Glen Carbon, Illinois

A Multiple Ascending Dose Study of DT-216P2 in Patients With Friedreich's Ataxia

Safety, Tolerability and Efficacy of Intravitreal KIO-104 in Patients With Macular Edema

In Part A of the study, the safety and efficacy of up to three doses (administrations) of KIO-104, administered every 2 weeks, will be investigated in 2 possible dose cohorts. Cohort 1: Low dose KIO-104 administered to the study eye Cohort 2: High dose KIO-104 administered to the study eye Part B of the study will investigate the safety and efficacy of up to three doses (administrations) of KIO-104 with 2 different dosing regimens (every 2 weeks or every 4 weeks), in participants with macular edema at a dose selected from Part A. Cohort 3A: KIO-104 administered to the study eye every 2 weeks Cohort 3B: KIO-104 administered to the study eye every 4 weeks

A Study to Learn More About How Well Treatment With BAY2927088 Tablets Works and How Safe it is in Participants Who Have a Solid Tumor With Mutations of the Human Epidermal Growth Factor Receptor 2 (HER2)

A Study to Evaluate the Efficacy and Safety of Orally Administered VX-01

This is a Phase 2, multi-center, double-masked, randomized, placebo-controlled, parallel group study to evaluate the efficacy of oral doses of VX-01 in subjects with moderate to severe NPDR, without CI-DME. Approximately 100 male and female subjects aged ≥ 18 years with a documented diagnosis of Type 1 Diabetic Mellitus or Type 2 Diabetic Mellitus with moderate to severe NPDR (without CI-DME) will be enrolled, if they meet all the eligibility criteria for the study. Subjects will be randomized 1:1 to 1 of 2 study cohorts: - Cohort 1 (n = 50): VX-01 (film-coated tablets, 150 mg administered BID) - Cohort 2 (n = 50): Placebo (film-coated tablets, administered BID) Subjects will be stratified by the presence or absence of proliferative diabetic retinopathy (PDR) and by glycated hemoglobin (HbA1c) of ≥ 8.5% or < 8.5% at Screening. All subjects will take 1 tablet of VX-01 or placebo BID for 52 consecutive weeks. All subjects will be followed for 12 weeks after completion of treatment at Week 52. The Sponsor, study site staff, monitors, personnel, and subjects will be masked to treatment assignment during the entirety of the study.

A Phase I Study of CS2009 in Participants With Advanced Solid Tumors

Study of Novel Treatment Combination Therapies in Participants With Head and Neck Squamous Cell Carcinoma.

This platform study will begin with a substudy targeting first-line (1L) recurrent or metastatic (r/m) HNSCC regardless of programmed cell death ligand 1 (PD-L1) expression status (Substudy-01), and new substudies may be added in the future targeting different study populations of HNSCC. All substudies evaluating additional drugs will be added in a staggered manner when relevant nonclinical and/or clinical data become available. Additional drugs may also be added to Substudy-01 in the future. (GS-US-699-7184-01) Substudy-01 will evaluate the efficacy and safety of novel combination of treatment regimens, domvanalimab (DOM) and zimberelimab (ZIM) combined with chemotherapy vs ZIM combined with chemotherapy. The primary objective is to assess the efficacy of DOM and ZIM in combination with chemotherapy versus ZIM in combination with chemotherapy. Substudy-01 planned enrollment is approximately 100.

The Fifth INTEnsive pReventing Secondary Injury in Acute Cerebral Haemorrhage Trial Within ACT-GLOBAL

Intracerebral hemorrhage (ICH) is a severe type of stroke, responsible for substantial disability and death worldwide. It accounts for 6.5% to 19.6% of all strokes, with incidence rates increasing, especially in low- and middle-income countries. Survivors often face significant consequences, including functional impairments, recurrent strokes, cognitive decline, and depression. Despite advancements in acute stroke care, there are few effective treatments specifically targeting the brain damage caused by ICH. Previous research has identified that the formation of perihaematomal oedema (PHE) is a critical factor in poor recovery, making it a key focus for therapeutic development. INTERACT5 domain will focus on two promising medications. Deferoxamine, an iron-chelating agent, targets oxidative stress caused by iron released from damaged brain tissue. Studies suggest it may reduce brain swelling and secondary injury after ICH. Colchicine, an anti-inflammatory medication, inhibits pathways involved in inflammation, which may help minimize brain damage. INTERACT5 will enroll patients aged 18-80 with acute spontaneous supratentorial ICH, confirmed through imaging, who present to the hospital within 24 hours of symptom onset. Other domain-specific inclusion criteria: - Hematoma volume ≥≥10 mL or any volume post-surgery - NIHSS score >8 - GCS ≥8>7 Participants will be randomized to one of four groups: standard care, deferoxamine alone, colchicine alone, or both treatments combined. Deferoxamine will be administered intravenously (32 mg/kg/day within 1 hour and continued for 2 consecutive days), and colchicine will be given orally (0.5 mg daily for 30 days). The study's primary outcome is the improvements in functional outcomes at 6 months, measured by the modified Rankin Scale (mRS). Secondary outcomes include EQ-5D-5L at 6 months, changes in PHE size, NIHSS scores, length of hospital stay, ambulatory status at discharge and safety indicators such as mortality at 6 months/SAEs to 6 month, kidney and liver function. Response Adaptive Randomization (RAR) will be used in this domain to allow readjustment of recruitment towards treatment arms with more favorable emerging effects. Randomization will use minimization method to minimize the imbalance between the number of patients in each treatment group over a number of factors including region, location (deep vs cortical)age (>65 vs ≤65 years old), sex (male vs female), time from onset (>6 vs ≤6 hours), haematoma volume (10-29 vs ≥30 mL), receipt of any decompressive surgery (yes vs no), and intraventricular extension of ICH, etc.

A Study to Assess the Safety, Pharmacokinetics, and Tolerability of ABI-1179 in Healthy Subjects and in Subjects Seropositive for HSV-2 With Recurrent Genital Herpes

Nutrition in Paediatric Critical Care

A Study to Evaluate the Safety, Tolerability, Pharmacokinetics (PK), and Antitumor Activity of ALK202 in Participants with Advanced Solid Tumors