Novartis Pharmaceuticals

This study looks at how well and how safely the drug Ilaris (canakinumab) works for people with Schnitzler's syndrome, a rare condition that causes hives, fever, joint pain, and fatigue. About 5 participants will receive the drug and be monitored for up to 48 weeks. The goal is t…

This study looks at the long-term safety and effectiveness of the drug QCZ484 in people with high blood pressure. It is for patients who already completed a previous study with the same drug. Researchers will monitor side effects and track blood pressure changes over time.

This study tests a new drug called ITU512 in healthy people and those with sickle cell disease. The goal is to see if it is safe and can increase fetal hemoglobin, which may reduce sickle cell symptoms. The trial is in early phases (1 and 2) and involves about 161 participants. I…

This study tests a new drug called GVV858, alone or with hormone therapy, in people with advanced hormone receptor-positive, HER2-negative breast cancer and other solid tumors. The goal is to find the safest dose and see how well it works. About 205 adults whose cancer has progre…

This study is testing an oral medication called asciminib in children aged 1 to 18 with Philadelphia chromosome-positive chronic myeloid leukemia (Ph+ CML-CP) who have already tried other treatments. The goal is to find the right dose that works as well as it does in adults and t…

This early-phase study tests a new drug called IEV407, alone or with hormone therapy, in people with advanced HR+/HER2- breast cancer that has stopped responding to standard treatments. The main goals are to check safety and find the right dose. About 194 participants will take p…

This early-phase trial tests a single dose of YTB323, a CAR-T cell therapy, in 28 adults with non-active progressive multiple sclerosis. The study aims to check safety and track disability changes over time. Participants must be 18–60 and able to undergo lumbar punctures and MRIs…

This study tests an experimental drug called VHB937 in people with early Alzheimer's disease. About 400 participants will receive either the drug or a placebo by infusion over 72 weeks. The goal is to see if the drug can slow down memory and thinking problems. Researchers will al…

This Phase 2 trial tests FWY003, an experimental drug, in 272 people with geographic atrophy (a form of advanced age-related macular degeneration). The goal is to see if different doses can slow the growth of damaged areas in the eye and preserve vision. Participants receive eith…

This study offers continued treatment with remibrutinib for up to 3 years to people who have already benefited from it in earlier Novartis trials. It aims to collect long-term safety data. About 648 participants with skin or allergy conditions will take part.

This study tests a new biologic drug called ianalumab combined with a standard platelet-boosting drug (TPO-RA) in people with immune thrombocytopenia (ITP), a condition where the immune system destroys platelets, causing bleeding. The trial includes 164 adults who have tried at l…

This phase 3 trial is testing whether a daily pill called remibrutinib works as well as the standard infusion or injection ocrelizumab for people with relapsing multiple sclerosis. About 360 adults aged 40–70 who are stable on ocrelizumab will either switch to remibrutinib or sta…

This study follows 30 adults in Taiwan with relapsing-remitting multiple sclerosis who are starting fingolimod (Gilenya), a daily pill that helps control the disease. Over 12 months, researchers will watch for specific side effects like slow heart rate, vision problems, liver iss…

This study is monitoring the safety and effectiveness of the drug iptacopan (Fabhalta) in 21 Korean adults with paroxysmal nocturnal hemoglobinuria (PNH) or C3 glomerulopathy (C3G). Researchers will track side effects and lab results from routine medical records. The goal is to s…

This early-stage trial is testing a new treatment called YTB323 for people with generalized myasthenia gravis that hasn't responded to standard therapies. YTB323 is a CAR-T cell therapy, meaning a patient's own immune cells are modified in a lab to better attack the disease. The …

This phase 3 trial tests whether adding pelabresib to the standard drug ruxolitinib works better than ruxolitinib alone for people with myelofibrosis, a type of bone marrow cancer. About 460 adults who have not taken JAK inhibitors before will receive either the combination or a …

This study follows 142 Canadian adults with moderate-to-severe hidradenitis suppurativa who are prescribed secukinumab as part of their regular care. Researchers will track changes in disease severity over 12 months using a standard scoring system. The goal is to understand how w…

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 Phase 2 trial tests a new drug called HDY015, which combines two medicines (QCZ484 and inclisiran) into a single injection. The goal is to see if it can safely lower both blood pressure and cholesterol in people who have both conditions. About 120 adults aged 18-75 with hype…

This early-stage trial is testing a new type of radiation therapy that seeks out and attacks cancer cells while sparing healthy tissue. It's for people with advanced breast, lung, stomach, or bladder cancer who have run out of standard options. The study will check if the treatme…

This study tests a monthly injection called pelacarsen to see if it can slow the worsening of calcific aortic valve stenosis, a condition where the heart valve becomes stiff and narrow. About 502 adults aged 50-79 with high levels of lipoprotein(a) and mild-to-moderate valve dise…

This study looks at how well ribociclib, a targeted cancer drug, works when added to hormone therapy for people with HR+ HER2- stage II or III breast cancer. It will follow about 2,766 patients in Russia, comparing those who get ribociclib plus hormone therapy to those who got ho…

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

This study tests a drug called zigakibart in 32 adults with IgA nephropathy, a kidney disease. The goal is to see if it can reduce signs of kidney damage and slow disease progression. Participants must have a recent kidney biopsy and persistent protein in their urine despite stan…

This phase 3 trial is testing whether adding the targeted drug ribociclib to standard hormone therapy can help prevent invasive breast cancer from returning in people with HR+ HER2- early breast cancer. About 1,400 participants will receive the combination for up to 3 years. The …

This study tests alpelisib, a drug that targets a specific gene mutation (PIK3CA), in people with lymphatic malformations—abnormal growths of lymph vessels. The trial includes children and adults and compares alpelisib to a placebo to see if it can shrink the growths and ease sym…

This study tests an oral drug called iptacopan in people with generalized myasthenia gravis (gMG) who are already on standard treatment. About 146 adults will receive either iptacopan or a placebo for 6 months to see if it improves their ability to do daily activities and reduces…

This early-stage study tests a new drug called JSB462 (Luxdegalutamide) in 15 Japanese men whose prostate cancer has spread to other parts of the body. The main goal is to check the drug's safety and how the body processes it. Participants take the drug once daily with food.

This study tests a new 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 can slow can…

This study tests whether starting inclisiran early during a hospital stay for a heart attack can lower bad cholesterol more than a placebo, when both are added to standard care. About 300 adults hospitalized for a heart attack will receive either inclisiran or a placebo shot at t…

This study tests a new combination of two drugs (tulmimetostat and JSB462) in men with advanced prostate cancer that has spread and stopped responding to standard hormone therapy. The trial has two parts: first, finding the safest dose, then comparing the combination to usual car…

This Phase 3 trial tests whether the drug votoplam can slow the progression of Huntington's disease in people with early symptoms. About 770 adults aged 21 to 70 with a confirmed genetic diagnosis will receive either votoplam or a placebo. The main goal is to see if votoplam pres…

This study tests an experimental drug called NIO752 in 300 people with progressive supranuclear palsy (PSP), a rare brain disorder that affects movement and balance. Participants are randomly assigned to receive either the drug or a placebo. After the main study, everyone can rec…

This study tests a new medicine called MAS825 for people with Still's disease, a rare inflammatory condition causing fever, rash, and joint pain. About 20 children and adults with active disease will receive the drug to see if it safely controls symptoms. The goal is to improve d…

This Phase 3 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 and recent disability progression will receive either remibrutinib or a placebo. The main g…

This study tests a new treatment called DJI136, which uses a patient's own immune cells engineered to target a protein (DLL3) found on small cell lung cancer cells. The trial enrolls 80 adults with extensive-stage small cell lung cancer whose disease has progressed after standard…

This study tests a new drug, AMO959, combined with a targeted radiation therapy (Pluvicto) and a hormone therapy in men with a specific type of advanced prostate cancer (PSMA-positive mCRPC) that has stopped responding to prior hormone treatment. The trial has two phases: first t…

This phase 3 trial is testing a CAR-T cell therapy called tisagenlecleucel that didn't quite meet commercial release standards. It's for people with B-cell acute lymphoblastic leukemia or diffuse large B-cell lymphoma who have no other approved options. The study will check if th…

This study tests a daily oral drug called alpelisib in people aged 2 and older with PIK3CA-related overgrowth spectrum (PROS), a rare condition causing abnormal tissue growth. The goal is to see if the drug can shrink overgrown tissue and improve symptoms. About 104 participants …

This study tests a drug called crizanlizumab in people aged 12 and older with sickle cell disease who have frequent pain 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 their usual treatment…

This study follows patients who have already been taking ruxolitinib alone or with panobinostat in earlier trials and are still benefiting. Researchers will track side effects over time to see how safe these drugs are for long-term use. About 296 participants with conditions like…

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 restore skin color and to find the best dose for future studies. Participants will receive e…

This study tests an experimental drug called Tulmimetostat (DZR123) in people with advanced solid tumors or lymphomas that have not improved with standard treatments. The drug is taken as a daily pill, either alone or combined with another drug (enzalutamide) for certain prostate…

This study tests whether adding pelacarsen to inclisiran can further lower a type of cholesterol called Lp(a) in people with heart disease. About 340 adults with high Lp(a) and LDL-C will receive either pelacarsen or a placebo, plus inclisiran. The main goal is to see if Lp(a) le…

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 genetic change called BRAF V600E. About 110 adults and children will be followed to track side effects and tumor shrinkage. …

This study tracks the safety and effectiveness of Piqray (Alpelisib) in people with a specific type of advanced breast cancer (HR+, HER2-, with a PIK3CA mutation). It includes postmenopausal women and men whose cancer worsened after hormone therapy. Researchers observe patients i…

This program provides access to pelacarsen, a drug for atherosclerotic cardiovascular disease, for patients who have no other treatment options and cannot join a clinical trial. It is for those with serious or life-threatening conditions where the potential benefit outweighs the …

This early-stage trial is testing a new radioactive drug (177Lu-DFC413) to see if it is safe and can shrink tumors in people with advanced solid cancers like pancreatic, lung, breast, colorectal, and sarcoma. About 180 adults whose cancer has progressed after standard treatments …

This early-phase study tests whether adding a new drug called pelabresib to the standard treatment ruxolitinib is safe and helpful for Japanese adults with myelofibrosis, a type of bone marrow cancer. Only 6 people will take part, and the main goal is to check for side effects an…

This early-stage study tests a new drug called DCY636 for safety and tolerability in healthy volunteers and people with moderate to severe atopic dermatitis (eczema). The trial has two parts: a single dose in healthy adults and multiple doses in eczema patients. It aims to gather…

This study looks at the long-term safety and benefits of the drug pelabresib in people with blood cancers, solid tumors, or advanced cancers. It includes up to 50 adults who were in earlier pelabresib studies and are still benefiting from the drug. Some participants who received …

This study is for people with high levels of Lp(a), a genetic risk factor for heart disease, who have already completed a prior pelacarsen trial. It will give them continued access to the drug while monitoring long-term safety and how well it keeps Lp(a) levels low. About 600 par…

This rollover study allows up to 40 people with KRAS G12C-mutated lung or other solid tumors who are already benefiting from opnurasib in a Novartis trial to keep receiving the drug. Participants continue their current dose of opnurasib alone or with TNO155 or trametinib. The goa…

This study tests a new drug, JSB462, combined with standard therapy (abiraterone) in 150 men whose prostate cancer has spread but still responds to hormone treatment. The goal is to see if the combination lowers PSA levels more effectively and delays cancer growth. Participants w…

This Phase 3 trial tests whether adding a radioactive drug called AAA817 to standard hormone therapy can slow cancer growth better than current treatments alone. About 940 men with PSMA-positive metastatic castration resistant prostate cancer will be randomly assigned to receive …

This study follows people with chronic myeloid leukemia (CML) who have already benefited from the drug asciminib in earlier Novartis trials. Researchers want to see how safe the drug is over the long term. About 347 participants will continue taking asciminib or other similar dru…

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

This study is watching how well asciminib works and how safe it is in adults with a type of blood cancer called chronic myeloid leukemia. It includes people who are newly diagnosed or have already tried one other treatment. The main goal is to see how many patients reach a very l…

This phase 3 trial tests a radioactive drug called lutetium-177 vipivotide tetraxetan in 450 men whose prostate cancer has returned and spread to a few spots after initial treatment. The drug targets a protein on cancer cells and delivers radiation directly to them. The goal is t…

This study tests whether a second course of ianalumab can help adults with two rare blood disorders—immune thrombocytopenia (ITP) and warm-antibody autoimmune hemolytic anemia (wAIHA)—who previously responded to the drug but then stopped responding. About 60 participants will rec…

This study offers continued access to crizanlizumab, a drug that helps reduce painful crises in sickle cell disease, for 130 patients who benefited from it in earlier Novartis trials. Participants must have completed a prior study and lack other access to the drug. The main goal …

This study looks at how patients with Familial Mediterranean Fever (FMF) and Still's disease (including systemic juvenile idiopathic arthritis and adult-onset Still's disease) use the drug canakinumab (Ilaris) over the long term. Researchers will track 160 patients across Europe …

This study is tracking the safety and effectiveness of the drug JAKAVI (ruxolitinib) in 395 patients with graft-versus-host disease (GVHD), a complication of bone marrow transplants. Researchers are monitoring infections and other side effects in patients receiving the drug in ro…

This study tests a drug called 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 condition where donor immune cells attack the patient's body after a stem cell transplant. The goal i…

This study tests a drug called remibrutinib in teenagers aged 12 to 17 who have chronic spontaneous urticaria (hives that come and go for no clear reason) that isn't controlled by antihistamines. About 100 participants will receive either the drug or a placebo for 24 weeks, with …

This study tests a new once-daily inhaler called QMF149 (a combination of indacaterol and mometasone) against a standard asthma drug, budesonide, in 200 children aged 6 to 12 with asthma. The goal is to see if the new drug works better at improving lung function and controlling a…

This study tests whether the drug ianalumab can help people with diffuse cutaneous systemic sclerosis, a condition that causes skin thickening and can damage internal organs like the lungs. About 200 adults will receive either ianalumab or a placebo as a shot under the skin. Rese…

This study looks at the long-term safety and effectiveness of the drug iptacopan in people with a rare blood disorder called atypical hemolytic uremic syndrome (aHUS). Participants must have completed a previous iptacopan study and still benefit from the drug. The goal is to moni…

This study follows 700 adults with prostate cancer who have already received at least one dose of lutetium-177 PSMA-617 in earlier Novartis trials. Researchers will monitor long-term side effects such as dry mouth, eye dryness, bone marrow problems, kidney damage, and new cancers…

This study tests if a drug called inclisiran can reduce fatty plaque buildup in the arteries of people who recently had a heart attack and have high cholesterol. About 318 adults will receive either inclisiran or standard care, and doctors will use special imaging to measure plaq…

This study follows about 3,250 people with HR+/HER2- early breast cancer who are at high risk of recurrence. Participants take ribociclib (Kisqali) plus an aromatase inhibitor as part of their routine care. Researchers will track how well the treatment works in real life, its sid…

This study tests an experimental drug called EDI048 for cryptosporidiosis, a diarrheal disease caused by a parasite. Healthy adults will be deliberately infected with the parasite and then given either EDI048 or a placebo to see if the drug reduces diarrhea severity. The goal is …

This study follows 1,000 patients with autoimmune or inflammatory conditions who have already completed a Novartis trial with secukinumab and are judged to benefit from continued treatment. Participants receive secukinumab injections to monitor long-term safety, including side ef…

This study tests whether adding the drug iptacopan to standard treatment helps people with active lupus nephritis (kidney inflammation caused by lupus) achieve better kidney function and fewer flares. About 240 adults with confirmed lupus nephritis will receive either iptacopan o…

This study tests an experimental 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 and how t…

This study tests a new drug called remibrutinib (a tablet) against a placebo in 44 adults with chronic hives (either spontaneous or triggered by things like pressure or cold). The goal is to see if it reduces symptoms like itching and burning, and how it works in the body. Partic…

This study tests a new treatment called rapcabtagene autoleucel, made from a patient's own immune cells, for people with severe scleroderma that hasn't improved with standard care. About 96 participants will receive either this one-time cell therapy or the current drug rituximab.…

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

This study tests a drug called secukinumab in Chinese adults with moderate to severe hidradenitis suppurativa, a painful skin condition that causes boils and tunnels under the skin. The goal is to see if the drug reduces skin lesions and prevents flare-ups over 16 weeks. About 36…

This study tests a one-time treatment called rapcabtagene autoleucel, made from a patient's own immune cells, for people with active lupus or lupus kidney disease that hasn't improved with standard therapies. About 179 adults will receive the treatment after a short chemotherapy …

This Phase 3 trial tests whether iptacopan can reduce protein in the urine and preserve kidney function in people with complement 3 glomerulopathy (C3G), a rare kidney disease. About 98 adults and adolescents will receive either iptacopan or a placebo for 6 to 12 months. The stud…

This study tests an experimental drug called LXE408 in 130 adults with chronic Chagas disease who do not have severe organ damage. The goal is to see if LXE408 can clear or reduce the parasite level in the blood over 6 months, compared to placebo and standard treatment. Participa…

This study is testing a drug called secukinumab, given as an IV infusion, in 20 children aged 2 to 17 with juvenile psoriatic arthritis. The main goals are to see how the drug moves through the body and to check its safety over up to 6 years. This is an early-stage trial, so it f…

This Phase 3 study tests whether remibrutinib can improve daily function in adults with generalized myasthenia gravis, a condition that causes muscle weakness. About 180 participants will receive either the drug or a placebo for 6 months, followed by an open-label phase where eve…

This study tests a drug called iptacopan in people with a rare kidney disease (IC-MPGN). About 106 adults and teens will receive either iptacopan or a placebo. The main goal is to see if iptacopan reduces protein in the urine and helps preserve kidney function over 18 months.

This study looks at 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 in 80 patients across China. The goal is to see if iptacopan impro…

This study follows 44 people in South Korea who have a specific type of lung cancer (NSCLC with MET exon 14 skipping mutation) and are taking the drug Capmatinib (Tabrecta). Researchers will monitor side effects and how well the drug shrinks tumors in real-world settings, not in …

This study tests a gene therapy called DFT383 in 30 children aged 2 to 5 with nephropathic cystinosis, a rare disease that damages kidneys and other organs. The treatment aims to fix the genetic problem so children may no longer need daily cysteamine medication. Researchers will …

This early-phase study tests an experimental cell therapy called YTB323 in about 28 people with relapsing multiple sclerosis whose disease is still active despite taking strong medications. The goal is to see if YTB323 is safe and can control the disease. Participants receive a s…

This early-stage trial tests a new oral drug called INR731, alone or with standard hormone therapy drugs, in 208 men with metastatic prostate cancer that no longer responds to hormone treatment. The main goals are to check safety, find the right dose, and see if the drug shows an…

This early-phase study tests a personalized cell therapy (rapcabtagene autoleucel) in 27 adults with difficult-to-treat rheumatoid arthritis or severe Sjogren's disease. The treatment uses a patient's own immune cells, modified to target and calm the overactive immune system. The…

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 control the cancer by reducing the number of cancer cells. About 50 children will take the drug, and researchers will check how well it work…

This study tests an experimental drug called AAA817 in adults with a specific type of advanced prostate cancer (PSMA-positive) that has worsened after prior treatments, including a targeted therapy. About 443 participants will be randomly assigned to receive either AAA817 or the …

This Phase 2 trial tests a one-time infusion of rapcabtagene autoleucel, a CAR-T cell therapy made from a patient's own immune cells, against standard treatments for severe active granulomatosis with polyangiitis (GPA) or microscopic polyangiitis (MPA). About 126 adults aged 18-7…

This study watches children and adults who are already taking or about to start Ilaris for hereditary periodic fever syndromes or systemic juvenile idiopathic arthritis. Researchers will track side effects and how well the drug controls symptoms over 16 weeks. No new medicine is …

This Phase 2 trial tests an oral drug called PKN605 in 165 adults with atrial fibrillation. Participants are randomly assigned to receive either the drug or a placebo, and neither they nor their doctors know which they get. The main goal is to see if PKN605 reduces the amount of …

This study tests a drug called inclisiran in children aged 6 to 12 who have a genetic condition that causes very high cholesterol. The drug is given as a shot under the skin and aims to lower 'bad' LDL cholesterol. The trial compares inclisiran to a placebo for one year, then eve…

This study looks at the long-term safety of inclisiran, a cholesterol-lowering injection, in children with a genetic condition that causes very high cholesterol. About 195 kids who completed earlier inclisiran studies will receive the drug for an extended period. Researchers will…

This study tracks 350 people with chronic spontaneous urticaria (hives) who are starting remibrutinib, a new drug. Researchers will survey patients and their doctors to see how well the drug controls symptoms and how satisfied patients are. The goal is to understand real-world ef…

This study tests whether starting inclisiran early after a heart attack can lower bad cholesterol and reduce the risk of another heart attack or stroke. About 300 adults in Russia who had a recent heart attack and have high cholesterol will receive inclisiran injections. Research…

This early-stage trial tests a new drug called OJR520 in healthy volunteers and people with chronic kidney disease. The main goal is to check if the drug is safe and how the body processes it. About 112 participants will receive either OJR520 or a placebo. This is the first time …

This study tests whether a targeted lipid optimization program—combining clinic visits and virtual sessions—helps high-risk heart disease patients in Abu Dhabi achieve better LDL cholesterol control. About 326 adults with atherosclerotic cardiovascular disease will be randomly as…

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 early-phase study tests a new radioactive drug, 225Ac-ETN029, in 116 adults with advanced DLL3-positive cancers like small cell lung cancer or neuroendocrine tumors. The main goal is to check safety and find the right dose, while also seeing if the drug can shrink tumors. Pa…

This study tests whether adding a radioactive drug (177Lu-DOTA-TATE) to standard therapy (octreotide) helps control advanced neuroendocrine tumors of the gut or pancreas better than standard therapy alone. About 240 newly diagnosed patients with slow-growing but high-burden tumor…

This study tests iptacopan, an oral drug, in 20 adults with IgA nephropathy, a kidney disease. Researchers will use kidney biopsies and lab tests to see if the drug reduces signs of inflammation and damage over 9 months. The goal is to understand if iptacopan can slow disease pro…

This study is for adults with a rare blood disorder called aHUS who are currently receiving antibody infusions. Researchers want to see if switching to a daily pill called iptacopan is safe and works just as well to prevent dangerous blood clots. About 50 participants will take i…

This study tests an experimental drug called LTP001 for pulmonary arterial hypertension (PAH), a serious lung condition that makes it hard for the heart to pump blood. The trial has two parts: first, healthy volunteers receive single or multiple doses to check safety and how the …

This early-stage trial is testing a new type of targeted radiation therapy for people with advanced pancreatic, lung, breast, or colorectal cancers. The treatment uses a radioactive drug that seeks out cancer cells to deliver radiation directly, aiming to kill them while sparing …

This program provides early access to CTL019, a cell therapy that reprograms a patient's own immune cells to attack cancer. It is for people with lymphoma or leukemia who have no other treatment choices and cannot join a clinical trial. The goal is to offer a potential benefit wh…

This study tests a drug called 177Lu-FAP-2286, which delivers radiation directly to cancer cells. It is for people with advanced solid tumors (like pancreatic, lung, or breast cancer) that have a specific protein called FAP. The trial has two parts: Phase 1 finds the safest dose,…

This early-stage trial tests a new drug called EDK060 in 28 adults with Charcot-Marie-Tooth type 1A, a genetic nerve disorder. Participants receive a single dose of EDK060 or a placebo to check safety and how the drug moves through the body. The goal is to see if EDK060 is safe e…

This phase 2 trial is testing the drug asciminib in 20 adults with chronic myeloid leukemia who have a specific genetic change (T315I mutation) and cannot take the standard drug ponatinib. The goal is to see if asciminib can control the leukemia and improve outcomes. Participants…

This study follows 600 Korean adults with chronic myeloid leukemia who are taking Scemblix (asciminib) to see how safe and effective it is in everyday medical practice. Researchers will track side effects and how well the drug controls the disease at 12 and 24 weeks. The goal is …

This study looks at the long-term safety and effectiveness of a drug called iptacopan for people with two rare kidney diseases: C3 glomerulopathy and IC-MPGN. About 225 adults who have already completed a previous iptacopan study will take the drug for an extended period. The goa…

This study tests a medicine called iptacopan in children aged 2 to under 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. About 12 children will take the medicine, and doctors will monitor side effects and blood…

This program offers eltrombopag to patients with serious or life-threatening thrombocytopenia (low platelet counts) who have no other treatment options and cannot join a clinical trial. A licensed physician must request the drug, and the potential benefit must outweigh the risks.…

This program provides access to the drug alpelisib (BYL719) for patients with PIK3CA-related overgrowth spectrum or breast cancer who have no other treatment options and cannot join a clinical trial. It is not a study but a way for doctors to request the drug for individual patie…

This study follows about 3,280 adults with chronic spontaneous urticaria (CSU) to see how well remibrutinib controls their hives in everyday medical practice. Participants either continue standard antihistamines or switch to remibrutinib, and researchers track symptom control and…

This early-stage trial is testing an experimental drug called HJB647 in 12 adults with stable chronic heart failure and reduced pumping ability. The study aims to check the drug's safety and how the body processes it at two different doses. Participants will receive both the drug…

This program provides crizanlizumab, a drug that may help reduce pain crises, to people with sickle cell disease who have no other treatment options. It is for patients who cannot join a clinical trial. The goal is to offer potential benefit when standard therapies are not availa…

This study follows 30 children aged 1 to 17 with chronic heart failure who are taking Entresto for the first time. Researchers will monitor for side effects like low blood pressure, high potassium, kidney problems, and dehydration over 52 weeks. The goal is to understand how safe…

This program offers ribociclib, a targeted cancer drug, to patients with serious or life-threatening breast cancer who have no other treatment options and cannot join a clinical trial. Doctors must request the drug for their patients. The goal is to provide potential benefit when…

This study tracks 600 adults with asthma who are already prescribed Enerzair inhaler. Over 24 weeks, researchers will monitor side effects and how well the drug controls asthma in everyday life. No new treatment is given; it simply observes routine care.

This study tests a combination of two targeted drugs, dabrafenib and trametinib, in people with rare solid tumors that have a specific genetic change called BRAF V600E. The goal is to see how well the drugs shrink or control the tumors. The study includes both children (age 1 and…

This study follows 170 men in China with metastatic prostate cancer who are receiving a radioactive drug called lutetium-177. Researchers will track how much their PSA levels drop, how long the cancer stays controlled, and their quality of life during and up to one year after tre…

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 how well it opens airways and how safe it is compared to a placebo. Results will help decide the best dose for future combination tre…

This study looks at how well and how safely the drug asciminib works for people newly diagnosed with a type of leukemia called chronic myeloid leukemia (CML). About 200 adults in China will take the drug and be followed for at least 12 months. The main goal is to see how many ach…

This Phase 2 trial tests an experimental drug called EYU688 in 108 adults with dengue fever. Participants receive either the drug or a placebo to see if it lowers the amount of virus in the blood and shortens fever. The study is currently recruiting.

This program provides early access to the drug ianalumab (VAY736) for patients with serious or life-threatening conditions like immune thrombocytopenia or Sjögren's syndrome who have no other treatment options. It is not a clinical trial but a managed access program where doctors…

This study is testing the long-term safety of a drug called ianalumab in people with lupus who have already completed a previous study with the same drug. About 550 participants will receive either ianalumab or a placebo monthly or quarterly for up to several years. The main goal…

This 2-year extension study looks at lupus nephritis patients who finished a previous trial of ianalumab. Some patients who responded well will stop treatment to see if their kidney disease stays under control. Others who did not respond or were already on open-label ianalumab wi…

This study tracks the safety and effectiveness of the drug Cosentyx (secukinumab) in Korean patients with hidradenitis suppurativa, pediatric plaque psoriasis, or juvenile idiopathic arthritis. Researchers will monitor 76 participants for side effects and how well the drug works …

This study follows 70 Korean adults with ankylosing spondylitis who have never taken biologic drugs. They will receive secukinumab as part of routine care. Researchers will measure changes in disease activity and inflammation over time to see how effective the treatment is in rea…

This study tests a drug called inclisiran in children aged 2 to 12 who have a rare genetic condition that causes extremely high cholesterol. The goal is to see if the drug safely lowers their bad cholesterol compared to a placebo. Participants will receive either inclisiran or pl…

This study is tracking 10 children aged 6 to 17 with aplastic anemia, a condition where the bone marrow stops making enough blood cells. The children are receiving eltrombopag (Revolade) for the first time, combined with anti-thymocyte globulin. Researchers are monitoring for ser…

This study follows about 500 adults with chronic spontaneous urticaria (CSU) who are either starting remibrutinib or sticking with standard antihistamines. Researchers will track how well the drug controls hives and itching over 24 months, and monitor for side effects. The goal i…

This study follows 177 people with HR+/HER2- early breast cancer who are already taking ribociclib with hormone therapy as part of their routine care. Researchers will track how well the treatment works and any side effects over time. The goal is to see if the drug helps prevent …

This study follows 175 people with spinal muscular atrophy who previously received the gene therapy Zolgensma in clinical trials. Researchers will monitor them for 5 years to track serious side effects and developmental milestones. The goal is to understand how safe and effective…

This program provides access to EXV811, atrasentan for people with IgA nephropathy, a serious kidney disease. It is for patients who have no other treatment options and cannot join a clinical trial. The goal is to offer potential benefit when standard therapies are not available.

This early-stage trial is testing a radioactive drug called Lutetium-177 vipivotide tetraxetan in men with a specific type of advanced prostate cancer that has stopped responding to hormone therapy. The drug targets and delivers radiation directly to cancer cells. The study aims …

This study tests whether adding inclisiran (a twice-yearly injection) to standard care lowers LDL cholesterol more than standard care alone in 520 patients who recently had a heart attack, stroke, or urgent heart procedure. Participants are randomly assigned to receive inclisiran…

This study tracks 1,500 adults with high cholesterol who are prescribed Leqvio (inclisiran) in routine care. The goal is to monitor side effects and measure how well the drug lowers LDL cholesterol. It is a mandatory safety review required by Korean health authorities, not a test…

This study follows about 40 adults in Saudi Arabia with chronic-phase chronic myeloid leukemia (CML) who have already tried two other targeted therapies. Researchers will track how well asciminib controls the cancer, its side effects, and how consistently patients take the medica…

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

This program provides Lamprene (clofazimine), a drug normally used for leprosy, to adults with NTM infections that haven't responded to other treatments or caused intolerable side effects. Patients take 100 mg of clofazimine daily as part of a multi-drug regimen. The goal is to o…

This program provides early access to the drug iptacopan for people with serious or life-threatening C3 glomerulopathy (C3G) or paroxysmal nocturnal hemoglobinuria (PNH) who have no other treatment options. It is designed for patients who cannot join a clinical trial or have exha…

This program provides asciminib (ABL001) to people with chronic phase chronic myeloid leukemia who have no other treatment options and cannot join a clinical trial. It is a managed access program, not a formal study, and aims to offer potential benefit when standard therapies are…

This program provides remibrutinib, an experimental drug, to adults with chronic spontaneous urticaria (long-term hives) that haven't improved with antihistamines or omalizumab. Participants take the drug as prescribed to help control their symptoms. The goal is to offer access t…

This study tests the drug ruxolitinib in 36 Chinese adults and teens (12+) who have severe graft-versus-host disease after a stem cell transplant and did not improve with steroids. Participants take ruxolitinib pills daily for up to 56 days. The main goal is to see how many patie…

This program provides early access to the drug ruxolitinib for people with serious or life-threatening conditions like certain blood cancers, severe COVID-19, or graft-versus-host disease. It is for patients who have no other treatment options and cannot join a clinical trial. Th…

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 early-phase study is testing two radioactive imaging agents, 68Ga-DFC413 and 68Ga-NNS309, to see how well they highlight tumors on PET scans. About 66 adults with advanced pancreatic, lung, breast, colorectal, or soft tissue cancers will receive the agents. The goal is to co…

This early-phase study tests a new radioactive imaging agent called [68Ga]Ga-DWJ155 in 36 adults with advanced breast cancer or non-small cell lung cancer. The agent is designed to bind to a target on cancer cells and show up on PET scans, helping doctors see where the cancer has…

This Canadian study tests whether adding two questions about morning stiffness and lower back pain to a standard screening tool (PEST) can help dermatologists identify psoriatic arthritis earlier in people with moderate-to-severe plaque psoriasis. About 502 adults who are candida…

This study tests an experimental oral drug called GXV813 in 142 hospitalized adults with schizophrenia. The goal is to see if it safely reduces symptoms like hallucinations and withdrawal during an acute episode. Participants are randomly assigned to receive GXV813 or a placebo, …

This study tests a new daily pill (remibrutinib) against a standard injected drug (dupilumab) for adults with chronic hives that antihistamines can't control. About 400 participants will take either the pill or get the shot for 4 weeks to see which works faster. The goal is to re…

This study surveys 77 adults with moderate to severe hidradenitis suppurativa (HS) in Saudi Arabia who are starting the medication Cosentyx (secukinumab). Researchers will ask patients about their treatment satisfaction and quality of life after 24 weeks. The goal is to understan…

This Phase 2 trial is testing an experimental drug called GHZ339 in 224 adults with moderate to severe atopic dermatitis (eczema). Participants will receive either the drug or a placebo, and researchers will measure changes in eczema severity using the EASI score at 16 weeks. The…

This study looks at how well the drug iptacopan works for people with paroxysmal nocturnal hemoglobinuria (PNH) in everyday medical practice. Researchers will track 50 Italian patients for 12 months to see if their anemia improves and if they need fewer blood transfusions. The go…

This early-phase study is testing the cancer drug pelabresib in 24 people with advanced cancers who also have liver problems. The goal is to see how the liver affects the drug levels in the body. Participants will take the drug daily for 14 days, then have a week off. This resear…

This study looks at how a cancer drug called pelabresib changes the way the body processes three other medicines: repaglinide (for diabetes), midazolam (a sedative), and a birth control pill. It involves 24 adults with advanced cancers who have no standard treatment options. The …

This registry will follow 1,000 adults with chronic-phase CML who are taking approved TKIs (like Gleevec or Tasigna) as their first or second treatment. Over 5 years, researchers will track side effects, treatment changes, and patient-reported experiences to understand how these …

This study examines stored tumor and blood samples from 30 patients who developed a secondary T-cell cancer after receiving the CAR-T therapy tisagenlecleucel. Researchers will test whether the therapy's genetic material is present in the tumor cells, which could suggest a role i…

This study follows 160 people with relapsing multiple sclerosis in Greece who are already taking ofatumumab (Kesimpta®) as part of their routine care. Researchers will track how many patients have no signs of disease activity—like relapses, new brain lesions, or worsening disabil…

This study is not testing a new drug. Instead, it will observe 800 people with relapsing multiple sclerosis in Germany who are already taking approved injectable or oral medications. Researchers will track how long patients stick with their prescribed treatment over up to four ye…

This study follows 278 men with advanced prostate cancer who are already receiving Pluvicto as part of their routine care in South Korea. Researchers will monitor side effects and how well the drug controls the cancer over time. No new treatment is given—this is purely an observa…

This study follows 500 men with metastatic castration-resistant prostate cancer who are receiving lutetium-177 vipivotide tetraxetan (a radioactive drug) as part of their routine care. Researchers will measure quality of life, pain, and disease progression over time using patient…

This study follows 250 Italian adults with moderate to severe hidradenitis suppurativa (a chronic skin disease causing painful lumps) who are starting secukinumab. Researchers will track how well the drug reduces skin lesions over time and how it affects healthcare use and costs.…

This study follows 300 men with a type of advanced prostate cancer that no longer responds to hormone therapy. Participants receive a targeted radiation treatment called lutetium-177 vipivotide tetraxetan as part of their normal care. Researchers will track how long the cancer st…

This study looks at whether a new eye-tracking device can help doctors see how well multiple sclerosis (MS) treatment is working. About 224 Canadian adults with relapsing-remitting MS will use the device during routine care. The goal is to find out if eye movement changes can rep…

This study follows 500 people in South Korea who have received tisagenlecleucel, a CAR-T cell therapy, for certain blood cancers like acute lymphoblastic leukemia and lymphomas. Researchers will monitor side effects, risk of new cancers, and how well the treatment works over time…

This study is observing 3,000 people with early-stage HR+/HER2- breast cancer who are taking ribociclib or abemaciclib as part of their routine care. Researchers will look at how long patients stay cancer-free, how well they stick to their medication, and their quality of life. T…

This study looks at how patients with HR+/HER2- advanced or metastatic breast cancer are actually treated in China, and how well those treatments work. Researchers will analyze data from 14,000 patients to see which first-line therapies are used, how often patients switch treatme…

This study looks at whether special strategies can help doctors test more patients for Lp(a), a genetic risk factor for heart disease. Researchers will compare testing rates before and after these strategies are used in clinics. The goal is to see if simple changes can help follo…

This study follows 1,400 people who previously received CAR-T cell therapy for cancer. Researchers will monitor them for 15 years to check for delayed side effects like new cancers, infections, or immune disorders. The goal is to understand the long-term safety of this gene-based…