Novartis Pharmaceuticals

This study tests a new drug called tulmimetostat combined with standard hormone therapies (darolutamide or abiraterone) in men whose prostate cancer has spread but still responds to hormone treatment. The goal is to see if the combination is safe and works better than standard ca…

This study tests whether adding a radioactive drug (177Lu-DOTA-TATE) to standard hormone therapy can slow tumor growth in people newly diagnosed with advanced neuroendocrine tumors of the gut or pancreas. About 240 participants will receive either the combination or standard ther…

This study looks at the long-term safety of a drug called pelacarsen in people with high levels of lipoprotein(a) and heart disease. About 5,700 adults who finished a previous pelacarsen trial will take the drug and be monitored for side effects. The goal is to see if pelacarsen …

This program provides access to the drug asciminib for people with chronic-phase chronic myeloid leukemia who have no other treatment options and cannot join a clinical trial. Doctors can request the drug for their patients if they meet the criteria. The goal is to offer a potent…

This study is the first time the experimental drug OJR520 is being tested in humans. It aims to check the drug's safety and how the body processes it in both healthy volunteers and people with chronic kidney disease. The trial is currently recruiting 112 participants and is in Ph…

This study tests a new gene therapy called DFT383 in children aged 2 to 5 with nephropathic cystinosis, a rare disease that damages the kidneys and other organs. The treatment aims to fix the genetic problem and reduce the need for current medications. About 30 children will take…

This early-stage study tests a new drug that delivers radiation directly to cancer cells with a protein called DLL3. It is for adults with advanced lung, prostate, or gut cancers that have not responded to other treatments. The main goals are to check safety, find the right dose,…

This study looks at how the body processes secukinumab given through an IV in children aged 2 to 17 with juvenile psoriatic arthritis. Researchers will measure drug levels in the blood and monitor side effects over up to 6 years. The goal is to understand the drug's safety and ho…

This study tests if a monthly injection called pelacarsen can slow the worsening of aortic valve stenosis, a condition where the heart valve becomes stiff and narrow. It includes 502 adults aged 50-79 with high levels of Lp(a), a type of fat in the blood, and mild-to-moderate val…

This study tests if starting inclisiran early in the hospital can lower bad cholesterol more than a placebo in people who just had a heart attack. About 300 adults will receive either inclisiran or a placebo alongside standard care. The goal is to see if this approach helps contr…

This study follows 500 men with metastatic castration-resistant prostate cancer who are receiving a radioactive drug called lutetium (177Lu) vipivotide tetraxetan. Researchers want to see how the treatment affects patients' quality of life, pain, and cancer progression in everyda…

This study tests a new drug called LTP001 for pulmonary arterial hypertension (PAH), a condition where high blood pressure in the lungs makes it hard to breathe. First, healthy volunteers will check the drug's safety. Then, people with PAH will see if it lowers lung pressure and …

This study watches how well the drug Ilaris works and how safe it is for children and adults with rare hereditary fever syndromes (like CAPS, FMF, TRAPS, HIDS/MKD) and a type of childhood arthritis called sJIA. About 25 people will be followed during their regular treatment. The …

This study tests an experimental drug called NIO752 in 300 people with progressive supranuclear palsy (PSP), a rare brain disorder that affects movement, balance, and speech. Participants will receive either the drug or a placebo for a period, followed by an open-label phase wher…

This study tests an experimental drug called remibrutinib in 555 people aged 12 and older with moderate to severe hidradenitis suppurativa (HS), a condition causing painful boils and tunnels under the skin. Participants will receive one of two doses of the drug or a placebo for 6…

This study monitors long-term safety in 1,000 people with autoimmune or inflammatory diseases who have already completed a Novartis trial with secukinumab and still need the drug. Participants continue receiving secukinumab because their doctor believes it helps and they cannot g…

This study follows about 3,280 adults with chronic spontaneous urticaria (CSU) to see how well remibrutinib controls their hives in real-world settings. Participants either continue standard antihistamines or switch to remibrutinib, as decided by their doctor. The main goal is to…

This study tests a new drug called AAA817 against standard treatments for men with a specific type of advanced prostate cancer (PSMA-positive) that has worsened after prior therapies, including a targeted radiation drug. About 443 adult participants will be randomly assigned to r…

This study tests a combination of two targeted drugs, dabrafenib and trametinib, in people with rare solid tumors that have a specific genetic change (BRAF V600E) and cannot be removed by surgery or have spread. About 40 children and adults will receive the drugs to see how well …

This study follows 30 children aged 1 to 18 with chronic heart failure who are taking Entresto for the first time. Researchers will monitor safety issues like low blood pressure, high potassium, kidney problems, and dehydration over 52 weeks. The goal is to understand how safe th…

This study tests whether the drug remibrutinib can slow disability worsening in people with secondary progressive multiple sclerosis (SPMS). About 1,275 adults aged 18-65 with SPMS will receive either the drug or a placebo. The main goal is to see if remibrutinib delays confirmed…

This study tests a new drug called ianalumab in 200 adults with a severe form of scleroderma that affects the skin and internal organs. Participants receive either the drug or a placebo to see if it improves disease activity and lung function over 52 weeks. The goal is to find a …

This study tests a new drug called LXE408 in 130 adults with chronic Chagas disease who do not have severe organ damage. The goal is to see if the drug can clear the Chagas parasite from the blood for at least 6 months. Participants will receive either LXE408 or a placebo, and ne…

This study tests whether adding the drug ribociclib to standard hormone therapy can help prevent breast cancer from returning in people with high-risk, hormone receptor-positive, HER2-negative early breast cancer. About 1,400 adults will receive the combination treatment for seve…

This study watches 600 adults with asthma who are prescribed the Enerzair inhaler as part of their normal care. Over 24 weeks, doctors track side effects and how well the medicine controls asthma symptoms. The goal is to confirm the inhaler works safely in everyday use.

This study is testing how well the drug asciminib works for people with a type of leukemia called chronic myeloid leukemia (CML). It includes both newly diagnosed patients and those who have already tried one other treatment. The goal is to see if asciminib can control the diseas…

This study tests an experimental drug called EYU688 in 108 adults with dengue fever. The goal is to see if it can lower the amount of virus in the blood and shorten the time fever lasts compared to a placebo. Participants must have had a fever for less than 48 hours and test posi…

This study is for men with a certain type of advanced prostate cancer that has spread and no longer responds to hormone therapy. It tests a radioactive drug called lutetium-177 vipivotide tetraxetan to see how the body handles it when kidneys are working normally or are impaired.…

This study is for people with chronic myeloid leukemia who are already taking the drug asciminib and benefiting from it. The goal is to monitor long-term safety and side effects while they continue treatment. About 347 participants will be followed, and doctors will also check if…

This early-phase study tests a new combination treatment for people with a certain type of advanced breast cancer (ER+, HER2-, GRPR+) that has stopped responding to standard hormone therapy. The treatment adds a targeted radiation drug ([177Lu]Lu-NeoB) to two existing hormone-blo…

This program provides access to the drug VAY736 (ianalumab) for patients with serious conditions like primary immune thrombocytopenia or Sjögren's syndrome who have no other treatment options. It is for patients who cannot join a clinical trial. The goal is to offer potential ben…

This study tests a new drug called remibrutinib in teens aged 12 to 17 who have chronic hives that don't get better with standard allergy pills. The goal is to see if the drug reduces itching and hives better than a placebo. Participants will take the drug or placebo for 24 weeks…

This study follows 40 adults in Saudi Arabia with chronic-phase CML who have already tried two or more TKIs. Researchers will track how well asciminib controls the cancer, its side effects, and how it affects daily life. The goal is to see if this medication works in real-world s…

This study tests whether a new drug (AAA817) given with hormone therapy can slow the growth of advanced prostate cancer that no longer responds to standard hormone treatment. About 940 adults with PSMA-positive metastatic castration resistant prostate cancer will be randomly assi…

This study watches how well Kisqali (ribociclib) combined with an aromatase inhibitor works in real-world settings for people with HR+/HER2- early breast cancer at high risk of coming back. About 3,250 participants will be followed to see how long they stay cancer-free, what side…

This 2-year study looks at how well the drug ianalumab works for adults with lupus nephritis, a kidney disease caused by lupus. Some participants who already responded to treatment will stop the drug to see if they stay well, while others will continue or start open-label ianalum…

This early-stage study tests a new drug, PIT565, in 54 adults with active systemic lupus erythematosus (SLE) who haven't improved with standard treatments. The main goal is to check the drug's safety and how the body processes it. Participants will receive increasing doses of PIT…

This study is tracking 50 people with C3 glomerulopathy, a rare kidney disease, who are taking the drug Fabhalta. Researchers want to see how safe the drug is and how well it controls the disease in everyday medical practice. The study will monitor infections, side effects, and k…

This early-stage study tests whether adding the experimental drug pelabresib to the standard treatment ruxolitinib is safe for Japanese adults with myelofibrosis, a rare bone marrow cancer. Only 6 participants will receive the combination to check for side effects and how the dru…

This early-phase trial tests a new treatment called YTB323 in 28 adults with non-active progressive multiple sclerosis (MS). The goal is to check if the therapy is safe and to see how it affects disability and disease progression. Participants receive a single dose, and researche…

This study tests two experimental drugs, remibrutinib (a pill) and dupilumab (an injection), in adults with chronic hives that antihistamines can't control. About 400 participants will receive one of the treatments for 4 weeks to see which works faster at reducing itch and hives.…

This study tests an experimental drug called GIA632 in 210 adults with non-segmental vitiligo, a condition that causes white patches on the skin. The goal is to see if the drug can safely reduce the size of these patches, especially on the face, and to find the best dose for futu…

This study looks at how well and how safely the drug asciminib works for people in China who have just been diagnosed with chronic myeloid leukemia in its early phase. About 200 adults will take the drug and be followed for at least 12 months to see if their cancer cells drop to …

This study tests a drug called asciminib in people with chronic myeloid leukemia (CML) who have a specific genetic change (T315I mutation) that makes standard treatments less effective. The goal is to see if asciminib can control the disease and improve outcomes for about 20 part…

This study is observing men with metastatic prostate cancer who are receiving a drug called lutetium-177 vipivotide tetraxetan, a type of targeted radiation therapy. Researchers will track how well the treatment works in everyday medical practice, including changes in PSA levels,…

This study tests a new radioactive drug (177Lu-NeoB) combined with chemotherapy (capecitabine) in adults with a specific type of advanced breast cancer (ER+/HER2-) that has worsened after standard hormone therapy. The goal is to find the best dose and see if the combination can s…

This study tests a drug called iptacopan for people with a rare kidney disease (IC-MPGN). The goal is to see if it can reduce protein in the urine and protect kidney function better than a placebo. About 106 adults and teens will take part, and the study is currently recruiting.

This study follows 250 Italian adults with moderate to severe hidradenitis suppurativa (HS) who are starting secukinumab. Researchers will track how well the drug reduces skin abscesses and tunnels over time, as well as treatment patterns and healthcare costs. The goal is to unde…

This study tests a new drug, iptacopan, for people with a rare kidney disease called C3G. The goal is to see if iptacopan can reduce protein in the urine and protect kidney function better than a placebo. About 98 participants aged 12 to 60 will take either the drug or a placebo …

This program provides early access to crizanlizumab (SEG101) for people with sickle cell disease who have no other treatment options and cannot join a clinical trial. It is designed for patients with serious or life-threatening conditions where the potential benefit outweighs the…

This study looks at how well secukinumab works for people with moderate-to-severe hidradenitis suppurativa (a chronic skin condition causing painful lumps) in everyday Canadian clinics. About 142 adults who are already prescribed secukinumab by their doctor will be followed for 1…

This study tests a medicine called iptacopan in 12 children aged 2 to 18 who have a rare blood disease called PNH. The goal is to see how the drug works in their bodies and if it is safe. PNH causes red blood cells to break apart, and this treatment aims to control that damage.

This study is for people with B-cell acute lymphoblastic leukemia or diffuse large B-cell lymphoma whose CAR-T cell therapy didn't quite meet commercial quality standards. Researchers want to see if this slightly off-spec treatment is still safe and effective. About 200 participa…

This study tests a longer course of a radioactive drug (lutetium-177) that targets prostate cancer cells. It is for adults with a specific type of advanced prostate cancer that has stopped responding to hormone therapy but who have not yet had chemotherapy. The goal is to see if …

This study tests a drug called FWY003 in people with geographic atrophy, a late stage of dry age-related macular degeneration that causes blind spots. About 272 participants will receive either the drug or a placebo to see if it can slow the growth of damaged areas in the eye. Th…

This early-stage study tests a single dose of EDK060 in 28 adults with Charcot-Marie-Tooth type 1A, a genetic nerve disorder. The main goal is to see if the drug is safe and how it moves through the body, compared to a placebo. Participants are randomly assigned and neither they …

This study is for adults with two rare blood disorders—immune thrombocytopenia (ITP) and warm-antibody autoimmune hemolytic anemia (wAIHA)—who have already benefited from the drug ianalumab in a previous trial. Researchers want to see if a second course of ianalumab can control t…

This study looks at how well secukinumab works for people with active ankylosing spondylitis who have never taken certain biologic drugs before. Researchers will track changes in disease activity and inflammation over time. The goal is to see if this treatment helps control sympt…

This study looks at the long-term safety of a drug called pelacarsen in about 600 people with high levels of Lp(a) and established heart disease. Participants have already completed a prior study and will continue taking the open-label drug. The main goal is to track side effects…

This study watches 100 people with paroxysmal nocturnal hemoglobinuria (PNH) who are taking Fabhalta. Researchers will track infections, side effects, and what happens if the drug is stopped. The goal is to see how safe the medicine is in everyday use over 48 weeks.

This study tests an experimental drug called MAS825 in 20 people with Still's disease, a rare inflammatory condition. The goal is to see if the drug safely controls symptoms like fever, rash, and joint pain. Participants must have active disease and be at least 1 year old.

This study looks at the long-term safety and effectiveness of the drug iptacopan in people with atypical hemolytic uremic syndrome (aHUS), a rare and serious kidney condition. About 65 adults who already completed a previous iptacopan study and are benefiting from the drug will c…

This study tests the drug ruxolitinib in Chinese patients aged 12 and older who have chronic graft-versus-host disease (cGVHD) that did not improve with steroids. cGVHD is a complication after a stem cell transplant where the donor cells attack the patient's body. The main goal i…

This program provides access to the drug ruxolitinib (INC424) for patients with serious or life-threatening conditions like certain blood cancers, severe COVID-19, or graft-versus-host disease when no other treatments are available. It is for people who cannot join a clinical tri…

This study tests an oral drug called iptacopan in people with generalized myasthenia gravis (gMG), a condition causing muscle weakness. About 146 adults with a specific antibody (AChR+) will receive either iptacopan or a placebo for 6 months, alongside their usual treatments. The…

This study follows 505 adults with chronic spontaneous urticaria (CSU) to see how well remibrutinib controls hives in everyday medical practice. Participants either continue standard antihistamines or switch to remibrutinib. The goal is to measure symptom control and safety over …

This study tests whether a new drug called remibrutinib works as well as the current drug ocrelizumab for people with relapsing multiple sclerosis. About 360 adults aged 40-70 who are already on ocrelizumab will either switch to remibrutinib or stay on ocrelizumab. The goal is to…

This study tests a new medicine called DII235 in 200 adults with high levels of lipoprotein(a), a genetic risk factor for heart disease. Participants will receive either the drug or a placebo to see if it safely lowers their lipoprotein(a) levels over 6 to 12 months. The goal is …

This program provides access to the drug EXV811 (atrasentan) for people with a serious kidney disease called IgA nephropathy who have no other treatment options and cannot join a clinical trial. Doctors can request the drug for their patients if they believe the potential benefit…

This study tests whether inclisiran, a twice-yearly injection, can reduce fatty plaque buildup in heart arteries of people who recently had a heart attack and have high cholesterol. About 334 adults aged 18-75 will receive inclisiran or standard care, and doctors will use special…

This study tests an oral drug called asciminib in children aged 1 to 18 with a specific type of leukemia (Ph+ CML-CP) who have already tried other treatments. The goal is to find the right dose that works as well in kids as it does in adults, and to make sure it is safe. About 34…

This study follows children who previously took dabrafenib and/or trametinib for certain brain tumors. It aims to see how safe these drugs are over the long term by tracking side effects and monitoring growth. About 163 children are enrolled, and the study is no longer recruiting…

This study looks at how well the drug ofatumumab (Kesimpta®) controls relapsing multiple sclerosis (MS) in 160 people in Greece who are receiving it as part of their normal medical care. Researchers will compare results to a previous clinical trial to see if the drug prevents rel…

This study tests two doses of an inhaled drug called glycopyrronium in 42 children aged 6 to 12 with moderate to severe asthma. The goal is to see if it improves lung function and to find the right dose for a future combination medicine. Participants will receive the drug or a pl…

This early-stage study tests a new drug, ITU512, in healthy volunteers and people with sickle cell disease. The goal is to see if it safely boosts fetal hemoglobin, a natural protein that can reduce painful sickle cell complications. The trial first checks safety and dosing in he…

This study looks at the long-term safety of the drug ianalumab in people with systemic lupus erythematosus (SLE). It is for patients who have already completed a previous ianalumab study. About 550 participants will receive either ianalumab or a placebo to monitor side effects an…

This program gives adults with chronic spontaneous urticaria (long-term hives) access to an experimental drug called remibrutinib. It is for people whose hives did not improve with standard treatments. The goal is to control symptoms, not cure the condition.

This study tests a targeted program to help people at high risk for heart attacks or strokes lower their LDL (bad) cholesterol to recommended levels. About 326 adults with high cholesterol and existing heart or blood vessel disease will be randomly assigned to the program or usua…

This study watches how safe and effective two drugs (dabrafenib and trametinib) are for people with advanced or recurrent solid tumors that have a specific gene change (BRAF V600E). About 110 participants will be followed to check side effects and tumor shrinkage. The goal is to …

This study tests the drug ruxolitinib in Chinese patients aged 12 and older who have a serious immune reaction called acute graft-versus-host disease (aGvHD) after a stem cell transplant, and whose condition did not improve with steroids. The goal is to see if ruxolitinib can con…

This study tests a new drug (AMO959) combined with a targeted radiation therapy (177Lu-PSMA-617) and a standard hormone therapy in men whose prostate cancer has spread and is no longer controlled by hormone treatment alone. About 123 adults with PSMA-positive metastatic castratio…

This study is testing how well the drug ribociclib, combined with standard hormone therapy, prevents breast cancer from coming back in people with HR+ HER2- stage II or III breast cancer. About 2,766 participants in Russia will be followed for up to 5 years. The goal is to see if…

This program offers early access to the drug iptacopan for people with serious conditions like C3 glomerulopathy (a kidney disease) or paroxysmal nocturnal hemoglobinuria (a blood disorder) who have no other treatment options. Doctors can request the drug for patients who are not…

This study tests a new medicine called pelacarsen in people with heart disease who have high levels of a blood fat called Lp(a), which increases heart attack risk. Participants will also receive another cholesterol-lowering drug, inclisiran. The goal is to see if pelacarsen can s…

This early-stage study tests a new drug called JSB462 in 15 Japanese men whose prostate cancer has spread to other parts of the body and hasn't responded to standard treatments. The main goal is to check the drug's safety and how the body processes it. This is not a cure but aims…

This early-phase study tests a new drug, INR731, in about 208 men with metastatic prostate cancer that no longer responds to hormone therapy. The drug is given alone or with standard treatments to find a safe dose and check for side effects. The goal is to control the disease, no…

This study tests how well the drug iptacopan works for adults with paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder. Researchers will track changes in hemoglobin levels and other health outcomes over time. About 80 participants in China who have not used similar t…

This study tests whether adding the drug ianalumab to a standard platelet-boosting therapy is tolerable for adults with primary immune thrombocytopenia (ITP), a condition where the immune system destroys platelets, causing bleeding risks. Participants must have tried at least one…

This early-stage study tests a new cell therapy called YTB323 in about 28 adults with relapsing multiple sclerosis (MS) who still experience disease activity despite taking strong MS medications. The goal is to see if YTB323 is safe and can help control the disease. Participants …

This study tests whether alpelisib, a drug that targets a specific gene mutation (PIK3CA), can shrink lymphatic malformations—abnormal fluid-filled growths—and improve symptoms in children and adults. About 232 participants will receive either alpelisib or a placebo, and doctors …

This study tests a new medicine called ianalumab for people with active lupus nephritis, a kidney problem caused by lupus. About 462 adults will receive either ianalumab or a placebo, added to their usual treatment. The goal is to see if ianalumab can help the kidneys work better…

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

This program provides early access to the drug alpelisib (BYL719) for people with PIK3CA-related overgrowth spectrum (PROS) or breast cancer who have no other treatment options and cannot join a clinical trial. Doctors must request the drug for individual patients who may benefit…

This program provides ribociclib (LEE011) to people with serious or life-threatening breast cancer who have no other treatment options and cannot join a clinical trial. Doctors can request the drug for eligible patients who may benefit. The goal is to offer a potential treatment …

This program provides early access to ETB115 (eltrombopag) for people with thrombocytopenia (low blood platelets) who have no other treatment options and cannot join a clinical trial. It is for patients with serious or life-threatening conditions where the potential benefit outwe…

This study monitors the safety and effectiveness of the drug Cosentyx in Korean patients with three conditions: hidradenitis suppurativa (painful skin boils), pediatric plaque psoriasis (scaly skin patches in children), and juvenile idiopathic arthritis (childhood arthritis). Abo…

This study tests a new treatment called rapcabtagene autoleucel for people with severe idiopathic inflammatory myopathies (IIM), a group of diseases causing muscle inflammation and weakness. About 123 adults aged 18 to 75 who haven't improved with standard therapies will be rando…

This early-phase study tests a new drug (177Lu-DFC413) that delivers radiation directly to cancer cells in adults with advanced solid tumors like pancreatic, lung, breast, colorectal, and soft tissue cancers. The main goals are to check safety, find the right dose, and see if it …

This study tests an experimental drug called QCZ484, given as a shot under the skin every 6 months, to see if it can safely lower blood pressure in people with mild to moderate hypertension. About 380 adults aged 18 to 75 who are not on blood pressure meds or are on up to two med…

This study tests a new drug, 177Lu-FAP-2286, that delivers radiation directly to cancer cells in people with advanced solid tumors. The trial has two parts: Phase 1 finds the safest dose, and Phase 2 checks if the drug shrinks tumors in pancreatic, lung, and breast cancers, alone…

This study tests a radioactive drug (177Lu-PSMA-617) in men whose prostate cancer has returned in a few spots after initial treatment. The goal is to see if the drug can delay cancer spread and the need for hormone therapy, which often causes side effects. About 450 men will be r…

This study is a required safety check by Korean health authorities for the drug Leqvio (inclisiran), used to lower cholesterol. It will follow 1,500 adults with high cholesterol who are already prescribed Leqvio in regular clinics. The main goal is to track any side effects and s…

This study tests a one-time treatment called rapcabtagene autoleucel for people with active lupus or lupus kidney disease that hasn't responded to standard therapies. The treatment uses a patient's own immune cells, modified in a lab, to target and calm the overactive immune syst…

This study offers ongoing access to crizanlizumab for people with sickle cell disease who have already completed a Novartis-sponsored trial and are benefiting from the drug. About 130 participants will continue receiving treatment to manage their condition. The goal is to provide…

This study tests a new combination of two drugs (tulmimetostat and JSB462) against standard care in men with advanced prostate cancer that has spread and no longer responds to hormone therapy. The trial has two parts: first finding the safest dose, then comparing the combo's effe…

This study offers continued access to dabrafenib and/or trametinib for up to 100 patients with melanoma, lung cancer, or other solid tumors who were already benefiting from these drugs in a prior Novartis or GSK trial. The main goal is to monitor long-term safety and side effects…

This study looks at the safety and effectiveness of the drug Revolade (eltrombopag) in children aged 6 to 17 with aplastic anemia, a rare condition where the bone marrow stops making enough blood cells. The children in this study have not received a standard treatment called anti…

This study tests an experimental oral drug, PKN605, to see if it can reduce the amount of time people with atrial fibrillation spend in an irregular heart rhythm. About 165 adults with a history of AF will receive either the drug or a placebo. The main goal is to measure AF burde…

This early-phase study tests a new radioactive drug, [177Lu]Lu-NNS309, in adults with advanced pancreatic, lung, breast, or colorectal cancers that have not responded to standard treatments. The main goals are to check safety, find the right dose, and see if the drug can shrink t…

This study tests whether adding pelabresib to the standard drug ruxolitinib works better than ruxolitinib alone for people with myelofibrosis, a rare bone marrow disorder. About 460 adults who have not taken JAK inhibitors before will receive either the combination or a placebo p…

This study monitors 600 Korean adults with chronic myeloid leukemia who are taking Scemblix (asciminib) as part of their regular care. Researchers will track side effects and how well the drug controls the disease over time. The goal is to confirm the drug's safety and effectiven…

This study tests a new drug, GVV858, in people with advanced hormone receptor-positive, HER2-negative breast cancer and other solid tumors that have not responded to standard treatments. The goal is to find a safe dose and see if it works better when given alone or with hormone t…

This study offers continued treatment with remibrutinib to about 212 people who have already completed a Novartis-sponsored remibrutinib study and are benefiting from the drug. The main goal is to monitor long-term safety over up to three years. Participants must have a dermatolo…

This study tests a drug called inclisiran in children aged 2 to under 12 who have a severe inherited form of high cholesterol (homozygous familial hypercholesterolemia). The goal is to see if the drug safely lowers their LDL (bad) cholesterol compared to a placebo over one year, …

This early-phase trial tests a personalized cell therapy called rapcabtagene autoleucel in 27 adults with severe rheumatoid arthritis or Sjögren's disease that hasn't improved with standard treatments. The therapy uses a patient's own immune cells, modified to target and calm the…

This study looks at the long-term safety of a drug called inclisiran in children with a genetic condition that causes very high cholesterol (familial hypercholesterolemia). About 195 kids who completed earlier studies will receive inclisiran to see if it remains safe and helps co…

This study tests a one-time cell therapy called rapcabtagene autoleucel against the standard drug rituximab in 96 people with severe, hard-to-treat scleroderma. The goal is to see if the cell therapy can better improve skin hardening, daily function, and lung health over 52 weeks…

This study looks at the long-term safety of a drug called iptacopan in adults with a kidney disease called primary IgA nephropathy. Participants have already completed an earlier study with iptacopan and can continue receiving the drug until it becomes commercially available. The…

This study watches how well the cholesterol-lowering drug inclisiran (Leqvio®) works in everyday medical practice in Spain. It includes 200 adults with heart disease or an inherited form of high cholesterol. The main goal is to see how many patients reach their target LDL cholest…

This study tests a new drug called remibrutinib against a placebo in 180 adults with generalized myasthenia gravis, a condition causing muscle weakness. Participants must already be on standard treatments. The goal is to see if the drug improves daily activities and muscle streng…

This early-stage study tests a new drug called PIT565 in people with rheumatoid arthritis (RA). The main goal is to check if the drug is safe and how the body processes it. About 57 adults with RA will take part. The study is open-label, meaning everyone knows they are getting th…

This study is tracking the safety and effectiveness of the drug Pluvicto in men with advanced prostate cancer that has spread and no longer responds to hormone therapy. About 278 participants in South Korea will be observed during their normal treatment. The main goal is to recor…

This program provides access to a cancer treatment called tisagenlecleucel (Kymriah) for people with lymphoma, leukemia, or other conditions where standard treatments have failed. It is for patients who cannot join a clinical trial and have no other good options. The goal is to o…

This study tests a new treatment called rapcabtagene autoleucel (a CAR-T cell therapy) against standard care for people with severe active vasculitis (GPA or MPA). About 126 adults aged 18-75 with confirmed ANCA antibodies and active disease will take part. The goal is to see if …

This program provides Lamprene (clofazimine), a drug originally approved for leprosy, to adults with non-tuberculous mycobacterial (NTM) infections who have no other treatment options. Patients must be 18 or older and have failed or cannot tolerate standard therapies. The drug is…

This study tests an experimental drug called iptacopan added to usual treatment for people with active lupus nephritis, a serious kidney complication of lupus. The goal is to see if it helps more patients achieve complete kidney recovery within 24 weeks. About 240 adults with con…

This study tests an oral drug, alpelisib, in children and adults with PIK3CA-related overgrowth spectrum (PROS), a rare condition causing abnormal tissue growth. The goal is to shrink overgrown tissue and improve symptoms. About 104 participants aged 2 and older will take the dru…

This study tests an oral drug called iptacopan in 31 children aged 2 to under 18 with IgA nephropathy, a kidney disease that can lead to kidney failure. The goal is to see if the drug reduces protein in the urine, a sign of kidney damage, and to check its safety. Participants tak…

This phase 3 trial tests whether the drug votoplam can slow the progression of Huntington's disease in people with early symptoms. About 770 participants will receive either votoplam or a placebo, and researchers will measure changes in movement, thinking, and daily function over…

This study tests a new drug called EDI048 in healthy adults who are intentionally infected with the parasite that causes cryptosporidiosis, a diarrheal disease. The goal is to see if the drug can reduce the severity and duration of diarrhea compared to a placebo. The results will…

This study tests a drug called asciminib in children aged 1 to 18 with a specific leukemia (Ph+ CML-CP). The goal is to see if the drug can lower the number of cancer cells in the blood. About 50 children will take the drug, either newly diagnosed or if other treatments stopped w…

This study looks at how well the drug iptacopan works for people with a rare blood disease called PNH. Researchers will track 50 patients in Italy for 12 months to see if their blood health improves and if they need fewer blood transfusions. The goal is to understand the drug's r…

This study looks at how well the drug ribociclib works with hormone therapy in everyday practice for people with a common type of early breast cancer (HR+/HER2-). Researchers will follow about 177 patients in Saudi Arabia for several years to see if the cancer comes back and to c…

This study tests two doses of a drug called remibrutinib against a placebo in 555 people aged 12 and older with moderate to severe hidradenitis suppurativa (HS), a condition causing painful lumps under the skin. The main goal is to see if the drug reduces the number of inflamed n…

This study tests an experimental drug called ECI830, alone or with other cancer drugs (ribociclib and fulvestrant), in people with advanced hormone receptor-positive, HER2-negative breast cancer or certain solid tumors. The goal is to find safe doses and see if the combination ca…

This study looks at whether using a mobile app helps people with HR+ HER2- stage II or III breast cancer stick to their prescribed ribociclib and aromatase inhibitor therapy for a full year. The app provides reminders and information about side effects and relapse risks. About 24…

This study tests whether adding a new drug, JSB462 (luxdegalutamide), to standard hormone therapy (abiraterone) works better than current treatments for men whose prostate cancer has spread and still responds to hormones. About 150 men will receive either the combination or stand…

This study tests a drug called inclisiran in children aged 6 to 12 who have a genetic condition that causes very high cholesterol. The goal is to see if it safely lowers bad cholesterol more than a placebo. About 51 children will take part, and the study lasts two years.

This early-stage trial tests a new treatment called YTB323, a CAR-T cell therapy, for people with generalized myasthenia gravis that hasn't improved with standard treatments. The study will enroll 15 participants to check if the therapy is safe and how well it works. The goal is …

This study tests whether giving a cholesterol-lowering drug called inclisiran soon after a heart attack can reduce the chance of another heart problem within a year. About 300 adults in Russia who have had a heart attack and high cholesterol will receive the drug early. Researche…

This study looks at whether adults with aHUS (a rare blood disorder) can safely switch from their current infusion treatment to a daily pill called iptacopan. About 50 participants will take iptacopan for 12 months to see if it prevents dangerous blood clots. The goal is to find …

This study compares a new combination inhaler (QMF149) to a standard asthma inhaler (budesonide) in 200 children aged 6 to 12 with asthma. Each child will try both treatments for 12 weeks to see which one improves lung function and symptom control more. The goal is to find a more…

This study tests a drug called zigakibart in 32 adults with IgA nephropathy, a kidney disease. The goal is to see if it can slow disease progression by reducing harmful deposits in the kidneys. Participants will receive the drug and be monitored for changes in kidney function and…

This study tests whether adding a drug called inclisiran to standard care can lower 'bad' LDL cholesterol more than standard care alone in people who have recently had a heart attack, stroke, or urgent heart procedure. About 520 adults will be randomly assigned to receive either …

This program provides secukinumab (AIN457) to people with hidradenitis suppurativa who have no other treatment options. It is for patients with serious disease who cannot join a clinical trial or use approved therapies. Doctors must request access for their patients.

This study tests an investigational drug called crizanlizumab in people aged 12 and older with sickle cell disease who have frequent painful crises. The goal is to see if the drug can reduce the number of these crises compared to a placebo. Participants may also continue taking h…

This study tests an experimental drug called VHB937 in people with early Alzheimer's disease. The goal is to see if it can slow down memory and thinking problems. About 407 participants will receive either the drug or a placebo for 72 weeks, and researchers will track changes in …

This study is testing the long-term safety and effectiveness of a daily oral drug called iptacopan for people with rare kidney diseases (C3G and IC-MPGN). It will follow up to 225 patients who have already been taking the drug in earlier studies, tracking their kidney function an…

This program provides access to the drug alpelisib for adults with advanced hormone-positive, HER2-negative breast cancer that has a specific genetic mutation (PIK3CA) and has progressed after standard hormone therapy. It's designed for patients who meet specific health criteria …

This study tests whether adding two simple questions about morning stiffness and lower back pain to a standard screening tool can help doctors spot psoriatic arthritis earlier in people with moderate-to-severe plaque psoriasis. About 502 adults in Canada who are candidates for bi…

This study tests a new drug called remibrutinib for adults with chronic hives (urticaria) that are not well controlled by standard antihistamines. About 44 participants will receive either the drug or a placebo for 12 weeks. The goal is to see if the drug reduces itching, pain, a…

This study tests several experimental compounds in people with moderate to severe atopic dermatitis (eczema). The goal is to see if these treatments can reduce the severity and extent of eczema over 16 weeks. About 224 adults with eczema for at least one year will participate. Th…

This study tests whether a new drug called GXV813 can safely reduce symptoms of schizophrenia during an acute episode. About 142 hospitalized adults aged 18-65 will receive either GXV813 or a placebo. The main goal is to see if the drug improves scores on a standard symptom scale…

This early-stage study tests a new drug called HJB647 in 12 adults with chronic stable heart failure and reduced pumping ability. The main goal is to check the drug's safety and how the body processes it. Participants will receive two different doses and a placebo in a random ord…

This study looks at how satisfied people with moderate to severe hidradenitis suppurativa (HS) are after starting a medication called secukinumab (Cosentyx). Researchers will ask about 77 adults in Saudi Arabia about their treatment experience and quality of life over 24 weeks. T…

This study looks at how well the drug remibrutinib works for people with chronic spontaneous urticaria (long-term hives with no clear cause). Researchers will survey 350 patients in the U.S. to see if the drug improves symptom control and quality of life. The goal is to understan…

This study looks at how the drug pelabresib is processed in the body of people with advanced cancer who also have liver problems. It involves about 24 adults whose cancer has not responded to standard treatments. The goal is to understand if liver function affects drug levels, wh…

This study follows about 175 people with spinal muscular atrophy (SMA) who already received the gene therapy OAV101 in a previous clinical trial. Researchers will monitor them for 5 years to check for any serious side effects and to see how their motor skills develop. The goal is…

This study investigates whether the CAR-T cell therapy tisagenlecleucel may lead to new T-cell cancers in patients who received it. Researchers will analyze stored tumor and blood samples from 30 patients to look for signs that the therapy itself caused these secondary malignanci…

This study watches for side effects in 30 Taiwanese adults with relapsing-remitting multiple sclerosis who are starting fingolimod. It does not test a new treatment but collects safety data over one year. Participants must not have used fingolimod before and must meet other healt…

This study looks at how the drug iptacopan affects the kidneys of 20 adults with IgA nephropathy, a kidney disease. Researchers will use kidney biopsies and lab tests to measure changes in immune system activity and kidney damage over 9 months. The goal is to understand how iptac…

This study looks at whether tracking eye movements can help doctors see how well multiple sclerosis (MS) treatment is working. About 224 Canadian adults with relapsing-remitting MS who are starting the drug ofatumumab will have their eye movements measured over time. The goal is …

This study watches 3,000 people with hormone-positive, HER2-negative stage II or III breast cancer who are already taking ribociclib or abemaciclib with hormone therapy. Researchers will look at medical records and ask patients about their quality of life, side effects, and how w…

This study follows 300 men with advanced prostate cancer that no longer responds to hormone therapy. Participants receive a standard treatment called lutetium-177 vipivotide tetraxetan, which delivers radiation directly to cancer cells. The goal is to see how well it works in rea…

This study follows 800 people with relapsing multiple sclerosis in Germany to see how long they stay on their prescribed injectable or oral medications. Researchers will collect information from medical records and questionnaires over up to four years. The goal is to understand r…

This study follows about 1,400 people who have already received CAR T-cell therapy for cancer. Researchers will monitor them for 15 years to check for delayed side effects, such as new cancers or infections. No new treatment is given—this is purely an observation to understand lo…

This study looks at how women with a common type of advanced breast cancer (HR+/HER2-) are treated in real-world clinics across China. Researchers will analyze medical records from 14,000 patients to see which treatments are used and how well they work. The goal is to fill gaps i…

This study follows 500 people in South Korea who have already received a treatment called tisagenlecleucel for certain blood cancers (like leukemia and lymphoma). The goal is to track long-term side effects, risk of new cancers, and how well the treatment works over time. No new …

This study looks at ways to help doctors test more patients for a genetic heart risk factor called Lp(a). High Lp(a) increases heart disease risk, but testing is not done often enough. The study will involve 4500 adults in clinics and compare testing rates before and after new st…

This study is watching how doctors use a blood test that measures nerve damage (called sNfL) to make decisions for people with relapsing multiple sclerosis. About 700 adults in Germany will take part, and researchers will track how often the test is used and what changes in treat…