune checkpoint inhibitors work better for people with solid tumors. Participants are asked to avoid acetaminophen and cannabis/THC/CBD, while taking aspirin and loratadine (or similar antihistamine…

This study tests a gene therapy called ABBV-RGX-314 for people with wet age-related macular degeneration (AMD), a leading cause of vision loss. The goal is to see if a single treatment can reduce the need for frequent eye injections while keeping vision stable. About 561 adults a…

This study tests a new drug called AZD7760 in healthy volunteers and people with end-stage kidney disease who are on dialysis. The goal is to see if the drug is safe and how the body processes it. If it works, it could help prevent serious staph infections that often occur in dia…

This study tests whether adding the drug pembrolizumab to a treatment that uses a patient's own immune cells (called TIL therapy) can shrink tumors in people with advanced melanoma. About 53 adults aged 18 to 72 who have already tried at least one other treatment will take part. …

This trial tests whether giving patients special immune cells, called TAA-T, after a stem cell transplant can help prevent high-risk blood cancers like acute myeloid leukemia from coming back. The cells are designed to recognize three common tumor markers and attack any remaining…

This study compares two treatments for people with high-stage, estrogen-positive, HER2-negative breast cancer that has a low genetic risk of returning. One group gets standard hormone therapy plus a targeted pill (ribociclib), while the other gets chemotherapy first, then the sam…

This clinical trial is testing several combinations of immunotherapy drugs to see if they can shrink or control advanced liver cancer. The study is for people with a type of liver cancer called hepatocellular carcinoma (HCC) that has spread or cannot be removed by surgery. Partic…

This study tests a new drug called CR-001 in adults with advanced solid tumors that have spread. The goal is to find the safest dose and see if it can shrink tumors. About 290 people will take part in this early-phase trial.

This phase 3 trial tests whether a new drug (MK-2870), alone or with another drug (pembrolizumab), works better than standard treatments for people with a common type of advanced breast cancer (HR+/HER2-). About 1,200 participants whose cancer has worsened after hormone therapy w…

This early-phase trial is testing an experimental oral drug called GIM-531, alone or with an immunotherapy (anti-PD-1), in 117 adults with advanced solid tumors that have stopped responding to standard treatments. The goal is to see if the drug is safe, tolerable, and can help th…

This study tests whether giving a second dose of an RSV vaccine to pregnant women who already received it in a prior pregnancy is safe and boosts immunity. About 550 women aged 18-49, between 24-36 weeks pregnant, will receive either the vaccine or a placebo. Researchers will tra…

This study tests a new drug called elranatamab, alone or with another drug (daratumumab), against a standard three-drug combo for people with multiple myeloma that came back or didn't respond after at least one prior treatment. About 944 participants will be randomly assigned to …

This study tests whether adding the experimental drug mevrometostat to the standard treatment enzalutamide can slow down cancer growth better than enzalutamide alone. It involves 1000 men with metastatic prostate cancer that still responds to hormone therapy. Participants will be…

This early-phase trial tests a new way to give radiation to people whose glioblastoma (a severe brain cancer) has come back after initial treatment. About 28 adults will receive focused radiation over 1 to 3 weeks, with different schedules to find the safest dose. The goal is to …

This study follows 200 adults in Japan with moderate to severe ulcerative colitis who are already prescribed risankizumab by their doctor. Researchers will track how well the drug controls symptoms like diarrhea and rectal bleeding over up to 156 weeks. The goal is to see if the …

This early-phase study tests a new drug called RO7851624 in 160 people with relapsed/refractory multiple myeloma who have tried several standard treatments. The main goal is to check the drug's safety and how the body processes it. Researchers will also look for signs that the dr…

This study tests an experimental immunotherapy called MK-1045 in 200 people with two types of non-Hodgkin lymphoma: follicular lymphoma (slow-growing) and diffuse large B-cell lymphoma (fast-growing). Participants have already tried at least two other treatments without success. …

This study tests an experimental pill called enpatoran for people with lupus who have active skin rashes. About 202 participants will receive either the drug or a placebo for 24 weeks, while continuing their usual treatments. The goal is to see if enpatoran can significantly redu…

This study tests a new drug, patritumab deruxtecan, against standard chemotherapy or another drug for people with a common type of advanced breast cancer (HR+/HER2-). The goal is to see if the new drug helps people live longer or keeps the cancer from growing. About 1000 particip…

This early-stage trial tests an investigational drug called ABBV-438 in about 127 adults with multiple myeloma that has come back or stopped responding to treatment. The study has two parts: first, finding a safe dose, then testing that dose in more people. The main goal is to ch…

This phase 3 trial tests whether a combination of two antibodies (teropavimab and zinlirvimab) plus the drug lenacapavir, given as injections twice a year, can keep HIV under control in adults whose virus is already well-suppressed by daily oral medication. The study compares thi…

This trial is testing an experimental once-daily pill called ASC30 in adults who are overweight or have obesity and also have type 2 diabetes. The study aims to see if ASC30 can help with long-term weight loss and improve blood sugar control compared to a placebo. Participants wi…

This early-phase study is testing a new oral drug called BG-75202, alone or with other medications, in people with advanced breast cancer or other solid tumors that have not responded to standard treatments. The main goals are to check the drug's safety, find the right dose, and …

This study compares a new immunotherapy, MK-1045, to the standard drug blinatumomab in people whose B-cell acute lymphoblastic leukemia has returned or stopped responding to treatment. About 340 adults with CD19-positive leukemia will receive one of the two treatments. The goal i…

This Phase 3 trial is testing an experimental once-daily oral medication called ASC30 in adults with obesity or overweight who also have weight-related health conditions like high blood pressure or sleep apnea. The study aims to see if ASC30 can lead to significant weight loss ov…

This phase III trial tests whether giving an extra round of chemotherapy and immunotherapy (pembrolizumab) before the usual chemoradiation can help people with high-risk, locally advanced cervical cancer live longer without the cancer growing. About 336 participants will be rando…

This study tests a new drug called ABBV-706 combined with the immunotherapy atezolizumab as a first treatment for people with extensive-stage small cell lung cancer, a fast-growing cancer that has spread. About 180 adults will receive either the new combination at one of two dose…

This trial compares two ways of closing the abdomen after open colorectal surgery: a device-assisted method (SutureTOOL) versus the traditional manual technique. The goal is to see if the device helps reduce the chance of an incisional hernia forming within one year. Participants…

This study enrolls 950 men with suspected but undiagnosed prostate cancer who are scheduled for a biopsy. Researchers will compare the accuracy of prostate MRI against a panel of blood and urine biomarkers (PCA3, PSA, TMPRSS2:ERG) in detecting clinically significant prostate canc…

This phase 3 trial is testing a new radioactive imaging agent called 68Ga-NYM032, given as an injection before a PET/CT scan, in 365 men newly diagnosed with prostate cancer. The goal is to see if this scan can accurately detect whether the cancer has spread to pelvic lymph nodes…

This study is testing whether a blood test that measures a substance called miRNA-371 can accurately detect active testicular cancer in people who have already had their primary tumor removed. Researchers will compare the blood test results with surgical findings to see how well …

This study is working on a new, non-invasive test to help rule out uterine cancer in women aged 45 and older who have abnormal bleeding. Researchers will collect samples from 4,500 women to see how well the test detects cancer and identifies benign cases. If successful, it could …

This study looks at whether a simple urine test for tumor DNA can tell which bladder cancer patients truly need a second surgery after their first tumor removal. About 196 adults with non-muscle-invasive bladder cancer will be randomly assigned to have their repeat surgery decisi…

This large study is testing whether a combined screening approach—using a blood test for DNA markers plus stool tests for hidden blood and inflammation—can catch gastrointestinal cancers earlier and save lives. Researchers will randomly assign 10,000 adults aged 45–74 to either r…

This study collects video recordings and data from 500 patients undergoing blue light cystoscopy, a procedure where a drug makes cancer cells glow. The goal is to train an artificial intelligence (AI) algorithm to detect bladder lesions in real time, helping doctors make more acc…

This trial is testing an experimental imaging agent called 89Zr-olaratumab in people with soft tissue sarcoma or other solid tumors that express a protein called PDGFRα. The agent is a radioactive-labeled antibody designed to bind to PDGFRα and show up on PET scans, potentially r…

This study enrolls 400 women at high risk for breast cancer to see if a shorter, abbreviated MRI scan works as well as a full-length MRI for early detection. Participants will get MRI scans every 6 months. The goal is to develop faster, personalized screening that could make regu…

This trial tests whether rinsing the bladder with sterile water during surgery works as well as a chemotherapy drug (gemcitabine) placed directly into the bladder to prevent cancer from coming back. People with upper tract urothelial carcinoma who are having surgery to remove the…

This pilot study tests whether giving a malaria drug (dihydroartemisinin piperaquine) to household members of pregnant women can help prevent malaria in the mothers. About 300 participants in Uganda will receive either a single dose or monthly doses. The goal is to see if this ap…

This study tests whether taking progesterone early in pregnancy can prevent preeclampsia, a condition that causes high blood pressure and organ damage. Over 600 low-risk pregnant women will either receive nightly vaginal progesterone or standard care. Researchers will track who d…

This trial tests three ways to screen adolescents and young adults for HIV in emergency departments: targeted screening based on risk, universal opt-in screening offered to everyone, and universal opt-out screening where testing is done unless declined. The goal is to see which a…

This study tests a community-based approach to make cervical cancer screening easier for women who haven't had a Pap test in over 3.5 years. It involves 920 women in low-resource areas of Texas. The goal is to see if this strategy is safe and works well.

This study tests whether mobile health (m-Health) patient navigation—using phone apps for education, reminders, and motivation—can help more women in Kenyan primary care clinics get screened for breast cancer. The trial involves 800 women and aims to improve access to mammography…

This study observes about 700 people with lung or liver cancer who are already receiving atezolizumab as a shot under the skin. Researchers want to see how satisfied patients are with this treatment and how it affects their quality of life. The study does not test a new drug but …

This study is testing a program called FOCUS ON US, designed to help sexual and gender minority (LGBTQ+) cancer patients and their caregivers. The program includes five sessions covering topics like family involvement, coping skills, and symptom management. Researchers want to se…

This trial tests whether adding acupoint application—a herbal plaster placed on specific body points—to a standard anti-nausea drug can better control nausea and vomiting caused by chemotherapy in adults with lung cancer. Participants receiving first-line platinum-based chemother…

This phase II trial tests whether adding BMX-001 to standard symptom management can reduce severe mouth sores in head and neck cancer patients receiving chemoradiation. About 98 participants will receive either BMX-001 or a placebo alongside usual care. The goal is to see if BMX-…

This study looks at possible eye-related side effects in people with cervical cancer that has returned or spread, who are being treated with the drug TIVDAK. About 100 participants will receive TIVDAK every 3 weeks and undergo regular eye exams before, during, and after treatment…

This study tests whether using a special imaging technique called Red Dichromatic Imaging (RDI) throughout the entire ESD procedure can reduce serious complications like bleeding and perforation, and shorten surgery time. About 640 adults with early stomach cancer or precancerous…

This trial tests whether two physical therapies—Kinesio Taping and compression therapy—can relieve breast lymphedema, a common side effect of breast-conserving surgery and radiation that causes swelling, pain, and heaviness. About 99 women who have completed radiation and have ul…

This study tests a program called Grow with Grit, where school nurses screen children aged 8-12 in rural Montana for anxiety using a standard questionnaire. Children who show signs of anxiety receive a brief, 3-5 minute coping-skills session during a regular nurse visit, along wi…

This study tests whether a nebulizer or a standard inhaler delivers albuterol better to improve breathing in women with LAM, a rare lung disease. About 100 women with reduced lung function will stay overnight for 3 days to try each method. The goal is to see which approach opens …

This study tests whether a short psychotherapy called brief behavioral activation (BBA) can reduce depression and anxiety and improve well-being in older adults who have survived cancer. BBA encourages people to set goals and engage in enjoyable or rewarding activities to change …

This Phase 3 study tests a once-weekly injection called NNC0487-0111 in 400 adults who are overweight or obese and have knee osteoarthritis. The goal is to see if it helps with weight loss and reduces knee pain compared to a placebo. Participants receive either the active drug or…

This study tests a drug called fezolinetant to see if it can reduce hot flashes in men with prostate cancer who are on hormone therapy (androgen deprivation therapy). About 60 men will take either the drug or a placebo daily for 4 weeks. Researchers will track hot flash frequency…

This pilot study tests whether VM100, a nutritional supplement made from ten natural ingredients, can improve quality of life for people with Duchenne muscular dystrophy (DMD). About 20 participants aged 8 and older will take VM100 or a placebo for 10 weeks. Researchers will meas…

This trial tests whether a depigmenting gel cream called Blancy Pro can reduce facial hyperpigmentation, including dark spots and melasma. Forty women aged 30 to 60 will apply the product daily for 90 days. Researchers will measure skin lightening using a colorimeter and clinical…

This study compares a shorter muscle cut (4-5 cm) during a procedure called POEM to the standard longer cut (8-10 cm) for people with certain swallowing disorders. The goal is to see if the shorter cut works just as well at relieving symptoms like trouble swallowing and chest pai…

This study tests whether two different exercise programs—one combining resistance training with cardio, the other with conditioning—have similar effects on muscle mass, fat mass, and blood pressure in women who have completed breast cancer treatment. Participants will exercise th…

This study tests whether alteplase, a drug that dissolves blood clots, can improve drainage of fluid around the lungs and reduce shortness of breath in people with cancer-related pleural effusion that is not draining well. About 30 participants will receive either alteplase or a …

This study looks at whether acupuncture can help young women (ages 15-40) with cancer get their periods back after chemotherapy caused them to stop. About 60 participants will receive acupuncture or usual care. Researchers will measure how many people join and finish the study, a…

This Phase 3 trial tests whether fenfluramine (Fintepla) can reduce behavioral and emotional symptoms in people with Rett syndrome. About 200 participants will receive either the drug or a placebo for 14 weeks. The study measures changes in behavior, sleep, and overall condition …

This study tests a mobile app called PACT that helps people with advanced cancer talk about and plan the care they would want if they couldn't speak for themselves. The app involves patients and their loved ones in these discussions. Researchers want to see if the app is easy to …

This study tests whether custom 3D-printed external breast prostheses improve comfort, fit, and emotional well-being for women who have had a mastectomy without reconstruction. Fifty participants will receive a personalized prosthesis made from 3D scans of their chest. The goal i…

This study explores whether a brief behavioral program can help cancer patients and their sleep partners get better rest. The program teaches techniques like limiting time in bed and going to bed only when sleepy, aiming to improve sleep efficiency. Participants, who are couples …

This study tests whether adding electroacupuncture and ear acupoint pressing beans to a standard opioid painkiller can better relieve severe cancer pain that doesn't respond to usual treatments. About 30 adults with refractory cancer pain will receive the combination for two week…

This study tests whether a non-invasive earbud that gently stimulates a nerve in the ear can help adult survivors of childhood leukemia who have trouble sleeping. Forty participants will be randomly assigned to receive either active or sham stimulation nightly for 2 to 8 weeks. T…

This study tests a non-invasive device called transcutaneous electrical acustimulation (TEA) in 190 elderly patients (65+) recovering from colorectal cancer surgery. The device delivers mild electrical pulses to the skin to stimulate bowel function. Participants receive TEA or a …

This study tests whether Acceptance and Commitment Therapy (ACT), delivered through a smartphone app, can help caregivers of children with RASopathies (like Neurofibromatosis type 1 and Noonan syndrome) cope with parenting stress. The trial is fully remote and involves 70 adult c…

This pilot trial explores whether a couple-based mindfulness program, called Mindful PaCT, is practical and helpful for people with metastatic colorectal cancer and their partners. The program involves four weekly video sessions that teach mindfulness skills like awareness, accep…

This pilot study will test whether a mobile phone app can help reduce depression symptoms in African American adults. Eight participants will attend six weekly 90-minute classes through the app and complete surveys over four months. The goal is to see if this approach is effectiv…

This trial tests two ways to bring nature to older adults stuck in the hospital for weeks: watching 360-degree nature videos through a virtual reality headset, or listening to audio guides that prompt them to imagine the same scenes. Both approaches use identical nature themes—me…

This study tests an investigational drug called CSL889 in 70 adults and adolescents with sickle cell disease who are having a painful vaso-occlusive crisis. The goal is to see if the drug can safely shorten the crisis and reduce the need for strong pain medicines. Participants wi…

This pilot trial tests whether a supervised exercise program is safe, helpful, and practical for adults who have had breast or gynecologic cancer. Participants will take part in a 12-week program combining strength and aerobic training under trained guidance. The study focuses on…

This study observes how doctors decide the length of the muscle cut during POEM surgery for people with swallowing disorders like achalasia. Researchers will follow 200 adults for 2 years, tracking symptoms and whether retreatment is needed. The goal is to see if tailoring the su…

This study looks at why some people with HIV develop Immune Reconstitution Inflammatory Syndrome (IRIS) after starting HIV medications. Researchers will use PET scans and lymph node biopsies to track inflammation and immune recovery in 300 adults. The goal is to better understand…

This study follows 500 people with neurocysticercosis, a brain infection caused by the pork tapeworm. Participants receive standard antiparasitic drugs and anti-inflammatories. Researchers will track their health over many years to understand what factors lead to better or worse …

This study follows 500 people with geographic atrophy, an advanced form of age-related macular degeneration that causes permanent vision loss. Participants receive avacincaptad pegol (IZERVAY) as part of their routine care. Researchers will collect data on treatment patterns, sid…

This natural history study follows up to 4,000 people with inherited bone marrow failure syndromes (IBMFS) and their families to learn why they are prone to certain cancers. Researchers will track health over time, collect genetic samples, and look for clues that separate those w…

This study investigates whether measuring whole-body metabolism—how the body uses energy—can reflect the metabolic changes in ovarian tumors and predict how well patients respond to chemotherapy. Researchers will measure resting metabolic rate and other metabolic markers in women…

This study is building a large collection of blood, saliva, urine, stool, and tissue samples from people who are at increased risk of cancer, including those with genetic predispositions, survivors of childhood or adult cancer, or those with precancerous conditions. The goal is t…

This study is collecting long-term safety and effectiveness data on treatments for various blood disorders, including hemophilia, sickle cell disease, and clotting disorders. It aims to enroll 3000 people of any age with these conditions. Researchers will monitor side effects, co…

This study aims to understand what happens in the brain when someone thinks about or attempts suicide. Researchers will use brain scans, sleep monitoring, blood tests, and questionnaires to compare four groups: people with recent suicidal thoughts or attempts, those with past sui…

This study aims to better understand trisomy 8 mosaicism, a rare genetic condition where some cells have an extra copy of chromosome 8, and its link to autoinflammatory disease (TRIAD). Researchers will follow affected individuals and their healthy relatives over time, collecting…

This study is creating the first large database for Niemann-Pick type C (NPC), a rare disease that damages the brain, liver, and spleen. Researchers will collect blood samples and medical records from 100 people with NPC to link their genes with their symptoms. The goal is to und…

This study aims to create a large collection of tissue samples from people with head and neck disorders, such as hearing loss, mouth diseases, and throat cancers. Researchers will use leftover surgical tissue and additional samples like blood, saliva, and cheek swabs to study the…

This study aims to describe the physical and behavioral traits of overweight and obese adults compared to lean individuals. Researchers will measure hormones, metabolism, food preferences, fitness, sleep, and thinking patterns in 2,000 participants. The goal is to better understa…

This study aims to learn more about the genetics and natural history of autoinflammatory diseases, which cause repeated fevers and inflammation. Researchers will study up to 5,000 people, including patients, their relatives, and healthy volunteers. Participants provide blood and …

This study looks at a rare inherited condition called Birt-Hogg-Dube (BHD) syndrome, which raises the risk of kidney cancer. Researchers will collect blood, saliva, and tissue samples from up to 950 participants to find the genes involved and understand how kidney tumors grow. Th…

This study looks at how the brains of children with anxiety disorders work differently from healthy children. Researchers will use brain scans and thinking tasks to track changes over a year, before and after standard treatments like therapy or medication. The goal is to find pat…

This large observational study in Mali is following up to 15,000 pregnant women and children to understand why some get malaria and others don't. Researchers are looking at immune responses, parasite features, and other factors that influence infection and disease severity. The g…

This study tests a redesigned surgical tool for removing bladder tumors. Researchers will use the device on bladder specimens from 25 patients who need their bladder removed. The goal is to see if the modified device provides better tissue samples for pathology, helping doctors a…

This study aims to understand how Staphylococcus aureus bacteria interact with human cells to cause infections, especially in the spine. Researchers will use tonsil tissue discarded after routine tonsillectomies to create lab models. About 500 participants aged 2 and older who ar…

This study aims to improve how doctors evaluate and manage people who have been exposed to bioterrorism agents (like anthrax) or emerging infectious diseases (like SARS or new flu strains). Up to 200 participants, including patients and healthcare workers, will be monitored and t…

This study looks at the tiny organisms living on our skin—like bacteria and fungi—and how they might differ in people with eczema (atopic dermatitis). Researchers will collect skin samples from healthy volunteers and patients aged 2 to 40 with moderate to severe eczema, as well a…

This study aims to find the genetic causes of severe combined immunodeficiency (SCID), a rare condition where the immune system doesn't work properly. Researchers will collect blood samples from 100 patients and their relatives to analyze DNA and other factors. The goal is to bet…

This study collects white blood cells from HIV patients through a process called leukapheresis to examine how the immune system responds to early treatment. Researchers aim to enroll 60 adults—30 with early HIV and 30 with chronic HIV—who are not yet on antiretroviral therapy. Pa…

This study uses special PET scans to measure inflammation in the brains of people with Alzheimer's, Parkinson's, ALS, and other neurodegenerative diseases. Researchers will compare these scans to those from healthy volunteers to see if inflammation levels differ. The goal is to d…

This study follows people of all ages who have or may have a RUNX1 gene variant, which can cause bleeding problems and increase the risk of blood cancers. Researchers will collect medical history, blood samples, and bone marrow over many years to understand how the condition deve…

This study monitors 202 adults in South Korea with advanced bladder cancer who are receiving the drug PADCEV (enfortumab vedotin) as part of their normal care. Researchers will track side effects and how well the cancer responds for up to a year. No new treatment is given; the go…

This natural history study is observing up to 250 people with Smith-Lemli-Opitz syndrome and related cholesterol disorders, as well as their relatives. Researchers will track symptoms, development, and lab results over several years to find better ways to measure disease progress…

This study will follow 1,000 people receiving Medtronic spinal or cranial devices during routine surgery. Researchers will track outcomes like fusion success, pain relief, and device safety for up to two years. The goal is to gather real-world data on how these already-approved p…

This study aims to understand the genetic causes of brain development disorders, including Aicardi syndrome, polymicrogyria, and Dandy-Walker malformation. Researchers will analyze genetic data from 2000 participants with these conditions, all confirmed by MRI. The goal is to use…

This study aims to improve how doctors find and understand pheochromocytomas and paragangliomas, rare tumors that can cause dangerous high blood pressure. Researchers will use advanced imaging scans and genetic testing in up to 3,000 participants to learn more about these tumors.…

This study aims to understand how and why eosinophils (a type of white blood cell) become active in conditions like allergies, asthma, and parasitic infections. Researchers will observe up to 800 people aged 1 to 100 with high eosinophil levels. No experimental treatments are giv…

This study creates a registry for children, teens, and young adults (ages 1 month to 39 years) diagnosed with very rare solid tumors (fewer than 2 cases per million people per year). Researchers will collect medical records, tumor samples, and patient questionnaires to better und…

This study at the National Institutes of Health looks at how infections can worsen symptoms in people with mitochondrial disease, a group of disorders that affect energy production in cells. Researchers will evaluate participants' immune systems through blood tests, physical exam…

This study looks at whether certain genes make some people more likely to develop eye damage from the drug Plaquenil, which is used for autoimmune diseases like lupus and rheumatoid arthritis. Researchers will compare people who have had eye damage from Plaquenil with those who h…

This study follows 300 children and adults with chordoma, a rare bone cancer of the skull base or spine. Researchers will track how the disease progresses over time using imaging, physical exams, and surveys. The goal is to better understand chordoma's behavior to design more eff…

This study collects tissue and blood samples from adults with HIV, other immune disorders, or cancers thought to be caused by viruses. Researchers will compare genes in cancer tissue to normal tissue to find genetic changes that drive these cancers. The goal is to better understa…

This study is following children and adults with olfactory neuroblastoma, a rare cancer of the nasal cavity, to learn more about how the disease behaves over time. Researchers will collect medical records, blood samples, and survey responses from 525 participants to track symptom…

This study collects skin, hair, blood, urine, or saliva samples from people with certain eye diseases (like macular degeneration) and from healthy volunteers. Researchers will turn these samples into stem cells in the lab to study how the diseases develop and to test potential ne…

This study looks at how people's immune systems react to infections from parasitic worms, which can cause serious health problems like swelling, blindness, or heart disease. Researchers will evaluate and treat up to 500 participants aged 3 to 100 using standard medical tests and …

This study looks at people with unusual or repeated infections and their family members to find the genetic and biochemical causes of immune system problems. Researchers will collect blood, saliva, urine, and other samples over time to better understand these conditions. The goal…

This study aims to better understand why some people experience dangerously low blood sugar (hypoglycemia), especially after fasting. Researchers will admit 1,500 adults to the NIH Clinical Center for controlled fasting and a series of blood tests to measure insulin and other fac…

This study is for people with cervical or pancreatic cancer who are scheduled to receive chemoradiotherapy. Researchers will collect tumor samples, blood, and imaging data before, during, and after treatment to see how the tumor environment changes over time. The goal is to learn…

This study aims to understand the causes and progression of Common Variable Immunodeficiency (CVID) and related inborn errors of immunity. Researchers will observe up to 500 patients over time using blood tests, imaging, and other exams. The goal is to find better ways to diagnos…

This study aims to learn more about childhood-onset behavioral, psychiatric, and developmental disorders by observing people of all ages who have these conditions, along with their family members. Researchers will collect medical histories, perform psychiatric assessments, and ma…

This study follows 2,800 unpaid caregivers of people with chronic conditions like diabetes or inherited disorders. Researchers will survey and interview caregivers over up to 5 years to track changes in their health, stress, and social support. The goal is to understand how careg…

This study enrolls adults with cancer or benign tumors who are already participating in other trials at the National Cancer Institute. Researchers will collect and link medical records and research samples from all of a patient's past, current, and future studies. The goal is to …

This study aims to uncover the genetic causes of pituitary and hypothalamic tumors in children. Researchers will collect blood and tumor samples from up to 2,000 participants to analyze DNA and look for inherited patterns. The study also trains doctors and tests new MRI technique…

This study tests two ways of preparing people with advanced thyroid cancer for a special PET scan that uses a radioactive form of iodine (I-124). The goal is to see which preparation method gives better information for calculating the right dose of radiation therapy. Thirty parti…

This study tests a new way to screen patients with acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS) by analyzing their bone marrow and blood for specific biomarkers. The goal is to quickly match patients with a treatment trial that targets their cancer's unique mark…

This study is a disease registry that will follow about 700 people with advanced non-small cell lung cancer that has a specific genetic change called METex14 skipping. Researchers will collect information on patient characteristics, treatments, and outcomes over time to better un…

This study is testing whether wearable devices like smartwatches can accurately track movement problems in people with Huntington's disease. About 100 participants, including those with Huntington's and healthy volunteers, will wear sensors and complete surveys and interviews at …

This study collects tissue and fluid samples from up to 5,000 cancer patients to help researchers learn more about cancer and develop new tests and treatments. Samples are taken from routine procedures or from extra biopsies done just for research. The goal is to build a resource…

This study looks at whether a structured recovery program called Enhanced Recovery After Surgery (ERAS) can improve outcomes for people undergoing endoscopic surgery to remove pituitary tumors. Researchers will track complications, pain, nausea, hospital stay length, and readmiss…

This study looks at proteins in lung fluid from 750 people with pneumonia to find biomarkers that could reveal what's causing the lung inflammation. Participants undergo a standard bronchoscopy with lung washings, plus a blood draw. The goal is to better understand different type…

This study is building a global patient registry to collect real-world information from people diagnosed with appendix cancer or pseudomyxoma peritonei (PMP), a rare condition where tumors spread in the abdomen. Participants and caregivers share details about their symptoms, trea…

This study aims to understand the genetic and biological causes of Proteus syndrome, a rare condition causing abnormal growth of bones, skin, and other tissues. Researchers will follow up to 1,500 participants over time, using medical exams, imaging, and blood tests to track how …

This study tests a new approach called RIGHTimeCPV, designed to help children with poor-prognosis cancer and their families talk more openly with doctors about the future. Participants choose a communication companion and attend a special visit to share their preferences. The goa…

This study aims to learn more about heart and blood vessel diseases by observing people who have them or are at risk. Researchers will collect samples and perform tests like imaging and stress tests on up to 5,000 participants, including healthy volunteers and relatives of affect…

This study looks at people who have had unusually severe or long-lasting viral infections, like serious cases of herpes, HPV, or the flu. Researchers want to find out if these people have hidden immune system or genetic problems that make them more vulnerable. Up to 600 participa…

This study follows up to 500 people with polyostotic fibrous dysplasia or McCune-Albright syndrome to learn how these conditions change over time. Researchers will collect medical data and tissue samples to better understand the disease. The goal is to improve future diagnosis an…

This study looks at whether a special MRI scan (mpMRI) can better monitor prostate cancer in men who choose active surveillance instead of immediate treatment. About 500 men with low-risk prostate cancer will get regular MRIs, biopsies, and blood tests over several years. The goa…

This study collects blood or cheek swab samples from 1,100 cancer patients at the National Cancer Institute to analyze genetic differences that may influence how their bodies process cancer treatments. The goal is to understand why some patients experience more side effects or le…

This study looks at people who have a gene variant (called a secondary finding) that was found during genetic testing for another reason. Researchers want to understand how these variants affect health, how people respond to the news, and whether they follow medical advice. Up to…

This study enrolls people with endocrine tumors (such as thyroid, parathyroid, adrenal, or pancreatic tumors) who are scheduled for surgery or biopsy. Researchers collect samples of the tumor, nearby healthy tissue, blood, and urine to analyze genetic and molecular changes. The g…

This study looks at how common certain antibodies and inflammatory markers are in people with chronic granulomatous disease (CGD). Researchers will measure levels of adenovirus-fighting antibodies and inflammatory cytokines in blood samples from 60 adults and children with CGD. T…

This study collects biological samples like blood, saliva, and urine from 300 adults aged 18-70, both with and without blood disorders such as sickle cell disease and thalassemia. The goal is to use these samples for research to better understand these conditions and related dise…

This study gathers fluid and tissue from the lungs and nose of healthy volunteers and people with a history of lung infections. Researchers will compare samples to find differences in immune function that might explain why some people are more prone to infections. Participants un…

This study aims to collect blood, nose, and saliva samples from up to 20,000 people aged 3 and older who have or may have a respiratory virus infection like flu or COVID-19. The samples will be stored and used in future research to better understand these viruses and how the immu…

This study aims to learn more about liver diseases like hepatitis B, C, and D by observing up to 8,050 participants over time. Participants will have yearly check-ups, blood and urine tests, and liver ultrasounds. Some may be offered experimental treatments if they qualify. The g…

This study follows 400 people aged 6 and older who have a rare stomach cancer called GIST. Researchers will collect tumor tissue during surgery and study it in the lab to learn more about the disease. Participants are monitored every 6 to 12 months for up to 10 years before surge…

This study aims to find genetic causes of rare diseases in children aged 2 to 18 who live outside the United States and have limited access to genetic testing. Researchers will collect blood, saliva, or cheek swab samples from affected children and their family members. The goal …

This study is testing whether PET scans and blood tests can predict memory and thinking problems that often occur after radiation treatment for high-grade glioma. Sixteen participants will have scans and cognitive tests before, during, and after radiation. The goal is to see if t…

This study aims to understand how genetics and environmental factors contribute to melanoma, a serious skin cancer. Researchers will enroll up to 3,000 people with a personal or family history of melanoma or related conditions. Participants will fill out questionnaires, provide b…

This study follows 600 people with GATA2 deficiency, a genetic disorder affecting the immune system and other body systems. Researchers aim to understand the full range of health problems it causes and why it affects people differently. Participants will have yearly check-ups for…

This study aims to learn more about myositis, a group of diseases that cause muscle inflammation and damage. Researchers will observe up to 1,200 children and adults with myositis, along with healthy volunteers, to track changes in the immune system and other health problems over…

This study interviews 40 young adults aged 18-30 who use both tobacco (cigarettes or vaping) and cannabis at least weekly. Researchers want to learn how and why people start, increase, and keep using both substances together. The goal is to better understand these behaviors, not …

This study looks at anxiety related to cancer screenings in adults with Fanconi anemia, a rare condition that raises cancer risk. Researchers will observe 20 participants during their regular screening visits, noting body language, worries, and the clinic environment. No treatmen…

This study aims to understand why some people are more prone to fungal infections, especially from Candida yeast. Researchers will collect blood, saliva, urine, and tissue samples from people with immune disorders, their family members, and healthy volunteers. No treatment is giv…

This study collects blood, urine, saliva, and tissue samples from nearly 6,000 people who have or may have cancer, including rare inherited forms. The samples are stored and used for research to understand the genetic and molecular basis of cancer. No new treatment is being teste…

This study follows up to 30 children (up to age 16) with a rare genetic condition called methylmalonic acidemia (MMA), which affects how the body processes food. The goal is to observe how the disease progresses over time by tracking specific blood tests and health signs. This in…

This study follows up to 3,500 people with sickle cell disease to learn more about how the condition progresses and what complications arise. Participants receive their usual medical care outside the hospital and visit the clinic for check-ups and research tests. No experimental …

This study aims to understand what causes primary sclerosing cholangitis (PSC), a rare chronic liver disease. About 143 adults with PSC will provide blood, saliva, urine, stool, and tissue samples, plus undergo imaging and procedures like colonoscopy and liver biopsy. No treatmen…

This registry study follows pregnant individuals aged 10 to 25 who received PENBRAYA, a vaccine against meningococcal disease, during pregnancy or within 30 days before their last period. The goal is to collect information on pregnancy outcomes and infant health during the first …

This pilot study tests whether a special type of PET/MRI scan can monitor how triple-negative breast cancer responds to immunotherapy. Twenty patients will get three scans over their first two treatment cycles. The results won't change their care but could help design larger stud…

This study uses detailed MRI scans and targeted biopsies to investigate why some prostate cancers come back after radiation therapy. Researchers will follow 300 men who are scheduled for radiation or whose cancer has returned after earlier treatment. The goal is to better underst…

This study collects blood, bone marrow, and tumor samples from 500 adults with cancer or pre-cancerous conditions. Researchers will use these samples to learn more about how cancers behave and why some respond differently to treatments. Participants provide samples over time and …

This study collects blood stem cells and immune cells from healthy volunteers and patients with primary immune deficiencies or blood disorders. The cells are used in the lab to develop new gene and cell therapies. Up to 850 adults aged 18-70 will participate. The goal is to advan…

This study tests whether regular blood and bone marrow tests using advanced DNA sequencing can better monitor children with B-cell acute lymphoblastic leukemia (B-ALL) after they achieve remission with CAR T therapy. The goal is to see if these tests can identify who is truly cur…

This study looks at how obesity and body fat affect heart disease risk in 2,000 healthy African American men and women. Researchers will measure body fat, blood fats, and insulin function to see if risk factors differ by sex. The goal is to understand why heart disease rates are …

This study aims to uncover genetic changes that cause autoimmune diseases in people native to Peru. Researchers will collect blood, urine, and stool samples from 300 participants, including patients with conditions like lupus and rheumatoid arthritis, their family members, and he…

This study collects blood, urine, bone marrow, and tumor samples from people with hairy cell leukemia and other blood cancers, as well as from healthy volunteers. Researchers will use these samples to study the diseases and develop new treatments called immunotoxins, which use an…

This study aims to build a digital early-warning system that uses machine learning to predict acute lung injury (ALI) in patients undergoing major liver surgery. By analyzing cardiopulmonary interaction characteristics, the model could help doctors identify at-risk patients early…

This study aims to collect information and bone samples from up to 1,000 people with bone or mineral disorders, such as tumor-induced osteomalacia or familial tumoral calcinosis. Participants receive standard medical evaluations and may provide bone tissue from surgery or a biops…

This trial compares two methods of examining colon cancer tissue after surgery: the European method, which relies solely on pathologists, and the Japanese method, which involves surgeons in finding lymph nodes. The goal is to see if the Japanese method is as accurate as the Europ…

This study looks at how differences in the PKLR gene might change sickle cell disease symptoms. Researchers will collect blood samples from 800 adults of African descent, with and without sickle cell disease, to measure certain proteins and energy molecules in red blood cells. Th…

This study collects wisdom teeth and surrounding tissue that are normally thrown away after oral surgery. Researchers will use these samples to learn more about oral health and disease. Up to 3,000 people aged 16 to 50 who need their wisdom teeth removed can join. The study also …

This study follows 950 people with idiopathic CD4+ lymphocytopenia (ICL), a rare condition where low CD4+ white blood cells raise infection risk. Researchers will track participants for up to 10 years, collecting medical history, blood samples, and infection data. The goal is to …

This study aims to learn more about primary immunodeficiency disorders (PIDs), which weaken the immune system and make people more prone to infections. Researchers will compare genetic and health data from people with PIDs, their healthy relatives, and healthy volunteers to find …

This study looks at whether tiny particles released by fat cells, called adiposomes, carry harmful fats that damage blood vessels in people with obesity and type 2 diabetes. Researchers will test if a 12-week aerobic exercise program or weight-loss surgery can reduce these harmfu…

This study is testing whether a special PET/MRI scan using an investigational tracer called [18F]FMISO can help monitor how well chemotherapy works in people with HER2-positive breast cancer. The study involves 25 patients and is for imaging purposes only—it will not change their…

This study follows 150 people with active or latent tuberculosis (TB) to collect blood, sputum, and tissue samples. The goal is to better understand how TB behaves in the body and how the immune system responds. Participants receive standard TB treatment as part of the study, but…

This study is testing whether artificial intelligence can accurately classify skin pigment, sun sensitivity (phototype), and sun damage from standard photos of the arm. Researchers will enroll 1,500 adults from diverse backgrounds, combining objective color measurements, expert a…

This study tests a decision-support tool called PRIME for adults aged 60 and older with blood cancers like leukemia or lymphoma. The tool helps patients clarify their values and shares that information with their doctors. The goal is to see if using PRIME leads to treatment decis…

This study is watching for side effects in 50 children in India who are prescribed somatrogon, a long-acting growth hormone injection for growth hormone deficiency. The goal is to see how safe the drug is during routine care over 3 years. Participants do not have to visit the cli…

This study looks at whether fungi moving from the gut into the blood can cause inflammation and worsen lung function in people with HIV. Researchers will measure a fungal marker called BDG in blood and urine, and track lung health over three years. The goal is to better understan…

This study looks at whether freezing ovarian tissue can help preserve fertility in people aged 2-21 with conditions like Turner syndrome, galactosemia, or premature ovarian insufficiency. Participants may have surgery to remove and freeze a gonad, with a small piece used for rese…

This study aims to create a simple, minimally invasive test to find pancreatic cancer at an early stage and track how well treatment is working. Researchers will study 700 people with pancreatic cancer, pancreatitis, or pancreatic cysts. The goal is to identify biological markers…

This study looks at how the immune system reacts during and after sepsis in 400 intensive care patients. Researchers will analyze blood samples to identify different immune response types, called immunotypes. The goal is to understand which immune patterns lead to better or worse…

This study aims to develop a standardized questionnaire that lets patients with venous malformations report their own symptoms and treatment outcomes. Venous malformations are abnormal veins that can cause pain, swelling, and emotional distress, and current assessments may miss w…

This study uses special MRI scans to see how radiation treatment affects the brain's white matter and thinking skills in people with cancer that has spread to the brain. Researchers will follow 15 patients to find early signs of memory or concentration problems. The goal is to be…