Tsuchiura-shi,ibaraki, Japan

A Study of Efgartigimod PH20 SC Given by Prefilled Syringe in Adults With Thyroid Eye Disease.

A Multicenter Multinational Observational Study of Children With Hypochondroplasia

A Study of Repotrectinib Versus Crizotinib in Participants With Locally Advanced or Metastatic Tyrosine Kinase Inhibitor (TKI)-naïve ROS1-positive Non-Small Cell Lung Cancer (NSCLC) (TRIDENT-3)

Evaluate the Efficacy and Safety of KK8398 in Patients With Achondroplasia（AOBA Study）

iPSC-based Drug Repurposing for ALS Medicine (iDReAM) Study

Phase 1 part: The study consists of a 12-week observation period, a 1-week (acceptable window: 5-9 days) transitional period, a 12-week study treatment period, and a 4-week follow-up period. Subjects who have been receiving riluzole since before the enrollment are allowed to continuously receive riluzole during the 12-week observation period (with the dosage remaining unchanged), and stop receiving riluzole from the beginning of the 1-week (acceptable window: 5-9 days) transitional period. After the completion of the transitional period, subjects whose total ALSFRS-R score decreased by 1 to 3 points during the 12-week observation period will receive bosutinib for 12 weeks to evaluate the safety and tolerability of bosutinib in ALS patients. All ALS drugs including riluzole will be prohibited during the bosutinib treatment period. In this study, 3 to 6 ALS patients will be enrolled in each of the 4 bosutinib dose lelvels [100 mg/day (dose level 1), 200 mg/day (dose level 2), 300 mg/day (dose level 3), or 400mg/day (dose level 4)] to evaluate the safety and tolerability of the investigational drug (bosutinib) under a 3+3 dose escalation study design. The dose will be escalated by 1 dose level at a time; no skipping will be allowed. Dose escalation and MTD will be determined by the safety assessment committee comprising oncologist, hematologist, ALS Expert based on the incidence of DLT in 4 weeks of treatment among 3 subjects enrolled (6 subjects if additionaly enrolled) in each dose level. RP2D will be determined by the safety assessment committee upon completion of 12-week study treatment in all subjects in all dose levels. Phase 2 part: The phase 2 part consists of 4 periods including a 12-week observation period, a 1-week (±2 days) transitional period, a 24-week study treatment period, and a 4-week safety follow-up period. After the completion of the transitional period, subjects whose total ALSFRS-R score decreased by 1 to 4 points during the 12-week observation period will receive bosutinib treatment during the 24-week study treatment period. In this study, 25 ALS patients will be enrolled; patients will be randomly assigned to the following groups: 12 patients in 200 mg/day group and 13 patients in 300 mg/daygroup of the investigational drug (bosutinib). The efficacy and the safety of bosutinib in ALS patients for 24 weeks will be assessed. The efficacy using ALSFRS-R score will be also compared with the external published data from edaravone study (MCI186-19). In order to compare with the edaravone study (MCI186-19) , the eligibility criteria of the phase 2 part is similar to those in MCI186-19. By statical allocation, approximately 85% of patients in each 200 mg and 300 mg group will have a decrease of 1-2 points in ALSFRS-R, and 15% will have a decrease of 3-4 points in ALSFRS-R, during the observation period, in accordance with MCI186-19. The efficacy using ALSFRS-R score will be also compared with matched control of Japanese Consorsium for Amyotrophic Lateral Sclerosis (JaCALS), a registory of ALS, in an exploratory manner.

A Study of Repotrectinib (TPX-0005) in Patients With Advanced Solid Tumors Harboring ALK, ROS1, or NTRK1-3 Rearrangements

In Phase 2, study subjects will be enrolled into 6 distinct expansion (EXP) cohorts: - EXP-1: ROS1 TKI-naïve ROS1+ NSCLC. Up to one prior line of chemotherapy OR immunotherapy is allowed - EXP-2: 1 Prior ROS1 TKI AND 1 Platinum-based Chemotherapy ROS1+ NSCLC. Disease progression, or intolerant to one prior line of a ROS1 TKI. Must have received one prior line of platinum based chemotherapy OR one prior line of platinum based chemotherapy in combination with immunotherapy before or after a ROS1 TKI - EXP-3: 2 Prior ROS1 TKIs AND NO Chemotherapy ROS1+ NSCLC. Disease progression, or intolerant to 2 prior lines of a ROS1 TKI treatment. No prior lines of chemotherapy or immunotherapy are allowed. - EXP-4: 1 Prior ROS1 TKI and NO Chemotherapy or Immunotherapy. Disease progression or intolerant to one prior line of a ROS1 TKI. No prior lines of chemotherapy or immunotherapy are allowed. - EXP-5: TRK TKI-naïve NTRK+ solid tumors. Any number of prior lines of chemo or immunotherapy is allowed. - EXP-6: TRK TKI-pretreated NTRK+ solid tumors. Disease progression, or intolerant to 1 or 2 prior TRK TKIs. Any number of prior lines of chemo- or immunotherapy are allowed.

Product Surveillance Registry

A Study of First-Line Olomorasib (LY3537982) and Pembrolizumab With or Without Chemotherapy in Patients With Advanced KRAS G12C-Mutant Non-small Cell Lung Cancer

Dose Optimization, Part A, and Part B are randomized. Safety Lead-In for Part B is single arm, non-randomized.

A Study to Learn More About How Well BAY 2927088 Works and How Safe it is Compared With Standard Treatment, in Participants Who Have Advanced Non-small Cell Lung Cancer (NSCLC) With Mutations of the Human Epidermal Growth Factor Receptor 2 (HER2)

A Study to Test the Safety and Tolerability of Brivaracetam in Children and Adolescents With Seizures

EP0156 is designed to assess the long-term safety and tolerability of BRV in pediatric study participants with epilepsy who participated in the neonatal study N01349 [NCG03325439] and/or have participated in the open-label, long-term, follow-up pediatric study N01266 [NCT01364597]. EP0156 will also assess the long-term safety and tolerability of BRV in Japanese pediatric study participants with partial-onset seizures who will be directly enrolled into the study in Japan. Pharmacokinetic data will also be evaluated in Japanese study participants.