Sumida City

Researchers are evaluating MF1, a new drug aimed at targeting the underlying pathological mechanisms of alpha-synucleinopathies, primarily Parkinsons disease PD. This Phase I first-in-human study seeks to assess the safety, tolerability, and pharmacokinetics of MF1, addressing a critical unmet medical need as current therapies only treat symptoms without modifying disease progression. The study involves healthy Japanese male adults and patients with idiopathic PD, reflecting different stages of clinical development. The trial has three parts Part A tests single ascending oral doses of MF1 and includes a food-effect assessment in healthy males aged 18 to under 45 years. Part B administers once-daily MF1 or placebo for 7 days in the same healthy population. Part C is an open-label study giving once-daily MF1 for 14 days to patients aged 40 to under 85 years with idiopathic PD Hoehn & Yahr stage 3 or below, with or without stable background antiparkinsonian treatments. The study also evaluates cerebrospinal fluid penetration and exploratory biomarkers related to neurodegeneration. Participants will undergo safety monitoring including adverse event assessments and pharmacokinetic sampling of plasma, urine, and cerebrospinal fluid. Biomarkers such as alpha-synuclein and others will be measured to explore pharmacodynamic effects. The study includes randomized, placebo-controlled parts in healthy volunteers and an open-label part in PD patients, with follow-up for up to 12 days after dosing. The total participation duration varies by study part, with careful review before progressing between stages.

Primary immune thrombocytopenia ITP is a condition where the immune system mistakenly destroys platelets, leading to a lower number of platelets and increased risk of bruising or bleeding. This Phase 3 study evaluates the long-term safety, tolerability, and effectiveness of mezagitamab in adults with chronic primary ITP. The study also investigates how the body processes mezagitamab over an extended period. Participants who completed previous mezagitamab studies TAK-079-3002 or TAK-079-1004 will be invited to join this continuation trial. Eligible participants may receive mezagitamab injections on demand, with treatment courses repeated as needed based on specific criteria and the investigators clinical judgment. The treatment is administered subcutaneously. During the study, participants will visit the clinic several times for assessments. Researchers will monitor safety by tracking treatment-emergent adverse events, and evaluate effectiveness through platelet response and remission rates. Measurements of drug levels and antibodies will also be taken. The study may last up to approximately 108 weeks, allowing detailed long-term follow-up.

Researchers are evaluating oral icotrokinra as a treatment for adults and adolescents with moderately to severely active ulcerative colitis, a chronic inflammatory disease of the large intestine causing ulcers in the colon lining. The study aims to assess how well icotrokinra works, along with its safety and tolerability in this population. This is a Phase 3, randomized, double-blind, placebo-controlled trial with a parallel group design including both adults and adolescents. Adult participants will be randomly assigned to receive either icotrokinra or placebo daily by mouth during a 12-week induction phase. At Week 12, those showing clinical response will enter a maintenance phase where they will continue icotrokinra or placebo daily for 40 weeks. Adults who do not respond will also enter the maintenance phase and receive icotrokinra. Adolescents will receive open-label icotrokinra during induction and then continue on icotrokinra during maintenance regardless of response. After completing the 40-week maintenance phase, eligible participants may join a long-term extension study. Participants will be monitored regularly through clinical assessments at specified time points including Week 12 for induction and Week 40 for maintenance. Outcomes measured include rates of clinical remission, symptom improvement, endoscopic and histologic healing, and quality of life scores. Safety will be evaluated by tracking adverse and serious adverse events throughout the study. The total study duration may extend up to approximately 6 years, ending in 2032, allowing long-term evaluation of icotrokinra in ulcerative colitis management.

Researchers are evaluating the safety and effectiveness of the drugs eltrekibart and mirikizumab in adults with moderately to severely active ulcerative colitis UC, a chronic inflammatory bowel disease. This Phase 2 study aims to find out how these treatments work alone or in combination to improve UC symptoms and health. The study is sponsored by Eli Lilly and Company and will last about 4 to 5 years, including screening. Participants will be randomly assigned to receive one of several treatments eltrekibart with a placebo, mirikizumab with a placebo, both drugs together, or placebo alone. The study is double-blinded, meaning neither the participants nor the researchers know which treatment is given to ensure unbiased results. The treatment period includes dosing with the study drugs or placebo, and participants will be monitored for safety and response. Participants will be involved in the trial for around 69 weeks, including a screening period of up to 35 days before starting treatment. Throughout the study, they will undergo assessments to measure clinical remission, response, endoscopic improvement, and quality of life using questionnaires. Blood samples will be taken to study drug levels, and researchers will monitor safety and disease activity regularly. The main outcome is the percentage of participants achieving clinical remission at Week 12, with additional measures assessed up to Week 52.

Researchers are evaluating the effectiveness and safety of JNJ-78934804 compared to guselkumab in people with moderately to severely active ulcerative colitis UC, a chronic condition where the colon lining becomes inflamed and develops ulcers. This Phase 3 study aims to measure clinical remission and other health improvements by Week 48. Participants receive an induction dose of either JNJ-78934804 or guselkumab at Weeks 0, 4, and 8, followed by maintenance doses every 4 weeks starting Week 12. Those meeting rescue criteria will receive additional induction doses of JNJ-78934804 at Weeks 16, 20, and 24, then maintenance doses every 4 weeks from Week 28. After completing the 48-week double-blind treatment phase, participants benefiting from the intervention may join a long-term extension phase. Throughout the study, participants will be monitored for clinical remission, endoscopic improvement, corticosteroid-free remission, fatigue, abdominal pain, quality of life, and mental health responses. Safety is assessed by tracking adverse events up to about 3 years. The study involves randomized assignment and double-blinding, with all treatments given by subcutaneous injection. Participation may last up to several years including follow-up and extension phases.

Primary immune thrombocytopenia ITP is a condition where the immune system mistakenly destroys platelets, the cells responsible for blood clotting, resulting in increased bleeding and bruising. This trial evaluates whether mezagitamab given as a subcutaneous injection can effectively maintain stable platelet counts in adults with chronic ITP compared to a placebo. The study is a phase 3, randomized, double-blind, placebo-controlled trial designed to assess both the efficacy and safety of mezagitamab in this population. Participants are randomly assigned to receive either mezagitamab or a matching placebo by subcutaneous injection once weekly. The treatment schedule consists of 8 weekly doses followed by an 8-week break, then another 8 weekly doses, totaling up to 6 months of treatment. Those who finish this trial or do not respond to treatment by week 16 may have the option to join a continuation study to receive open-label mezagitamab if eligible and if the site is open. During the study, participants will visit the clinic multiple times for treatment and monitoring. Researchers will measure platelet responses, symptom changes using patient questionnaires, bleeding events, and blood concentrations of mezagitamab. Safety assessments include monitoring for anti-drug antibodies and adverse events. The primary outcome is the percentage of participants with a durable platelet response up to week 24. The trial is expected to complete in March 2028.

Polycythemia vera PV is a rare blood cancer where the body produces too many red blood cells, causing thicker blood and raising the risk of serious problems like blood clots. This study aims to see if rusfertide can help Japanese adults with PV keep their hematocrit levels under control and reduce the need for regular blood removal procedures called phlebotomies. The study is a phase 2, open-label trial, meaning both participants and researchers know the treatment being given. Participants will receive rusfertide injections under the skin once a week for an initial 52-week period, followed by a long-term extension period of 182 weeks where treatment continues. After the treatment phases, there is a 4-week safety follow-up to monitor participants. All participants receive rusfertide, and no placebo or comparator group is involved. During the study, participants will have regular hematology tests to monitor their blood levels and hematocrit control. Researchers will track the number of phlebotomies needed and measure the percentage of participants maintaining hematocrit below 45%. Follow-up visits and assessments will continue for up to about 244 weeks, including screening, treatment, extension, and safety follow-up to evaluate the treatments impact and safety.

Narcolepsy Type 2 NT2 is a lifelong condition causing excessive daytime sleepiness, difficulty staying awake, and problems with concentration and memory, impacting daily life activities. Researchers are evaluating TAK-360, a drug that acts like orexin, a brain chemical that promotes wakefulness, to understand its safety, tolerability, and ability to help adults with NT2 stay awake. This is a Phase 2, randomized, double-blind, placebo-controlled study to find the right dosage of TAK-360. Participants will be randomly assigned to receive either TAK-360 tablets or matching placebo tablets orally for 4 weeks. TAK-360 is being studied to assess its effects compared to placebo. The study uses a parallel design and includes a 4-week treatment period where participants take the assigned tablets daily. During the study, participants will be monitored for treatment-emergent adverse events for up to 15 weeks. Researchers will assess wakefulness using tools like the Epworth Sleepiness Scale and the Maintenance of Wakefulness Test at baseline and after 4 weeks of treatment. Safety, tolerability, and efficacy data will be collected throughout the study, which runs until November 2026.

Researchers are evaluating the safety and pharmacokinetics PK of AZD7760, a combination of two monoclonal antibodies, in healthy Japanese adults. This phase I, randomized, double-blind, placebo-controlled study focuses on assessing how the body processes AZD7760 and monitors safety in adults aged 18 to 55 years without significant health issues. The trial aims to better understand the effects of this investigational drug for potential prevention of Staphylococcus aureus bloodstream infection. Participants will receive a single intravenous IV infusion of one of two doses of AZD7760 or a matching placebo on Day 1. The study consists of a screening period lasting up to 28 days, followed by the dosing period of 3 days during which the infusion occurs on the first day. After dosing, participants will be followed for 12 months to monitor safety and drug levels in the body. During the study, participants will undergo various safety assessments including monitoring for adverse events, medically attended adverse events, and special interest events for up to 361 days. Blood samples will be collected to measure drug concentration and immune response. Participants will be evaluated regularly through laboratory tests, physical exams, and questionnaires to ensure safety and gather pharmacokinetic data throughout the follow-up period.

Researchers are evaluating the levels of drug exposure in adults with locally advanced unresectable or metastatic gastric or gastroesophageal junction adenocarcinoma. This Phase 3 study compares tislelizumab given by subcutaneous injection versus intravenous infusion as first-line therapy combined with chemotherapy. The study involves approximately 351 participants and aims to understand the treatments effects in this patient group. Participants will be randomly assigned to receive either tislelizumab 300 mg by subcutaneous injection or 200 mg by intravenous infusion on Day 1 of each 21-day cycle. Both groups will also receive chemotherapy tailored to each patient. The study includes a screening period, a treatment period with repeated cycles, and a follow-up period to assess long-term outcomes. During the study, participants will be monitored through various assessments, including tumor evaluations and biomarker tests. Researchers will measure drug concentration levels, response rates, progression-free survival, overall survival, and adverse events for up to two years. Safety and treatment effectiveness will be closely followed throughout the study duration, which ends in April 2028.