University of Kansas Medical Center

The primary purpose of the study is to evaluate the safety and tolerability of single intravenous (IV) doses of PGN-EDODM1 administered to participants with Myotonic Dystrophy Type 1 (DM1). The study consists of 2 periods: A Screening Period (up to 30 days) and a Treatment and Observation Period (16 weeks).

Type: Interventional

Start Date: Dec 2023

open study

The purpose of this study is to evaluate the safety, and tolerability and efficacy of VX-522 in participants 18 years of age and older with cystic fibrosis and a cystic fibrosis transmembrane conductance regulator (CFTR) genotype not responsive to CFTR modulator therapy.

Type: Interventional

Start Date: Feb 2023

open study

This phase I trial tests the safety, side effects, and best dose of combination therapy with tazemetostat and belinostat in treating patients with lymphomas that have returned (relapsed) or resisted treatment (refractory). Tazemetostat is in a class of medications called EZH2 inhibitors. The EZH2 gene provides instructions for making a type of enzyme called histone methyltransferase which is involved in gene expression and cell division. Blocking EZH2 may help keep cancer cells from growing. Belinostat is in a class of medications called histone deacetylase inhibitors. Histone deacetylases are enzymes needed for cell division. Belinostat may kill cancer cells by blocking histone deacetylase. It may also prevent the growth of new blood vessels that tumors need to grow and may help make cancer cells easier to kill with other anticancer drugs. There is some evidence in animals and in living human cells that combination therapy with tazemetostat and belinostat can shrink or stabilize cancer, but it is not known whether this will happen in people. This trial may help doctors learn more about treatment of patients with relapsed or refractory lymphoma.

Type: Interventional

Start Date: Mar 2023

open study

A Randomized, Double-blind, Placebo-controlled, Parallel Group Study of BPN14770 in Male Adults (Aged 18 to 45) with Fragile X Syndrome

Type: Interventional

Start Date: Nov 2022

open study

This is a 24-month, observational study of 50 participants with Becker muscular dystrophy (BMD)

Type: Observational

Start Date: Apr 2022

open study

This is a Phase 1/2 multicenter, open-label, single dose trial of 4D-710 investigational gene therapy in adults with cystic fibrosis.

Type: Interventional

Start Date: Mar 2022

open study

A multi-center, longitudinal, prospective, non-comparative study to investigate the long-term safety and effectiveness of risdiplam, prescribed based on clinician judgment as per the Evrysdi® U.S. Package Insert (USPI) in adult and pediatric participants with spinal muscular atrophy (SMA). In this study, participants will be followed for up to 5 years from enrollment or until withdrawal of consent, loss to follow-up, or death. Participants who discontinue risdiplam may still remain in the study, if they agree to continue participating in the follow-up assessments.

Type: Interventional

Start Date: Jun 2022

open study

The number of older Americans will double in the next 4 decades to nearly 90 million, placing an unprecedented financial and resource burden on the health care system. Exercise has clear and demonstrable physical benefits, but a more precise understanding of how exercise supports cognitive function is essential. Demonstrating definitively that exercise as recommended by public health entities has benefits for cognition would have enormous public health implications, encourage the public to adapt more active lifestyles, and stimulate the development of effective exercise delivery programs.

Type: Interventional

Start Date: Oct 2021

open study

The objective of this study is to evaluate the effects of sotatercept (MK-7962, formerly called ACE-011) treatment (plus background pulmonary arterial hypertension (PAH) therapy) versus placebo (plus background PAH therapy) on time to clinical worsening (TTCW) in participants who are newly diagnosed with PAH and are at intermediate or high risk of disease progression.

Type: Interventional

Start Date: Mar 2022

open study

This trial will look at a drug called SEA-CD70 with and without azacitidine, to find out if it is safe for patients with myelodysplastic syndrome (MDS) and acute myeloid leukemia (AML). It will study SEA-CD70 to find out what its side effects are and if it works for AML and MDS. A side effect is anything the drug does besides treating cancer. This study will have six groups or "parts." - Part A will find out how much SEA-CD70 should be given to patients. - Part B will use the dose found in Part A to find out how safe SEA-CD70 is and if it works to treat patients with MDS. - Part C will use the dose found in Part A to find out how safe SEA-CD70 is and if it works to treat patients with AML. - Part D will find out how much SEA-CD70 with azacitidine should be given to patients. - Part E will use the dose found in Part D to find out how safe SEA-CD70 with azacitidine is and if it works to treat patients with MDS or MDS/AML that has not been treated. - Part F will use the dose found in Part D to find out how safe SEA-CD70 with azacitidine is and if it works to treat patients with MDS or MDS/AML.

Type: Interventional

Start Date: Aug 2020

open study

Spinal muscular atrophy (SMA) is a neurogenetic disorder caused by a loss or mutation in the survival motor neuron 1 gene (SMN1) on chromosome 5q13, which leads to reduced SMN protein levels and a selective dysfunction of motor neurons. SMA is an autosomal recessive, early childhood disease with an incidence of 1:10,000 live births. SMA is the leading cause of infant mortality due to genetic diseases. The purpose of this registry is to assess the long term outcomes of patients with SMA in the context of advances in treatment options and also to characterize and assess long-term safety and effectiveness of OAV-101.

Type: Observational [Patient Registry]

Start Date: Sep 2018

open study

The purpose of the clinical study is to investigate whether the local delivery of bisphosphonate as a surgical adjuvant can decrease the chance of a giant cell tumor of bone coming back to the same location. The hypothesis is that the local administration of bisphosphonate will decrease the rate of the tumor returning compared to traditional aggressive surgical removal of the tumor.

Type: Interventional

Start Date: May 2018

open study

The goal of this observational study is to better understand links between patient or tumor characteristics and outcomes in HER2-positive breast cancer.

Type: Observational [Patient Registry]

Start Date: Sep 2024

open study

Lung transplant is an option for treating end-stage lung disease in cystic fibrosis (CF). In the United States, more people with CF and low lung function die each year than undergo lung transplant. More than half of people with CF who die without a lung transplant were never referred for consideration. Patient preference not to undergo lung transplant may account for 25-40% of decisions to defer referral. Rates of death without transplant are higher for people with CF who are members of marginalized communities, including those with Black race, Hispanic ethnicity, or low socioeconomic status. Increasing awareness of lung transplant among people with CF, and promoting understanding of the risks and benefits of transplant, can potentially reduce the number of people with CF who die without a lung transplant. The CF Foundation (CFF) lung transplant referral guidelines were developed to optimize the timing of referral for lung transplant. These guidelines recommend annual conversations with people with CF once their forced expiratory volume in one second (FEV1) is <50% predicted. Considering lung transplant as a treatment option before it is medically needed will allow more time to learn about lung transplant and address any barriers to lung transplant that may exist. Investigators are interested in understanding how people with CF use lung transplant educational resources and how one prepares for having discussions and/or making decisions about lung transplant as a treatment option for advanced CF. The purpose of this study is to test whether a research website improves patient preparedness for discussions about lung transplant. Investigators also aim to understand whether there are unique factors that affect people with CF from communities with decreased access to transplant ("communities of concern"). Study involvement will span 6 months and study activities will involve the following: - Four Zoom research sessions (15-30 minutes each) - Survey assessments - Access to a research website that contains educational resources about lung transplant - Audio recording of a routine CF clinic visit to determine if and how lung transplant is discussed between a participant and his/her/their CF doctor

Type: Interventional

Start Date: Sep 2023

open study

This study will evaluate the efficacy of TNX-103 (oral levosimendan) compared with placebo in subjects with PH-HFpEF as measured by the change in 6-Minute Walk Distance (6 MWD; Day 1 to Week 12).

Type: Interventional

Start Date: Jan 2024

open study

This is a Phase 2, double-blind, parallel-group, placebo-controlled study to assess the safety, tolerability, pharmacokinetics, pharmacodynamics, and efficacy of multiple doses of ONO-2808 in patients with MSA. This is the first study of ONO-2808 in patients with MSA.

Type: Interventional

Start Date: Sep 2023

open study

The proposed project will evaluate the musculoskeletal outcomes of quadriceps and hamstring muscle size and function following orthopedic knee surgery involving anterior cruciate ligament (ACL) repair or reconstruction. Currently, the research team collaborates with a team of orthopedic specialists at the University of Kansas Health System and monitor muscle size post-knee repair and follow the standard of care (SOC) practices of the licensed physical therapists (PT). The proposed project will include a randomized clinical trial to observe the muscular outcomes following the current SOC plus supplementation of calcium-β-hydroxy-β-methylbutyrate (caHMB) or placebo. CaHMB has been shown to improve rates of muscle protein synthesis while suppressing muscle protein breakdown in healthy adults. The use of caHMB has also provided evidence of muscular protection from atrophy during prolonged bed rest. This evidence supports the utility in clinically injured athletes that are subjected to disuse atrophy from the inability to bear weight or participate in typical daily physical activity. Additionally, matched for activity-related knee injuries, female athletes are more susceptible to incurring a significant injury due to a variety of genetic, hormonal, biological, anatomical, and biomechanical predispositions. Therefore, the proposed study will recruit approximately 30 females over the age of 18 that have sustained an injury to the ACL and will plan to undergo reconstructive knee surgery involving the ACL. Subjects will be monitored and measured prior to their surgical date (T0), at 2-weeks post operative (T1), and every 6-weeks until they are cleared to return to sport (T2-TRTS). Participants will be randomly assigned 1:1 in a double-blind manner to either an experimental (EXPHMB) or placebo (CONPLA) group. Doses will be provided to the participants in coded containers and will complete their dosing and a record log of intake for the duration of their rehabilitation. Three 3-day food, exercise, and health record logs will be collected to monitor nutritional intake, activity, and menstrual patterns at T0, T3, and TRTS. Participant's assessments will include body composition analysis via bioelectrical impedance analysis for total and segmental muscle and fat mass, skeletal muscle mass, and body fat percent. We will collect ultrasound images of the quadriceps and hamstrings of the operative-involved limb (OPIL) and non-operative limb (NOPL) limbs for muscle cross-sectional area (mCSA), thickness (mT), subcutaneous fat thickness (TFAT), and corrected echo intensity (EICOR) at all time points. Strength and functional assessments will occur upon entrance to the study (T0), and after loaded exercise is indicated by the practitioner (T3-TRTS) to the tolerance of the athlete. These assessments include maximal voluntary isometric contractions (MVIC) for leg extension and leg curl, standing balance tests, single-leg and double-leg jump assessment, and drop landing deviation, all on dual force plates. Data will be analyzed using multiple three-way analyses of variance [surgical leg (OPIL vs. NOPL) x treatment (EXPHMB vs. CONPLA) x time (T0 vs. T1 vs. T2 vs. T3 vs. T4 vs. T5 vs. TRTS) for the dependent variables. Significance is established at p≤0.05 and follow-up ANOVAS, T-tests, and post-hoc analyses will be conducted when significance is present. The evidence from this study will support the practitioners and coaches' abilities to maximize recovery and training outcomes, respectively, in previously injured female athletes.

Type: Interventional

Start Date: May 2023

open study

The purpose of this study is to evaluate the efficacy of aticaprant compared with placebo as adjunctive therapy to an antidepressant in improving depressive symptoms in adult participants with major depressive disorder (MDD) with moderate-to-severe anhedonia (ANH+) who have had an inadequate response to current antidepressant therapy with a selective serotonin reuptake inhibitor (SSRI) or serotonin-norepinephrine reuptake inhibitor (SNRI).

Type: Interventional

Start Date: Jun 2022

open study

CKJX839D12303 is a research study to determine if the study treatment, called inclisiran, in comparison to placebo taken in addition to statin medication can effectively reduce the total amount of plaque formed in the heart's vessels as measured by coronary computed tomography angiography (CCTA) from baseline to month 24. This study is being conducted in eligible participants with a diagnosis of non-obstructive coronary artery disease (NOCAD), where the coronary arteries are blocked less than 50%, and with no previous cardiovascular events.

Type: Interventional

Start Date: Jul 2022

open study

INSIGHTS is a registry research study that collects key information on medical history for girls and women with Turner syndrome and the clinical care they receive. This includes genetic tests, imaging, medications, and more for hundreds of patients seen at a number of clinics across the US. In addition to learning a lot about the current state of health for individuals with TS, INSIGHTS serves as an infrastructure to conduct future studies are meaningful to patients and their families.

Type: Observational [Patient Registry]

Start Date: May 2020

open study

This phase III trial compares the effect of usual treatment of chemotherapy and steroids and a tyrosine kinase inhibitor (TKI) to the same treatment plus blinatumomab. Blinatumomab is a Bi-specific T-Cell Engager ('BiTE') that may interfere with the ability of cancer cells to grow and spread. The information gained from this study may help researchers determine if combination therapy with steroids, TKIs, and blinatumomab work better than the standard of care.

Type: Interventional

Start Date: Jan 2021

open study

The objective of this randomized controlled trial (RCT) is to compare the clinical outcome of MitraClip™ device versus surgical repair in patients with severe primary MR who are at moderate surgical risk and whose mitral valve has been determined to be suitable for correction by MV repair surgery by the cardiac surgeon on the local site heart team.

Type: Interventional

Start Date: Jul 2020

open study

This phase I/II trial studies the safety, side effects and best dose of M3814 and to see how well it works when given together with radiation therapy in treating patients with pancreatic cancer that has spread to nearby tissue or lymph nodes (locally advanced). M3814 may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. Hypofractionated radiation therapy delivers higher doses of radiation therapy over a shorter period of time and may kill more tumor cells and have fewer side effects. Giving M3814 and hypofractionated radiation therapy together may be safe, tolerable and more effective than radiation therapy alone in treating patients with locally advanced pancreatic cancer.

Type: Interventional

Start Date: Jan 2021

open study

This screening and multi-sub-study randomized phase II/III trial will establish a method for genomic screening of similar large cancer populations followed by assigning and accruing simultaneously to a multi-sub-study hybrid Master Protocol (Lung-MAP). The type of cancer trait (biomarker) will determine to which sub-study, within this protocol, a participant will be assigned to compare new targeted cancer therapy, designed to block the growth and spread of cancer, or combinations to standard of care therapy with the ultimate goal of being able to approve new targeted therapies in this setting. In addition, the protocol includes non-match sub-studies which will include all screened patients not eligible for any of the biomarker-driven sub-studies.

Type: Interventional

Start Date: Feb 2019

open study

The investigators are doing this study to see if a treatment called dry needling improves muscle spasticity (muscle tightness) in people who have Multiple Sclerosis. Dry needling involves using tiny needles, like those in acupuncture, to target some muscles, like calf muscles. It differs from traditional acupuncture as it focuses on treating or managing muscle spots, aiming to reduce muscle stiffness and pain. Dry needling may offer a minimally-invasive and medication-free approach to improve muscle spasticity. The investigators hope to see if dry needling also helps enhance balance and walking abilities. This might provide potential improvements inoverall mobility and balance.

Type: Interventional

Start Date: May 2024

open study