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
A Modular Phase I/II Open-label Multicentre Study to Evaluate the Safety PK PD Immunogenicity and Efficacy of AZD4512 Monotherapy or in Combination with other Anticancer agent(s) in subjects with Relapsed/Refractory B-cell Non-Hodgkin Lymphoma
This is a Phase I/II study testing whether the new medicine AZD4512 is safe, effective, and well-tolerated in patients with B-cell Non-Hodgkin Lymphoma (B-NHL) whose cancer has come back after treatment (recurrence) or become resistant to initial treatment (refractory). AZD4512 is a new experimental antibody-drug conjugate (ADC) that works by targeting cancer cells that have a protein called CD22 on their surface and killing them. Patients will be divided into groups to receive different dose levels of AZD4512 alone or in combination with other cancer treatments. The study team will gradually increase the dose levels for AZD4512 to find out the most effective dose with the fewest side effects. Blood samples will be collected from all patients to see how their bodies are handling the study medicines and monitor their overall well-being. Specialized blood tests will be done to monitor changes in the patient's tumor DNA, RNA, and proteins (biomarkers), and to learn more about the patient’s cancer, in the hope of helping them further with treatments. All patients will have special scans done to check how their tumors are responding to the study medicine. The study team will monitor all patients for potential side effects and safety concerns.
A Molecularly Driven Phase 1b Dose Escalation and Dose Expansion Study of the DNA-PK Inhibitor Peposertib (M3814) in Combination with the ATR Inhibitor M1774
This study tests the effectiveness of two drugs, peposertib (M3814) and M1774, in patients with advanced solid tumors who have certain genetic changes or signs of DNA stress. The study has two parts: first, a phase to find the highest dose with few side effects (maximum tolerated dose, or MTD), and second, to test the most effective dose (recommended Phase 2 dose, or RP2D) in more detail. In the dose escalation phase, groups of patients will receive different doses, and doctors will check for serious side effects over a 28-day period to decide if the dose should go up, stay the same, or go down. The expansion phase will have two groups: one for patients with ATM gene mutations and another for patients with signs of DNA replication stress, to study how the drugs affect these specific markers. The final dose chosen will aim for a side effect rate close to 25%, based on safety data. Rules are in place throughout the study to adjust doses and keep patients safe.
A Multi-Center Observational Study Assessing the Utility of an Immune Cell Function Assay in a Cardiac Sarcoid Population
Sarcoid is an autoimmune disease that can be found in any and every organ system. The greatest degree of morbidity and mortality comes from cardiac involvement where it can lead to sudden cardiac death, heart block, and heart failure. The basics of the treatment for cardiac sarcoid include appropriate preventative treatment for ventricular arrhythmias, guideline directed medical treatment for heart failure, and immunosuppression to decrease active cardiac inflammation. The amount of immunosuppression and with which medication is of expert opinion. Using PET scan, we can help assess for successful suppression of the disease, but often 2nd or 3rd line treatments are required after initial therapy strategy is deemed insufficient. As of now, there are no predictors of who will fail initial treatment. For this study, there are three different hypotheses. First, we suspect the baseline Immuknow tertile (low, medium, high) will correlate with the rate of response to first line immunosuppression with low being more likely than the combined medium/high grouping to be responsive on first clinically indicated PET follow up. Second, that a subsequent decrease in ImmuKnow grade, for those that start in the medium or high range, will be predictive of resolution of inflammation on cardiac PET. Lastly, we would like to assess whether an intermediate ImmuKnow assay, drawn at 1 month, would be predictive of response at 3 month follow up.The study population will include adults with a clinical or histological diagnosis of cardiac sarcoidosis found to have active inflammation on cardiac PET scan who are not currently on immunosuppressive therapy. This should be their first time undergoing treatment for cardiac sarcoid (i.e. not with disease recurrence). This will give us a uniform patient population that will not have effects of prior immunosuppression as to their response to current treatment, and not possibly already have treatment failure. We will also be storing biologic samples for future analysis. We believe that a change in the immunophenotype of an individual in response to treatment will be the hallmark of successful immunosuppression. We will be using these samples to assess this in the future.