Regeneron Pharmaceuticals

This study tests a new gene therapy called DB-OTO for children with hearing loss caused by changes in the otoferlin gene. The treatment is injected into the inner ear to help restore hearing. Up to 30 children and infants will take part to check safety and how well it works.

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

This study tests a new drug, linvoseltamab, alone or with another drug called carfilzomib, against standard treatments for people with multiple myeloma that has returned or not responded to prior therapy. About 915 participants who have had 1 to 3 prior treatments will be enrolle…

This early-stage study tests BPX-601, a personalized CAR T cell therapy made from a patient's own immune cells, for advanced prostate cancer that has spread and stopped responding to other treatments. The trial will enroll 36 people to find the safest dose and check for side effe…

This study tests whether a weekly dose of alirocumab (Praluent) is safe and effective for adults with high cholesterol. Alirocumab is already approved for use every two weeks, but this trial explores if giving it weekly works better. About 420 participants will receive different …

This expanded access program offers continued treatment with dupilumab to adults who have severe IgE-mediated food allergies and were previously treated with linvoseltamab and dupilumab in an earlier study. The goal is to provide ongoing access to the medication for those who com…

This Phase 2a trial is testing a new drug called ALN-PNP, alone or with a weight-loss drug (tirzepatide), in 204 adults with a specific genetic form of fatty liver disease (MASLD). The goal is to see if lowering a certain liver protein can reduce liver fat. Participants must have…

This Phase 2 trial tests two experimental drugs, ALN-ANG3 and evinacumab, in 270 people with diabetic kidney disease. The drugs block a protein called ANGPTL3 to lower blood fats. Researchers will measure changes in urine protein and kidney function over time to see if the drugs …

This study is the first time REGN20423 is being tested in humans. It will enroll 144 adults, first healthy volunteers and then people with atopic dermatitis (eczema). The goal is to check safety, side effects, how the drug moves through the body, and whether it helps eczema sympt…

This Phase 2 trial tests whether adding fianlimab to cemiplimab works better than cemiplimab alone for people with advanced head and neck cancer that has come back or spread. About 120 participants whose tumors have a specific marker (PD-L1 positive) will receive either the drug …

This phase 3 trial tests an experimental drug called odronextamab for people with previously untreated follicular lymphoma, a slow-growing blood cancer. The study compares odronextamab alone or with standard chemotherapy to see if it leads to better remission rates. About 822 par…

This phase 2 trial is testing an experimental drug called ubamatamab, alone or combined with other cancer drugs, in 220 people with advanced ovarian, fallopian tube, or primary peritoneal cancer that has stopped responding to platinum-based chemotherapy. The study aims to see how…

This study tests two experimental drugs, REGN7508 and REGN9933, to prevent strokes in people with atrial fibrillation (an irregular heartbeat) who cannot or choose not to take standard blood thinners. About 2,628 participants will receive one of the drugs or a placebo to see if t…

This study tests a new drug called REGN5678, given alone or with another drug (cemiplimab), in men with advanced prostate cancer that no longer responds to hormone therapy. The goal is to see if the combination can safely shrink tumors by helping the immune system attack cancer c…

This study tests an experimental drug called ALN-SOD in people with ALS caused by a mutation in the SOD1 gene. The drug is given via spinal injection to target the root cause of the disease. The main goal is to check safety, but researchers will also measure effects on biomarkers…

This study tests an experimental drug called REGN13335 in 99 adults with pulmonary arterial hypertension (PAH), a condition where high blood pressure in the lungs makes the heart work harder. Participants will receive either the drug or a placebo, on top of their usual PAH medica…

This study tests whether a new combination of drugs (fianlimab and cemiplimab) works better than an already approved treatment (Opdualag) for people with advanced melanoma that cannot be removed by surgery or has spread. About 560 adults with previously untreated melanoma will re…

This study tests a new experimental drug, REGN7945, combined with another experimental drug, linvoseltamab, in people with multiple myeloma that has returned or not responded to at least three prior treatments. The goal is to see if the combination is safe and can shrink tumors b…

This study tests an experimental drug called linvoseltamab in 220 people with AL amyloidosis that has returned or not improved after prior therapy. The drug aims to reduce abnormal proteins that damage organs like the heart and kidneys. The trial first finds the safest dose, then…

This study is testing the long-term safety and effectiveness of two experimental drugs, pozelimab and cemdisiran, taken together for paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder. About 202 participants who completed a prior study will continue treatment to see…

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

This study tests an experimental drug called REGN7508 (cenvacibart) against aspirin to prevent blood clots in 2000 adults having knee replacement surgery. The drug targets a clotting factor to reduce clot risk. Researchers will compare how many people develop clots or die from cl…

This study tests an experimental drug called mibavademab in 34 women with Functional Hypothalamic Amenorrhea (FHA), a condition where periods stop due to stress or intense exercise. The goal is to see if the drug can help the body produce the hormones needed for ovulation and imp…

This early-stage study tests an experimental drug called pozelimab, given as an injection into the eye, for people with geographic atrophy (GA) — a condition where parts of the retina stop working, leading to vision loss. The main goal is to check the drug's safety and tolerabili…

This study tests two experimental drugs, REGN7508 and REGN9933, in about 1,200 people with atrial fibrillation (an irregular heartbeat). The goal is to see if these drugs can prevent blood clots and strokes with less bleeding compared to the standard blood thinner apixaban. Parti…

This study tests two experimental drugs, pozelimab and cemdisiran, together in 35 adults with paroxysmal nocturnal hemoglobinuria (PNH) whose red blood cell destruction is not well-controlled by current C5 inhibitors. The goal is to see if the combination can lower hemolysis (bre…

This study tests an experimental drug called ALN-CFB in people with paroxysmal nocturnal hemoglobinuria (PNH) who still have low red blood cells despite standard treatment. The goal is to see if adding ALN-CFB is safe and can improve anemia. About 24 participants will receive eit…

This study tests an experimental drug called REGN7508 (cenvacibart) to prevent venous thromboembolism (blood clots) in adults after elective total knee replacement. About 2,000 participants will receive either REGN7508, apixaban, or enoxaparin to compare effectiveness and safety.…

This study tests an experimental drug called REGN5093 in people with advanced non-small cell lung cancer that has a specific change in the MET gene. The first part finds a safe dose, and the second part checks if the drug can shrink tumors. About 231 participants will take part.

This early-stage trial is testing two experimental drugs, vonsetamig and odronextamab, in people with lupus nephritis that has not responded to standard treatments. The main goal is to check safety and side effects over the long term. Researchers will also see if the drugs can he…

This study tests the drug dupilumab in children as young as 6 months who have active eosinophilic esophagitis (EoE), a condition that causes inflammation in the esophagus and can make eating difficult. The goal is to see if the drug is safe and effective over 24 weeks. About 20 c…

This phase 3 trial is testing two experimental drugs, REGN7508 and REGN9933, to prevent serious blood clots in people with peripheral artery disease who recently had leg surgery to improve blood flow. The study will enroll about 7,050 participants and compare the drugs to a place…

This early-phase study tests a new experimental drug, REGN17372, combined with another drug called linvoseltamab in people with relapsed or refractory multiple myeloma (cancer that has returned or stopped responding to treatment). The main goals are to check safety, find the best…

This Phase 2 trial is testing an investigational drug called linvoseltamab in 116 people with precancerous blood conditions that raise their risk of developing multiple myeloma. The goal is to see if the drug can eliminate abnormal plasma cells and lab signs of disease. Researche…

This phase 2 trial is testing two experimental drugs, ubamatamab and REGN7075, alone or together, in 300 people with advanced or metastatic non-small cell lung cancer who have already had standard treatments. A third drug, sarilumab, may be used to manage immune-related side effe…

This phase 3 trial tests an experimental drug called odronextamab, given together with standard chemotherapy (CHOP), for people with diffuse large B-cell lymphoma who have not been treated before or whose cancer has come back. The study compares this new combination to the curren…

This phase 3 trial compares an experimental drug, linvoseltamab, to an existing drug, daratumumab, in 270 people with smoldering multiple myeloma at high risk of developing active multiple myeloma. The goal is to see if linvoseltamab can delay the disease longer. Participants wil…

This study tests an experimental drug, REGN7508, to see if it works better than the standard blood thinner apixaban for treating and preventing blood clots in people with cancer. The trial will include about 1,600 participants with or without cancer who have deep vein thrombosis …

This study tests an experimental gene insertion therapy called REGV131-LNP1265 for people with severe or moderately severe hemophilia B. The therapy uses CRISPR/Cas9 to add a working copy of the clotting factor IX gene into the body's DNA, aiming to enable long-term production of…

This early-stage trial tests an experimental drug called REGN5837 combined with the approved drug odronextamab for people with aggressive B-cell lymphoma that has come back or not responded to prior treatments. The study first finds a safe dose, then checks how well the combo wor…

This Phase 2 trial tests a drug called ALN-HSD, which uses gene silencing to target liver scarring in people with MASH, a fatty liver disease that can lead to cirrhosis. The study enrolls 120 adults with genetic risk factors and moderate-to-severe liver fibrosis. Researchers will…

This study tests an experimental drug called linvoseltamab in people newly diagnosed with multiple myeloma, a type of blood cancer. The drug is designed to help the immune system attack cancer cells. The trial will check for side effects and see if the drug can shrink tumors. Abo…

This study is testing a new drug called ubamatamab, given alone or with another drug called cemiplimab, in people with ovarian cancer or other cancers that have come back after treatment. The main goals are to check safety, find the right dose, and see if the drugs can shrink tum…

This study tests two experimental drugs, REGN4336 and REGN5678, in men with metastatic castration-resistant prostate cancer that has worsened despite prior treatment. The first part finds safe doses, and the second part checks how well the combination shrinks tumors. About 228 pa…

This phase 3 trial tests an experimental drug, odronextamab, combined with lenalidomide in people with follicular or marginal zone lymphoma that has returned or not responded to treatment. The study first checks safety and dosing, then compares the new combo against the standard …

This study tests a new drug called mibavademab for people with generalized lipodystrophy, a rare condition where the body lacks fat tissue. The goal is to see if the drug can improve blood sugar, lower blood fats, and reduce liver fat. About 28 children and adults will take part …

This study tests an experimental drug called REGN10597, alone or with another drug (cemiplimab), in people with advanced solid tumors like melanoma and kidney cancer. The goal is to see if the treatment is safe and can shrink tumors. About 240 participants will receive the drug i…

This study tests an experimental drug called ALN-CIDEB in adults with fatty liver conditions (MASLD or MASH). The main goal is to check safety and tolerability, and also to see if the drug can reduce liver fat. About 132 participants will receive either the drug or a placebo. Thi…

This study tests a new drug called linvoseltamab in people with multiple myeloma that has returned or stopped responding to other treatments. The goal is to find the safest and most effective dose and to see if the drug can shrink or control the cancer. About 387 participants wil…

This study tests an experimental drug called ALN-SNCA in 46 people with early Parkinson's disease. The drug is designed to lower levels of a harmful protein, alpha-synuclein, in the brain. The main goal is to check if the drug is safe and tolerable, and to see how it moves throug…

This study tests an experimental drug called ALN-PNP in healthy adults and people with a liver condition called MASLD who have a specific gene variant. The main goal is to check safety and how the body handles the drug. It is a first-in-human trial, so the drug is not yet approve…

This study tests a drug called pozelimab in children aged 1 to 5 who have CHAPLE disease, a very rare genetic condition that causes severe stomach and heart problems. The main goal is to see if the drug is safe and how it affects the body. Only 5 children will take part, and the …

This study tests a new drug called marlotamig (REGN7075) alone or with cemiplimab (Libtayo) and sometimes chemotherapy in people with advanced solid tumors. The goal is to find the safest dose and see if the combination can shrink tumors. About 933 participants are being recruite…

This program provides early access to ubamatamab for people with two rare and aggressive cancers: renal medullary carcinoma and epithelioid sarcoma. The drug is given on a case-by-case basis to those who have no other treatment options. The goal is to offer a potential treatment …

This program provides expanded access to REGN4461 (mibavademab) for people with rare diseases caused by deficient leptin signaling, such as generalized lipodystrophy and monogenic obesity. The drug aims to help control metabolic issues linked to these conditions. Participants rec…

This study tests an experimental drug called mibavademab in people with a rare form of obesity caused by a faulty leptin gene. The drug aims to help control hunger and body weight. Only 4 participants will be enrolled to see how well it works and if it is safe.

This phase 3 study tests a new drug, linvoseltamab, against a standard three-drug combo (EPd) in people with multiple myeloma that has returned after 1-4 prior treatments. About 410 adults will be randomly assigned to receive either linvoseltamab or EPd. The goal is to see which …

This study tests whether adding a third drug to standard immunotherapy (cemiplimab) plus chemotherapy works better for people with early-stage non-small cell lung cancer who can have surgery. About 120 adults will receive treatment before and after their operation. The main goal …

This early-phase trial tests an experimental treatment called 27T51, a type of immune cell therapy (CAR T) designed to attack cancer cells that carry a protein called MUC16. The study includes about 90 women with ovarian, peritoneal, or fallopian tube cancer that has come back or…

This study tests an experimental drug called linvoseltamab combined with other cancer treatments for people with multiple myeloma that has come back after previous therapy. The main goal is to see if the combinations are safe and to find the right dose. About 317 participants wil…

This program provides compassionate use of odronextamab for people with relapsed or refractory follicular lymphoma, diffuse large B-cell lymphoma, or other B-cell non-Hodgkin lymphomas who have no other treatment options. Odronextamab is a drug that helps the immune system attack…

This program provides emergency access to REGN-EB3, a combination of three antibodies, for people with confirmed Ebola virus disease. It is available to both adults and children in Ebola-affected regions, including pregnant women. The goal is to offer a potential treatment option…

This study tests an experimental drug called REGN5668, alone or combined with other drugs, in people with ovarian, fallopian tube, peritoneal, or endometrial cancer. The trial has two parts: first to find the safest dose, then to see if the drug shrinks tumors. About 612 particip…

This study tests an experimental imaging agent (89Zr-DFO-REGN5054) that may help visualize immune cells called CD8+ T cells in tumors. About 50 people with advanced solid cancers will receive the imaging agent along with the immunotherapy drug cemiplimab. The main goal is to chec…

This phase 3 trial tests an experimental drug called REGN7508 to prevent blood clots in people with solid tumors who are receiving cancer treatment. About 1,120 participants will receive either the drug or a placebo. The study measures how well the drug prevents clots and checks …

This study tests two experimental drugs (REGN5713 and REGN5715) given alone or together to reduce eye allergy symptoms caused by birch pollen. About 350 adults with birch pollen allergy will receive the drugs or a placebo and then be exposed to pollen in a controlled setting. The…

This study follows 500 European patients with advanced non-small cell lung cancer who are already prescribed cemiplimab (Libtayo) as part of their normal care. Researchers will track how long patients live, how well the cancer responds, and any side effects. The goal is to see if…

This study is a long-term follow-up for 20 people with chronic kidney disease who received an experimental drug called vonsetamig and then had a kidney transplant in an earlier study. No new treatment is given here. Researchers will track how the drug affects the body and monitor…

This study watches how people with eosinophilic esophagitis (EoE) use the medicine Dupixent in everyday care. Researchers will collect information from about 350 patients to learn more about the disease and how treatment affects symptoms and quality of life. No new treatments are…

This study follows up to 50 people who previously received gene-modified T cell therapy in another trial. No new treatment is given. Researchers will check for delayed side effects, how long the modified cells last, and overall health. The goal is to better understand long-term s…