Higashimatsuyama

Researchers are evaluating the effects of iptacopan compared with a placebo in adults aged 18 to 85 years who have generalized Myasthenia Gravis positive for acetylcholine receptor antibodies (AChR+ gMG). This Phase III, randomized, double-blind, placebo-controlled, multicenter study aims to assess the efficacy, safety, and tolerability of iptacopan while participants continue their stable standard of care treatments. The study includes participants with moderate to severe gMG symptoms and positive diagnostic criteria. Participants will be randomly assigned in a 1:1 ratio to receive either iptacopan or a matching placebo in the form of hard gelatin capsules for six months (180 days). During this time, they will continue their stable standard of care treatments. After the double-blind treatment period, a maximum 60-month open-label extension phase is offered. Safety follow-up assessments will occur one week and one month after the last dose of study treatment. During the study, participants will be evaluated for changes in their Myasthenia Gravis Activity of Daily Living (MG-ADL) total score from baseline to month 6. Researchers will monitor safety and tolerability throughout the treatment and extension periods. Vaccination status, infection monitoring, and regular clinical assessments will be part of participant evaluations to ensure safety and track disease symptoms over time.

Researchers are evaluating the pharmacokinetic (PK) comparability between TAK-881 and HYQVIA, both given as subcutaneous (SC) infusions, for maintenance treatment of adults with Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIDP). This Phase 3 trial includes participants who have previously received intravenous or subcutaneous immunoglobulin G treatments and aims to compare these two therapies' behavior in the body. Participants must have a confirmed diagnosis of CIDP and have responded to IgG treatment before, consistent with established diagnostic criteria. The study consists of multiple phases: screening, a ramp-up phase if needed, a HYQVIA treatment phase, a TAK-881 treatment phase, and finally, an extension phase. Participants who previously received conventional subcutaneous or intravenous immunoglobulin will start with a HYQVIA ramp-up phase 1 to 2 weeks after their last dose. Those already on HYQVIA proceed straight to treatment. Participants receive SC infusions of HYQVIA for 20 weeks, then switch to TAK-881 for 24 weeks. During the extension phase, home infusions are preferred, with clinic visits spaced between 12 and 24 weeks. Throughout the study, participants visit the clinic every 3 or 4 weeks during the initial phases. Researchers will monitor immunoglobulin G levels through blood tests at specified intervals to assess drug exposure. Safety and treatment adherence are tracked, and participants complete disability assessments. The total duration includes these treatment phases and the extension, with careful follow-up to evaluate the therapies' pharmacokinetic profiles and participant well-being.

Researchers are evaluating the effectiveness, safety, and tolerability of remibrutinib compared to a placebo in adults with Generalized Myasthenia Gravis (gMG) who are already receiving stable standard-of-care treatment. This Phase III study is randomized, double-blind, placebo-controlled, and involves multiple centers. The study aims to provide new insights into treatment options for people living with gMG. Participants will be randomly assigned in equal numbers to receive either remibrutinib or a placebo during the Core Part, which lasts 6 months and is double-blinded. After this, there is an Extension Part lasting up to 60 months where all participants may receive open-label remibrutinib. The study treatments are administered according to the study protocol, and the trial compares the active drug with placebo to monitor their effects. During the study, participants will be assessed regularly, including measuring changes in their daily living activities affected by myasthenia gravis over six months. Researchers will monitor safety, tolerability, and adherence to treatment. The total participation can extend up to 66 months, including the core and extension periods, with ongoing evaluations and safety follow-up to understand the long-term effects of remibrutinib.

Researchers are evaluating the effectiveness of riliprubart compared to the usual treatment of intravenous immunoglobulin (IVIg) in adults with Chronic Inflammatory Demyelinating Polyneuropathy (CIDP) who are already receiving maintenance IVIg treatment. This Phase 3 study aims to assess how well riliprubart works and its safety in this population. Participants must meet specific CIDP diagnostic criteria and have a history of responding to IVIg treatment. The study involves administering riliprubart or placebo through intravenous (IV) or subcutaneous (SC) solutions, alongside the usual IVIg treatment. The treatment phase includes a randomized, double-blind comparison of riliprubart versus IVIg, followed by an open-label extension period for continued evaluation. The entire study lasts up to 109 weeks, encompassing screening, treatment, and follow-up phases. Participants will have regular assessments to monitor their response to treatment, including measurements of disability and disease activity scores. Researchers will track the percentage of participants showing a response from baseline to week 24 and those maintaining response through week 48 during the extension. Safety and long-term effects will also be observed throughout the study duration, ensuring comprehensive monitoring of participant health and treatment outcomes.