Iruma

Researchers are evaluating the oral azacitidine formulations combined with cedazuridine (CED) tablets in patients with Myelodysplastic Syndrome (MDS) to find doses that achieve similar drug exposure levels to standard azacitidine injections. This Phase 1, multi-center, open-label trial focuses on patients diagnosed with various subtypes of MDS, including refractory anemia and chronic myelomonocytic leukemia. Treatment begins with a single oral dose of azacitidine formulations on day -3 of Cycle 1, followed by subcutaneous azacitidine injection on day 1. From day 2 to day 7 of Cycle 1, and throughout subsequent cycles, patients receive both oral azacitidine formulations and CED tablets daily for seven days per cycle. This dose-escalation study aims to assess pharmacokinetics and drug exposure over these treatment periods. Participants will undergo assessments including monitoring of drug levels to compare oral and injection forms, safety evaluations, and organ function tests. The primary outcome is the total area under the curve (AUC) ratio of azacitidine after oral administration compared with the injection, measured up to one month. Patients are expected to be followed through treatment cycles with monitoring for adverse events, survival, and treatment response during the study.

Researchers are evaluating whether baricitinib can delay the onset of clinical stage 3 type 1 diabetes (T1D) in children and adults at high risk of developing the disease. This phase 3, double-blind, randomized, placebo-controlled study includes participants aged 1 to under 36 years who have early stages of T1D or multiple diabetes-related autoantibodies indicating increased risk. The study aims to measure the time from the start of the trial to diagnosis of stage 3 type 1 diabetes, with participation lasting up to approximately 5 years. Participants will be randomly assigned to receive either baricitinib or a placebo, both administered orally. The trial compares these two groups to assess the impact of baricitinib on delaying progression to stage 3 T1D. The study's design includes careful monitoring of participants over time to evaluate the effects of the medication or placebo on disease development. During the study, participants will undergo regular assessments to detect the progression of diabetes, including laboratory tests for autoantibodies and clinical evaluations. Researchers will track the time it takes for participants to develop stage 3 T1D, along with monitoring safety and any adverse effects. The total duration of participation can be up to 5 years, ensuring thorough observation of long-term outcomes related to the study interventions.

Researchers are evaluating the effectiveness and safety of brenipatide when given along with standard care compared to a placebo with standard care in adults with bipolar disorder. This Phase 2 study aims to see if brenipatide can delay the worsening of bipolar symptoms. The trial includes participants aged 18 to 75 years and involves a careful assessment of how well the treatment works and its safety profile. The trial has three main periods: a screening period lasting about one month, a treatment period of at least six months, and a follow-up period of around two months. Participants receive either brenipatide or placebo, both given by subcutaneous injection, alongside their usual bipolar disorder medications. The study may end earlier if symptoms worsen or if participants withdraw for any reason. Participants will be asked to self-inject the study medication, maintain diaries, complete questionnaires, and attend regular visits throughout the study. Researchers will monitor the time to relapse, defined as the number of days from randomization until symptoms worsen according to specific criteria, over at least six months. Safety and adherence to treatment will also be closely observed during the study.

Researchers are evaluating the long-term safety and tolerability of KarXT in treating mania or mania with mixed features in adults with Bipolar-I disorder. This phase 3, open-label extension study aims to better understand how KarXT performs over an extended period in this population. The study includes participants who either completed previous double-blind placebo-controlled studies or are newly diagnosed with Bipolar-I disorder experiencing manic symptoms. Participants receive KarXT at specified doses on certain days, with some also taking therapeutic doses of Lithium, Valproate, or Lamotrigine as part of their treatment. The study does not mention a placebo group during this extension, focusing instead on monitoring the long-term effects of KarXT alone or in combination with these established therapies. During the study, participants are monitored for adverse events up to week 54 to assess safety. Evaluations include psychiatric assessments using scales such as the Young Mania Rating Scale and CGI-BP score at screening and baseline. Researchers will track treatment-emergent adverse events and overall tolerability throughout the study duration, which lasts up to 54 weeks for each participant.

Researchers are evaluating the effectiveness and safety of a new drug combination called Mezigdomide (CC-92480) with bortezomib and dexamethasone (MeziVd) compared to an existing combination of pomalidomide, bortezomib, and dexamethasone (PVd). This study focuses on adults with relapsed or refractory multiple myeloma (RRMM) who have previously received between one and three lines of therapy, including prior lenalidomide treatment. The trial is a Phase 3, randomized, multicenter, open-label study aiming to improve outcomes for this condition. Participants will be assigned to receive either the MeziVd or PVd treatment regimen, with specified doses of each drug given on certain days. The study involves two treatment groups: one receiving mezigdomide, bortezomib, and dexamethasone, and the other receiving pomalidomide, bortezomib, and dexamethasone. Both regimens follow precise dosing schedules as determined by the study protocol. During the study, participants will be monitored regularly for disease progression or death, with the primary outcome being progression-free survival over up to approximately five years from the date of randomization. Ongoing assessments will include evaluations of safety and effectiveness. The total participation time may vary, and researchers will closely follow participants to gather detailed information on treatment responses and adverse effects.

Researchers are investigating the safety and effectiveness of KarXT in treating manic episodes in adults with Bipolar-I Disorder. This Phase 3 study focuses on individuals experiencing acute mania or mania with mixed features who require hospitalization. The study aims to compare KarXT with a placebo to see how well it reduces manic symptoms during a three-week inpatient period. Participants will be randomly assigned to receive either KarXT or a placebo at specified doses during the inpatient treatment phase. The study is double-blind, so neither the participants nor the researchers know who receives which treatment. The total study duration, including screening, treatment, and safety follow-up, will not exceed seven weeks. During the study, participants will be closely monitored through psychiatric evaluations and rating scales, including the Young Mania Rating Scale (YMRS) to measure changes in mania symptoms by week 3. Other assessments include the Clinical Global Impressions-Bipolar scale and safety evaluations. Researchers will track adherence, symptoms, and any side effects throughout the study period.

Researchers are investigating the safety and effectiveness of oral rilzabrutinib compared to a placebo in adults aged 18 and older diagnosed with primary warm autoimmune hemolytic anemia (wAIHA). This Phase 3, double-blind, parallel-group study aims to assess how well rilzabrutinib can achieve a durable hemoglobin response in about 90 male and female participants with this condition, who have had it for at least six months and have specific treatment histories related to corticosteroids. After a 4-week screening period, participants are randomly assigned in a 2:1 ratio to receive either oral rilzabrutinib or placebo for up to 24 weeks during the primary analysis period. Following this, all participants who complete this phase enter an open-label period where they receive rilzabrutinib for 28 weeks. Those who show an increase in hemoglobin during the last 8 weeks of this open-label phase may continue into a long-term extension lasting until the last participant has completed 52 weeks in this phase. During the study, participants will undergo regular assessments including measuring hemoglobin levels to determine response, safety evaluations, and pharmacokinetic and pharmacodynamic monitoring. The main outcome is the proportion of participants achieving a durable hemoglobin response defined as an increase of at least 2 g/dL from baseline on two-thirds of visits between Week 12 and Week 24. Safety follow-up will continue for 2 weeks after treatment completion or discontinuation, ensuring close monitoring throughout the study duration.

Researchers are evaluating the safety and effectiveness of a 14-day intravenous infusion of teplizumab in Japanese participants aged 8 to 34 years who have Stage 2 Type 1 Diabetes. This Phase 2, two-arm study aims to assess teplizumab's ability to delay progression to Stage 3 Type 1 Diabetes. The study also examines the drug's pharmacokinetics, pharmacodynamics, and immunogenicity based on previous clinical data from Western countries. Participants will receive teplizumab through intravenous infusion following a dosing regimen consistent with the FDA-approved schedule for delaying Stage 3 Type 1 Diabetes onset. The study includes a treatment period of 14 days and lasts approximately 756 days in total to monitor long-term safety and effectiveness outcomes. Throughout the study, participants will undergo multiple assessments including monitoring for adverse events, vital signs, ECGs, and laboratory tests. Researchers will measure changes in C-peptide levels and endogenous insulin from baseline up to week 104 to evaluate beta-cell function. The total study duration involves continuous safety monitoring and collection of data to determine the number of participants progressing to Stage 3 Type 1 Diabetes.