University of Kansas Medical Center

The safety, tolerability, and antileukemic response of ziftomenib in combination with standard of care treatments for patients with relapsed/refractory acute myeloid leukemia will be examined with the following agents: FLAG-IDA, low-dose cytarabine, and gilteritinib.

Type: Interventional

Start Date: Feb 2024

open study

This is a Phase 2, open-label, multi-center, 2-part study of NS-089/NCNP-02 administered by weekly IV infusion to ambulant boys aged ≥4 to <15 years with DMD due to mutations amenable to exon 44 skipping. Participants will receive a selected dose of NS-089/NCNP-02 administered once weekly. The study consists of 2 parts: Part 1 and Part 2. Six participants (Cohort 1) will participate in both Part 1 and Part 2, and 14 participants (Cohort 2) will be added for Part 2.

Type: Interventional

Start Date: Feb 2024

open study

This is a Phase 3 Randomized, double-blind, Multiregional Study of Ivonescimab Combined with Chemotherapy Versus Pembrolizumab Combined with Chemotherapy for the First-line Treatment of Metastatic Non-small Cell Lung Cancer. The primary endpoint is overall survival and progression free survival assessed by investigator. The key secondary endpoints include response and safety.

Type: Interventional

Start Date: Oct 2023

open study

Phase 1 study comprised of open-label, dose escalation, multiple cohorts of P-BCMA-ALLO1 allogeneic T stem cell memory (Tscm) CAR-T cells in subjects with relapsed / refractory Multiple Myeloma (RRMM).

Type: Interventional

Start Date: May 2022

open study

The purpose of this study is to better understand how people feel after a radiofrequency ablation standard of care surgery using a different type of needle (multi-tined expandable electrode/MEE) in comparison to receiving conventional medical management (CMM) techniques.

Type: Interventional

Start Date: Dec 2020

open study

The Pompe Registry is a global, multicenter, international, longitudinal, observational, and voluntary program for patients with Pompe disease, designed to track the disease's natural history and outcomes in patients, both treated and not. Data from the Registry are also used to fulfill various global regulatory commitments, to support product development/reimbursement, and for other research and non-research related purposes. The objectives of the Registry are: - To enhance understanding of the variability, progression, identification, and natural history of Pompe disease, with the ultimate goal of better guiding and assessing therapeutic intervention. - To assist the Pompe medical community with the development of recommendations for monitoring patients, and to provide reports on patient outcomes, to optimize patient care. - To characterize the Pompe disease population. - To evaluate the long-term effectiveness of alglucosidase alfa.

Type: Observational [Patient Registry]

Start Date: Sep 2004

open study

This phase I trial tests the safety, side effects, and best dose of triapine in combination with radiation therapy in treating patients with glioblastoma or astrocytoma that has come back after a period of improvement (recurrent). Triapine may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. Radiation therapy uses high energy x-rays, particles, or radioactive seeds to kill cancer cells and shrink tumors. Giving triapine in combination with radiation therapy may be safe, tolerable, and/or effective in treating patients with recurrent glioblastoma or astrocytoma.

Type: Interventional

Start Date: Jul 2025

open study

This study is researching an experimental drug called ubamatamab, also referred to as "study drug". The study is focused on patients who have advanced ovarian cancer. The aim of the study is to see how safe, tolerable, and effective the study drug is on its own and in combination with other anti-cancer drugs (bevacizumab, cemiplimab, fianlimab and a standard chemotherapy drug, pegylated liposomal doxorubicin [PLD]), referred to as "combination drugs'. The study is looking at several other research questions, including: - What side effects may happen from taking the study drug and its experimental combinations - How much study drug and fianlimab is in the blood at different times - Whether the body makes antibodies against the study drug (which could make the drug less effective or could lead to side effects) and its combinations

Type: Interventional

Start Date: May 2025

open study

The purpose of this study is to determine if Clenbuterol is a therapeutic option for FSHD by determining the safety and tolerability of the medication at three different dose levels.

Type: Interventional

Start Date: Jun 2025

open study

This phase I trial studies the side effects and best dose of CA-4948 when given together with fluorouracil, leucovorin, oxaliplatin (FOLFOX) plus bevacizumab in treating patients with colorectal cancer that has spread from where it first started (primary site) to other places in the body (metastatic). CA-4948 may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. The chemotherapy drugs used in FOLOX, fluorouracil and oxaliplatin, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Leucovorin is used with fluorouracil to treat colorectal cancer. Bevacizumab is in a class of medications called anti-angiogenic agents. It works by stopping the formation of blood vessels that bring oxygen and nutrients to the tumor. This may slow the growth and spread of the tumor. Giving CA-4948 with FOLFOX plus bevacizumab may be safe, tolerable and/or effective in treating patients with metastatic colorectal cancer.

Type: Interventional

Start Date: Jun 2025

open study

The goal of this Phase 2 clinical trial is to investigate the efficacy and safety of NEU-411 in men and women aged 40-80 years with early Parkinson's Disease (PD) who have predicted elevations in the activity of the "leucine-rich repeat kinase 2" ("LRRK2" for short) pathway based on their genetic profile. A DNA test will be used to identify the "LRRK2-driven" population with predicted elevation in the LRRK2 pathway. Participants will: • Take NEU-411 or placebo every day for 52 weeks

Type: Interventional

Start Date: Jan 2025

open study

The purpose of TEAM-HF IDE clinical trial is to evaluate safety and effectiveness of the HeartMate 3 LVAS compared to guideline directed medical therapy (GDMT) in a population of ambulatory advanced heart failure patients who are not dependent on intravenous inotrope.

Type: Interventional

Start Date: Dec 2024

open study

BMT CTN 2207 will investigate the use of marrow transplantation for treatment of severe aplastic anemia that has not previously been treated.

Type: Interventional

Start Date: May 2025

open study

The goal of this clinical trial is to test in moderately symptomatic chronic subdural hematoma (CSDH) patients if middle meningeal artery embolization (MMAE) can be used as an alternative to conventional open surgery. The main questions it aims to answer are: - Compared to open conventional surgery, does MMAE reduce the need for rescue surgery or deaths? - What is the safety of MMAE and conventional open surgery in these patients? Participants will be asked to: - Share their medical history and undergo physical examinations - Have blood drawn - Have CT scans of the head - Answer questionnaires - Undergo MMAE or conventional open surgery - Provide information about possible adverse events Researchers will compare participants in the MMAE group with those in the conventional open surgery group to see if there is a reduced need for rescue surgery or deaths and evaluate safety.

Type: Interventional

Start Date: Nov 2024

open study

RESET-Myositis: Open-Label Study to Evaluate the Safety and Efficacy of CABA-201 in Subjects with Active Idiopathic Inflammatory Myopathy or Juvenile Idiopathic Inflammatory Myopathy

Type: Interventional

Start Date: Dec 2023

open study

Study GLB-001-01 is a first-in-human (FIH), Phase 1, open-label, dose escalation and expansion clinical study of GLB-001 in participants with relapsed or refractory acute myeloid leukemia (R/R AML) or in participants with relapsed or refractory higher-risk myelodysplastic syndromes (R/R HR-MDS). The dose escalation part (Phase 1a) of the study will evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD) and preliminary efficacy of GLB-001 administered orally. Approximately 24 participants (up to 42 participants) may be enrolled in Phase 1a of the study. The dose expansion part (Phase 1b) will be followed to understand the relationships among dose, exposure, toxicity, tolerability and clinical activity, to identify minimally active dose, and to select the recommended dose(s) for phase 2 study. Up to 24 participants (12 participants per dose level) may be enrolled in Phase 1b of the study.

Type: Interventional

Start Date: Jan 2024

open study

The purpose of this randomized, double-blind, placebo-controlled, parallel group study is to determine the efficacy of frexalimab in delaying the disability progression and the safety up to 36 months double-blind administration of study intervention compared to placebo in male and female participants with nrSPMS (aged 18 to 60 years at the time of enrollment). People diagnosed with nrSPMS are eligible for enrollment as long as they meet all the inclusion criteria and none of the exclusion criteria. Study details include: - This event-driven study will end when the target number of 6-month cCDP events is achieved, and the study is expected to last 43 months from randomization of the first participant to the common study end. - The number of scheduled visits will be up to 25 (including 3 follow-up visits) with a visit frequency of every month for the first 6 months and then every 3 months. - If the prespecified number of events for 6-month cCDP is not reached by V21/W180, scheduled visits will continue every 3 months.

Type: Interventional

Start Date: Dec 2023

open study

The purpose of the study is to understand how the study medicine PF-06823859 (dazukibart) works in people with idiopathic inflammatory myopathies (DM and PM). These disorders cause inflammation that weakens the muscles that are important for movement and may also cause skin rash in people with DM. This study is seeking participants who: - Are 18 years of age or older or minimum legal adult age as defined per local regulation, whichever is greater - Have active DM or active PM. - Are receiving a stable dose of 1 corticosteroid taken by mouth and/or 1 traditional immunosuppressant. - Note: Corticosteroids and immunosuppressants are medicines that help reduce inflammation and may signal to the immune system not to attack the body. Dermatomyositis (DM) is a rare disease that causes muscle inflammation that results in muscle weakness and low muscle stamina. Patients with DM have a characteristic skin rash. Polymyositis (PM) is a rare disease that involves mainly muscle inflammation resulting in muscle weakness, that can sometimes be painful. Patients with DM and PM may have trouble going up the steps, walking or getting to a standing position. Some of the participants will receive the study medicine (dazukibart) and some will receive placebo (which is similar to study medicine but contains no medicine in it). The study medicine or placebo will be given as an intravenous (IV) infusion (directly into the veins), which takes about 1 hour; every 4 weeks from Day 1 to Week 48 of the study. Both dazukibart and placebo and will be given at the study site. The study will compare the experiences of people receiving study medication to those of the people who do not. This will help to see if dazukibart is safe and effective. Participants will take part in this study for about 13 months. During this time, participants will have 15 study visits. These visits will be performed at the study site.

Type: Interventional

Start Date: May 2023

open study

The purpose of this study is to evaluate the efficacy, safety, tolerability and pharmacokinetics of NMD670 in the treatment of ambulatory adults with spinal muscular atrophy type 3

Type: Interventional

Start Date: Sep 2023

open study

The objectives of this application are to 1) compare short- and long-term harm reduction and abuse liability potential of a nicotine salt pod-based electronic cigarettes (EC) in African American (AA) exclusive EC, dual cig-EC, and exclusive cig users, 2) characterize factors that predict who switches fully, partially, or not at all, and 3) examine if harm reduction can be further enhanced by treating dual users with varenicline (VAR) to eliminate cigarette smoking.

Type: Interventional

Start Date: Jul 2023

open study

This study will assess if the presence of immune system cells in and around the tumor impacts tumor shrinkage in patients receiving neoadjuvant chemoimmunotherapy for triple-negative breast cancer.

Type: Interventional

Start Date: Dec 2022

open study

The goal of this clinical study is to compare the study drug, axicabtagene ciloleucel, versus standard of care (SOC) in first-line therapy in participants with high-risk large B-cell lymphoma.

Type: Interventional

Start Date: Feb 2023

open study

The purpose of this study is to compare the efficacy and safety of mezigdomide (CC-92480), bortezomib and dexamethasone (MeziVd) versus pomalidomide, bortezomib and dexamethasone (PVd) in participants with relapsed or refractory multiple myeloma (RRMM) who received between 1 to 3 prior lines of therapy and who have had prior lenalidomide exposure.

Type: Interventional

Start Date: Sep 2022

open study

This phase II/III trial studies how well sentinel lymph node biopsy works and compares sentinel lymph node biopsy surgery to standard neck dissection as part of the treatment for early-stage oral cavity cancer. Sentinel lymph node biopsy surgery is a procedure that removes a smaller number of lymph nodes from your neck because it uses an imaging agent to see which lymph nodes are most likely to have cancer. Standard neck dissection, such as elective neck dissection, removes many of the lymph nodes in your neck. Using sentinel lymph node biopsy surgery may work better in treating patients with early-stage oral cavity cancer compared to standard elective neck dissection.

Type: Interventional

Start Date: Sep 2020

open study

The intent of this study is to establish a registry of post-surgical outcomes in patients undergoing radical cystectomy at MD Anderson Cancer Center and the collaborating institutions. The goals of this initiative are to obtain a detailed baseline of multiple patient-reported outcomes (PRO) and clinician-reported outcomes (CRO) as well as various presenting conditions associated with them, so that future quality improvement interventions can be evaluated accurately as to their relative contribution to improved outcomes.

Type: Observational [Patient Registry]

Start Date: Apr 2015

open study