University of Kansas Medical Center

This is a Phase 1/2 study of Multiple-Ascending Dose (MAD) levels for 12 weeks of treatment followed by 24 weeks of open-label treatment with a selected dose of NS-050/NCNP-03 administered once weekly to ambulant boys with DMD, who have a DMD exon deletion amenable to exon 50 skipping.

Type: Interventional

Start Date: Sep 2024

open study

This phase II/III trial examines whether patients who have undergone surgical removal of bladder, kidney, ureter or urethra, but require an additional treatment called immunotherapy to help prevent their urinary tract (urothelial) cancer from coming back, can be identified by a blood test. Many types of tumors tend to lose cells or release different types of cellular products including their DNA which is referred to as circulating tumor DNA (ctDNA) into the bloodstream before changes can be seen on scans. Health care providers can measure the level of ctDNA in blood or other bodily fluids to determine which patients are at higher risk for disease progression or relapse. In this study, a blood test is used to measure ctDNA and see if there is still cancer somewhere in the body after surgery and if giving a treatment will help eliminate the cancer. Immunotherapy with monoclonal antibodies, such as nivolumab and relatlimab, can help the body's immune system to attack the cancer, and can interfere with the ability of tumor cells to grow and spread. This trial may help doctors determine if ctDNA measurement in blood can better identify patients that need additional treatment, if treatment with nivolumab prolongs patients' life and whether the additional immunotherapy treatment with relatlimab extends time without disease progression or prolongs life of urothelial cancer patients who have undergone surgical removal of their bladder, kidney, ureter or urethra.

Type: Interventional

Start Date: Feb 2024

open study

This is a Phase 3, multicenter, randomized, double-blind, placebo-controlled study to evaluate the clinical efficacy, safety, and tolerability of XEN1101 administered as adjunctive treatment in primary generalized tonic-clonic seizures (PGTCS).

Type: Interventional

Start Date: Feb 2023

open study

This phase III trial compares the effect of adding stereotactic body radiation therapy (SBRT) to the usual treatment (conventional image guided radiation therapy [IGRT] and chemotherapy followed by immunotherapy with durvalumab or osimertinib) versus the usual treatment alone in treating patients with non-small cell lung cancer that has spread to nearby tissue or lymph nodes (locally advanced) and cannot be treated by surgery (inoperable). SBRT uses special equipment to position a patient and deliver radiation to tumors with high precision. This method may kill tumor cells with fewer doses over a shorter period and cause less damage to normal tissue. IGRT is a type of radiation that uses a computer to create a picture of the tumor, to help guide the radiation beam during therapy, making it more accurate and causing less damage to healthy tissue. Usual chemotherapy used in this trial consists of combinations of the following drugs: cisplatin, carboplatin, paclitaxel, nab-paclitaxel, pemetrexed, and etoposide. Cisplatin and carboplatin are in a class of medications known as platinum-containing compounds. Cisplatin works by killing, stopping, or slowing the growth of tumor cells. Carboplatin works in a way similar to the anticancer drug cisplatin but may be better tolerated than cisplatin. Carboplatin works by killing, stopping, or slowing the growth of tumor cells as well. Paclitaxel is in a class of medications called antimicrotubule agents. It works by stopping the growth and spread of tumor cells. Nab-paclitaxel is an albumin-stabilized nanoparticle formulation of paclitaxel which may have fewer side effects and work better than other forms of paclitaxel. Pemetrexed is in a class of medications called antifolate antineoplastic agents. It works by blocking the action of a certain substance in the body that may help tumor cells multiply. Etoposide is in a class of medications known as podophyllotoxin derivatives. It blocks a certain enzyme needed for cell division and deoxyribonucleic acid (DNA) repair and may kill tumor cells. Immunotherapy with durvalumab, may induce changes in the body's immune system and may interfere with the ability of tumor cells to grow and spread. Osimertinib is in a class of medications called kinase inhibitors. It works by blocking the action of a protein called EGFR that signals cancer cells to multiply. This helps slow or stop the spread of tumor cells. Adding SBRT to the usual treatment of IGRT with chemotherapy and immunotherapy may be more effective at treating patients with locally-advanced non-small cell lung cancer than giving the usual treatment alone.

Type: Interventional

Start Date: Jul 2023

open study

Claudin 18.2 protein, or CLDN18.2 is a protein found on cells in the digestive system. It is also found on some tumors. Researchers are looking at ways to attack CLDN18.2 to help control tumors. ASP2138 is thought to bind to CLDN18.2 and a protein on a type of immune cell called a T-cell. This "tells" the immune system to attack the tumor. ASP2138 is a potential treatment for people with stomach cancer, gastroesophageal junction cancer (GEJ cancer) or pancreatic cancer. GEJ is where the tube that carries food (esophagus) joins the stomach. Before ASP2138 is available as a treatment, the researchers need to understand how it is processed by and acts upon the body. In this study, ASP2138 will either be given by itself, or given together with standard treatments for gastric, GEJ and pancreatic cancer. Pembrolizumab and mFOLFOX6, and ramucirumab and paclitaxel are standard treatments for gastric and GEJ cancer. mFOLFIRINOX is a standard treatment for pancreatic cancer. This information will help find a suitable dose of ASP2138 given by itself and together with the standard cancer treatments and to check for potential medical problems from the treatments. The main aims of the study are: - To check the safety of ASP2138 and how well people can tolerate medical problems during the study. - To find a suitable dose of ASP2138 to be used later in the study. - These are done for ASP2138 given by itself and when given together with the standard cancer treatments. Adults 18 years or older with stomach cancer, GEJ cancer, or pancreatic cancer can take part. Their cancer is locally advanced unresectable or metastatic. Locally advanced means the cancer has spread to nearby tissue. Unresectable means the cancer cannot be removed by surgery. Metastatic means the cancer has spread to other parts of the body. There should also be the CLDN18.2 marker in a tumor sample. People cannot take part if they need to take medicines to suppress their immune system, have blockages or bleeding in their gut, have specific uncontrollable cancers, have specific infections, have a condition such as hemophagocytic lymphohistiocytosis (HLH) which is when the body over-reacts to a "trigger" such as infection, or have a specific heart condition ("New York Heart Association Class III or IV"). Phase 1: Lower to higher doses of ASP2138 - ASP2138 is either given through a vein (intravenous infusion) or just under the skin (subcutaneous injection). - Different small groups are given lower to higher doses of ASAP2138. - ASP2138 is either given by itself, or given with 1 of 3 standard treatments: - Pembrolizumab and mFOLFOX6 (first treatment for gastric GEJ cancer) - Ramacirumab and paclitaxel (Second treatment for gastric or GEJ cancer) - ASP2138 with mFOLFIRINOX (first treatment for pancreatic cancer) Phase 1b: doses of ASP2138 worked out from Phase 1 - ASP2138 is either given through a vein or just under the skin. This depends on the findings from Phase 1. - People with gastric cancer, GEJ cancer or pancreatic cancer are given doses of ASP2138, worked out from Phase 1. - This includes doses of ASP2138 given by itself and ASP2138 given with the standard cancer treatments. - The standard cancer treatments given depends on the type of cancer they have. End of treatment visit: This is 7 days after final dose of study treatment or if the study doctor decides to stop the person's treatment. People who have locally advanced unresectable pancreatic cancer will not receive ASP2138 by itself.

Type: Interventional

Start Date: Jun 2022

open study

The primary aim is to test whether abatacept, as compared to placebo, is associated with a reduction in major adverse cardiac events (MACE) among participants hospitalized with myocarditis secondary to an immune checkpoint inhibitor (ICI). The primary outcome, MACE, is a composite of first occurrence of cardiovascular death, non-fatal sudden cardiac arrest, cardiogenic shock, significant ventricular arrythmias, significant bradyarrythmias, or incident heart failure.

Type: Interventional

Start Date: Jul 2022

open study

This is a multicenter, single-blinded, randomized controlled clinical trial evaluating the safety and efficacy of the AGN1 LOEP SV Kit for the treatment of painful vertebral compression fragility fractures (VCFs). The objective of this study is to demonstrate non-inferiority of the AGN1 LOEP SV Kit for the treatment of VCFs to standard of care vertebroplasty treatment using bipedicular injection of PMMA bone cement.

Type: Interventional

Start Date: May 2022

open study

This trial will look at a drug called sigvotatug vedotin (SGN-B6A) alone and with pembrolizumab, with or without chemotherapy, to find out whether it is safe for people who have solid tumors. It will study sigvotatug vedotin to find out what its side effects are. A side effect is anything the drug does besides treating cancer. It will also study whether sigvotatug vedotin works to treat solid tumors. The study will have four parts. - Part A of the study will find out how much sigvotatug vedotin should be given to participants. - Part B will use the dose found in Part A to find out how safe sigvotatug vedotin is and if it works to treat solid tumors. - Part C of the study will find out how safe sigvotatug vedotin is in combination with these other drugs. - Part D will include people who have not received treatment. This part of the study will find out how safe sigvotatug vedotin is in combination with these other drugs and if these combinations work to treat solid tumors. - In Parts C and D, participants will receive sigvotatug vedotin with either: - Pembrolizumab or, - Pembrolizumab and carboplatin, or - Pembrolizumab and cisplatin.

Type: Interventional

Start Date: Jun 2020

open study

This phase Ib trial studies the side effects of nivolumab and to see how well it works in treating patients with autoimmune disorders and cancer that has spread to other places in the body or cannot removed by surgery. Immunotherapy with monoclonal antibodies, such as nivolumab, may help the body's immune system attack the cancer, and may interfere with the ability of tumor cells to grow and spread.

Type: Interventional

Start Date: Jul 2019

open study

53-Week Study Evaluating the Safety and Efficacy of Intra-articular (IA) Injections of TPX-100 vs Placebo in Subjects with Mild to Severe Tibio-Femoral Osteoarthritis of the Knee

Type: Interventional

Start Date: May 2025

open study

The main goals of this study are to evaluate the safety and efficacy of casdozokitug in combination with toripalimab plus bevacizumab and to define a recommended dose for casdozokitug in combination with toripalimab plus bevacizumab.

Type: Interventional

Start Date: Dec 2024

open study

The purpose of this study is to determine if vimseltinib is safe, tolerable and works effectively to treat adults with active moderate to severe cGVHD. Participants will be treated with vimseltinib in 28-day treatment cycles for approximately 2 years.

Type: Interventional

Start Date: Nov 2024

open study

This is a first-in-human (FIH), open-label, multicenter, dose escalation and dose expansion study to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics, and preliminary antitumor activity of BGB-53038 as monotherapy in participants with advanced or metastatic solid tumors harboring KRAS mutations or amplification, as well as when used in combination with tislelizumab (also known as BGB-A317) in participants with nonsquamous non-small cell lung cancer (NSCLC) and used in combination with cetuximab in participants with colorectal cancer (CRC). The study consists of 2 phases: Phase 1a Dose Escalation and Safety Expansion and Phase 1b Dose Expansion.

Type: Interventional

Start Date: Nov 2024

open study

This phase III trial compares the effect of adding cemiplimab to standard therapy (surgery with or without radiation) versus standard therapy alone in treating patients with stage III/IV squamous cell skin cancer that is able to be removed by surgery (resectable) and that may have come back after a period of improvement (recurrent). The usual treatment for patients with resectable squamous cell skin cancer is the removal of the cancerous tissue (surgery) with or without radiation, which uses high energy x-rays, particles, or radioactive seeds to kill cancer cells and shrink tumors. Immunotherapy with monoclonal antibodies, such as cemiplimab, may help the body's immune system attack the cancer, and may interfere with the ability of tumor cells to grow and spread. Cemiplimab has been approved for the treatment of skin cancer that has spread or that cannot be removed by surgery, but it has not been approved for the treatment of skin cancer than can be removed by surgery. Adding cemiplimab to the usual treatment of surgery with or without radiation may be more effective in treating patients with stage III/IV resectable squamous cell skin cancer than the usual treatment alone.

Type: Interventional

Start Date: Feb 2025

open study

This study is a Phase 1 multicenter, open-label study evaluating the safety and efficacy of escalating doses of MT-401-OTS in 2 participant populations: 1) Those with intermediate or high-risk AML per 2022 ELN criteria who have evidence of MRD and/or </= 10% blast following prior induction therapy or at least 4 cycles of nonintensive therapy and 2) those with high- or very-high-risk MDS per 2023 IWG criteria and who have residual disease with </= 10% blasts following treatment with an HMA-based therapy.

Type: Interventional

Start Date: Jun 2025

open study

The main aims of this study are to understand why adults with Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIDP) chose a certain treatment, why they changed to HyQvia from another therapy, how satisfied they are with HyQvia and their previous treatment, how their work productivity and activity is impacted and learn about their CIDP signs and symptoms. Other aims are to collect information on any medical problems or side effects during the treatment with HyQvia, learn how effective treatment of CIDP with HyQvia is and understand details on the use of HyQvia in standard clinical routine as well on the need for healthcare intervention (such as emergency room visits or hospital visits or stays). During the study, data will be collected from medical records already available, interviews with participants at study start and study completion and via questionnaires completed by participants. Participants will be treated as per the doctor's or the clinic's routine.

Type: Observational

Start Date: Apr 2025

open study

This is a randomized, open-label study in adult patients who have completed standard first line therapy for large B-cell lymphoma (LBCL) and achieved a complete response or partial response suitable for observation, but who have minimal residual disease (MRD) as detected by the Foresight CLARITY™ Investigational Use Only (IUO) MRD test, powered by PhasED-Seq™. The purpose of the trial is to assess the efficacy and safety of consolidation with cemacabtagene ansegedleucel (cema-cel), an allogeneic CD19 CAR T product, as compared to standard of care observation. In this study, participants with MRD are randomized 1:1 to treatment with cema-cel or an observation arm. Treatment includes cema-cel following a lymphodepletion regimen of fludarabine and cyclophosphamide. Prior to August 2025, participants may also have received an anti-CD52 monoclonal antibody, ALLO-647, as part of their lymphodepletion regimen.

Type: Interventional

Start Date: Jun 2024

open study

This clinical trial is studying advanced solid tumors. Solid tumors are cancers that start in a part of your body like your lungs or liver instead of your blood. Once tumors have grown bigger in one place but haven't spread, they're called locally advanced. If your cancer has spread to other parts of your body, it's called metastatic. When a cancer has gotten so big it can't easily be removed or has spread to other parts of the body, it is called unresectable. These types of cancer are harder to treat. Patients in this study must have cancer that has come back or did not get better with treatment. Patients must have a solid tumor cancer that can't be treated with standard of care drugs. This clinical trial uses an experimental drug called PF-08052666/SGN-MesoC2. PF-08052666/SGN-MesoC2 is a type of antibody-drug conjugate (ADC). ADCs are designed to stick to cancer cells and kill them. They may also stick to some normal cells. This study will have 3 parts. Part A and Part B of the study will find out how much PF-08052666/SGN-MesoC2 should be given to participants. Part C will use the information from Parts A and B to see if PF-08052666/SGN-MesoC2 is safe and if it works to treat solid tumor cancers.

Type: Interventional

Start Date: Aug 2024

open study

Phase 1b, open-label study evaluating the safety, tolerability, pharmacokinetics, preliminary antitumor activity, and pharmacodynamics of OPN-6602 monotherapy and in combination with dexamethasone in subjects with relapsed and/or refractory MM.

Type: Interventional

Start Date: Aug 2024

open study

This clinical trial is designed to compare the efficacy and safety of Clofazimine Inhalation Suspension versus placebo when added to guideline-based therapy (GBT)

Type: Interventional

Start Date: Sep 2024

open study

This is a Phase 3 extension, global, multicenter study to assess the long-term safety and tolerability of tolebrutinib in adult participants (aged ≥18 years) with RMS, PPMS, or NRSPMS who were previously enrolled in the Phase 2b LTS (LTS16004) or 1 of the 4 Phase 3 tolebrutinib pivotal trials (GEMINI 1 [EFC16033], GEMINI 2 [EFC16034], HERCULES [EFC16645], or PERSEUS [EFC16035]). SUBSTUDY: ToleDYNAMIC substudy

Type: Interventional

Start Date: Apr 2024

open study

RESET-MG: A Phase 1/2 Open-Label Study to Evaluate the Safety and Efficacy of CABA-201 in Participants with Generalized Myasthenia Gravis

Type: Interventional

Start Date: Dec 2024

open study

The overall objective of the RESTORATiVE303 study is to evaluate the safety and the Clostridioides difficile infection (CDI) recurrence rate at Week 8 in participants who receive a 14-day course of VE303 or matching placebo. The objectives and endpoints are identical for Stage 1 (recurrent CDI) and Stage 2 (high-risk primary CDI).

Type: Interventional

Start Date: May 2024

open study

Study RIN-PF-305 is designed to evaluate the safety and efficacy of inhaled treprostinil in subjects with progressive pulmonary fibrosis (PPF) over a 52-week period.

Type: Interventional

Start Date: Oct 2023

open study

This phase II trial compares mosunetuzumab to the usual treatment (rituximab) for improving survival in patients with nodular lymphocyte-predominant Hodgkin lymphoma (NLPHL). Rituximab and mosunetuzumab are monoclonal antibodies. They bind to a protein called CD20, which is found on B cells (a type of white blood cell) and some types of cancer cells. This may help the immune system kill cancer cells. Mosunetuzumab may be more effective at extending survival in patients with NLPHL than the usual approach with rituximab.

Type: Interventional

Start Date: Jan 2024

open study