Shibuya

Researchers are conducting a long-term follow-up study for people previously treated with ciltacabtagene autoleucel (cilta-cel), a CAR-T cell therapy targeting B-cell maturation antigen (BCMA) used in multiple myeloma. The study aims to collect data on delayed side effects and better understand the long-term safety of cilta-cel over a period of up to 15 years after the last dose. Participants in this study will not receive any new treatments. Instead, the study will track their health in two phases: from year 1 to year 5 after their last cilta-cel dose, then from year 6 up to year 15. Safety evaluations will include monitoring for new or worsening malignancies, neurological, rheumatologic, autoimmune, hematologic disorders, infections, and any serious adverse events. Participants will be followed at least once a year during the study. Assessments will include reviewing adverse events, lab test results, physical exams including neurological checks, and other safety data. This comprehensive monitoring will help researchers understand long-term effects and ensure ongoing participant safety throughout the 15-year follow-up.

Researchers are evaluating how well the drug JNJ-79635322 works compared to an anti-B-cell maturation antigen (BCMA)xCD3 bispecific antibody in adults with relapsed or refractory multiple myeloma. This phase 3 study includes participants who have received at least three prior treatments including a proteasome inhibitor, an immunomodulatory drug, and an anti-CD38 antibody. The study aims to compare the effectiveness of these two treatments in this patient population. The study involves two treatment groups receiving either JNJ-79635322 or Teclistamab, both given as subcutaneous injections. Participants must have measurable disease and evidence of disease progression or lack of response to their most recent therapy. The study excludes those with certain infections, central nervous system involvement, allergies to the study drugs, recent major surgery, or recent live vaccine receipt. Participants will be monitored for overall response rate and progression-free survival for up to five years and four months. Throughout the study, performance status will be assessed, and participants will be regularly evaluated for safety and treatment response. The total duration of participation and follow-up allows for long-term evaluation of treatment effects and disease progression.

Researchers are evaluating the effectiveness and safety of brenipatide compared to a placebo in adults with Alcohol Use Disorder (AUD) and hazardous alcohol use. This Phase 3, multicenter, randomized, double-blind study aims to understand if brenipatide can help participants reduce or stop drinking. The study lasts approximately 56 weeks and focuses on changes in drinking patterns using the Timeline Followback Method (TLFB). Participants will receive either brenipatide (LY3537031) or a placebo, both administered by subcutaneous injection. Participants who cannot self-inject will have assistance from a trained support person. They are expected to store and use the blinded study drug as directed, maintain electronic and paper diaries, and complete questionnaires throughout the study. During the study, participants will have scheduled visits to monitor their progress, including assessments of drinking behavior and safety evaluations. Researchers will measure changes in alcohol use patterns up to 56 weeks. Participants must be motivated to reduce or stop drinking and be available for all study visits and procedures. Safety and adherence will be closely monitored throughout the trial.

Researchers are evaluating the safety and effectiveness of brenipatide compared to a placebo for adults with moderate-to-severe Alcohol Use Disorder (AUD). This phase 3 study aims to better understand if brenipatide can help reduce drinking in this population. Participants will be followed for about 56 weeks to gather comprehensive information. Participants will receive either brenipatide (LY3537031) or a placebo, both given by subcutaneous injection. The study involves a randomized, double-blind design, meaning neither the participants nor the researchers know who receives which treatment during the trial. This method helps provide reliable results about the effects and safety of brenipatide. During the study, participants will attend scheduled visits, self-inject the study drug, and complete electronic and paper diaries as well as questionnaires. Researchers will monitor changes in drinking patterns using the Timeline Followback Method for up to 56 weeks. Safety monitoring and regular assessments will be performed throughout the study to track participants' health and adherence.

The primary purpose of this study is to identify the recommended phase 2 dose (RP2D\[s\]) and schedule(s) to be safe for JNJ-79635322 in Part 1 (dose escalation), and to characterize the safety and tolerability of JNJ-79635322 at the RP2D(s) selected and in disease subgroups in Part 2 (dose expansion).

A Phase 3b, open-label, single-arm, rollover study to evaluate the long-term safety of luspatercept, to the following participants: * Participants receiving luspatercept on a parent protocol at the time of their transition to the rollover study, who tolerate the protocol-prescribed regimen in the parent trial and, in the opinion of the investigator, may derive clinical benefit from continuing treatment with luspatercept * Participants in the follow-up phase previously treated with luspatercept or placebo in the parent protocol will continue into long-term post-treatment follow-up in the rollover study until the follow-up commitments are met * The study design is divided into the Transition Phase, Treatment Phase and Follow-up Phase. Participants will enter transition phase and depending on their background will enter either the treatment phase or the Long-term Post-treatment Follow-up (LTPTFU) phase * Transition Phase is defined as one Enrollment visit * Treatment Phase: For participants in luspatercept treatment the dose and schedule of luspatercept in this study will be the same as the last dose and schedule in the parent luspatercept study. This does not apply to participants that are in long-term follow-up from the parent protocol * Follow-up Phase includes: \- 42 Day Safety Follow-up Visit * During the Safety Follow up, the participants will be followed for 42 days after the last dose of luspatercept, for the assessment of safety-related parameters and adverse event (AE) reporting \- Long-term Post-treatment Follow-up (LTPTFU) Phase * Participants will be followed for overall survival every 6 months for at least 5 years from first dose of luspatercept in the parent protocol, or 3 years of post-treatment from last dose, whichever occurs later, or until death, withdrawal of consent, study termination, or until a subject is lost to follow-up. Participants will also be monitored for progression to AML or any malignancies/pre-malignancies. New anticancer or disease related therapies should be collected at the same time schedule Participants transitioning from a parent luspatercept study in post-treatment follow-up (safety or LTPTFU) will continue from the same equivalent point in this rollover study. The ACE-536-LTFU-001 rollover study will be terminated, and relevant participants will discontinue from the study when all participants fulfill 5 years on the study, including treatment and follow-up.

This research aims to evaluate the effect of a 24-week treatment with Treprostinil Palmitil Inhalation Powder (TPIP) compared to placebo on exercise capacity in adults with pulmonary hypertension associated with interstitial lung disease (PH-ILD). This is a Phase 3, randomized, double-blind, placebo-controlled study involving participants diagnosed with PH WHO Group 3 linked to fibrotic interstitial lung disease. Participants will receive either TPIP or a placebo through oral inhalation using a capsule-based dry powder inhaler device once daily for 24 weeks. The study compares the impact of these treatments on exercise ability over the treatment duration. During the study, participants' exercise capacity will be assessed by changes in the 6-minute walk distance from baseline to week 24. Researchers will monitor safety and effectiveness through regular evaluations, including lung function tests, heart catheterization results, and walking tests to measure endurance and functional status. The total participation duration includes the 24-week treatment period with assessments at baseline and week 24.

This research aims to observe the safety and effectiveness of dabrafenib and trametinib in patients with BRAF V600E mutation-positive unresectable advanced or recurrent solid tumors, excluding colorectal cancer. It is a prospective, multicenter, single-arm, non-interventional observational study conducted through a central registration system using electronic data capture. The study includes both adult and pediatric patients, with long-term monitoring planned to collect comprehensive safety data. Patients already prescribed Tafinlar/Mekinist (dabrafenib and trametinib) before joining the study will be enrolled without treatment allocation or changes. The study targets 65 adult patients for effectiveness analysis and approximately 20 pediatric patients. Pediatric patients will be observed for up to 8 years after starting treatment to gather long-term information, while adult patients will be followed for one year post-treatment initiation. During participation, patient safety and treatment response will be monitored through reports of adverse events and overall response rates. Pediatric patients will have ongoing safety assessments related to skeletal and sexual development over the 8-year period. Adults will have their treatment response evaluated over one year. Data collection includes long-term follow-up regardless of treatment discontinuation, aiming to provide comprehensive post-marketing surveillance information on these medications.

This research observes patients with Paroxysmal Nocturnal Hemoglobinuria who are treated with Fabhalta capsules. It is a multicenter, single-arm, non-interventional study designed to monitor drug use and safety over time. The study uses a central registration and all-case surveillance system to collect data. Participants will be observed for 48 weeks after starting Fabhalta treatment. If treatment stops within this period, any adverse events and use of other medications will be tracked up to 30 days after the last treatment day. There are no additional interventions or comparison groups in this study. During the study, researchers will monitor the occurrence of infections and other adverse events through case report forms. Participants' health and drug usage will be recorded throughout the observation period. The total participation lasts for 48 weeks, focusing on safety and drug use in real-world settings.