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Researchers at Emory University are testing a new combination of medicines for children whose cancer has come back or stopped responding to previous treatments. The study combines three drugs: sirolimus (which slows cell growth), celecoxib (an anti-inflammatory), and two chemotherapy drugs given in low doses. The goal is to see if this combination works better than standard treatments for solid tumors and brain cancers in children.
WHY IT MATTERSThis trial offers a potential new treatment option for children with recurrent or refractory cancers who have limited alternatives, though enrollment is currently closed.
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Researchers are testing a combination of two immunotherapy drugs in patients with four types of rare cancers: neuroendocrine tumors, biliary tract cancers, ovarian clear cell carcinoma, and tumors with high microsatellite instability. This Phase 2 trial involves 240 patients and is based on earlier research showing these cancers may respond well to this type of treatment.
WHY IT MATTERSPatients with these rare cancers now have access to a targeted immunotherapy combination that showed promise in earlier studies, potentially offering a new treatment option for cancers that historically have limited therapeutic choices.
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Researchers are looking for 1,500 people with rare blood fat disorders to join a long-term study. The study will track how these conditions change over time and how people's bodies respond to different treatments. This research is sponsored by the National Heart, Lung, and Blood Institute, a major government health organization.
WHY IT MATTERSThis natural history study will help doctors better understand rare lipid disorders that are often genetically inherited, potentially leading to improved treatments and monitoring strategies for patients with uncommon dyslipidemias.
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Researchers are testing new cancer treatments for people with rare types of ovarian cancer that have come back or didn't go away after initial treatment. The study will test different medicines based on specific genetic markers found in each patient's tumor. About 176 patients will participate, and the trial is being run by Roche, a major pharmaceutical company.
WHY IT MATTERSThis trial offers personalized treatment options for patients with recurrent or persistent rare epithelial ovarian cancers, where standard treatments have failed or stopped working—a situation with very limited options.
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Researchers are looking for pregnant women or women planning to get pregnant who have rare blood vessel diseases like Hereditary Hemorrhagic Telangiectasia, Marfan syndrome, lymphedema, or arteriovenous malformations. This study will track their pregnancies to understand what complications might happen for the mother and baby, since doctors don't have good information about this yet.
WHY IT MATTERSThis is the first large study to prospectively track pregnancy outcomes in women with these rare vascular diseases, filling a critical gap in medical knowledge that could directly improve care and safety for pregnant patients with these conditions.
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Researchers are recruiting 2,000 people with rare genetic disorders to participate in a study using a new technology called genome sequencing. This technology reads a person's entire genetic code to find which gene is causing their disease. The goal is to help doctors better diagnose and treat patients with rare conditions that are hard to identify with current testing methods.
WHY IT MATTERSIf you or a family member has an undiagnosed rare disorder affecting development or birth defects, this trial could identify the genetic cause—potentially opening doors to targeted treatment and genetic counseling for relatives.
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Researchers in France are looking for 1,000 families with children who have intellectual disabilities or autism caused by genetic changes. Families will answer questions online about their child's health and development to help doctors better understand these conditions and create personalized treatment plans in the future.
WHY IT MATTERSThis study directly recruits families affected by genetic intellectual disability and autism spectrum disorder to build a database that could lead to personalized medicine approaches for these conditions.
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Researchers are testing a new drug called zipalertinib to treat advanced lung cancer in patients who have a specific genetic mutation called EGFR exon 20 insertion. This mutation makes lung cancer harder to treat with standard medications. The study is looking for 220 patients to see if zipalertinib is safe and effective for this type of cancer.
WHY IT MATTERSThis trial targets EGFR exon 20 insertion mutations in advanced NSCLC, a genetic subtype that historically has limited treatment options and poor response to standard EGFR inhibitors.
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Researchers are testing whether two cancer-fighting drugs called atezolizumab and bevacizumab work better together for treating rare cancers. These drugs help the body's immune system recognize and attack cancer cells. The study includes 133 patients with various uncommon solid tumors and is currently enrolling participants.
WHY IT MATTERSThis trial offers patients with rare cancers like Merkel cell carcinoma, nasopharyngeal cancer, and mesothelioma access to a combination immunotherapy treatment that may not be available outside of clinical research.
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Researchers at Vanderbilt University are studying how the nervous system controls blood vessels in the abdomen and how this might cause high blood pressure in people with certain rare nervous system disorders. The study focuses on three conditions where the nervous system doesn't work properly: Multiple System Atrophy, Orthostatic Hypotension, and Supine Hypertension. By understanding this connection, doctors may find new ways to treat high blood pressure in these patients.
WHY IT MATTERSThis trial directly addresses a poorly understood cause of high blood pressure in patients with Multiple System Atrophy and related autonomic nervous system disorders, potentially leading to better treatment options for this hard-to-manage symptom.
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Researchers are testing a new drug called furmonertinib in patients with advanced lung cancer that has specific genetic mutations. This is an early-stage study (Phase 1) with 160 patients to see if the drug is safe and how well it works. The drug targets uncommon mutations in EGFR and HER2 genes that some lung cancer patients have.
WHY IT MATTERSThis trial addresses treatment options for patients with advanced NSCLC carrying uncommon EGFR or HER2 mutations, which typically have fewer targeted therapy options than common mutations.
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Researchers completed a study testing two cancer drugs given together: imatinib mesylate and cyclophosphamide. The study involved 26 patients and aimed to find the safest and most effective dose of imatinib mesylate when combined with a fixed dose of cyclophosphamide. This was an early-stage study (Phase 1) to understand how these drugs work together in the body.
WHY IT MATTERSThis completed trial provides important safety and dosing information for doctors considering combination therapy with imatinib and cyclophosphamide for certain cancer patients, potentially improving treatment options.
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Researchers are testing a new way to help young people with rare bone diseases get better. The program combines sailing therapy (being out on the water) with online rehabilitation exercises that patients can do at home. This study builds on earlier successful results and will compare two different treatment approaches to see which works best.
WHY IT MATTERSThis trial offers young patients with rare skeletal disorders access to an innovative rehabilitation model combining in-person sailing therapy with remote maintenance care, potentially improving outcomes beyond traditional therapy alone.
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Researchers are testing a new cancer drug called CLR 131 in children and young adults whose cancers have come back or stopped responding to standard treatments. The study includes kids with several types of hard-to-treat cancers like neuroblastoma, sarcomas, and brain tumors. This is an early-stage study to figure out the right dose and see if the drug is safe and effective.
WHY IT MATTERSThis trial offers a potential treatment option for children with relapsed or refractory neuroblastoma, sarcomas, and other pediatric solid tumors where standard treatments have failed—conditions with very limited curative options.
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Researchers are testing a new experimental cancer treatment called BNT326 to see if it is safe and works well for people with advanced solid tumors (cancers that have spread or come back after treatment). The study will first test BNT326 alone, then test it combined with other experimental treatments. About 980 people will participate in this Phase 1 and Phase 2 trial.
WHY IT MATTERSThis trial is actively recruiting patients with advanced solid tumors across multiple sites, offering access to a novel immunotherapy approach from BioNTech before it becomes widely available.
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Doctors are building a national database to collect information from 500 patients with pulmonary alveolar proteinosis (PAP), a rare lung disease. This registry will help researchers better understand PAP, improve how doctors diagnose it, and test new treatments. Patients who join will have a voice in deciding what research gets done.
WHY IT MATTERSThis is an active recruitment opportunity for PAP patients to directly influence research priorities and gain access to new diagnostic tests and experimental therapies being evaluated through the registry.
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Researchers are looking for 20,000 people with rare diseases that doctors haven't been able to diagnose yet. Many patients spend years going to different doctors and getting repeated tests without finding answers. This study, run by the National Human Genome Research Institute, aims to help solve medical mysteries by using genetic testing and careful evaluation to figure out what's causing people's symptoms.
WHY IT MATTERSIf you or a family member has spent years seeking a diagnosis without answers, this large national study offers free genetic evaluation and expert medical review specifically designed to identify undiagnosed rare diseases.
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Researchers at the National Heart, Lung, and Blood Institute are looking for 1,000 people to join a study about heart and blood vessel diseases that run in families. They want to collect information and blood samples from people who have these genetic diseases, their relatives, and healthy volunteers to better understand how genes affect the heart and blood vessels. This study is open to anyone age 2 and older who may have a genetic condition affecting their heart or blood vessels.
WHY IT MATTERSThis is a large-scale discovery study actively recruiting now that could help identify new genetic causes of inherited heart and vascular diseases, potentially leading to earlier diagnosis and treatment options for affected families.
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Researchers are looking for people with melorheostosis, a rare disease where bones become abnormally thick and hard, to join a study. The study will follow participants over time to understand how the disease develops and what causes it. Doctors will do physical exams and collect medical information from people with the disease and their unaffected family members.
WHY IT MATTERSThis is an active recruiting trial sponsored by the National Institutes of Health that could help researchers understand melorheostosis progression and identify disease mechanisms, potentially leading to future treatments for this condition with limited research.
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Researchers are testing a combination of two cancer-fighting treatments called talimogene laherparepvec and nivolumab in patients with rare lymphomas (blood cancers) and skin cancers that didn't respond to previous treatments. Talimogene laherparepvec is a modified virus that helps the immune system fight cancer cells, while nivolumab is an immunotherapy drug that removes the brakes on the immune system. This study involves 68 patients and is currently enrolling participants.
WHY IT MATTERSThis trial offers a potential new treatment option for patients with refractory (treatment-resistant) rare lymphomas like mycosis fungoides and Merkel cell carcinoma, which have very limited options after standard therapies fail.