A Double-Blind Randomized Multicenter Trial Evaluating the Efficacy and Safety of PRAX-628 in Adults with Focal Seizures (POWER 1)
The main purpose of this research is to find out how well PRAX-628 works and how safe it is when used to treat focal seizures compared to placebo (inactive drug). The study also looks at the side effects of taking PRAX-628 compared with placebo and how the body processes it. The active study drug or placebo will be taken in addition to your regular anti-seizure medications.
A DOUBLE-BLIND RANDOMIZED PLACEBO-CONTROLLED MULTICENTER OUTPATIENT PARALLEL-GROUP STUDY TO ASSESS THE EFFICACY AND SAFETY OF STACCATO ALPRAZOLAM IN PARTICIPANTS 12 YEARS OF AGE AND OLDER WITH EPILEPSY WITH A PREDICTABLE SEIZURE PATTERN.
Staccato alprazolam is being developed for the indication of rapid cessation of a prolonged focal or generalized seizure that has not progressed to status epilepticus in patients with epilepsy 12 years of age and older. After completing the study, eligible study participants will be allowed to enroll in an open-label extension (OLE) study.
A Feasibility Pilot Study of Home-Based Intermittent 60Hz Light Therapy for the Treatment of Depression
The purpose of this study is to see how easy it is to use an intermittent light therapy device in adults with a major depressive episode (MDE), and to see how mood is impacted by the light therapy. Participants will complete 15 non-invasive light stimulation sessions, online surveys, and assessments.
A First-in-human (FIH) Study of Inhibitory Interneurons (NRTX- 1001) in Drug-Resistant Unilateral Mesial Temporal Lobe Epilepsy (MTLE)
The purpose of this two part study is to evaluate the safety (Stage 1 and Stage 2) and efficacy (Stage 2) of NRTX-1001 in subjects with drug resistant mesial temporal lobe epilepsy (MTLE).
A First-in-Human Phase 1a/1b Trial to Assess the Safety Tolerability and Preliminary Efficacy of LY4170156 an Antibody-Drug Conjugate Targeting Folate Receptor a Expressing Tumor Cells in Participants with Selected Advanced Solid Tumors
This study will test a new drug called LY4170156, an antibody drug conjugate (ADC) to see how it works in the body, and if it helps patients with different types of cancer, like ovarian, endometrial, cervical, lung, breast, pancreatic, and colorectal cancers. The study has two phases. In Phase 1a, doctors will find the best dose of LY4170156 by giving it to patients and checking for side effects and how well it works. If needed, they will try different doses to see which one is the safest and most effective. In Phase 1b, the best dose from Phase 1a will be given to more patients to see how well it works as a single treatment. Patients will be divided into groups based on the type of cancer and a specific antigen in their tumors to which the ADC binds, which will be tested in a lab. The study will make sure that the drug is given at the right dose and checks how patients feel during the treatment.
A First in Human Study of theMenin-KMT2A (MLL1)Inhibitor JNJ-75276617 in Participants with Acute Leukemia
A First in Human Study of theMenin-KMT2A (MLL1)Inhibitor JNJ-75276617 in Participants with Acute Leukemia
A Follow-Up Open-Label Research Evaluation of Sustained Treatment with Aficamten (CK-3773274) in Hypertrophic Cardiomyopathy (HCM)
This is an open-label study of CK-3773274 in patients with HCM. The treatment duration is anticipated to be multiple years, concluding when marketing authorization is achieved in the patient’s country or Cytokinetics terminates the study. Approximately 600 patients will be enrolled in this study. After (up to) 56-days of screening, eligible patients will be administered a daily dose of CK-3773274. The highest maximum tolerated dose of CK-3773274 in CY 6022 will be informed by the ongoing conduct of other studies of CK-3773274. Each patient will start at the lowest prespecified dose and undergo echocardiography-guided dose titration to their maximum tolerated dose (not to exceed the highest prespecified dose). Dose adjustment may be made no more frequently than every 2 weeks.Approximately 95 investigative sites worldwide.
A Global Prospective Observational Registry of Patients with Pompe Disease
The purpose of this registry is to evaluate clinical outcomes in participants with Pompe disease. This includes what happens to participants who are not on any treatment and the long-term safety and effects of different types of treatments for Pompe disease.
A Global Randomized Open-label Multicenter Phase 2b/3 Trial Evaluating BJT-778 vs Delayed Treatment for the Treatment of Chronic Hepatitis Delta Infection (AZURE-1)
The main goal of this study is to determine the effectiveness of BJT-778 as a long-term treatment in patients with hepatitis delta virus (HDV) infection.
A Modular Open-label Phase I/II Study to Evaluate the Safety Tolerability Pharmacokinetics and Efficacy of EP0031 in Patients with Advanced RET-altered Malignancies
EP0031-101 is an interventional, modular, multi-arm, multi-centre, open-label Phase I/II study to investigate the safety, tolerability, PK, and PD of EP0031, to determine the RP2D of EP0031, and todetermine preliminary efficacy of EP0031 in defined patient populations with RET-altered malignancies.The design consists of a core study protocol and individual Modules, as follows:? Module A: Monotherapy dose escalation and RP2D optimisation in patients with RETaltered solid tumours (including a paired biopsy cohort)? Module B: Dose-expansion cohorts will be opened to further explore the safety andtolerability and provide preliminary efficacy data in selected patient populations withRET-altered tumours? Module C: Further dose expansion and initial efficacy investigation in patients who haveprogressed following first-generation SRI therapy