Iruma

Researchers are evaluating ASTX030, an oral azacitidine AZA formulation combined with cedazuridine CED tablets, in patients with Myelodysplastic Syndrome MDS. This Phase 1 study aims to identify doses of these oral formulations that achieve a similar total exposure AUC to that of the standard subcutaneous AZA injection at 75 mgm2, including separate parts focusing on tolerability and expansion assessments. During the tolerability assessment part, patients receive subcutaneous AZA on Day 1 of Cycle 1 followed by oral AZA and CED on Days 2 to 7, with oral dosing continued on Days 1 to 7 in subsequent cycles. The expansion assessment part uses a crossover design where patients alternate between ASTX030 and subcutaneous AZA in the first two cycles, then continue ASTX030 from Cycle 3 onward, with each cycle lasting 28 days. Participants will be monitored through pharmacokinetic measurements comparing total AUC of AZA between oral and injection forms up to 2 months for the expansion part and up to 1 month for the tolerability part. Safety and tolerability are assessed, and participants must meet specific organ function and performance status criteria. The study spans multiple cycles with scheduled dosing and evaluations to compare the investigational oral treatment to standard injection therapy.

Researchers are evaluating whether baricitinib can delay the development of clinical stage 3 type 1 diabetes T1D in children and adults at high risk. This phase 3, double-blind, randomized, placebo-controlled study includes participants aged 1 to under 36 years who have early stages of T1D or specific diabetes-related autoantibodies. The study aims to better understand preventing or delaying the onset of clinical diabetes in this at-risk population. Participants will be randomly assigned to receive either baricitinib or a placebo orally. The study lasts up to approximately 5 years, during which participants take the assigned treatment and are monitored regularly. The trial includes a control group receiving placebo to compare with the baricitinib group. The dosing schedule and exact treatment duration depend on the study protocol and participant response. Throughout the study, participants will undergo assessments including monitoring the time to diagnosis of stage 3 T1D, blood tests measuring glucose and C-peptide levels, body measurements, and health surveys. Researchers will track changes in these measures over time, including pharmacokinetics of baricitinib. Safety and response to treatment will be closely observed throughout the study duration to evaluate the effects of the medication.

Researchers are studying baricitinib to see if it can help preserve beta-cell function in children and adults aged 1 to 35 years who have been newly diagnosed with type 1 diabetes. This Phase 3 study aims to evaluate the treatments impact on preserving insulin production shortly after diagnosis. Participants will be followed for about 60 weeks to assess changes in key diabetes-related measures. Participants will be randomly assigned to receive either baricitinib or a placebo, both taken orally. The study compares these two groups to evaluate the effects of baricitinib on beta-cell function. The main measurement is the change in C-peptide area under the curve over 52 weeks, which indicates insulin production. Additional outcomes include changes in blood sugar control, insulin use, hypoglycemia events, and other health indicators. During the study, participants will attend visits for assessments and monitoring over approximately 60 weeks. Tests will include blood measurements like C-peptide and hemoglobin A1c, insulin dose tracking, and health surveys. Safety and drug levels will be monitored. Researchers will use these data to understand if baricitinib can help maintain beta-cell function in people newly diagnosed with type 1 diabetes.

Researchers are evaluating the efficacy and safety of brenipatide combined with standard of care compared to placebo plus standard of care in delaying the worsening of symptoms in adults with bipolar disorder. This Phase 2, randomized, double-blind study aims to understand if brenipatide can help delay relapse in bipolar disorder patients. The study is sponsored by Eli Lilly and Company and focuses on adults aged 18 to 75 years diagnosed with bipolar disorder I or II. Participants will be randomly assigned to receive one of two doses of brenipatide or a placebo, each administered by subcutaneous injection alongside their standard of care medication. The trial is divided into three periods a screening period lasting about one month, a treatment period lasting at least six months, and a follow-up period lasting approximately two months. The total duration of participation may vary and can be shortened if symptoms worsen or if the participant withdraws. During the study, participants will self-inject the study medication, maintain study diaries, and complete questionnaires assessing their condition. Researchers will monitor time to relapse, changes in functional impairment, mood symptoms using specific rating scales, quality of life, patient global impressions, body weight, and pharmacokinetics. Safety will be closely observed, including the presence of treatment-emergent anti-drug antibodies. Participants are expected to attend regular visits throughout the treatment and follow-up periods.

Researchers are conducting a phase 3, open-label extension study to assess the long-term safety and tolerability of KarXT for treating mania or mania with mixed features in adults with Bipolar-I disorder. The study focuses on evaluating how participants respond to KarXT over an extended period, emphasizing safety measurements such as adverse events and symptom changes. Participants will receive KarXT at specified doses over a treatment period lasting up to 54 weeks. This study includes participants previously involved in related placebo-controlled studies as well as new participants diagnosed with Bipolar-I disorder with manic symptoms. The treatment may be given alongside standard therapeutic doses of lithium, valproate, or lamotrigine as applicable. Throughout the study, participants will undergo regular assessments including monitoring of treatment emergent adverse events, serious adverse events, and psychiatric symptom scales like the Columbia-Suicide Severity Rating Scale, Young Mania Rating Scale, and others. Safety and tolerability will be closely tracked, with evaluations occurring up to week 54. The entire participation may last until the study end date in June 2028, ensuring comprehensive long-term follow-up.

This trial investigates the effectiveness and safety of two treatment combinations for people with relapsed or refractory multiple myeloma who have received one to three prior treatments and were previously treated with lenalidomide. It compares mezigdomide, bortezomib, and dexamethasone MeziVd against pomalidomide, bortezomib, and dexamethasone PVd to see which is better for this condition. The study is a Phase 3, randomized, open-label trial sponsored by Celgene. Participants receive either the MeziVd combination or the PVd combination, with specified doses given on certain days according to the study plan. These treatments are given as drugs, and participants are randomly assigned to one of these two groups to compare their effects. The study will continue for up to approximately five years to evaluate long-term outcomes. During the study, participants will be monitored regularly through various assessments including measuring disease progression, survival, response to treatment, and quality of life using specific questionnaires EORTC QLQ-C30 and QLQ-MY20. Blood samples may be checked for drug levels, and adverse events will be tracked. The main focus is progression-free survival, measured from randomization until disease worsening or death. Participants health and responses will be followed for up to five years, with ongoing visits and evaluations throughout this period.

Researchers are evaluating KarXT for the treatment of manic episodes in adults with Bipolar-I Disorder. This Phase 3, randomized, double-blind, placebo-controlled study involves participants experiencing an acute episode of mania or mania with mixed features. The study aims to compare the effectiveness and safety of KarXT against a placebo during a 3-week inpatient treatment period. Participants will receive flexible dosing of either KarXT or placebo during the 3-week double-blind inpatient phase. Before treatment, psychotropic medications must be washed out within 14 days. The study includes screening, the treatment period, and a safety follow-up, totaling no more than seven weeks. During the study, participants will have their symptoms assessed using tools such as the Young Mania Rating Scale and Clinical Global Impressions-Bipolar scale. Researchers will monitor changes in mania symptoms and overall clinical impression at week 3. Safety follow-up continues after treatment to ensure participant well-being throughout the study duration.

Researchers are evaluating oral rilzabrutinib in a Phase 3 study involving adults aged 18 and older with primary warm autoimmune hemolytic anemia wAIHA. The study aims to compare the drugs effectiveness, safety, pharmacokinetics, and pharmacodynamics to a placebo in achieving a durable hemoglobin response. Approximately 90 participants will take part, with a focus on those who have a confirmed diagnosis of primary wAIHA and meet specific criteria related to corticosteroid treatment history and health status. Participants will first undergo a 4-week screening period. Eligible individuals will then be randomly assigned in a 21 ratio to receive either oral rilzabrutinib or a matching placebo twice daily during the primary analysis period lasting up to 24 weeks. Those who complete this phase will enter an open-label period for 28 weeks, during which all receive rilzabrutinib. Following this, participants who show an increase in hemoglobin during the last 8 weeks of the open-label period may continue in a long-term extension phase lasting up to 52 weeks. A safety follow-up will occur 2 weeks after treatment ends or is stopped. During the study, participants will have scheduled visits to assess hemoglobin levels, fatigue, dyspnea, and other health indicators using questionnaires and laboratory tests. Researchers will monitor adverse events, vital signs, physical exams, and electrocardiograms throughout the study and extension periods. The primary outcome is the proportion of participants achieving a durable hemoglobin response, defined as an increase of at least 2 gdL from baseline in most visits between weeks 12 and 24. Secondary outcomes include overall hemoglobin response, fatigue changes, and need for rescue therapy. Total study duration varies by participant based on their progression through the study phases.

Researchers are evaluating the safety and effectiveness of a 14-day intravenous infusion of teplizumab in Japanese children and adults aged 1 to 34 years who have Stage 2 Type 1 Diabetes T1D. This Phase 2, parallel study aims to confirm the effects of teplizumab on delaying progression to Stage 3 T1D, building on prior studies conducted in Western countries. The study also assesses pharmacokinetics, pharmacodynamics, and immunogenicity of this treatment regimen. Participants are randomly assigned to receive either teplizumab via intravenous infusion for 14 days or no treatment as a control. The dosing regimen matches the FDA-approved schedule for delaying Stage 3 T1D onset in patients aged 8 years and older. The study duration is about 756 days, during which participants are monitored for clinical outcomes and safety. During the study, participants undergo regular assessments including blood tests for C-peptide, insulin, glucose tolerance, and hemoglobin A1c, as well as monitoring for adverse events and vital signs. The primary outcomes include the number of participants progressing to Stage 3 T1D within 104 weeks and changes in insulin production measures. Safety is monitored throughout the study period with laboratory tests, electrocardiograms, and recording any treatment-emergent adverse events.