Inherited disease susceptibility

This study tests a vaccine called Nous-209 in 45 people with Lynch syndrome, a genetic condition that raises the risk of colon and other cancers. The vaccine is made from man-made copies of proteins that cancer cells produce, aiming to teach the immune system to recognize and att…

This phase 2 trial tests whether a daily pill called miransertib can slow or stop the abnormal tissue overgrowth seen in Proteus syndrome, a rare genetic condition. About 38 people aged 3 and older will take the drug for up to 4 years. Researchers will measure changes in foot ove…

This phase 3 trial is testing whether adding the immunotherapy drug atezolizumab to standard chemotherapy can help prevent cancer from coming back in people with stage III colon cancer that has a specific DNA repair defect (dMMR). About 712 participants will receive either chemo …

This Phase 2 trial tests a combination of pembrolizumab (Keytruda) and quavonlimab against other drug combos in 302 people with stage IV colorectal cancer that has a specific genetic feature (MSI-H or dMMR). The goal is to see if the new combo shrinks tumors better and for longer…

This study follows 165 children who previously took dabrafenib and/or trametinib for certain brain tumors. Researchers want to see if these drugs are safe over the long term and how they affect growth. The children continue their treatment and are monitored for side effects and d…

This Phase 3 trial compares the drug niraparib to the standard chemo temozolomide in 450 adults with newly diagnosed, aggressive glioblastoma that has a specific genetic marker (MGMT unmethylated). Participants take the assigned drug daily during radiation, then continue until th…

This study investigates whether combining the medication lovastatin with intensive reading tutoring can improve reading abilities in children and young adults (ages 8-20) with Neurofibromatosis Type 1 (NF1) who have reading disabilities. Participants first receive either lovastat…

This study compares two types of implants used in spinal fusion surgery: 3D-printed cages and titanium-coated cages. The goal is to see which leads to better bone fusion and less disability for patients with herniated discs or degenerative spine disease. About 78 adults undergoin…

This study tests whether giving a higher radiation dose to a brain area called the subventricular zone (where stem cells live) can delay glioblastoma from coming back. About 60 adults with newly diagnosed glioblastoma will receive standard radiation and chemo, but half will get e…

This study tests a program called Share plus for adults aged 60+ with type 1 diabetes and their care partners. The program helps them better use continuous glucose monitor data together. Researchers want to see if it improves blood sugar control and reduces diabetes-related stres…

This study observes how well insulin icodec controls blood sugar in 245 people with type 1 diabetes who have never used it before. Participants take the insulin as prescribed by their doctor, and researchers track changes in HbA1c and treatment satisfaction over about 22 to 30 we…

This study tests whether giving the BCG vaccine (traditionally used for tuberculosis) twice, four weeks apart, can help children aged 8 to 18 with type 1 diabetes. The goal is to see if it improves blood sugar control, reduces insulin use, and lowers the risk of dangerously low b…

This trial tests a combination of donor immune cells (targeting a common virus) and the drug pembrolizumab (Keytruda) in 58 people with glioblastoma, an aggressive brain cancer. The goal is to see if this approach can shrink tumors and help patients live longer. The study has two…

This phase 2 trial tests a drug called VK-2019 in 13 patients with Epstein-Barr virus (EBV)-positive nasopharyngeal cancer that has returned or spread and has no standard treatment. VK-2019 works by blocking a key viral protein, EBNA1, which the virus needs to survive. The main g…

This early-stage trial is testing an experimental drug called APX005M in 32 children with brain tumors that have come back or are hard to treat, including a type of brain stem glioma. The drug works by activating the immune system to attack cancer cells. The main goals are to fin…

This study compares a new three-drug combination (NABPLAGEM) against a standard two-drug chemo regimen for people with advanced pancreatic cancer that has spread and carries a BRCA or PALB2 gene mutation. The trial enrolls 10 participants whose cancer worsened after initial treat…

This study compares a targeted drug called selumetinib to standard chemotherapy (carboplatin/vincristine) in children aged 2 to 21 with neurofibromatosis type 1 (NF1) and low-grade glioma, a type of brain tumor. The goal is to see if selumetinib works as well or better at control…

This trial tests a new drug called Cadonilimab in 28 people with advanced colorectal cancer that has not responded to standard immunotherapy. The drug is a bispecific antibody that targets two immune checkpoints (PD-1 and CTLA-4) to potentially reactivate the immune system agains…

This phase 3 trial tests ravulizumab, a drug that blocks part of the immune system, against a placebo in 579 adults with IgA nephropathy—a kidney disease that can lead to kidney failure. The goal is to see if ravulizumab reduces protein in the urine and preserves kidney function …

This study tests a one-time gene therapy called CAP-003 in adults with Parkinson's disease caused by a GBA1 gene mutation. The goal is to see if it is safe and if it can help control the disease. Participants receive a single IV infusion and are monitored for two years.

This study tests a donor stem cell transplant for people with severe immune system problems, like SCID and Wiskott-Aldrich syndrome. The goal is to help the body make healthy blood cells and fight infections. Participants receive donated stem cells to rebuild their immune system.…

This study tests a stem cell transplant method for people with acquired or inherited bone marrow failure. Donor stem cells are specially processed to remove certain immune cells, which may lower the risk of graft rejection and graft-versus-host disease. The goal is to see if this…

This phase II trial tests whether intensive blood pressure management (targeting systolic BP below 120 mmHg) is better than standard care (below 140 mmHg) for patients with advanced kidney or thyroid cancer starting anti-angiogenic TKI therapy. The study enrolls 61 participants a…

This early-stage study tested the safety and best dose of vorinostat when given with the chemotherapy drug temozolomide in 83 adults with aggressive brain tumors called malignant gliomas. The goal was to see if adding vorinostat could make the tumor cells more sensitive to chemot…

This study tests a drug called selumetinib in children and young adults (ages 3-18) with neurofibromatosis type 1 (NF1) who have nerve tumors that cannot be removed by surgery. The goal is to see if the drug can shrink or slow the growth of these tumors. Participants take the dru…

This early-stage study tests whether a mix of three drugs—tacrolimus, nivolumab, and ipilimumab—can shrink or control certain skin cancers in people who have had a kidney transplant. The main goal is to see if the cancer responds without causing the body to reject the transplante…

This phase II trial is studying a combination of chemotherapy (Hyper-CVAD) and the targeted drug dasatinib, with or without a donor stem cell transplant, in 97 adults with Philadelphia chromosome-positive acute lymphoblastic leukemia (ALL). The goal is to see how well this approa…

This early-stage study tests a new drug called ADX-850 in 28 people with mild to moderate high blood pressure. The drug uses siRNA technology to target genes involved in blood pressure regulation. The main goal is to check safety and how the drug moves through the body, with a se…

This early-stage study tests a combination of two drugs, avutometinib and defactinib, in children and young adults (ages 3 to 30) with advanced or recurrent solid tumors that have specific genetic changes. The main goal is to find the safest dose with the fewest side effects. The…

This phase 2 trial tests whether the drug TPST-1495 can reduce the number and size of polyps in the small bowel and colon of people with familial adenomatous polyposis (FAP), an inherited condition that greatly raises colon cancer risk. About 38 participants will take the drug by…

This study was designed to test two immunotherapy drugs, nivolumab and ipilimumab, in children and young adults with cancers that returned or didn't respond to treatment and had many genetic mutations. The goal was to see if the drug combination was safe and could shrink tumors. …

This phase 2 trial tests the drug cabozantinib in children and young adults with rare cancers like sarcomas, Wilms tumor, and others that have come back or not responded to treatment. Cabozantinib works by blocking enzymes that help tumors grow and form blood vessels. The study a…

This study tests whether combining two immunotherapy drugs (nivolumab and ipilimumab) with radiation therapy can control or shrink tumors in people with colorectal or pancreatic cancer that has specific genetic features (MSS or MSI-high). About 84 adults with advanced disease are…

This study tests whether low-dose sirolimus can safely improve blood cell function in adults with RUNX1 familial platelet disorder, a genetic condition that raises bleeding and leukemia risks. Six participants receive the drug and are monitored for side effects and changes in blo…

This study offers continued treatment with the drug ceritinib to people with ALK-positive cancers who were already benefiting from it in a previous Novartis study. The main goal is to track safety and side effects over time. About 233 participants will receive the drug as long as…

This study looks at how well women with low estrogen (like after menopause) stick to using vaginal estrogen treatments to prevent repeat urinary tract infections. About 111 women will be randomly assigned to use a cream, tablet, or ring. Researchers will track adherence over 6 mo…

This phase 2 trial tests the drug talazoparib in 150 people with advanced cancers that have not responded to standard treatments. Participants must have specific gene changes in BRCA1, BRCA2, or related DNA repair genes. The drug works by blocking an enzyme that helps cancer cell…

This study looks at how safe and effective the drug everolimus is for Taiwanese adults with tuberous sclerosis complex (TSC) who have kidney tumors called angiomyolipomas. Four participants will take the drug and be monitored for side effects and tumor shrinkage. The goal is to c…

This study tests a new drug called Cetrelimab (JNJ-63723283) in 234 people with advanced solid tumors like lung, melanoma, bladder, and colorectal cancers. The drug works by blocking PD-1, a protein that stops the immune system from attacking cancer cells. The trial has two parts…

This study planned to test a new treatment combining CYNK-001 (natural killer cells from donated cord blood) with interleukin-2 in adults whose glioblastoma had returned and could be surgically removed. The goal was to see if this combination was safe and could extend the time be…

This study tests a granule (sprinkle) form of the drug selumetinib in children aged 1 to under 7 years who have neurofibromatosis type 1 (NF1) with painful, inoperable tumors. The goal is to find the right dose, check safety, and see if it shrinks tumors. About 36 children will t…

This study tests an oral medication (decitabine/cedazuridine) in people with BAP1 gene mutations who have early-stage mesothelioma that is not yet causing symptoms. The goal is to see if the drug can stop the cancer from growing or shrink it. About 9 participants will receive tre…

This early-phase study tests a vaccine called SurVaxM in 35 children with certain brain tumors that have come back or are hard to treat. The vaccine is designed to teach the immune system to find and destroy tumor cells that have a protein called survivin. The goal is to see if i…

This study follows about 111 people with Lennox-Gastaut syndrome, Dravet syndrome, or tuberous sclerosis complex who are taking Epidyolex (a CBD oral solution) for seizures. Researchers track how many stay on the drug for a year and how many seizure-free days they have. It's an o…

This phase 2 study tests a personalized immune cell therapy called tumor-infiltrating lymphocytes (TIL) in 20 people with advanced solid cancers (like stomach, colon, or pancreatic cancer) that have not responded to standard treatments. Patients first receive chemotherapy to prep…

This study tests a new insulin infusion set designed to last up to 7 days for people with type 1 diabetes. Twenty adults currently using a Tandem insulin pump and Dexcom CGM will try the SteadiSet at home. The goal is to see if the set can survive a full week without causing high…

This early-phase study tests whether adding a drug called S-Gboxin to standard treatment can help people with glioblastoma, a fast-growing brain cancer. The drug works by cutting off the energy supply to cancer cells. Ten patients with recurrent or progressive disease will receiv…

This study tests a technique that delivers two cancer drugs, bevacizumab and carboplatin, directly into the brain's arteries for people with recurrent glioblastoma or anaplastic astrocytoma. The goal is to see if this method is safe and can slow tumor growth better than standard …

This early-stage trial tests a new surgical approach for recurrent glioblastoma, an aggressive brain cancer. After standard tumor removal, surgeons place a piece of abdominal tissue (omentum) into the brain cavity to bypass the blood-brain barrier. The study involves 10 adults an…

This study tests whether a two-drug combination can help the immune system attack ovarian cancer that has returned after standard treatments. The drugs are E7777, which removes certain immune cells that block the body's defenses, and pembrolizumab, which reactivates cancer-fighti…

This phase 2 trial tests two oral drugs, abrocitinib and ritlecitinib, in 78 people aged 12-35 who were diagnosed with type 1 diabetes within the last 100 days. The goal is to see if these JAK inhibitors can help preserve the body's ability to produce insulin, measured by a stimu…

This pilot study tests a fludarabine-based drug regimen to prepare children with bone marrow failure syndromes for a bone marrow transplant from a matched sibling donor. The goal is to help the donor cells successfully take root while reducing serious side effects. The study incl…

This study tested a combination of the immunotherapy drug blinatumomab with either standard chemotherapy or dasatinib and prednisone in adults aged 65 and older with acute lymphoblastic leukemia (ALL). The goal was to see if the combination could improve survival and control the …

This study compares a new blended therapy (online sessions plus in-person visits) to standard in-person therapy for teens with conduct disorder or antisocial behavior. Researchers want to see if the blended approach works just as well at reducing aggression, delinquency, and out-…

This study follows 1,600 people with type 1 diabetes, including young children aged 2-6, who use hybrid closed-loop insulin pumps (Medtronic, Tandem, or Omnipod). These devices automatically adjust insulin based on blood sugar readings but still require users to enter meal inform…

This study tests a program called MIAPP that adds a patient navigator, smartphone check-ins, and small rewards to usual care for people who start buprenorphine for opioid use in the hospital and also use methamphetamine. The goal is to help them connect to outpatient treatment wi…

This study tests a drug called trametinib in children with a rare blood cancer (juvenile myelomonocytic leukemia) that has returned or not responded to treatment. The drug works by blocking certain enzymes that help cancer cells grow. The goal is to see if it can shrink or contro…

This phase 2 pilot study is testing whether giving two immunotherapy drugs, botensilimab and balstilimab, before surgery can shrink tumors in people with colorectal cancer. The trial includes 26 participants across three groups, with some receiving more doses and one group having…

This study looks at whether taking selumetinib with a low-fat meal reduces stomach problems in teenagers with neurofibromatosis type 1 (NF1) who have tumors that cannot be removed by surgery. About 24 teens will take the drug under both fed and fasted conditions to compare drug l…

This early-stage study tests a new drug called MEDI5752, given alone or with chemotherapy, in adults with advanced solid tumors that have not responded to other treatments. The main goals are to check safety, find the right dose, and see if the drug can shrink tumors. About 400 p…

This phase 2 trial tests whether giving selpercatinib before standard surgery can shrink tumors in people with RET-altered thyroid cancer. About 30 participants with advanced or recurrent disease will receive the drug and then undergo surgery. The goal is to see if the drug impro…

This study tests an experimental drug called BIA 28-6156 in people with Parkinson's disease who have a specific change in the GBA1 gene. The goal is to see if the drug can delay the worsening of movement-related daily living skills over 78 weeks. About 237 participants will recei…

This early-stage trial is testing the safety and potential benefit of pembrolizumab, an immunotherapy drug, in 71 children whose brain tumors (like DIPG, high-grade gliomas, or medulloblastoma) have come back or not responded to standard treatments. The drug works by helping the …

This study tests a drug called selumetinib in 32 Chinese children and adults with neurofibromatosis type 1 (NF1) who have nerve tumors that cannot be surgically removed. The goal is to check the drug's safety and how well it works at shrinking these tumors. Participants take the …

This study tests a drug called selumetinib in children and young adults with low grade glioma that has come back or not responded to other treatments. The drug works by blocking certain enzymes that help tumor cells grow. The goal is to find the best dose and see if it can shrink…

This phase II trial is testing whether a combination of talimogene laherparepvec (a modified herpes virus that infects and kills cancer cells) and nivolumab (an immunotherapy drug) can shrink tumors in people with rare skin cancers or lymphomas that haven't responded to standard …

This study tests an experimental drug called KL590586, taken as a capsule, in people with advanced solid tumors that have specific RET gene changes. The trial has two phases: Phase I checks safety and the right dose, while Phase II looks at how well the drug shrinks tumors. Up to…

This early-phase trial tests an investigational drug called ABBV-706, alone or combined with other drugs, in about 288 adults with advanced solid tumors like small cell lung cancer, high-grade brain tumors, and neuroendocrine cancers. The main goals are to check safety, find the …

This early-stage trial tested a personalized DNA vaccine designed to train the immune system to attack glioblastoma, a fast-growing brain cancer. Nine patients with a specific type of glioblastoma (unmethylated MGMT) received the vaccine along with an immune booster. The main goa…

This study tests a drug called inaxaplin in 42 people with a genetic form of kidney disease linked to the APOL1 gene. The goal is to see if the drug can reduce protein in the urine, a sign of kidney damage, and to check for side effects. Participants take the drug as tablets, and…

This phase II trial tests the safety and effectiveness of a drug called XmAb20717 in 140 people with advanced rare cancers, including mesothelioma, lymphoma, and small cell lung cancer. The drug is given by IV. The main goal is to see if tumors shrink. The trial is currently susp…

This phase II trial is testing whether adding the drug asciminib to standard treatment can improve outcomes for people newly diagnosed with Philadelphia chromosome positive acute lymphoblastic leukemia (Ph+ ALL). The study will enroll 55 participants and involves several phases o…

This study tests a device that delivers specific light and sound patterns to the brain, aiming to slow the progression of mild-to-moderate Alzheimer's disease. About 670 participants will use the device at home for an hour daily over a year, with half receiving a sham (inactive) …

This study tests a drug called mirdametinib in 114 adults and children with a genetic condition called NF1 that causes nerve tumors (plexiform neurofibromas) that cannot be removed by surgery and cause serious problems. The drug works by blocking a protein that helps tumors grow.…

This study tests two ways to surgically repair spina bifida in the womb using a tiny camera. The goal is to close the spinal defect and reverse brain changes caused by the condition. About 110 pregnant women carrying babies with spina bifida will take part. The two methods are co…

This study tests whether adding the targeted drug pazopanib to standard chemotherapy (temozolomide) can safely control glioblastoma, an aggressive brain cancer. About 51 adults who had surgery and chemoradiation will receive the combination. The main goal is to find the best dose…

This phase 3 trial tests imatinib, a targeted cancer drug, with two different chemotherapy backbones in children and adolescents with Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL). The goal is to see if a less intense chemo regimen works as well as the s…

This study tests a milder chemotherapy and immune-suppressing regimen before a stem cell transplant for children and young adults with non-malignant blood disorders like sickle cell disease or immune deficiencies. The goal is to safely achieve donor cell engraftment with fewer si…

This early-phase trial is testing whether adding the drug venetoclax to a standard treatment regimen can help adults with a specific type of leukemia (Philadelphia chromosome-positive ALL). The study involves 20 participants and aims to find the safest dose of venetoclax when com…

This early-phase study tests a radioactive drug (177Lu-PP-F11N) that seeks out and attaches to cancer cells in people with advanced medullary thyroid cancer and certain other neuroendocrine tumors. The goal is to see if the drug can help image tumors and deliver targeted radiatio…

This phase 3 trial tests a procedure called renal denervation, where a small amount of alcohol is injected around the kidney nerves to calm overactive signals that raise blood pressure. 142 people with uncontrolled hypertension will either get the real procedure or a sham (fake) …

This early-stage trial tests whether injecting stem cells from donated umbilical cords into the spine can help repair degenerated discs in people with lower back and leg pain. Twenty patients who have not improved with other treatments will receive the injection right after a min…

This study tests whether taking vitamin B1 tablets for a year can reduce the size and number of colon polyps in people with familial adenomatous polyposis (FAP), a genetic condition that raises colon cancer risk. Ten adults with FAP who still have their colon will take vitamin B1…

This early-phase study tests a new treatment for glioblastoma, an aggressive brain cancer. The approach uses a patient's own immune cells, modified in the lab to better recognize and attack the tumor, combined with chemotherapy. The main goal is to check the safety of this therap…

This phase 2 trial tests the drug trametinib in children and young adults (ages 1 month to 25 years) with low-grade glioma or plexiform neurofibroma that has not responded to prior treatment. Participants take a daily oral dose for up to 18 cycles. The study aims to see if the dr…

This early-stage trial tests whether combining a precise form of radiation (stereotactic radiosurgery) with two immunotherapy drugs (MBG453 and spartalizumab) is safe for people whose glioblastoma has come back. The study includes 16 adults with recurrent GBM. The main goal is to…

This early-stage trial tests whether a single infusion of umbilical cord stem cells can safely preserve insulin production in adults newly diagnosed with type 1 diabetes. Sixty participants will receive either the stem cells or a placebo. The main goal is to see if the treatment …

This study tests a weight management and health behavior program for people with BRCA or Lynch syndrome mutations, who have a higher risk of certain cancers. Participants receive diet tracking, exercise guidance, phone coaching, and online support to help them lose weight and ado…

This study tests a powerful mix of chemotherapy drugs plus a targeted pill (ponatinib) for people with a fast-growing leukemia called Philadelphia chromosome-positive ALL. The goal is to see if this combination can stop the cancer from coming back. About 88 adults with newly diag…

This study tests whether taking a probiotic (a 'good' bacteria) can help women who get frequent urinary tract infections (UTIs). About 90 women with a history of repeat UTIs will take either a probiotic or a placebo (a dummy pill) for 6 months. The goal is to see if the probiotic…

This study tests a minimally invasive fetoscopic surgery to repair spina bifida in unborn babies. The goal is to close the spinal defect and reverse brain herniation while reducing risks like uterine rupture and preterm birth that come with open fetal surgery. Thirty pregnant wom…

This trial tests an implantable ultrasound device that temporarily opens the blood-brain barrier, allowing chemotherapy to reach brain tumors. It includes 57 adults with recurrent glioblastoma who have already tried 1-2 treatments. The goal is to find a safe dose and see if this …

This phase 2 trial tests whether dulaglutide, a drug used for type 2 diabetes, can improve small blood vessel function in adults with type 1 diabetes. The study involves 47 participants aged 18-40 with long-standing type 1 diabetes. Researchers measure blood flow and insulin sens…

This was a phase 2 trial testing a combination of niraparib and abiraterone plus prednisone for metastatic hormone-sensitive prostate cancer in Hispanic/Latino and Black patients with specific gene mutations. The study aimed to enroll 64 people but was withdrawn before any partic…

This phase 2 trial tests whether adding blinatumomab (an immunotherapy) to standard targeted therapy (dasatinib) can improve treatment for adults with Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL). The goal is to achieve a deep molecular response without…

This study looks at whether injecting special stem-like cells called Muse cells directly into the ovaries can help women aged 28 to 70 who are in perimenopause. The cells may repair tissue, reduce inflammation, and improve hormone balance. Researchers will track safety, hormone l…

This early-stage trial tests a new weekly injection called NNC0194-0499, alone or with another drug (semaglutide), in 96 adults with type 1 diabetes. The goal is to see if it improves blood sugar levels compared to placebo. All participants continue their usual insulin. The study…

This study looks at the long-term safety of everolimus in people with tuberous sclerosis complex (TSC) who have seizures that are hard to control. Participants are already taking everolimus and benefiting from it in an earlier study. The goal is to see if the drug remains safe an…

This phase 2 trial tests whether pembrolizumab (Keytruda) can shrink or control advanced solid tumors that have certain gene mutations (BRCA1, BRCA2, POLE, POLD1). The study enrolled 21 adults with cancers that have spread or come back. Researchers measure how many patients see t…

This early-phase trial tests an experimental drug called radiprodil in 30 people with tuberous sclerosis complex or focal cortical dysplasia type II whose seizures have not responded to at least two other medications. The study is open-label, meaning everyone gets the drug, and w…

This phase II study tests a vaccine called SurVaxM, designed to train the immune system to attack glioblastoma cells, when given alongside standard chemotherapy (temozolomide). The trial includes 66 patients with newly diagnosed, survivin-positive glioblastoma or gliosarcoma. The…

This phase 2 study tests two drugs, sunitinib and temsirolimus, in 137 people with advanced rare tumors that have no cure. The goal is to see if these drugs can shrink tumors or slow their growth. Participants take one of the two drugs, and researchers measure how long the cancer…

This study tests a bone marrow transplant method that avoids harsh chemotherapy and radiation for people with dyskeratosis congenita, a genetic disorder causing bone marrow failure. The goal is to fix the blood system without worsening lung or liver disease or raising cancer risk…

This study tests a drug called ACP-196 (acalabrutinib) in 24 adults with recurrent glioblastoma, an aggressive brain cancer that has returned after standard treatment. Participants take the drug by mouth once or twice daily. The main goal is to see if the drug can shrink tumors o…

This early-phase trial tests a new drug called HRO761, alone or combined with pembrolizumab or irinotecan, in people with advanced solid tumors that have high microsatellite instability (MSI-high) or mismatch repair deficiency (dMMR). The main goals are to check safety and find t…

This study tests the drug selumetinib in adults with neurofibromatosis type 1 (NF1) who have painful, inoperable nerve tumors called plexiform neurofibromas. About 145 participants will receive either selumetinib or a placebo to see if the drug can shrink the tumors and reduce pa…

This Phase II trial tests whether combining lenvatinib and everolimus can shrink or slow advanced carcinoid tumors that cannot be removed by surgery. The study enrolls 36 adults with confirmed unresectable carcinoid tumors. Both drugs are taken orally and work by blocking enzymes…

This study was designed to test the drug alrizomadlin (APG-115) in people with BAP1 cancer syndrome who have early-stage mesothelioma that does not yet need standard treatment. The goal was to see if the drug could stabilize or improve the disease. However, the trial was withdraw…

This study tests a drug called vosoritide in 56 children with short stature caused by certain genetic conditions. The drug targets the growth plate to help children grow faster. Participants are observed for 6 months, then treated with daily injections for 12 months to check safe…

This phase 2 trial tests whether combining two immunotherapy drugs, nivolumab and relatlimab, can shrink or control advanced solid tumors with a specific genetic feature (MSI-H) that have stopped responding to prior immunotherapy. About 38 adults with these hard-to-treat cancers …

This early-stage trial tests a small device placed under the skin that contains insulin-producing cells. The goal is to see if it is safe and tolerable for people with type 1 diabetes who already receive standard islet transplants. Only 10 participants are enrolled, and the main …

This early-phase trial tests whether adding the drug AZD1390 to standard radiation therapy can help people with a fast-growing brain tumor called glioblastoma (grade 4 glioma). The study includes both newly diagnosed and recurrent patients. First, researchers measure how much dru…

This early-phase trial tests the drug niraparib (Zejula) in people with two types of brain cancer: newly diagnosed glioblastoma and recurrent glioma with specific genetic changes (IDH mutation and ATRX loss). The study first checks if the drug reaches the tumor tissue, then gives…

This large global trial is testing whether two experimental drugs—volrustomig (an immunotherapy) and casdatifan (a pill)—work better than the current standard immunotherapy combination for people with advanced clear cell kidney cancer. About 1,100 participants who have not had pr…

This study tests a new gel, MegaShield-SP, to see if it prevents scar tissue after spine surgery better than an existing gel. About 80 adults aged 20-70 with a herniated disc or spinal stenosis will receive one of the two gels during surgery. Researchers will measure scar formati…

This trial tests whether the drug ribociclib can reach brain tumors in people with recurrent high-grade glioma or meningioma. About 48 participants take the drug before their scheduled tumor-removal surgery. The goal is to measure how much drug gets into the tumor and surrounding…

This early-stage study tests a genetically engineered virus (rQNestin34.5v.2) that is injected directly into recurrent brain tumors to see if it is safe and what dose works best. About 62 adults with certain types of brain cancer that have come back or worsened are taking part. T…

This study tests a drug called selumetinib in adults with neurofibromatosis type 1 (NF1) who have nerve tumors that cannot be removed by surgery. The goal is to see if the drug can shrink these tumors or slow their growth. Participants take the drug twice daily and are monitored …

This phase 2b trial tests whether the immunotherapy drug tremelimumab can help people with advanced mesothelioma live longer. About 571 patients who have already tried standard chemotherapy will receive either tremelimumab or a placebo. The main goal is to see if the drug improve…

This early-phase study tests a new drug called E7386 in about 70 people with advanced solid tumors, including colorectal cancer. The main goal is to check safety and find the right dose. Participants have tumors that no longer respond to standard treatments.

This study tests whether using a continuous glucose monitor (CGM) helps dialysis patients with type 1 or type 2 diabetes control their blood sugar better than standard finger-prick tests. 96 participants will be split into three groups: standard care, intermittent CGM use, or con…

This phase 2 study tests pembrolizumab (Keytruda) in 40 men with metastatic castration-resistant prostate cancer that has specific genetic flaws (mismatch repair deficiency or CDK12 inactivation). The goal is to see if the drug can shrink tumors or slow the cancer. Researchers wi…

This early-phase study tests whether a personalized immune cell therapy (dendritic cells) is safe and can help control advanced nasopharyngeal cancer. The treatment uses the patient's own immune cells and donor cell secretions to boost the body's cancer fight. The study involves …

This early trial is testing whether the drug olaparib can shrink tumors in people with newly diagnosed BRCA-mutant ovarian, fallopian tube, or peritoneal cancer before they have surgery. Fifteen participants will receive olaparib for two cycles, then undergo standard chemotherapy…

This study tests whether a device called Sonablate can safely use high-intensity focused ultrasound (HIFU) to treat vein problems like varicose veins, leg ulcers, and certain birth defects. About 30 adults with these conditions will receive the treatment, which does not involve c…

This study tests a new way to treat adults with a specific type of leukemia (Ph+ ALL) that has come back or not responded to treatment. The approach combines low-intensity chemotherapy with two targeted drugs, ponatinib and blinatumomab, to kill cancer cells and boost the immune …

This study tests a stem cell transplant using a milder chemotherapy-like regimen to treat children and young adults (up to age 28) with various immune system disorders. The goal is to help the donor stem cells settle in the body with fewer side effects. The study involves 20 part…

This early-phase trial is testing whether giving two immune-boosting drugs (nivolumab and ipilimumab) before surgery can help fight recurrent glioblastoma, a fast-growing brain cancer. About 63 adults whose tumor has returned after standard treatment will receive the drugs or a p…

This Phase 2 trial tests a genetically modified herpes virus (RP1) alone or with the immunotherapy drug nivolumab in people with advanced solid tumors, including melanoma, non-small cell lung cancer, and certain skin cancers. The study aims to see if the combination is safe and c…

This study was designed to test the safety and effectiveness of the Supira System, a temporary heart pump, in patients undergoing high-risk coronary interventions. The device is inserted through a leg artery to help pump blood from the heart to the body during the procedure. Howe…

This study aimed to test an experimental virus therapy called G207, combined with a single dose of radiation, in children whose high-grade glioma had come back after prior treatment. The virus was injected directly into the brain tumor to kill cancer cells and trigger an immune r…

This early-phase trial is testing a new type of cell therapy called P-MUC1C-ALLO1 for people with advanced solid tumors (like breast, ovarian, lung, or pancreatic cancer) that have not responded to other treatments. The therapy uses donor immune cells engineered to recognize and …

This study tests an experimental drug called ST101 in people with advanced solid tumors that cannot be removed by surgery or have spread. The trial includes cancers like glioblastoma, melanoma, breast, and prostate cancer. It aims to find a safe dose and see if the drug can shrin…

This study follows 100 people who received a new type of knee replacement device called EVOLUTION® NitrX™. The goal is to see how safe and effective it is over time. Participants have conditions like arthritis or knee damage. The main measure is a patient-reported score of knee f…

This pilot study tests whether an energy-restricted ketogenic diet (ERKD) can help control glioblastoma, an aggressive brain cancer. Sixteen adults with newly diagnosed glioblastoma will follow the diet while receiving standard radiation and chemotherapy. The goal is to see if th…

This study tests two minimally invasive fetal surgery techniques for repairing neural tube defects (spina bifida) in the womb. One method uses a Durepair patch to reinforce the repair, while the other does not. Researchers aim to see if the patch leads to a thicker repair and few…

This trial is testing whether adding hydroxychloroquine to two targeted cancer drugs (dabrafenib and trametinib) can help children and young adults with certain brain tumors that have come back or not responded to treatment. The study includes patients with specific genetic chang…

This study tests a two-year follow-up program for 200 adults with hereditary cancer syndromes like BRCA or Lynch syndrome. Participants get personalized care plans and regular check-ins with a genetics doctor to help them follow cancer prevention and screening guidelines. The goa…

This early-phase study tests an experimental oral drug (GSK4418959) in adults with advanced solid tumors that have specific genetic changes (dMMR or MSI-H). The drug works by blocking a protein (WRN) that these tumors need to survive. The study aims to see if the drug, alone or w…

This study tests a drug called teplizumab in 20 children under 8 who have early signs of type 1 diabetes (Stage 2). The goal is to check if the drug is safe and how the body handles it. Teplizumab works by calming the immune system to protect insulin-making cells. If successful, …

This study tests whether adding the experimental drug BPM31510 (with vitamin K1) to standard radiation and chemotherapy can help people with newly diagnosed glioblastoma live longer without their cancer growing. About 50 adults with this aggressive brain tumor will receive the co…

This pilot study aimed to test whether a combination of four drugs—ATG, low-dose IL-2, adalimumab, and exenatide—could help people newly diagnosed with type 1 diabetes keep producing their own insulin. The trial planned to enroll adults aged 18-35 diagnosed within the last 120 da…

This study tests a combination of an experimental immune booster (N-803) with standard checkpoint inhibitors in people with advanced solid tumors (like lung, bladder, or skin cancer) whose disease progressed after prior immunotherapy. The goal is to see if the combo can shrink tu…

This study tests whether combining an AI smartphone app, intermittent fasting (16 hours of fasting daily), and resistance training can improve blood sugar control and physical fitness in people with type 2 diabetes. Sixty adults with diabetes will either follow this program or re…

This study tests a new artificial disc implant (PerQdisc) placed during surgery for a herniated disc. The goal is to see if it is safe and helps reduce back and leg pain. About 50 adults with a single herniated disc in the lower back will take part. The implant is meant to replac…

This early-phase study tests a combination of two drugs, niraparib and dostarlimab, in 18 adults with BRCA-mutated breast, pancreas, or ovary cancer that has spread or cannot be surgically removed. Niraparib blocks a DNA repair protein in cancer cells, while dostarlimab helps the…

This phase 2 study tests the drug olaparib in 114 people with metastatic breast cancer whose tumors have mutations in certain DNA repair genes (like BRCA1, BRCA2, ATM, PALB2, and others). Olaparib is a PARP inhibitor that may kill cancer cells by blocking their ability to fix dam…

This phase II trial is testing whether combining two drugs—niraparib and panitumumab—can help people with advanced colorectal cancer that has spread. The study includes 26 patients whose cancer has not responded to at least one prior treatment. The goal is to see if the combinati…

This early-phase study tests whether a new type of cell therapy, made from a patient's own immune cells that are genetically modified to resist chemotherapy, can be safely given alongside standard chemotherapy for newly diagnosed glioblastoma. About 22 adults with this aggressive…

This study tests a treatment plan for people with stage IVA nasopharyngeal cancer (a type of nose and throat cancer). Participants receive a combination of the immunotherapy drug pembrolizumab and chemotherapy before and during radiation, followed by maintenance pembrolizumab for…

This phase 2 study tests whether adding the antiviral drug valganciclovir to standard treatment (radiation and chemotherapy) can help people with glioblastoma live longer. The idea is that many glioblastomas carry a virus called CMV, which may make the cancer more aggressive. Abo…

This phase II trial tests whether aspirin can delay tumor growth and maintain hearing in people with vestibular schwannoma (acoustic neuroma), a non-cancerous brain tumor. About 97 participants are randomly assigned to take aspirin or a placebo twice daily. The study tracks how l…

This study tests whether methotrexate, a drug used for other types of arthritis, can reduce pain and improve hand function in people with a severe form of hand osteoarthritis. About 163 adults with moderate to severe finger pain will receive either methotrexate or a placebo for 6…

This study tests a new approach for people aged 5 to 45 with primary immune deficiencies and end-stage lung disease. Participants receive a double lung transplant followed by a stem cell transplant from the same donor. The goal is to see if this combined procedure is safe and can…

This study tests whether adding high-dose vitamin C to the usual treatment (radiation and chemotherapy) helps adults with a newly diagnosed aggressive brain tumor called glioblastoma. About 90 participants will receive the vitamin C infusions alongside standard care. The goal is …

This early-stage study tests whether adding an experimental drug called OKN-007 to standard chemoradiotherapy is safe and tolerable for people with newly diagnosed glioblastoma, a fast-growing brain cancer. About 27 participants will receive the combination after surgery. The mai…

This study follows 188 children and adolescents who completed the PROTECT study to check the long-term safety of teplizumab, an antibody treatment for recent-onset type 1 diabetes. Researchers will monitor side effects, insulin use, and blood sugar control for an additional 42 mo…

This study tests whether injecting doxycycline directly into skin schwannomas can shrink them in people with neurofibromatosis type 2 (NF2). Nineteen participants will receive up to three injections per tumor, with the dose based on tumor size. The goal is to find a less invasive…

This study tests if transplanting donor islet cells into the liver can safely help people with difficult-to-control type 1 diabetes achieve normal blood sugar levels without insulin. Up to 50 adults aged 18-58 who have had diabetes for at least 5 years and still struggle with blo…

This early-phase trial tests whether adding high-dose vitamin C and an iron supplement (ferumoxytol) to standard radiation and chemotherapy can help adults with newly diagnosed glioblastoma, an aggressive brain tumor. The study includes 17 participants and focuses on finding a sa…

This phase 3 trial tests whether ponatinib (Iclusig) works better than imatinib (Gleevec) for adults newly diagnosed with Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL). Both drugs are taken as pills alongside reduced-intensity chemotherapy for up to 20 m…

This early-phase trial tests whether a drug called rhIL-7-hyFc can safely increase lymphocyte counts in people with high-grade glioma who have low immune cells after standard radiation and chemotherapy. About 42 participants will receive either the drug or a placebo by injection.…

This study looks at 360 adults aged 18-75 who need a hip replacement due to arthritis or pain. It compares three different types of artificial hip parts to see which one causes less bone loss and gives better pain relief and movement over 5 years. Participants will have CT scans …

This study tests whether adding the experimental drug LAM561 to standard radiation and chemotherapy can help people with newly diagnosed glioblastoma, a fast-growing brain tumor. About 144 adults aged 18 to 75 will receive either LAM561 or a placebo alongside their usual treatmen…

This study tests whether eating wild blueberry powder every day can lower blood pressure and improve blood vessel health in middle-aged and older men and postmenopausal women with slightly high blood pressure. Researchers will measure artery function and stiffness over time. The …

This early-stage study tests a treatment that uses a patient's own immune cells, modified to recognize and attack cancer cells, for adults with B-cell blood cancers that have returned or not responded to standard therapy. The treatment is given with chemotherapy and sometimes an …

This study tests a combination of two drugs, cobolimab and dostarlimab, in children and young adults (up to age 21) with advanced solid tumors that have not responded to other treatments. The goal is to find the safest dose and see if the combination can shrink tumors. The study …

This Phase II trial tests a stem cell transplant from unrelated or partially matched family donors for children and young adults with severe immune deficiencies. The stem cells are specially processed to remove certain immune cells, aiming to reduce complications. The goal is to …

This study tested a new treatment called SR-02, which involves implanting insulin-producing cells into the abdomen of adults with type 1 diabetes who experience dangerously low blood sugar. Participants would need to take lifelong anti-rejection drugs to prevent the body from att…

This phase 3 trial tests whether giving a dose of radiation directly to the tumor site during surgery (intraoperative radiotherapy) can help people with a newly diagnosed aggressive brain cancer called glioblastoma live longer without their cancer growing. The study involves 314 …

This Phase 3 study tests whether a gel containing patidegib can lower the number of new basal cell carcinomas (a type of skin cancer) on the face of adults with Gorlin syndrome. Participants apply the gel or a placebo to their face twice daily for a year. The study will compare h…

This study tests whether adding an experimental drug called NanO2 to standard radiation and chemotherapy can help people with newly diagnosed glioblastoma, an aggressive brain cancer. NanO2 is designed to deliver more oxygen to tumors, which may make radiation work better. About …

This study tests a combination of three drugs—cisplatin, romidepsin, and nivolumab—in people with advanced triple-negative breast cancer or BRCA-related breast cancer that has spread. The goal is to see if the combination can shrink tumors and control the disease. About 51 partic…

This early-stage trial tests a single dose of M032, a genetically modified herpes simplex virus that produces IL-12, in 29 patients with recurrent or progressive brain tumors (glioblastoma, anaplastic astrocytoma, or gliosarcoma). The virus is infused directly into the tumor area…

This trial tests a gene therapy called OTL-103 for people with Wiskott-Aldrich syndrome, a rare genetic disorder that weakens the immune system and causes bleeding. The therapy uses the patient's own blood stem cells, modified in a lab to carry a working copy of the faulty gene, …

This study tests two drugs, nivolumab and cabozantinib, together in 60 people with advanced or metastatic non-clear cell kidney cancer. The goal is to see how well the combination shrinks tumors. Participants take cabozantinib daily and receive nivolumab infusions every two weeks…

This study compares two types of knee implants (NexGen CR and Persona CR) in 160 people with knee osteoarthritis. Researchers will measure how well the implants stay in place, how satisfied patients are, and how active they become after surgery. The goal is to find out which impl…

This early-phase study tests an oral drug called DSP-0390 in 39 adults whose high-grade glioma (a fast-growing brain tumor) has come back after prior treatment. The main goals are to check the drug's safety, find the best dose, and see if it can shrink tumors. Participants must b…

This phase 2 trial tests two experimental immunotherapy drugs, botensilimab and balstilimab, alone or together, against standard chemotherapy in 234 people with metastatic colorectal cancer that has not responded to prior treatments. The goal is to see if these drugs can shrink t…

This early-stage trial is testing an oral drug called LOXO-260 in about 70 people with advanced cancers that have a specific change in the RET gene. The cancers have stopped responding to other targeted treatments. The study aims to find the safest dose and see if the drug can sh…

This study offers voluntary screening for newborns in North Carolina to detect a wide range of rare health conditions early. Using a small blood sample already collected at birth, the program tests for dozens of disorders, including spinal muscular atrophy, cystic fibrosis, and m…

Li-Fraumeni syndrome is a rare genetic condition that greatly increases the risk of developing many types of cancer. This pilot study will test three advanced MRI techniques—whole body STIR MRI, DW-MRI, and PET-MRI—in 30 people with the condition to see how well they can detect v…

This study tests whether a special PET scan using [11C]acetate can detect early signs that the drug rapamycin is working in people with lymphangioleiomyomatosis (LAM), a rare lung disease. Seven adults with LAM and kidney tumors will get one or two scans over 3–6 months. The goal…

This study is developing a blood test to find early-stage tumors in people with hereditary cancer syndromes (high genetic risk for cancer). Researchers will collect blood samples and medical data from about 1,400 participants across Canada. They will also interview patients and d…

This study is testing whether whole body MRI scans can help find cancers early in children and adults with Li-Fraumeni syndrome, a genetic condition that greatly raises cancer risk. About 150 participants will receive annual whole body MRI scans for four years to see if they keep…

This large study is testing whether a new laser-based blood test can help screen for breast cancer. Researchers will compare blood samples from women with and without breast cancer to see if the test can accurately spot the disease. If it works, it could offer a less invasive way…

This study tests an advanced ultrasound otoscope, a device that uses sound waves to look inside the ear and detect fluid behind the eardrum. Researchers will compare its readings to the surgeon's findings during ear tube surgery in 300 children up to 12 years old. The goal is to …

This phase IIb trial tests whether a vaccine called Tri-Ad5, combined with an immune booster N-803, can prevent colon cancer in people with Lynch syndrome. The vaccine targets proteins found in precancer and cancer cells, aiming to train the immune system to destroy them. 186 par…

This phase II trial tests a drug called Diamyd, given as injections into lymph nodes, to see if it is safe and can prevent type 1 diabetes in children aged 8 to 17 who are at high risk. Only 5 participants are enrolled, and the study focuses on safety and whether the drug can sto…

This study tests a surgery called radical fimbriectomy in women with BRCA mutations who are at high risk for pelvic cancer but want to keep their ovaries. The procedure removes the fallopian tubes, where many of these cancers start, while leaving the ovaries to produce natural ho…

This study tests different programs to help people at high risk for cancer (such as those with BRCA or Lynch syndrome) or cancer survivors increase their physical activity and eat healthier. The goal is to see which programs work best to reduce cancer risk. About 337 participants…

This early-stage study tests an experimental vaccine designed to prevent cancer in adults with BRCA1 or BRCA2 gene mutations. The vaccine is given alone or with another drug, followed by a mild electric pulse to help it enter cells. Researchers want to see if it is safe and wheth…

This study tests whether taking aspirin can prevent ovarian cancer in women with BRCA1 or BRCA2 gene mutations, who are at high risk. About 117 participants will take either aspirin or a placebo daily for up to 2 years before their scheduled risk-reducing surgery. Researchers wil…

This study tests whether using a chlorhexidine rinse after bowel movements can reduce urinary tract infections (UTIs) in post-menopausal women who already use vaginal estrogen and have frequent UTIs. About 136 women aged 55-89 will be randomly assigned to use either the chlorhexi…

This phase 4 trial tests whether taking metformin daily before 20 weeks of pregnancy can reduce the risk of high blood pressure disorders, like preeclampsia, in pregnant women with type 1 diabetes. About 60 women will receive either metformin plus standard care or standard care a…

This study looks at two types of surgery to prevent ovarian cancer in women aged 30-50 who have gene mutations that raise their risk. One surgery removes both the fallopian tubes and ovaries, while the other removes only the tubes first and delays removing the ovaries. The goal i…

This study looks at two forms of vaginal estrogen—cream and tablet—to see which one post-menopausal women prefer for preventing recurrent urinary tract infections (UTIs). About 21 women who have had multiple UTIs will try both treatments and then choose their favorite. The goal i…

This study looks at whether showering once or twice with a special antiseptic soap before lumbar disc herniation surgery reduces the risk of surgical site infections. 86 participants will be randomly assigned to shower either the night before surgery or both the night before and …

This study looks at whether putting vancomycin powder directly into the joint during hip or knee replacement surgery can prevent infections better than standard IV antibiotics alone. About 1,800 adults having primary or revision joint replacement are being followed for two years.…

This study tests whether a specific ultrasound-guided nerve block can reduce pain after elective lower back surgery better than the usual method of injecting pain medicine into the wound. Adults aged 18-65 undergoing surgery for conditions like a herniated disc or spinal stenosis…

This study tests whether a targeted nerve block (retrolaminar block) provides better pain relief after lumbar discectomy than the standard method of injecting pain medicine into the surgical wound. 130 adults having elective single-level herniated disc surgery will be randomly as…

This study looks at whether auto-captioning glasses are easy and helpful for adults with NF2-related schwannomatosis who have hearing loss. Eighteen participants will use the glasses for 12 weeks and report their experience through surveys and interviews. The goal is to see if th…

This study tests whether monthly alexandrite laser treatments can safely shrink or improve the look of skin tumors in adults with neurofibromatosis type 1 (NF1). Ten participants will receive up to six monthly laser sessions, with some areas treated with cooling and others withou…

This study tests whether taking a special olive oil (high in a compound called oleocanthal) along with curcumin (the active part of turmeric) is safe and might help shrink skin tumors in adults with neurofibromatosis type 1 (NF1). About 23 participants take the supplements twice …

This study explores whether a supervised, circuit-based resistance training program can help glioblastoma patients maintain muscle mass and perform daily tasks more easily. Researchers will enroll 24 adults with glioblastoma who are currently on treatment. Participants will exerc…

This study looks at two different ways to manage pain after lower back surgery without relying on drugs. About 267 adults will be randomly assigned to either standard care or an enriched pain management plan. The goal is to see which approach better reduces pain and helps people …

This study tests a new treatment called BRC-OA, made from donated human fat tissue, for people with mild to severe knee osteoarthritis. The goal is to see if it safely reduces pain and improves knee function. About 42 adults aged 18-80 will receive one of two doses or a control, …

This study looks at 600 hip or knee replacement patients to compare three different wound closure products: Sylke adhesive dressing, Exofin skin glue, and Suture Strip Plus. The goal is to see which method causes the fewest allergic skin reactions and gives the best scar appearan…

This study looks at two ways to numb people for lower back surgery: general anesthesia (being fully asleep) plus a nerve block, or spinal anesthesia (numbing only the lower body) plus a nerve block. The goal is to see which approach helps patients recover better and feel less pai…

This study compares two common procedures for chronic back pain caused by a slipped disc or narrowed spine: a steroid injection and a nerve-targeting radiofrequency treatment. Researchers will track pain levels in 200 adults right after the procedure and three weeks later. The go…

This early-stage trial tests whether monthly injections of Kybella (a drug that dissolves fat) can safely shrink skin tumors in people with Neurofibromatosis Type 1. Up to 15 adults will receive up to 6 monthly treatments, and researchers will compare treated and untreated tumors…

This study tests whether a program that combines a smartphone app with face-to-face therapy sessions can improve psychological well-being in children and adolescents with type 1 diabetes. The goal is to reduce diabetes-related distress and help young patients better manage their …

This study looks at whether 8 weeks of Pilates exercises can help middle-aged women (45-60 years old) with long-term lower back pain from a herniated disc. Researchers will measure balance, ankle strength, and body awareness in 30 women. Half will do Pilates, and the other half w…

This study adapts a proven PTSD therapy (STAIR-NT) for people in methadone treatment who also use cocaine and opioids. Researchers will test if the adapted program is practical and acceptable, and whether it reduces substance use and PTSD symptoms. About 80 participants will be r…

This study was designed to see if two drugs, empagliflozin and anakinra, could help people with prediabetes who experience low blood sugar after eating. The trial planned to give a single dose of each drug before a test meal and measure symptoms. However, the study was withdrawn …

This study looks at whether a specific nerve block (called an erector spinae plane block) can improve pain control after surgery for a slipped disc in the lower back. Researchers will track pain scores, opioid use, how soon patients can walk, and how long they stay in the hospita…

This trial tests a 6-month peer mentorship program for adolescents with type 1 diabetes who struggle to use continuous glucose monitors (CGM). Participants are randomly assigned to standard care or the Type 1 Together program, which pairs them with mentor families and provides ed…

This study looks at whether virtual reality (VR) therapy or a simple deep rest relaxation technique can help older adults feel less stressed and recover better after hip or knee replacement surgery. About 90 people aged 60 and older who recently had surgery will try one of these …

This study tests a small device that delivers mild electrical stimulation to the perineum (the area between the scrotum and anus) to help men who leak urine after prostate removal surgery. Thirty men who are scheduled for robotic prostate surgery and already use at least one pad …

This study tests a 6-week group program called Working Out Dads (WOD) to see if it helps reduce mental health struggles in fathers of children aged 0-4. About 293 dads with mild to moderate distress will either join the WOD peer-support group or receive usual care. The goal is to…

This study tested whether shortwave diathermy, a treatment that uses heat deep in the tissue, can help people with hand osteoarthritis. 36 adults aged 35-75 with hand osteoarthritis received either real or placebo treatment. Researchers measured pain, grip strength, and daily han…

This study tests whether adding laser therapy to standard physiotherapy and exercise reduces knee osteoarthritis pain more than a sham (fake) laser plus exercise. Sixty-four adults with knee osteoarthritis will receive either real or sham laser treatments twice a week for six wee…

This study tests whether adding virtual reality (VR) to standard physical therapy helps people recover better after lumbar disc surgery. About 46 adults who had surgery on their lower back will either get standard therapy or standard therapy plus a 20-minute VR session. Researche…

This study tested a stem cell treatment for one person with severe, long-term knee pain from osteoarthritis who had no other options left. The patient received stem cells from their own fat, given both through a vein and directly into the knee joints. The goal was to see if the t…

This study tests a 6-week virtual group program that teaches parents strategies to encourage their toddler's vocalizations and shared smiling. The program is for children aged 12-42 months who have early signs or a diagnosis of autism or related social communication challenges. R…

This study tests whether adding high-intensity laser therapy to a standard exercise program can better reduce pain and improve function in people with thumb osteoarthritis. 48 participants with stage I-III thumb joint arthritis will receive laser treatment three times a week for …

This pilot study tests a short online program called 'Well-Beans for Caregivers' for adults caring for someone with tuberous sclerosis complex (TSC). The program consists of three weekly 2-hour sessions based on Acceptance and Commitment Therapy to help caregivers cope with diffi…

This study is developing and testing a mobile app that guides people with lumbar disc herniation through fascia exercises. Participants will be taught exercises by a physiotherapist and then use either the app, a home brochure, or standard lumbar stabilization exercises. The goal…

This study compares two types of nerve blocks—quadratus lumborum block and retrolaminar block—for pain after lumbar disc surgery. About 84 adults having elective back surgery will receive one of the two blocks before surgery. Researchers will measure pain scores, opioid use, and …

This study tests whether adding mental health care to standard diabetes treatment improves mood and blood sugar control in adults with type 1 diabetes. Eighty participants will be randomly assigned to usual care or a collaborative care model that includes a behavioral health cons…

This study tests whether offering online educational videos and mailed saliva genetic testing kits helps more first-degree relatives of people with BRCA mutations get tested, compared to standard care with a family letter. About 820 participants will be enrolled across several me…

This study follows 250 male endurance athletes who were part of an earlier study to see how their heart arteries have changed over time. Researchers will use CT scans and AI to examine plaque buildup and determine if it is stable and harmless or likely to cause heart attacks. The…

This study follows 154 people taking Epidiolex (cannabidiol) for seizures caused by Lennox-Gastaut syndrome, Dravet syndrome, or tuberous sclerosis complex. Researchers will monitor for signs of chronic liver injury and fibrosis over time using blood tests and liver scans. The go…

This observational study collects extra tumor tissue from 416 people with various cancers (bladder, kidney, colorectal, head and neck, liver, lung, skin, endometrial) during routine biopsies. The goal is to develop and train a 'live tumor diagnostic platform' that can predict whe…

This Phase 2 trial tests whether dichloroacetate (DCA), a drug that affects cell energy use, can alter tumor metabolism in people with recurrent glioblastoma. Forty patients scheduled for surgery will either receive DCA or no drug for one week before their operation. Researchers …

This natural history study follows 259 children, adolescents, and adults with Neurofibromatosis Type 1 (NF1) for up to 10 years. Researchers will track tumor growth, monitor quality of life, and perform genetic testing to better understand how the disease changes over time. The g…

This study looks at people and families who have a high risk of cancer to learn what genes and environmental factors might increase that risk. Over 5,000 participants are followed over time with questionnaires, medical records, and optional genetic testing. No treatment is given,…

This study follows 350 people who survived a brain bleed (intracerebral hemorrhage) and were part of earlier trials. Researchers will test memory, thinking, and movement skills over time, and collect blood samples to look for signs of ongoing inflammation. The goal is to see if s…

This study looks at how the menstrual cycle changes blood sugar and energy use in women with type 1 diabetes. About 40 women will use a closed-loop insulin pump and continuous glucose monitor to track these changes. The goal is to understand how hormone shifts affect insulin need…

This study tests a web-based tool called gp-multitool.de that helps general practitioners follow guidelines for older patients with multiple chronic conditions. About 659 patients from GP practices in Germany are taking part. The tool lets patients fill out questionnaires online …

This study is testing a digital health tool to help more people of Ashkenazi Jewish descent get tested for BRCA gene mutations, which increase cancer risk. Over 5,400 participants will use a web portal to learn about testing and decide whether to proceed. The goal is to see if th…

This study follows over 1,000 women in Singapore from before pregnancy through their child's early years. Researchers are looking at how nutrition, lifestyle, and emotional health before and during early pregnancy may influence the child's body composition, brain development, and…

This study looks at whether the natural balance of bacteria in the uterus affects embryo implantation and pregnancy success in women undergoing IVF. Researchers will analyze existing data from 80 women with a history of repeated IVF failure or pregnancy loss. No extra treatments …

This study looks at whether offering genetic testing to family members of people with hereditary breast and ovarian cancer syndrome helps more relatives learn about their own cancer risk. Researchers will follow 118 participants and their relatives to see how many choose to get t…

This study looks at whether a 25-minute hydration education session plus a self-assessment worksheet can help wildland firefighters and other active people drink more fluids and stay hydrated. About 38 participants who are underhydrated will be split into two groups: one gets the…

This study tests whether virtual peer groups can help Latinx teenagers with type 1 diabetes use insulin pumps and continuous glucose monitors more effectively. About 120 teens and their caregivers will join online sessions covering topics like family relationships, school, and wo…

This pilot study aimed to see if a diet low in two specific nutrients (cysteine and methionine) could affect metabolism in patients with recurrent gliomas. Up to 30 participants were to be randomly assigned to either a normal diet or the special diet for 2, 4, or 7 days before st…

This study evaluates a program that aims to improve diets by making healthy foods like fish, chicken, and eggs more available and affordable for low-income households in Mozambique. Researchers will survey over 5,000 men and women to see if the program increases how often they ea…

This study gathers health questionnaires from up to 1,500 people with multiple endocrine neoplasia (MEN1 or MEN2) and their close relatives. The goal is to build a database that helps researchers better understand these rare inherited conditions. No new treatments or procedures a…

This study is creating a web-based program to help people with hereditary cancer mutations share their genetic test results with family members. The goal is to increase awareness among at-risk relatives so they can take steps to prevent cancer. The program is designed for patient…

This study uses brain imaging to understand why some people with type 1 diabetes lose their ability to sense dangerously low blood sugar. Researchers will temporarily induce low blood sugar in participants and scan their brains to see how glucose levels change. The goal is to lea…

This study tests a program called Launch for young adults (ages 18-26) who misuse multiple drugs and their supportive parent. The program provides peer support to the young adult and helps parents use a reward-based system at home. The goal is to see if the program is practical a…

This pilot study examines proteins in the vitreous (the gel-like fluid inside the eye) of patients with pseudoexfoliation syndrome (PEX), a condition that can lead to glaucoma. Researchers will compare protein levels between eyes with and without PEX in 24 adults undergoing eye s…

This study collects health information and test results from 317 people at high risk for pancreatic cancer, including those with cysts, genetic syndromes, or other risk factors. Researchers aim to build a database to find better ways to detect the disease early or prevent it. No …

This observational study will follow 384 patients in Brazil who are using cannabis-based products (like CBD or THC oils) prescribed by their own doctors for chronic pain, anxiety, or depression. Researchers will not provide the medication but will track patients' quality of life,…

This study follows 500 people with urinary tract infections (UTIs) for up to 5 years. Researchers will collect health, lifestyle, and lab data to learn which treatments work best and how to reduce antibiotic resistance. The goal is to improve patient care and guide future studies…

This study is looking for genes that might make some people more likely to get an infection after hip or knee replacement surgery. Researchers will collect blood or saliva samples from 150 people in families with a history of such infections, plus unaffected relatives. The goal i…

This observational study at Mayo Clinic aims to understand how genetics and environment contribute to disease before it begins. Researchers will collect biological samples, voice recordings, and survey data from 100,000 adult Mayo Clinic patients. The goal is to build a comprehen…

This study monitors the safety of selumetinib in children aged 3 to 18 with neurofibromatosis type 1 and symptomatic, inoperable plexiform neurofibromas. Researchers will track potential side effects on the heart, bones, muscles, liver, eyes, and puberty. The goal is to better un…

This study looks at how diet and exercise affect the epigenome—chemical marks on DNA—in sperm of overweight Hispanic men. Researchers will compare sperm from 20 healthy active men and 80 obese inactive men before and after a 12-week program of diet, exercise, or both. They will a…

This study is a national registry collecting data from 1500 people with familial adenomatous polyposis (FAP) and related conditions across 28 Italian centers. It aims to better understand how these diseases progress, how they are currently managed, and what factors influence outc…

This study looks at whether a website and a genetic family navigator can help more relatives of people with hereditary cancer get genetic testing. About 205 participants will use the online hub and work with a navigator to learn about their cancer risk. The goal is to see if this…

This early study tests whether sirolimus, an FDA-approved drug, can improve blood flow to the brain in healthy adults with a genetic risk for Alzheimer's disease. Researchers will use MRI scans to measure changes after 4 weeks of daily low-dose sirolimus. The study also explores …

This study looks at whether short educational videos can help people with inherited cancer gene mutations learn more about their condition. About 43 adults who carry mutations in genes like BRCA1 or BRCA2 will watch the videos and then take a quiz. The goal is to see if this simp…

This study tests two simple, scalable methods to help people with inherited cancer risk follow through on recommended screenings. Researchers will provide online educational resources and personalized phone outreach to 900 participants. The goal is to see if these approaches impr…

This study looks at how a mother's stress, anxiety, and mood during pregnancy might influence her child's brain development and behavior. Researchers are following 587 families in Singapore, including mothers, fathers, and children. They use brain scans and developmental tests to…

This study follows about 1,400 pregnant women and their children for at least 4 years after birth. Researchers want to learn how stress, social support, and genetics affect a mother's mental health during pregnancy and her child's development. The study does not test any treatmen…

This study looks at whether watching an online genetics video, with or without phone counseling from a genetic counselor, can reduce the stress women feel about their cancer risk. It involves 5,200 women aged 30 or older who have had triple-negative breast cancer and still have a…

This study is testing a new smartphone app called ENHANCe that helps family doctors manage patients with high blood pressure using home blood pressure readings. The app scans home readings and gives the doctor a recommendation on how to adjust treatment. The study will enroll 116…

This study follows about 1,200 mothers from early pregnancy and their children for at least 20 years. Researchers want to learn how genetics, diet, lifestyle, and environment during pregnancy and early childhood affect the risk of metabolic and mental health conditions later in l…

This study looks at how bacteria living in the gut and diet might influence colorectal cancer risk in people with Lynch syndrome and other inherited colon conditions. Researchers will collect stool samples, colon biopsies, and diet questionnaires from 77 participants during routi…

This study looks at a specific genetic change found in some people with high blood pressure. Researchers want to see if this change is linked to a condition called primary aldosteronism, which can sometimes be cured with surgery. About 76 men from a larger Swedish health study wh…

This study looks at how a person's genes and lifestyle choices, like smoking, affect their risk of getting bladder cancer. Researchers will compare over 6,000 people with and without bladder cancer to find patterns. The goal is to better understand who is most at risk, which coul…

This study follows 80 people with Neurofibromatosis type 1 (NF1) who are at high risk for developing malignant peripheral nerve sheath tumors (MPNST). Researchers will use yearly whole-body MRI scans, blood samples, and checkups to track changes in nerve lesions over time. The go…

This study looks at whether people with Lynch Syndrome—a condition that raises the risk for several cancers—follow recommended screening guidelines after genetic counseling. Researchers will track about 1,530 participants to see who gets the suggested tests and what factors help …

This study tests whether providing an information letter plus a booklet helps relatives of people with Lynch syndrome decide about genetic counseling and testing. About 185 untested relatives will be randomly assigned to receive either a basic letter or a letter plus a detailed b…

This study follows 400 adults aged 65 and older with type 1 diabetes to understand how they age compared to people without diabetes. Researchers will measure physical ability, thinking skills, and emotional well-being. The goal is to learn what helps some people age well despite …

This study is testing a chatbot and online portal called AYA-RISE to help adolescents and young adults (ages 12-24) who have genetic conditions that raise their cancer risk. The goal is to see if the tool helps them communicate with family and doctors and make informed decisions …

This study looks at the complete DNA and RNA of 10 people and their families who are at high risk for hereditary cancer but have no known genetic cause. Researchers will use whole genome sequencing and tumor analysis to discover new genetic changes that may lead to cancer. The go…

This study looks at 800 people with a type of breast cancer called lobular breast cancer. Researchers are searching for inherited gene changes that may raise cancer risk. Depending on the gene found, patients may get closer monitoring or preventive surgery. The goal is to tailor …

This study tests whether a Patient Navigator can help 150 people with type 1 diabetes use their continuous glucose monitors more effectively. The goal is to reduce health disparities and improve outcomes. Participants will receive extra support while using their standard CGM devi…

This study tests whether a culturally-informed telephone counseling program can help Latina women at high risk for hereditary breast or ovarian cancer learn more about their risk and feel more in control. About 493 women with a personal or family history of these cancers will tak…

This study looks at whether a web-based educational program can help relatives of people with BRCA1 or BRCA2 mutations understand their genetic testing options. About 240 adults who are close relatives of someone with a BRCA mutation will take part. Participants will either use t…

This study will collect and analyze samples from 10,000 Mayo Clinic patients to understand how genes, environment, and lifestyle interact. Researchers will build a large database to find links between these factors and various diseases. The goal is to improve future medical care,…

This study follows 500 people with neurofibromatosis type 1 (NF1) to see how their skin tumors (called cutaneous neurofibromas) grow and change over time. Researchers use 3D whole-body photography to track tumor number and size each year. The goal is to better understand these tu…

This study checks how well the immune system works in healthy people with BRCA1 or BRCA2 gene mutations. About 50 adults over age 25 who carry these gene changes but have not had cancer will get a seasonal flu vaccine. Researchers will measure their antibody and immune cell respo…

This study aimed to see if thyroid surgery changes the thickness of the carotid artery, which is a sign of heart disease risk. It planned to measure artery thickness before and after surgery in adults with various thyroid conditions. However, the study was withdrawn before enroll…

The IMPACT study is an international effort to screen men aged 40-69 who carry BRCA1, BRCA2, or mismatch repair gene mutations, which raise their risk of prostate cancer. Participants receive annual PSA tests and optional biopsies if levels are high. The goal is to learn how ofte…

The IMPACT Study is testing two online tools—GeneSHARE and LivingLabReport—to help people with inherited cancer gene variants share their results with family members and follow cancer screening guidelines. The study involves 720 adults who have a genetic variant linked to cancers…

This study aims to find out how often a blood clotting disorder called thrombophilia is the cause of recurrent pregnancy loss. Researchers will review the medical records of 1,000 women aged 18-40 who have had two or more miscarriages. The goal is to improve awareness and help do…

This study looks at whether yearly MRI scans can find new head and neck or brain cancers early in people who had hereditary retinoblastoma and were treated with radiation. Researchers will follow 88 participants for 10 years to see if early detection leads to better surgery outco…

This study aimed to see if a rapid whole genome sequencing test on amniotic fluid could give faster genetic diagnoses for fetuses with suspected genetic diseases. Researchers planned to compare results from amniotic fluid and blood samples from up to 90 families. However, the stu…

This study follows over 3,000 Black people with HIV in the UK to understand how genes and lifestyle affect their risk for kidney disease, heart disease, diabetes, and obesity. Researchers collect health data and blood/urine samples to find genetic markers that predict these condi…

This study aims to find blood-based biomarkers that signal the presence and size of plexiform neurofibromas in people with Neurofibromatosis type 1 (NF1). Researchers will collect blood samples and use whole-body MRI to compare biomarker levels in 200 participants with different …