Novartis Pharmaceuticals

This study tested the safety and effectiveness of the drug EGATEN (triclabendazole) in 301 people aged 6 and older with fascioliasis, a parasitic liver infection. Participants received two doses of EGATEN 12 hours apart and were followed for 90 days. The goal was to see if the dr…

This study tested the safety of combining two drugs, alpelisib and fulvestrant, in 40 men and post-menopausal women with a certain type of advanced breast cancer (HR+, HER2-, with a PIK3CA mutation) that had worsened after hormone therapy. The goal was to track side effects. The …

This study tested a single injection of tiny particles (NGI226) to see if it safely helps repair damaged Achilles tendons. 45 adults with mid-portion Achilles tendinopathy received either the study drug or a placebo. Researchers measured side effects and changes in tendon stiffne…

This study tested the safety and effectiveness of inclisiran sodium, a cholesterol-lowering injection, in 65 Indian adults with high cholesterol or mixed dyslipidemia. Participants received three doses over 270 days. The goal was to see how well it lowers LDL cholesterol and if a…

This study tested two doses of secukinumab (a shot) combined with a steroid taper in 381 adults aged 50+ with polymyalgia rheumatica (PMR) who had recently relapsed. The goal was to see if secukinumab helps patients achieve and maintain remission without needing steroids long-ter…

This study looks at the long-term safety of imatinib, a daily pill for certain cancers like GIST and CML. It includes 250 patients who were already benefiting from imatinib in a previous Novartis trial and continue taking it. The main goal is to track side effects over time, with…

This study tested a new drug called BION-1301 in 103 healthy volunteers and adults with IgA nephropathy, a kidney disease. The main goal was to check the drug's safety and how the body processes it. Researchers measured side effects, blood pressure, and kidney function over time.

This study tested a treatment called tisagenlecleucel, a type of CAR-T cell therapy, in 98 adults with follicular lymphoma that had come back or stopped responding to other treatments. The therapy works by reprogramming a patient's own immune cells to find and attack cancer cells…

This study tested a drug called PTC518 in 159 adults with Huntington's disease to see if it is safe and can lower a harmful protein in the blood. Participants received different doses of the drug or a placebo for up to 12 months. The goal is to slow disease progression by targeti…

This study follows about 1,100 Japanese adults with high blood pressure who are prescribed Entresto tablets. Researchers track side effects like low blood pressure, high potassium, and kidney issues over 52 weeks. The goal is to see how safe and effective Entresto is in everyday …

This study reviewed medical records of 37 adults with Philadelphia-positive acute lymphoblastic leukemia (Ph+ ALL) who took asciminib because other treatments stopped working or caused side effects. Researchers wanted to see how many patients achieved complete remission within th…

This study tested a drug called remibrutinib in 76 adults with peanut allergy. Participants took one of three doses or a placebo daily for one month. The main goal was to see if the drug helped them eat more peanut protein without having an allergic reaction. Results showed some …

This early-stage study tested a gene therapy called CPK850 in 12 adults with a rare inherited eye disease (RLBP1 retinitis pigmentosa) that causes progressive vision loss and night blindness. The treatment was injected under the retina to deliver a working copy of the faulty gene…

This Phase 3 trial tested whether the CAR-T cell therapy tisagenlecleucel works better than standard treatments for adults with aggressive B-cell non-Hodgkin lymphoma that came back or didn't respond to initial therapy. The study enrolled 330 participants and compared how long th…

This study tested a radioactive drug called 177Lu-PSMA-617 in 62 Chinese men with advanced prostate cancer that had spread and stopped responding to hormone therapy and chemotherapy. The drug targets a protein on cancer cells and delivers radiation directly to them. Researchers m…

This study looked at the safety and effectiveness of a drug called asciminib in 85 Indian adults with a specific type of leukemia (Ph+ CML-CP). Some participants had a genetic mutation (T315I) that can make treatment harder, while others had already tried two or more other drugs.…

This study tested a gene therapy called voretigene neparvovec in 4 Japanese patients with a rare inherited eye disease that causes vision loss. The treatment was given as an injection under the retina to replace the faulty gene. The goal was to see if it could safely improve ligh…

This study looked at the safety and effectiveness of a drug called ribociclib combined with hormone therapy for people with a certain type of advanced breast cancer (HR+/HER2-). Researchers collected real-world data from 175 patients in the Middle East to track side effects and h…

This study tested a new oral medication called iptacopan in 34 adults with a rare disease called atypical hemolytic uremic syndrome (aHUS), which causes blood clots, low platelets, and kidney damage. The goal was to see if the drug could improve blood counts and kidney function w…

This study tested a single intravenous dose of TIN816, an enzyme that breaks down ATP to reduce inflammation, in 316 ICU patients with sepsis-related acute kidney injury. The goal was to see if it improves kidney function over 8 days and reduces major kidney problems by day 90. T…

This study looked at how safe and effective Xolair is for adults with chronic rhinosinusitis and nasal polyps when used in routine care. 135 patients who were already prescribed Xolair were followed to track side effects and changes in symptoms. The goal was to see how the drug p…

This study tested a new drug called NIS793 added to standard chemotherapy for people with metastatic pancreatic cancer that has spread. The goal was to see if the combination could help patients live longer by reducing scar tissue in the tumor. About 511 adults with this advanced…

This study followed 134 adults in Italy with active secondary progressive multiple sclerosis who were prescribed siponimod (Mayzent) as part of their routine care. Over 36 months, researchers measured how many patients experienced a confirmed worsening of disability or cognitive …

This study tested a drug called pelacarsen in 422 Black/African American and Hispanic patients with high Lp(a) levels and existing heart disease. Lp(a) is a genetic risk factor for heart attacks and strokes. The goal was to see if pelacarsen could safely lower Lp(a) levels over 1…

This Phase 2 trial tested a drug called CFZ533 (iscalimab) in 273 adults with Sjögren's syndrome, an autoimmune disease causing dryness, pain, and fatigue. Participants received either the drug or a placebo for 24 weeks. The study measured changes in disease activity and symptoms…

This study tested two experimental drugs, VAY736 and CFZ533, in 107 adults with systemic lupus erythematosus (SLE). The goal was to see if these drugs could reduce lupus symptoms and allow patients to lower their steroid use. Participants received either one of the drugs or a pla…

This study tested whether sacubitril/valsartan (Entresto) works better than enalapril for people with heart failure caused by chronic Chagas disease. Over 900 adults with reduced heart function took part. The goal was to see if the drug could reduce heart-related deaths, hospital…

This study looked at over 2,600 people with multiple sclerosis to compare two drugs: Kesimpta and Ocrevus. Researchers used insurance claims data to see which drug led to fewer relapses and lower healthcare costs. The goal is to help patients and doctors choose the best treatment…

This study tested a new two-drug combination (KAF156 plus a special form of lumefantrine) in 295 children aged 6 months to 18 years with uncomplicated malaria. The goal was to see if the treatment safely clears the parasite and prevents the infection from coming back. The approac…

This early-phase study tested a new drug called siremadlin added to two existing drugs (venetoclax and azacitidine) for adults with acute myeloid leukemia (AML) who are too frail for standard chemotherapy. The study included 14 people whose cancer did not respond well to initial …

This study tested a combination of two anti-malarial drugs, cipargamin and KLU156, in 60 people with uncomplicated Plasmodium falciparum malaria. Participants were at least 12 years old and had a confirmed infection. The main goal was to see if the drugs could clear the malaria p…

This study tested a radioactive drug called 177Lu-PSMA-617 in 94 Japanese men with advanced prostate cancer that had spread and stopped responding to hormone therapy. The drug seeks out and delivers radiation directly to cancer cells. Researchers measured how well it shrank tumor…

This study followed 164 adults with advanced, progressive neuroendocrine tumors of the gut or pancreas who were treated with Lutathera (a radioactive drug) in normal hospital settings. Researchers measured how long patients lived without their cancer getting worse and how many tu…

This study looked at 781 adults with moderate-to-severe plaque psoriasis and metabolic syndrome (a cluster of conditions like high blood pressure and obesity). Researchers wanted to see if adding a lifestyle intervention (diet and exercise) to the drug Secukinumab (Cosentyx) impr…

This phase 2 trial tested whether adding asciminib to the standard drug imatinib helps people with chronic myeloid leukemia (CML) achieve deeper remission. 104 adults who had been on imatinib for at least a year without reaching very low cancer levels were randomly assigned to ad…

This study tested whether taking two targeted drugs (dabrafenib and trametinib) for 12 months after melanoma surgery could keep the cancer from returning. It included 870 adults with high-risk stage III melanoma that had a specific BRAF V600 mutation. Results showed the drug comb…

This study tested an experimental protein called LNA043, given as injections into the knee, to see if it can help regrow cartilage in people with knee osteoarthritis or cartilage injuries. 142 adults participated in two parts: one for cartilage lesions and one for osteoarthritis.…

This study followed 107 adults in Switzerland with relapsing multiple sclerosis who were already taking Kesimpta (ofatumumab) as part of their routine care. Researchers tracked how many patients had no signs of disease activity, including relapses, new brain lesions, or worsening…

This completed Phase 4 trial tested the drug inclisiran (given as a shot under the skin) against a placebo in 1770 adults with high cholesterol who were already on cholesterol-lowering therapy. The goal was to see if inclisiran helps more people reach their LDL cholesterol target…

This study tested the safety and effectiveness of brolucizumab, a drug injected into the eye, for treating diabetic macular edema (DME) in 66 Indian patients. DME is a complication of diabetes that can cause vision loss. The study aimed to see how well the drug works in real-worl…

This study looked at how well ofatumumab works in 562 people with relapsing multiple sclerosis who switched from other drugs (fumarates or fingolimod) because their disease was still active. The main goal was to see how many relapses they had per year. Researchers also tracked si…

This study tested two experimental drugs, DFV890 (a daily pill) and MAS825 (a single shot), in 31 adults with coronary heart disease and a blood condition called CHIP. The goal was to see if these drugs could reduce specific markers of inflammation linked to heart risk. The trial…

This study tested an experimental daily pill called DFV890 in 24 adults with coronary heart disease and high levels of inflammation (measured by hsCRP). The goal was to see if the drug could safely lower certain inflammatory markers linked to heart risk. Participants took the pil…

This study tested a drug called secukinumab in 137 Chinese adults with a type of spinal arthritis (non-radiographic axial spondyloarthritis) that causes back pain and stiffness. The goal was to see if the drug improves symptoms more than a placebo after 16 weeks, and to check its…

This study tested a new intravenous drug called cipargamin in 254 adults and children with severe malaria. The goal was to find a safe and effective dose that quickly clears the malaria parasite, especially in areas where standard treatments are becoming less effective. Researche…

This study tested a drug called capmatinib in 50 Indian adults with advanced non-small cell lung cancer that has a specific genetic change (MET exon 14 skipping mutation). The goal was to see how safe the drug is and how well it shrinks tumors. Participants took capmatinib pills,…

This study looked at 124 adults with chronic myeloid leukemia (CML) who had been on the drug nilotinib for at least 3 years and had very low levels of cancer. First, researchers tested if lowering the nilotinib dose by half for a year could help patients stop treatment entirely. …

This early-phase study tested a new drug called gevokizumab alongside standard cancer treatments in 167 adults with advanced colorectal, stomach, or kidney cancer that had spread. The goal was to find a safe and active dose and see if the combination could help control the diseas…

This study tested canakinumab, an anti-inflammatory biologic, in 14 Japanese adults with Adult-Onset Still's Disease (AOSD), a rare condition causing fevers and joint pain. Participants received injections every 4 weeks for at least 48 weeks. The goal was to see if the drug could…

This study tested an oral targeted drug called capmatinib in 36 Chinese adults with advanced non-small cell lung cancer that has a specific MET gene mutation (exon 14 skipping). The drug aims to block faulty signals that help cancer grow. Researchers measured how many patients' t…

This phase 3 trial tested secukinumab, a biologic drug, in 354 people with giant cell arteritis (GCA), a condition causing inflamed arteries. Participants received either secukinumab or a placebo, along with a steroid taper. The goal was to see if secukinumab could help more peop…

This study looked at how an intravenous (IV) form of secukinumab is processed by the body and whether it is safe for adults with giant cell arteritis (GCA) or polymyalgia rheumatica (PMR). These conditions cause inflammation in blood vessels or muscles, leading to pain and stiffn…

This Phase 2 trial tested whether an oral drug called RHH646 can help regrow cartilage in the knee for people with osteoarthritis. 82 adults aged 35 to 75 with knee osteoarthritis took either RHH646 or a placebo. The study measured changes in cartilage volume using MRI and tracke…

This study tested a new drug called CFZ533 in 418 kidney transplant patients to see if it could prevent organ rejection as well as the standard drug tacrolimus. Patients received either CFZ533 or tacrolimus along with other standard medications. The main goal was to compare rates…

This study tested the drug asciminib in 199 adults with chronic myeloid leukemia (CML) whose cancer was not well controlled after trying at least two other targeted therapies. The main goal was to see how many patients achieved a major molecular response (a key sign of disease co…

This Phase 3 study tested whether ofatumumab, a monthly injection, works better than standard first-line disease-modifying therapies (like glatiramer acetate or interferon) in people newly diagnosed with relapsing multiple sclerosis. The main goal was to see if more patients coul…

This study tested a monthly injection of ofatumumab in 180 people with very early relapsing-remitting multiple sclerosis (MS). The goal was to see if the drug could stop all signs of disease activity—like relapses, disability worsening, and new brain lesions—over 18 months. Resea…

This study tested the drug secukinumab (Cosentyx) in 151 adults with newly diagnosed giant cell arteritis, a condition causing inflamed blood vessels. Participants were in remission and received either secukinumab or a placebo, along with a steroid taper. The goal was to see if s…

This study reviewed medical records of 13 Korean children with severe aplastic anemia, a condition where the bone marrow stops making enough blood cells. Researchers wanted to see if adding eltrombopag to standard immunosuppressive therapy was safe and effective. Over 6 months, t…

This study tested a new biologic drug called VAY736 in 68 adults with autoimmune hepatitis who did not get better with or could not tolerate standard treatments. The goal was to see if VAY736 could safely help normalize liver enzyme levels. Participants received either VAY736 or …

This study tested a drug called ianalumab in 21 adults with Sjögren's syndrome, an autoimmune disease that causes dry eyes and mouth. The goal was to understand how the drug works in the salivary glands and check its safety. Participants received injections of ianalumab and had b…

This study looked at health records of 1,249 heart failure patients in Portugal to compare two types of medication: sacubitril/valsartan versus older drugs called ACE inhibitors or ARBs. The goal was to see which treatment better prevents heart failure hospitalizations or death f…

This phase 2 trial tested an experimental drug called LRX712, given as injections into the knee, to see if it can help regrow cartilage in people with mild to moderate knee osteoarthritis. 45 participants received either the drug or a placebo over 8 weeks. The main goal was to me…

This study tested whether adding the drug atrasentan to standard care (including SGLT2 inhibitors) can reduce protein in the urine of people with IgA nephropathy, a kidney disease. 54 adults with biopsy-confirmed IgA nephropathy participated. The main goal was to see if atrasenta…

This study tested a new malaria drug called KLU156 in over 1,700 adults and children with uncomplicated malaria. It compared KLU156 to a standard treatment, Coartem, to see if it works as well. Some participants continued taking KLU156 for up to two years to check its safety with…

This study looked at how well the drug ribociclib works in real-world settings for Indian women with a common type of advanced breast cancer (HR+, HER2-). Researchers reviewed medical records of 335 patients treated across 18 sites in India. The goal was to measure how long the d…

This phase 4 trial tested the drug nilotinib in 34 adults with advanced GIST that could not be removed by surgery. The goal was to see how many patients had their tumors shrink or stay stable after 6 months. Researchers measured response using standard tumor scans. The study prov…

This study tested whether a twice-yearly injection (inclisiran) lowers LDL cholesterol better than a daily pill (bempedoic acid) in 402 adults with heart disease who still have high cholesterol despite taking statins. Participants received either inclisiran or bempedoic acid on t…

This study tested a new drug called XXB750 in 189 people with resistant hypertension—high blood pressure that doesn't get better with standard treatments. Participants received either a placebo or one of several doses of XXB750 as a shot under the skin, on top of their usual medi…

This study looked at whether people with relapsing multiple sclerosis can safely switch from an intravenous (IV) anti-CD20 antibody treatment to a self-injected drug called ofatumumab. 111 adults took part, and researchers tracked their brain scans and side effects for a year. Th…

This study tested whether adding a daily pill called QAW039 to standard asthma treatments helps people with uncontrolled asthma breathe better. Over 700 adults and teens took either QAW039 or a placebo for 12 weeks. The main goal was to see if lung function (measured by how much …

This study tested an oral medication called iptacopan in 52 adults with Paroxysmal Nocturnal Hemoglobinuria (PNH) who were already stable on standard infusion treatments. Participants switched from their usual therapy to iptacopan pills taken twice daily. The main goal was to see…

This study tested a drug called inclisiran in teenagers aged 12 to 18 who have a rare inherited condition that causes extremely high cholesterol. The goal was to see if the drug safely lowers their LDL (bad) cholesterol. Participants received either inclisiran or a placebo for on…

This study tested a one-time gene therapy called OAV101 (Zolgensma) in 27 children aged 2 to 18 with spinal muscular atrophy (SMA) who had previously stopped taking other SMA medications (Spinraza or Evrysdi). The goal was to see if the treatment is safe and can help maintain or …

This study looked at how well and how safely the drug fingolimod (Gilenya) works for people in China with relapsing multiple sclerosis (RMS), a condition where the immune system attacks the nerves. Over 24 months, 98 participants took a daily 0.5 mg pill. The main goal was to see…

This study tested a new inhaler called QMF149, which combines two drugs (indacaterol and mometasone), against a standard steroid inhaler (mometasone alone) in 802 adults and teens with mild asthma. The goal was to see if the combination improved lung function and symptom control …

This study tested a drug called crizanlizumab in 36 men with sickle cell disease who suffer from priapism—painful erections lasting an hour or more. The goal was to see if the drug could reduce how often these events happen. Participants received the drug by IV and reported their…

This study tested the drug ofatumumab in 99 Chinese adults with relapsing multiple sclerosis (RMS). The goal was to see if monthly injections could reduce the number of relapses and monitor for side effects. Participants received the drug for 12 months, and researchers tracked re…

This study tested whether a daily pill called QAW039 can help people with uncontrolled asthma breathe better. 675 adults and teens took either the drug or a placebo for 12 weeks, on top of their usual inhalers. The main goal was to see if lung function improved.

This study tested whether adding amlodipine to the blood pressure drug LCZ696 works better than LCZ696 alone. About 718 adults with mild-to-moderate high blood pressure that wasn't well controlled by LCZ696 took part. The goal was to see if the combination lowers blood pressure m…

This study tested whether adding eltrombopag (a platelet booster) to standard immune-suppressing drugs (r-ATG and cyclosporine A) helps patients with severe aplastic anemia, a rare condition where the bone marrow stops making enough blood cells. Thirty-six East-Asian adults and c…

This study tested a one-time gene therapy called OAV101 in 126 children aged 2 to 18 with type 2 spinal muscular atrophy (SMA) who had never received other SMA treatments. The therapy was given as a spinal injection and compared to a sham procedure. The goal was to see if it coul…

This study tested a drug called inclisiran in teenagers aged 12 to 18 who have a genetic condition that causes very high cholesterol. The goal was to see if the drug is safe and can lower bad cholesterol. Participants received either inclisiran or a placebo for one year, then eve…

This study tested a drug called inclisiran in 207 Chinese adults with moderately high bad cholesterol (LDL-C) who were not taking other cholesterol medicines. Participants received either inclisiran or a placebo for 6 months, followed by 6 months where everyone got the drug. The …

This study tested a drug called alpelisib combined with hormone therapy (fulvestrant or letrozole) in 383 people with advanced breast cancer that has a specific genetic change (PIK3CA mutation). The goal was to see how many patients were alive without their cancer getting worse a…

This study tested a combination of two targeted drugs, dabrafenib and trametinib, in 40 Chinese adults with a specific type of advanced lung cancer (BRAF V600E mutation-positive NSCLC). The goal was to see if the drugs could shrink tumors and control the cancer. Participants rece…

This study tested a drug called eltrombopag in 20 Chinese adults with severe aplastic anemia, a condition where the bone marrow stops making enough blood cells. Participants had not responded to previous treatments. The drug was given daily for up to a year to see if it could imp…

This completed study in Spain looked at 310 people with relapsing multiple sclerosis who were taking ofatumumab (Kesimpta®). Researchers wanted to see how well the drug reduced relapses and how safe it was in everyday medical practice. The study collected information from medical…

This study followed 42 children aged 6 to 17 with moderate to severe plaque psoriasis in China who were treated with Cosentyx® (secukinumab) for up to 52 weeks. The goal was to see how safe and effective the drug is in real-world settings. Researchers tracked side effects and mea…

This early-stage trial tested a drug called NIO752 in 59 people with progressive supranuclear palsy (PSP), a rare brain disorder that affects movement and balance. The drug was given as a spinal injection to see if it is safe and how the body processes it. The main goal was to ch…

This phase 3 trial tested iptacopan (LNP023) in 518 adults with IgA nephropathy, a kidney disease that can lead to kidney failure. Participants received either the drug or a placebo, on top of standard care. The goal was to see if iptacopan reduces protein in the urine and slows …

This study tested a personalized treatment plan using the drug brolucizumab for people with a specific eye condition called polypoidal choroidal vasculopathy (PCV). The goal was to see if adjusting the treatment schedule based on each person's needs could maintain or improve visi…

This study tested whether adding the drug ruxolitinib to standard steroids helps children with chronic graft-versus-host disease (cGVHD), a complication after stem cell transplants. 46 children aged 28 days to 18 years took part. The main goal was to see how many had a complete o…

This early-stage study tested a new drug called QCZ484 in 56 healthy volunteers and people with mild hypertension. The main goal was to check safety and how the body handles the drug. Participants received a single injection of either QCZ484 or a placebo, and researchers monitore…

This early-stage study tested two versions of an experimental drug called ianalumab in 48 adults with active rheumatoid arthritis. The goal was to see if the two forms are similar enough in safety and how the body processes them. Participants received multiple injections, and res…

This Phase 2 trial tested an experimental drug called INE963 in 52 adults with uncomplicated Plasmodium falciparum malaria. The main goal was to see how quickly the drug clears malaria parasites from the blood. Researchers also checked the 28-day cure rate. The study is part of a…

This study tested whether adding the drug ribociclib to standard hormone therapy helps slow the growth of advanced breast cancer in Chinese women. About 327 women with a specific type of breast cancer (HR+, HER2-) took part. The main goal was to see how long the cancer stayed und…

This study tested two biologic drugs, secukinumab and ustekinumab, in 119 adults with active psoriatic arthritis who had not responded to or could not tolerate TNFα-inhibitor treatment. The main goal was to see which drug better improved physical function over 28 weeks. Researche…

This study tested a lower starting dose (400 mg) of the targeted drug ribociclib combined with standard hormone therapy (letrozole or anastrozole) in 376 women with advanced hormone receptor-positive, HER2-negative breast cancer. The goal was to see if the lower dose could contro…

This study tested a drug called remibrutinib in 144 adults with chronic spontaneous urticaria (hives lasting over 6 months) that standard antihistamines couldn't control. Participants took a 25 mg tablet twice daily for up to 12 weeks. The main goal was to see if the drug affecte…

This study looked at the long-term safety and effectiveness of the drug secukinumab in children with two types of juvenile idiopathic arthritis: juvenile psoriatic arthritis and enthesitis related arthritis. Fifty-five children who had already completed a previous study continued…

This study tested a new medicine called inclisiran in 350 people with high cholesterol who were not taking any other cholesterol drugs. The goal was to see if inclisiran could lower 'bad' LDL cholesterol more than a placebo or another drug called ezetimibe. Participants received …

This study tested whether adding a drug called inclisiran to standard care helps lower 'bad' LDL cholesterol more effectively in people who recently had a heart attack. About 400 participants received either the new care pathway including inclisiran or usual care alone. The goal …

This study tested a new drug called asciminib in 56 adults with chronic myeloid leukemia (CML) whose cancer had not responded well to other treatments. The drug is taken as a pill and aims to control the disease. The main goal was to check safety and side effects over 24 weeks, w…

This extension study tested the safety and tolerability of two doses of iscalimab in 206 people with Sjögren's syndrome who had already completed a prior study. Participants received weekly injections for three weeks, then bi-weekly doses. The goal was to see if the drug is safe …

This phase 3 trial tested whether a monthly injection of erenumab could reduce the number of migraine days in adults with chronic migraine (15 or more headache days per month). Over 550 Asian participants received either the drug or a placebo for 12 weeks. The main goal was to se…

This study tested a drug called crizanlizumab in 117 children aged 2 to 18 with sickle cell disease who have had painful vaso-occlusive crises. The goal was to find the right dose and check safety, with some children also taking hydroxyurea. The drug works by blocking a protein i…

This completed study looked at whether a dedicated clinic focused on guideline-directed medical therapy (GDMT) helps heart failure patients get the right combination of medications. Researchers analyzed data from 342 patients at Massachusetts General Hospital who attended the GDM…

This study tested whether an eye injection called brolucizumab works as well as laser treatment for people with proliferative diabetic retinopathy, a serious eye condition caused by diabetes. 689 participants received either the injection or laser, and researchers measured vision…

This study looked at the safety of the drug sacubitril/valsartan in Japanese children with heart failure. Eight children who had already been in a previous trial took the drug to see if any side effects occurred. The goal was to gather more safety information and provide the drug…

This study tested the safety of a drug called crizanlizumab in 140 Indian patients aged 16 and older with sickle cell disease who have had painful crises. The drug was given by IV infusion over several months, with or without standard hydroxyurea therapy. The main goal was to che…

This study compared two different dosing schedules of brolucizumab (Beovu) in 52 people with wet age-related macular degeneration (AMD) who had not responded well to previous treatment. Participants received the drug as an eye injection either on a fixed schedule or with adjustme…

This study tracked 109 patients with a specific genetic mutation (MET exon 14 skipping) causing advanced lung cancer who were prescribed Tabrecta tablets. Researchers monitored side effects and how well the drug controlled the cancer over up to one year. The goal was to see how T…

This study looked at whether people with chronic myeloid leukemia (CML) who have done very well on the drug nilotinib can safely stop taking it. 215 patients who had been on nilotinib for at least two years and had very low levels of cancer cells were enrolled. The main goal was …

This study looked at whether people with chronic myeloid leukemia (CML) who had very low levels of cancer cells could safely stop taking the drug nilotinib (Tasigna). Participants had already been treated with imatinib (Gleevec) and then nilotinib for at least 3 years. The main g…

This study tested a new imaging method called [68Ga]Ga-DOTA-TATE PET/CT to see how well it finds neuroendocrine tumors compared to standard scans. It included 71 adults in Japan, both with and without the disease. The goal was to prove the new scan works well for diagnosis in Jap…

This completed Phase 3 study tested a new radioactive tracer called [18F]CTT1057 for PET/CT scans in 190 men with prostate cancer whose PSA levels were rising after initial treatment. The goal was to see if the tracer could accurately locate where the cancer had returned. Partici…

This study tested a new imaging agent called [18F]CTT1057 for PET scans to see how well it can find prostate cancer tumors. 195 men with high-risk prostate cancer who were scheduled for surgery received the scan. The results were compared to tissue samples taken during surgery to…

This study tested an experimental drug called DFV890 in 115 adults aged 50-80 with painful knee osteoarthritis. Participants received either the drug or a placebo to see if it reduced pain and inflammation. The goal was to find a new way to ease symptoms and improve daily functio…

This study tested Xolair (omalizumab) in 50 teenagers aged 12 to 17 with severe seasonal allergic rhinitis (hay fever) that standard treatments couldn't control. Researchers tracked side effects and how much symptoms improved. The goal was to see if Xolair is safe and effective i…

This study looked at whether Xolair (omalizumab) is safe and works for Chinese teenagers with chronic spontaneous urticaria (long-term hives and itching) that doesn't get better with standard antihistamines. 55 teens took part, receiving Xolair for 12 weeks, followed by 4 weeks o…

This study tested whether secukinumab, an anti-inflammatory drug, can reduce pain and improve function in adults with moderate to severe rotator cuff tendinopathy (shoulder tendon pain) that didn't get better with standard treatments. 62 participants received either the drug or a…

This study tested a drug called ligelizumab in 49 teenagers aged 12 to 17 who had chronic hives (itchy welts) that didn't get better with standard allergy pills. The goal was to see if adding ligelizumab to their current treatment could reduce the number and severity of hives. Pa…

This study tested a drug called omalizumab in 337 adults and teens with severe Japanese cedar pollen allergy (hay fever) that standard medicines couldn't control. Participants received either omalizumab or a placebo on top of their usual allergy treatments for 12 weeks. The goal …

This study tested a drug called canakinumab (ACZ885) in 49 children and young adults aged 8-20 with sickle cell anemia. The goal was to see if it could reduce daily pain compared to a placebo. Participants rated their pain daily using a 0-10 scale, and researchers measured change…

This early-stage study tested a new drug called XXB750 in 27 adults with stable heart failure and reduced heart pumping ability. The main goal was to see if the drug is safe and tolerable. Participants received either the drug or a placebo, and researchers monitored for side effe…

This study followed 69 people in Taiwan with lupus nephritis who were newly prescribed the drug Myfortic (mycophenolate sodium). Researchers tracked side effects and reasons for stopping the drug over 12 months. The goal was to understand the drug's safety in this specific popula…

This study examines the patient profiles and treatment patterns of adults with melanoma in Japan who were prescribed either dabrafenib with trametinib or encorafenib with binimetinib. Researchers are looking at data from 2012 to 2021 to understand patient characteristics and how …

This study looked at 117 people with gastro-enteropancreatic neuroendocrine tumors (GEP-NETs) who received a type of radiation therapy called PRRT in German clinics. Researchers collected information on patient characteristics, treatment timing, and how PRRT was given in everyday…

This study aims to understand how satisfied both doctors and patients are with current multiple sclerosis (MS) treatments in everyday medical practice. Researchers will collect information from 261 patients and their physicians across several countries, including the UK, France, …

This study looks at medical records from 750 people with a common type of early breast cancer (HR+/HER2-). It aims to understand how doctors treat these patients in real life, especially after newer targeted drugs called CDK4/6 inhibitors became available. By analyzing treatment …

This study tested whether long-term digital education, like online lessons or app-based guidance, helps people who had a heart attack lower their LDL (bad) cholesterol more than standard care. Researchers followed 121 patients in the Czech Republic for 12 months. The goal was to …

This study followed 307 people with relapsing multiple sclerosis in Germany who had recently switched to the drug Kesimpta (ofatumumab) from another treatment. Researchers collected information on why patients made the switch and how many doses they missed over one year. The goal…

This completed study monitored pregnancy and infant outcomes in 13 women with sickle cell disease who were treated with the drug crizanlizumab (Adakveo) during pregnancy or shortly before. The goal was to track rates of fetal loss and birth defects. Because it was a small observa…

This completed study followed 1868 patients with high cholesterol to see how inclisiran (Leqvio®) and other cholesterol-lowering treatments work in real-world medical practice. Researchers tracked heart events, medication use, and lab results without assigning any treatments. The…

This study used blood and urine samples from 236 Japanese patients with chronic heart failure to see how biomarkers like BNP change during treatment with sacubitril valsartan or enalapril. Researchers looked for links between biomarker changes and improvements in heart failure sy…

This study examined how the drug omalizumab is prescribed in real-world practice for adults with chronic urticaria (long-term hives) in China. Researchers analyzed electronic health records from over 3,400 patients to see what doses were commonly used. The goal was to understand …

This completed study looked at 480 patients with HR+, HER2- metastatic breast cancer who were treated with CDK4/6 inhibitors as their first therapy. Researchers examined patient profiles, treatment patterns, and outcomes in everyday clinical practice. The goal was to understand h…

This study looked at medical records from over 218,000 adults in England with a common type of breast cancer (HR+/HER2-). Researchers tracked which treatments people received, how the disease progressed over time, and how healthcare resources were used. The goal was to understand…

This study looked at how the drug remibrutinib behaves in people with severe kidney problems compared to healthy people. Researchers measured drug levels in blood and urine to understand how the kidneys process it. The goal is to help doctors decide the right dose for patients wi…

This study observed over 2,400 women in Russia with advanced HR+/HER2- breast cancer to see how well different treatments work in everyday medical practice. Researchers tracked how long patients stayed on their therapy before it stopped working or caused side effects. The goal wa…

This study analyzed medical records of 213 children with spinal muscular atrophy (SMA) in the Czech and Slovak Republics. Researchers looked at what treatments were used and how the children's motor skills, breathing, and feeding changed over time. The goal was to understand real…

This study looked at over 1,200 men with advanced prostate cancer who received a radioactive drug called Lutetium-177 vipivotide tetraxetan in real-world clinics. Researchers tracked patient characteristics, treatment patterns, and PSA level reductions. The goal was to see how th…

This study looked at how well people with relapsing multiple sclerosis (RMS) follow their Kesimpta (Ofatumumab) treatment plan. Researchers used data from the MSGo patient support app to track when doses were taken. They also compared how patients felt (reported symptoms) with th…

This study examined medical records of over 6,400 prostate cancer patients who received a treatment called 177Lu-PSMA-617. Researchers wanted to understand the patients' characteristics, treatment patterns, and survival outcomes. The study did not test a new treatment but instead…

This completed observational study in India enrolled 595 people with HR-positive, HER2-negative advanced or metastatic breast cancer who were already taking alpelisib plus fulvestrant. Researchers looked for PIK3CA gene mutations in tumors and tracked how well the cancer responde…

This study followed over 2,300 adults with moderate to severe plaque psoriasis to see how safe and effective secukinumab is in everyday medical practice. Researchers recorded side effects and how well the drug cleared skin symptoms. The goal was to gather real-world evidence, not…

This study looked at medical records from over 11,900 adults in China who took either inclisiran or PCSK9 mAbs to lower their high cholesterol. Researchers wanted to see how well these drugs worked in everyday medical practice, not just in controlled trials. The study found that …

This study analyzed health records of nearly 5,000 people with spinal muscular atrophy (SMA) to see how well three approved treatments work in everyday medical practice. Researchers looked at complications, medical equipment use, and hospital visits before and after starting each…

This study looked back at medical records of 1,480 people with chronic-phase chronic myeloid leukemia (CML) treated in U.S. community oncology clinics between 2016 and 2022. Researchers examined which drugs were used, how doses were adjusted, and how well patients responded. The …

This study analyzed insurance claims from over 4,000 people with spinal muscular atrophy to see how three approved treatments (Zolgensma, Spinraza, and Evrysdi) are actually used in daily practice and what they cost over time. Researchers looked at dosing patterns, yearly drug co…

This study observed over 2,600 women in Germany with a common type of advanced breast cancer (HR+/HER2-). Researchers compared how long the cancer stayed under control when women started treatment with a drug combo (ribociclib plus hormone therapy) versus other hormone therapies …

This study reviewed medical records of 355 people in Turkey with myelofibrosis (a type of bone marrow cancer) who were treated with the drug ruxolitinib for at least 3 months. Researchers collected information on patient demographics, disease characteristics, and how the drug was…

This completed survey looked at whether healthcare professionals and people with multiple sclerosis (or their caregivers) understand the safety steps needed when using the drug Mayzent (siponimod). Over 300 participants from Europe answered questions about proper use, side effect…

This study looked at the real-world safety of LUTATHERA, a radioactive drug used to treat a type of nerve and hormone cell cancer called neuroendocrine tumors. Researchers tracked 347 patients who received the drug in regular medical practice, focusing on kidney problems, bone ma…

This study looked at how well the combination of dabrafenib and trametinib works in real-life settings for people with high-risk stage III melanoma that has been completely removed by surgery. It included 39 adults in Turkey whose melanoma had a specific genetic mutation (V600E).…

This completed study interviewed 28 people who had sepsis-associated acute kidney injury (SA-AKI) to learn about their symptoms, impacts, and treatment experiences. Participants shared their perspectives in 60-minute interviews. The goal was to better understand what patients go …

This study checked how well German hospitals and heart failure clinics follow recommended treatments for people with a certain type of heart failure (HFrEF) after a serious episode. Researchers looked at the care of 899 patients to see if guidelines were being used. The goal was …

This study tested an experimental drug called TIN816 in 20 adults in the intensive care unit who had kidney injury from sepsis. The main goal was to learn how the drug behaves in the body and whether it is safe. This was an early-stage study, so it focused on gathering informatio…

This completed study observed 572 patients with neovascular age-related macular degeneration or diabetic macular edema who were already receiving brolucizumab injections. Researchers collected routine medical data to see how well the drug resolved fluid in the eye and improved vi…

This completed study followed 127 adults with moderate to severe plaque psoriasis, psoriatic arthritis, ankylosing spondylitis, or non-radiographic axial spondyloarthritis who were already taking or starting secukinumab. Researchers observed them for two years to see how long the…

This completed observational study followed 367 people with relapsing-remitting or active secondary progressive multiple sclerosis who were taking Kesimpta for the first time. Researchers tracked side effects and how well the drug worked in everyday use. The goal was to gather re…

This completed study looked at how CDK4/6 inhibitors are used in everyday practice for advanced breast cancer in Portugal. Researchers analyzed data from 16 public hospitals to understand dosing patterns, treatment duration, and how often doses were adjusted. The study included n…

This study looked at hospital records from 2015 to 2022 to find out how many people in France have a group of rare genetic conditions called PROS. Researchers counted new cases each year, described patient ages and other health problems, and estimated costs. No treatments were te…

This study looked at how the drug JDQ443 behaves in people with different levels of liver function. It included 33 adults with small cell lung cancer and mild, moderate, or severe liver problems, plus healthy volunteers. The goal was to measure drug levels in the blood to guide s…

This study used national health survey data to estimate how many US adults with high cholesterol might be eligible for a new drug called inclisiran. Researchers also calculated how many heart attacks and strokes could be prevented over 10 years if these patients took the drug. Th…

This study looked at how 2,298 older adults with chronic myeloid leukemia (CML) were treated in everyday medical practice, using Medicare records. Researchers tracked which drugs patients took, how often they switched treatments, and how much healthcare they used. The goal was to…

This completed study surveyed 134 adults with multiple sclerosis who were starting treatment with either Kesimpta or the newer subcutaneous (under-the-skin) version of Ocrevus. The goal was to compare how patients felt about their first injection, including any pain, redness, swe…

This study looked back at medical records of 606 people with secondary progressive multiple sclerosis (SPMS) in Russia who were taking siponimod. Researchers wanted to understand patient characteristics and how the drug was used in everyday practice. The study collected data from…

This study analyzed insurance claims from over 2,500 people with chronic myeloid leukemia (CML) to see how often side effects from tyrosine kinase inhibitors (TKIs) occur and how they affect treatment changes and healthcare costs. Researchers looked at side effects that required …

This study looked at how 754 people with chronic myeloid leukemia (CML) in Japan were treated with drugs called tyrosine kinase inhibitors (TKIs) between 2011 and 2019. Researchers used a hospital database to track which TKIs patients received, how often they switched or stopped …