An observational multi-center study to validate outcome measures for future clinical trials of Multiple System Atrophy (MSA)
The purpose of this research study is to develop and validate new methods to measure if a potential drug being tested in future clinical trials of patients with multiple system atrophy (MSA) is effective. These methods include a scale or questionnaire (“clinical outcome assessment” or COA), brain neuroimaging (magnetic resonance imaging or MRI) and biochemical biomarkers (blood or cerebrospinal fluid (CSF) samples).
An Open-Label Multi-Center Retrospective and Prospective Evaluation of Shoulder Arthroplasty Clinical and Radiographic Outcomes
This is an open label, multi-center, retrospective and prospective clinical study. Patients who are to undergo shoulder arthroplasty may be screened for participation. Likewise, patients who have previously undergone shoulder arthroplasty with the Equinoxe®, or other arthroplasty systems, may be screened for enrollment. This study may include patients from multiple centers, both US and international, undergoing hemi- or total shoulder joint replacement. Patients will be screened for enrollment based upon the inclusion and exclusion criteria described in the protocol.
AN OPEN-LABEL MULTICENTER STUDY OF LOXO-435 (LY3866288) IN ADVANCED SOLID TUMOR MALIGNANCIES WITH FGFR3 ALTERATIONS
This is a study of a drug called LOXO-435 for patients with advanced solid tumors, specifically focusing on metastatic urothelial cancer (mUC) that has a change in a gene called FGFR3. The study will be conducted in two phases. Phase 1a is the first part and will test different doses of LOXO-435 to determine its safety, how well it is accepted by the patient's body, how the body processes it (pharmacokinetics), and its initial effectiveness. This phase will include patients with any type of solid tumor that has an alteration in the FGFR3 gene or its related proteins. The goal is to find the recommended dose for the next phase. Phase 1b is the second part and will involve four groups of patients receiving the recommended dose from Phase 1a. These groups will include patients with urothelial cancer, either as a single treatment or in combination with pembrolizumab (another drug), as well as patients with other types of advanced solid tumors who will receive LOXO-435 alone. The purpose is to evaluate the effectiveness and safety of LOXO-435 in these specific patient groups.
An Open-label Phase 1 Multicenter Study to Evaluate the Safety and Preliminary Anti-tumor activity of NT-112 in Human Leukocyte Antigen-C*08:02-Positive Adult Subjects with Unresectable Advanced and/or Metastatic Solid Tumors That Are Positive for the KRAS G12D Mutation
This study is testing a NT-112, a type of cellular therapy, in patients with advanced cancer, such as lung, pancreatic, or colorectal cancer, who have a specific genetic change called KRAS G12D. The study team wants to see if the medicine has few side effects and is effective in treating the cancer. They will try different amounts of the medicine to find thehighest dose that patients can handle without severe side effects. The researchers will carefully follow a plan to decide how much medicine each patient will get. The study team will keep a close eye on any side effects. The study has different periods, like screening, where they check if people meet the criteria, enrollment, treatment, and follow-up. Patients will go through various tests before joining the study, and the researchers will collect information about safety, effectiveness, and other factors.
An Open-Label Randomized Controlled Multi-Center Study of The Efficacy of Daromun (L19IL2 + L19TNF) Neoadjuvant Intratumoral Treatment Followed by Surgery and Adjuvant Therapy VS Surgery and Adjuvant Therapy in Clinical Stage IIIB/C/D Melanoma Patients
The primary objective of the study is to demonstrate that a neoadjuvant Daromun treatment followed by surgery and adjuvant therapy improves in a statistically significant manner the recurrence-free survival (RFS) of Stage IIIB/C/D melanoma patients with respect to the standard of care (surgery and adjuvant therapy).Primary endpoint of the study is recurrence free survival (RFS) in a time-to-event analysis in the Daromun plus surgery and adjuvant therapy treatment group (Arm 1) versus the surgery and adjuvant therapy control group (Arm 2). Analysis will be performed for the "Intention To Treat" population.
An Open-label Randomized Phase 3 Study of MK-2870 as a Single Agent and in Combination with Pembrolizumab Versus Treatment of Physician s Choice in Participants with HR+/HER2- Unresectable Locally Advanced or Metastatic Breast Cancer
This clinical study will evaluate MK-2870 either alone or in combination with pembrolizumab versus TPC in participants with HR+/HER2- (both HER2-zero and HER2-low) unresectable locally advanced or MBC, who have not been previously treatedwith chemotherapy in the metastatic setting.
An open-label randomized trial of zanidatamab with standard-of-care therapy against standard-of-care therapy alone for advanced HER2-positive biliary tract cancer (BTC)
This study is testing a new treatment called zanidatamab to see how well it works and how safe it is for people with a type of cancer called HER2-positive biliary tract cancer (BTC), which affects the gallbladder and bile ducts and cannot be removed by surgery or has spread to other parts of the body. Participants may receive up to two cycles of chemotherapy (cisplatin and gemcitabine, or “CisGem”) before being randomly assigned to one of two groups. In Group A, participants will get zanidatamab plus CisGem, with or without an immune therapy drug (like durvalumab or pembrolizumab). In Group B, participants will get CisGem, with or without the immune therapy drug. The treatment will continue until the cancer gets worse.
AN OPEN-LABEL TREATMENT WITH RANDOMIZATION OBSERVATION INVESTIGATOR-INITIATED STUDY ON THE DURATION AND EFFICACY OF JORNAY PM (METHYLPHENIDATE HYDROCHLORIDE EXTENDED-RELEASE CAPSULES) ON ADULT ADHD SYMPTOMS AND EXECUTIVE FUNCTION AND EMOTIONAL REGULATION THROUGHOUT THE DAY INTO EARLY EVENING
The purpose of this research study is to examine the efficacy of Jornay PM on Attention-Deficit/Hyperactivity Disorder (ADHD) symptoms and Executive Function throughout the day into early evening. This study will last approximately 11 weeks with will involve about 11 visits that are conducted both remote (WebEx/Telephone) and in the clinic.
ANBL1531: A Phase 3 Study of 131I-Metaiodobenzylguanidine (131I-MIBG) or Crizotinib Added to Intensive Therapy for Children with Newly Diagnosed High-Risk Neuroblastoma (NBL) (IND# 134379)
Despite recent improvements in outcome for children with newly diagnosed high-risk neuroblastoma, cure rates remain unsatisfactory. Further, these gains have been the result of interventions during the Consolidation (tandem autologous stem cell transplant) and Post-Consolidation (dinutuximab immunotherapy) phases of treatment, while rates of disease control during Induction have not improved in recent COG trials. The current phase 3 trial seeks to improve the event-free survival (EFS) for children with high-risk neuroblastoma through early integration of promising novel targeted therapies: targeted radiopharmaceutical therapy with 131I-MIBG or the ALK inhibitor, crizotinib. After enrollment, patients will receive one cycle of Induction chemotherapy. Subsequent therapy will be based upon MIBG avidity and ALK status. Patients with MIBG-avid, ALK wild type (or ALK unknown) disease will be randomized to one of three arms: A) current COG recommended high-risk therapy including four more cycles of Induction chemotherapy and surgical resection of the primary tumor, Consolidation with tandem transplant and focal external beam radiation, and dinutuximab immunotherapy with isotretinoin; B) current COG recommended high-risk therapy with the addition of a block of 131I-MIBG after the third Induction cycle; or C) current COG recommended high-risk therapy with the addition of a block of 131I-MIBG after the third Induction cycle and substitution of busulfan / melphalan (BuMel) single autologous stem cell transplant in place of tandem transplant. Patients with MIBG non-avid, ALK wild type (or ALK unknown) disease will be non-randomly assigned to receive current COG recommended high-risk therapy without the addition of 131I-MIBG. Patients with ALK aberrant tumors (ALK tyrosine kinase mutation or ALK amplification) will be non-randomly assigned to receive crizotinib added to current COG recommended high-risk therapy. The primary endpoint is EFS and 774 eligible and evaluable patients are anticipated to enroll over approximately 5 years. Key secondary endpoints are toxicity, end-Induction response, and overall survival. Late effects of therapy including targeted therapies will be compared with late effects of current COG recommended treatment. Embedded correlative studies seek to understand predictors of benefit and resistance to 131I-MIBG and crizotinib.
Andrew W. Brotman, MD | NYU Langone Health
Andrew W. Brotman is senior advisor to CEO and Dean Alec C Kimmelman, MD, PhD.