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 Phase III trial will examine the efficacy of computerized cognitive training methods on perceived cognitive impairment in breast cancer survivors.

Type: Interventional

Start Date: Feb 2024

open study

The purpose of this study is to assess the efficacy of metformin to improve airway ion channel function in those with CF-related diabetes (CFRD)

Type: Interventional

Start Date: Feb 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 primary aims of this study is to characterize exosomal biomarker levels in patients with locally advanced rectal cancer undergoing neoadjuvant chemoradiation therapy. Compare rates of exosomal expression before during and after chemoradiation therapy with pathologic response rates at the time of APR or LAR. The researchers will also examine the functionality of these exosomes in malignant colonic organoids and mouse models of colorectal cancer.

Type: Observational [Patient Registry]

Start Date: Feb 2018

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

This phase II/III trial studies how well radiation therapy works when given together with cisplatin, docetaxel, cetuximab, and/or atezolizumab after surgery in treating patients with high-risk stage III-IV head and neck cancer the begins in the thin, flat cells (squamous cell). Specialized radiation therapy that delivers a high dose of radiation directly to the tumor may kill more tumor cells and cause less damage to normal tissue. Drugs used in chemotherapy, such as cisplatin and docetaxel, work in different ways to stop the growth of tumor cells, either by killing the cells or by stopping them from dividing. Cetuximab is a monoclonal antibody that may interfere with the ability of tumor cells to grow and spread. Immunotherapy with monoclonal antibodies, such as atezolizumab, may help the body's immune system attack the cancer, and may interfere with the ability of tumor cells to grow and spread. The purpose of this study is to compare the usual treatment (radiation therapy with cisplatin chemotherapy) to using radiation therapy with docetaxel and cetuximab chemotherapy, and using the usual treatment plus an immunotherapy drug, atezolizumab.

Type: Interventional

Start Date: Mar 2013

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

The goal of this clinical trial is to treat male and female participants with two types of Multiple Sclerosis (MS) called primary progressive or secondary progressive MS. The main questions the trial aims to answer are the following: - Is TRX319 safe when administered to patients with progressive forms of MS? - At what dose does TRX319 work the best to treat participants with primary and or secondary progressive MS? - Is pre-conditioning (with Bendamustine) needed to allow TRX319 to better treat participants with primary and/or secondary progressive MS? Participants will be asked to be on study for up 1 year and may receive up to 3 total administrations of TRX319. While on study, participants will have blood tests and other assessments (MRI scans and lumbar punctures) done to understand the safety of TRX319 and how it may benefit their multiple sclerosis.

Type: Interventional

Start Date: Mar 2026

open study

Rationale: LTI-03 is an experimental medication breathed into the lungs using an inhaler. It is being studied for the treatment of Idiopathic Pulmonary Fibrosis (IPF). IPF is a progressive, fatal lung disease caused by the death of lung cells involved in oxygen uptake and by progressive fibrosis (scarring) of the lungs. As the disease progresses, patients experience loss of lung function and increased breathing problems. LTI-03 is hypothesized to treat IPF by protecting and restoring the function of the oxygen uptake cells and by controlling lung fibrosis which may result in improving lung scarring. The purpose of this research is to evaluate LTI-03 including: its safety, whether it causes side effects, whether it improves lung scarring, and whether it improves IPF symptoms. LTI-03 will be compared to placebo in patients diagnosed with IPF within the last 5 years. Patients on a stable dose of nintedanib, pirfenidone, or nerandomilast (if available by prescription) may participate. Trial Design: This is a Phase 2, randomized, double-blind, placebo-controlled, multi-center study that includes a 28-day Screening Period, a 24-week Treatment Period, and 4-week Follow-up Period. Study Assessments: Up to 9 visits to the study clinic will be required. Safety and tolerability will be evaluated with the following assessments: physical examination; collection of vital sign data (heart rate, blood pressure, respiratory rate and peripheral oxygen saturation [SpO2] via pulse oximetry); heart data collected by 12-lead electrocardiogram; and collection of blood samples for safety laboratory tests. In addition, participants will be asked about any adverse events (side effects) they have experienced between clinic visits, if they have changed any medications, and if they are able to properly use their study drug inhaler. Participants will undergo a lung function test (spirometry) at every visit, which will be used to evaluate both safety and efficacy. Another test measuring the diffusion capacity of the lungs for carbon monoxide (DLCO) will be required at Screening only. Blood samples will also be collected at each visit to measure disease biomarkers. At select visits patients will be asked to complete the Living with Pulmonary Fibrosis questionnaire to evaluate their IPF symptoms. Participants will also undergo a specialized lung scan (HRCT) at Baseline and at the End of Treatment to measure changes in lung fibrosis. Interventions: LTI-03 and placebo are provided in powder-filled capsules that participants will self- administer using an inhaler. Placebo capsules look like LTI-03 capsules but have no active ingredients. Approximately 120 participants will be randomly assigned in a blinded manner to one of study drug treatment groups.

Type: Interventional

Start Date: Feb 2026

open study

The goal of this clinical trial is to collect additional data to demonstrate if the Contour Neurovascular System (CNS) is a safe and effective treatment of wide-necked, saccular, intracranial aneurysms. Participants will: Undergo treatment with the Contour Neurovascular System Complete follow-up visits at 1month, 12months, and 24 months, following their procedure Report any adverse events to their study team

Type: Interventional

Start Date: Jun 2025

open study

This phase III trial compares the effect of adding cetuximab to pembrolizumab versus pembrolizumab alone in treating patients with head and neck squamous cell carcinoma (HNSCC) that has come back after a period of improvement (recurrent) and/or that has spread from where it first started (primary site) to other places in the body (metastatic). Cetuximab is in a class of medications called monoclonal antibodies. It binds to a protein called EGFR, which is found on some types of tumor cells. This may help keep tumor cells from growing. Immunotherapy with monoclonal antibodies, such as pembrolizumab, may help the body's immune system attack the tumor, and may interfere with the ability of tumor cells to grow and spread. Giving cetuximab and pembrolizumab together may be more effective at treating patients with recurrent and/or metastatic HNSCC than pembrolizumab alone.

Type: Interventional

Start Date: Mar 2025

open study

A first in human study to evaluate the safety and preliminary antitumor activity of BBO-8520, a KRAS G12C (ON and OFF) inhibitor, as a single agent and in combination with pembrolizumab and BBO-10203 in subjects with locally advanced and unresectable or metastatic non-small cell lung cancer with a KRAS (Kirsten rat sarcoma) G12C mutation.

Type: Interventional

Start Date: May 2024

open study

This phase II trial evaluates response-guided low-dose tamoxifen for reducing breast density in women who are at higher than average risk for breast cancer. Increasing breast density is a well established risk factor for breast cancer. Tamoxifen is a selective estrogen receptor modulator. It works by blocking the effects of the hormone estrogen in the breast. Tamoxifen has been shown to reduce breast density, even at reduced dosages, and is approved for the prevention of breast cancer.

Type: Interventional

Start Date: Sep 2024

open study

The main aim of this study is to learn about the effect of treatment with vedolizumab IV (vedolizumab) together with adalimumab or vedolizumab (VDZ) together with ustekinumab (UST) in adults with moderate to severe Crohn's Disease, and the effect of treatment with vedolizumab alone, after the dual targeted treatment. The study is conducted in two parts. In Part A, participants will receive the dual targeted treatment (vedolizumab together with either adalimumab or ustekinumab). In part B, participants will receive vedolizumab only. Part B will include participants who responded to the treatment in Part A. Each participant will be followed up for at least 26 weeks after the last dose of treatment.

Type: Interventional

Start Date: Apr 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

The purpose of this prospective observational study is to collect data from participants who have recently had an allogenic Stem Cell Transplant(alloSCT) and are at risk of Chronic Graft Versus Host Disease(cGVHD)

Type: Observational

Start Date: Aug 2023

open study

The primary objective is to quantify the degree of pain relief in patients undergoing gonadal vein embolization with coils as well as identify clinical or imaging factors that are predictive of a positive response to treatment, or poor response to treatment.

Type: Observational

Start Date: May 2019

open study

This study is designed with the interest to learn on the effects of estrogen levels and how it affects body compositions and muscle function in midlife women who are taking a GLP-1RA for weight loss.

Type: Observational

Start Date: Feb 2026

open study

This phase II study evaluates SABR as non-operative treatment for women aged 65+ with ER-positive, HER2-negative breast cancer ≤5 cm and no lymph node involvement. Eligible patients, including those previously on endocrine therapy, receive 5 SABR treatments, followed by monitoring of quality of life and assessment of treatment-related toxicity.

Type: Interventional

Start Date: Nov 2025

open study

This is an open-label extension study for participants who were previously enrolled in and completed an Avalyn Pharma Sponsored study with an inhaled antifibrotic, such as AP01. Eligible participants will have their final dose of drug at the end of study visit from the lead-in study and first AP-LTE-008 study visit on the same day.

Type: Interventional

Start Date: Apr 2025

open study

The purpose of this study is to find out if Berinert can improve kidney function in the first year after transplant and to find out what effects, good or bad, Berinert will have in the kidney recipient. This research study will compare Berinert to placebo. The placebo looks exactly like Berinert but does not contain any active drug. Placebos are used in research studies to see if the results are due to the study drug or due to other reasons. Neither you or the study doctor can choose or know which group is assigned. The primary objective is to test whether intrarenal artery C1 esterase inhibitor (C1INH) injection into the donor kidney prior to transplantation improves kidney function in recipients of high risk, deceased donor kidney transplants as measured by 12-month Estimated Glomerular Filtration Rate (eGFR) Chronic Kidney Disease Epidemiology Collaboration (CDK-EPI)

Type: Interventional

Start Date: Sep 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 FOLFOX, 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

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