Vanderbilt Clinical Trials

This study is an access and distribution protocol for unlicensed cryopreserved cord blood units (CBUs) in pediatric and adult patients with hematologic malignancies and other indications.

Type: Observational

Start Date: Oct 2011

open study

This study is a Phase III, Randomized, Controlled, Global Multicenter Study to Evaluate the Efficacy and Safety of Oral Tinengotinib versus Physician's Choice in Subjects with Fibroblast Growth Factor Receptor (FGFR)-altered, Chemotherapy- and FGFR Inhibitor-Refractory/Relapsed Cholangiocarcinoma

Type: Interventional

Start Date: Dec 2023

open study

This post-approval study (PAS) is designed to provide continued clinical evidence to confirm the long-term safety and effectiveness of the FlexAbilityTM Ablation Catheter, Sensor EnabledTM (FlexAbility SE) for the treatment of ventricular tachycardia in a post-market environment. This is a prospective, single arm, open-label, multi-center, observational study.

Type: Observational

Start Date: Jul 2023

open study

This research will establish a mg/kg dose for a future RCT to determine the efficacy of high-dose oral montelukast for children with moderate and severe acute asthma exacerbations. Aim: Perform an adaptive, double-masked randomized controlled trial (RCT) of high-dose oral montelukast, with escalating mg/kg dose levels determined by PK-guided dose modeling, added to standard treatment versus standard treatment alone, in children with exacerbations that are moderate or severe after initial treatment with inhaled albuterol. Hypothesis 1: High-dose oral montelukast achieves peak plasma concentration (Cmax) >1,700 ng/ml in >86% of at least one of three sequential participant groups with escalating weight-based (milligram/kilogram or mg/kg) doses between groups. Hypothesis 2: Participants randomized to high-dose oral montelukast have a 2 point or greater improvement of the validated Acute Asthma Intensity Research Score (AAIRS) at 4 hours post-treatment in comparison with control group participants. Hypothesis 3: Among montelukast recipients, Cmax correlates with change of the AAIRS at 4 hours, after adjustment for pre-treatment exacerbation severity and systemic leukotriene stress measured using urinary leukotriene E4 (LTE4).

Type: Interventional

Start Date: Oct 2023

open study

This phase I trial tests the safety, side effects, and best dose of ZEN003694 in combination with the usual treatment with capecitabine in treating patients with cancer that has spread from where it first started (primary site) to other places in the body (metastatic) or cannot be removed by surgery (unresectable) and that it has progressed on previous standard treatment. ZEN003694 is an inhibitor of a family of proteins called the bromodomain and extra-terminal (BET). It may prevent the growth of tumor cells that over produce BET protein. Capecitabine is in a class of medications called antimetabolites. It is taken up by cancer cells and breaks down into fluorouracil, a substance that kills cancer cells. Giving ZEN003694 in combination with capecitabine may be safe in treating patients with metastatic or unresectable solid tumors.

Type: Interventional

Start Date: Nov 2023

open study

The objective of this clinical study is to evaluate the safety and effectiveness of the Adagio VT Cryoablation System in the ablation treatment of Sustained Monomorphic Ventricular Tachycardia (SMVT)

Type: Interventional

Start Date: Sep 2023

open study

This is a multicenter, double-blind, parallel-group, placebo controlled, 2-part study to evaluate the efficacy and safety of ZX008 when used as adjunctive therapy for the treatment of uncontrolled seizures in children and adults with cyclin-dependent kinase like-5 (CDKL5) deficiency disorder (CDD).

Type: Interventional

Start Date: Mar 2022

open study

RATIONALE: Studying samples of tumor tissue from patients with cancer in the laboratory may help doctors learn more about changes that may occur in RNA and identify biomarkers related to cancer. PURPOSE: This research study is collecting and analyzing lung tissue samples from patients undergoing surgery for non-small cell lung cancer.

Type: Observational

Start Date: May 2001

open study

The goal of this Open-Label Study is to evaluate the safety and tolerability of PRTH-101 alone or in combination with pembrolizumab in adults with advance or metastatic solid tumors.

Type: Interventional

Start Date: Mar 2023

open study

The GAP Study is a prospective cohort study designed to comprehensively investigate genetic variations that may contribute to cancer development among individuals diagnosed with appendix/appendiceal cancer who are ages 18+ years.

Type: Observational

Start Date: Nov 2022

open study

This is an open-label, multicenter, two-arm Phase II clinical trial that will evaluate the impact of 2nd line chemotherapy (i.e. capecitabine) on survival in patients with non-Luminal A hormone receptor-positive (HR+) metastatic breast cancer (MBC)

Type: Interventional

Start Date: Sep 2023

open study

Study SPH4336-US-01 is an open-label (no placebo), multicenter clinical trial to evaluate the safety, blood levels (pharmacokinetics) and preliminary anti-tumor effects of SPH4336, a selective enzyme blocker, in patients with specific types of liposarcomas (tumors expressing the target of the study drug).

Type: Interventional

Start Date: Aug 2023

open study

The protocol describes a randomized, double-blind, placebo-controlled trial with independent sub-studies of setmelanotide in patients with obesity and at least one of the specific gene variants in the Melanocortin-4 Receptor pathway: - POMC or PCSK1 (Sub-study 035a) - LEPR (Sub-study 035b) - SRC1 (Sub-study 035c) - SH2B1 (Sub-study 035d) The objectives and endpoints are identical for these sub-studies.

Type: Interventional

Start Date: Dec 2021

open study

Benign central airway stenosis (BCAS) is an important cause of both pulmonary morbidity and mortality. Notable causes include post-intubation stenosis, collagen vascular diseases, airway trauma, infectious and idiopathic subglottic stenosis (iSGS). Surgery is the preferred definite option; however, the first therapeutic attempt is usually endoscopic to temporarily restore airway patency and symptomatic improvement. Several endoscopic modalities exist for treatment. Most commonly, thermal or laser therapy to make radial incisions into the stenotic lesion, followed by balloon dilation to increase the area of patency. Clinicians may also inject steroids or antineoplastic agents such as mitomycin C. All of these methods have benefits and associated risks. Symptomatic stenosis frequently reoccurs with these methods. For example, the investigators have been doing 3-4 ballon dilations procedures a week at our institution. Spray cryotherapy (SCT) is a novel FDA-cleared technique that allows for liquid nitrogen to be delivered through the working channel of a bronchoscope. Few retrospective studies exist without more robust clinical trial data to reduce the risk of bias and support its widespread use. The investigators postulate that SCT and standard of care techniques will improve airway patency volume at six months than the standard of care techniques alone. Some of the proposed advantages include improved wound healing which may translate to less scar tissue and thus improvements in airway patency for a longer duration of time.

Type: Interventional

Start Date: Oct 2021

open study

One-size-fits-all approaches have failed to demonstrate sustained effects on childhood obesity, especially among low-income minority families, who experience constantly changing barriers to engaging in health behavior. Addressing obesity in these populations requires intervening in early childhood and situating interventions in the context of families and communities. Developing personalized childhood obesity prevention interventions with sustained effectiveness that support families in health behaviors despite dynamic barriers could address chronic disease risk and health disparities in low-income and minority communities.

Type: Interventional

Start Date: Nov 2021

open study

The influenza virus is a significant cause of morbidity in adult solid organ transplant (SOT) recipients. However, these individuals show a suboptimal response to vaccines including the standard-dose (SD) inactivated influenza vaccine (IIV). Recent studies have investigated two strategies to overcome poor immune responses in SOT recipients: (1) administration of high-dose (HD)-IIV compared to SD-IIV and (2) two doses of SD-IIV compared to one dose of SD-IIV in the same influenza season. The first study compared HD-IIV vs. SD-IIV in adult SOT and noted HD-IIV was safe and reported higher immunogenicity; however, the median post-transplant period was 38 months. In another phase II trial of adult SOT recipients, two doses of SD-IIV a month apart compared to one-dose SD-IIV revealed increased immunogenicity, with a median post-transplantation period of 18 months. Therefore, these studies lack evaluation in the early post-transplantation period in this vulnerable population when influenza disease is most severe. The administration of two-doses of HD-IIV in the same influenza season has also not been studied in SOT recipients. Moreover, the vast majority of SOT influenza vaccinations studies have not substantively evaluated prolonged immunogenicity. Thus, the optimal immunization strategy for SOT recipients less than 12 months post-transplant is poorly-defined. In addition, the immunologic predictors and correlates of influenza vaccine immunogenicity in SOT recipients have not been defined. The investigators hypothesize that adult solid organ transplant recipients that are 1-11 months out from transplant and are receiving high-dose inactivated influenza vaccine will have higher hemagglutination inhibition (HAI) geometric mean titers to influenza A antigens compared to adult SOT recipients receiving standard-dose inactivated influenza vaccine. To test this hypothesis and address the above critical knowledge gaps, The investigators propose to conduct a phase II multicenter randomized controlled trial comparing either two doses HD-IIV, two doses of SD-IIV, or one-dose of HD-IIV in adult kidney, heart, and liver SOT recipients 1-11 months post-transplantation. The results of this study will address significant gaps in knowledge regarding influenza vaccine strategies and immune responses in adult SOT recipients and will guide vaccine recommendations in this vulnerable population.

Type: Interventional

Start Date: Jan 2021

open study

This involves development and application of magnetic resonance imaging (MRI) methods for visualizing hemodynamic and metabolic relationships in healthy volunteers with advanced meditation experience.

Type: Interventional

Start Date: Feb 2021

open study

This phase II trial studies how well combination chemotherapy works in treating patients with newly diagnosed stage II-IV diffuse anaplastic Wilms tumors (DAWT) or favorable histology Wilms tumors (FHWT) that have come back (relapsed). Drugs used in chemotherapy regimens such as UH-3 (vincristine, doxorubicin, cyclophosphamide, carboplatin, etoposide, and irinotecan) and ICE/Cyclo/Topo (ifosfamide, carboplatin, etoposide, cyclophosphamide, and topotecan) 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. This trial may help doctors find out what effects, good and/or bad, regimen UH-3 has on patients with newly diagnosed DAWT and standard risk relapsed FHWT (those treated with only 2 drugs for the initial WT) and regimen ICE/Cyclo/Topo has on patients with high and very high risk relapsed FHWT (those treated with 3 or more drugs for the initial WT).

Type: Interventional

Start Date: Oct 2020

open study

This study evaluates the progression of myopia in participants using microdosed atropine 0.01%, atropine 0.1%, or placebo ophthalmic solution. Eligible subjects will administer study medication daily in each eye for 48 months. Efficacy and safety assessments will be performed at visits scheduled for 1, 6, 12, 18, 24, 30 and 36 months after initiation of medication use. Subjects will be re-randomized at the 36 month visit, then followed at 6 month intervals for an additional year.

Type: Interventional

Start Date: Jun 2019

open study

This phase II trial studies how well the combination of dabrafenib and trametinib works after radiation therapy in children and young adults with high grade glioma who have a genetic change called BRAF V600 mutation. Radiation therapy uses high energy rays to kill tumor cells and reduce the size of tumors. Dabrafenib and trametinib may stop the growth of tumor cells by blocking BRAF and MEK, respectively, which are enzymes that tumor cells need for their growth. Giving dabrafenib with trametinib after radiation therapy may work better than treatments used in the past in patients with newly-diagnosed BRAF V600-mutant high-grade glioma.

Type: Interventional

Start Date: Feb 2020

open study

Pseudohypoparathyroidism is a genetic disorder with limited treatment options. Patients have early-onset obesity, short stature and increased risk of type 2 diabetes. This phase 2 clinical trial will test the efficacy of theophylline, a phosphodiesterase inhibitor, in pseudohypoparathyroidism. The investigators hypothesize that theophylline will cause weight loss, improve glucose tolerance and slow growth plate closure in children and young adults.

Type: Interventional

Start Date: Sep 2018

open study

The purpose of this study is to evaluate the safety and tolerability of extended dosing with eplontersen in participants with ATTR-CM.

Type: Interventional

Start Date: Nov 2022

open study

Study CDFV890G12101 is an open-label, phase 1b, multicenter study with a randomized two-dose optimization part, and a dose expansion part consisting of two groups evaluating DFV890 in patients with myeloid diseases. The purpose of this study is to assess the safety, tolerability, pharmacokinetics, pharmacodynamics, efficacy and recommended dose for single agent DFV890 in patients with lower risk (LR: very low, low or intermediate risk) myelodysplastic syndromes (LR MDS) and lower risk chronic myelomonocytic leukemia (LR CMML).

Type: Interventional

Start Date: May 2023

open study

This study aims to learn about the effects of continuous positive airway pressure (CPAP) on people with autonomic failure and high blood pressure when lying down (supine hypertension) to determine if it can be used to treat their high blood pressure during the night. CPAP (a widely used treatment for sleep apnea) involves using a machine that blows air into a tube connected to a mask covering the nose, or nose and mouth, to apply a low air pressure in the airways. The study includes 3-5 days spent in the Vanderbilt Clinical Research Center (CRC): at least one day of screening tests, followed by up to 3 study days. Subjects may be able to participate in daytime and/or overnight studies. The Daytime study consists of 2 study days: one with active CPAP and one with sham CPAP applied for up to 2 hours. The Overnight study consists of 3 study nights: one with active CPAP, one with sham CPAP, both applied for up to 9 hours and one night sleeping with the bed tilted head-up.

Type: Interventional

Start Date: Jun 2022

open study

SAR439459 is a human anti-Transforming growth factor β (TGFβ) monoclonal antibody. This phase 1 clinical study investigates the safety, tolerability, and activity of a single dose of SAR439459 in adult participants with OI. Participants will receive a single IV dose of SAR439459 with safety, pharmacokinetic (PK), and pharmacodynamic (PD) assessments over 24 weeks. There will be up to 3 dose cohorts. In addition to safety, tolerability, and PK assessments, bone mineral density (BMD) will be evaluated by dual-energy Xray absorptimetry (DXA) scan and a series of blood biomarkers will be monitored to document pharmacodynamic effects of the single dose of SAR439459.

Type: Interventional

Start Date: Aug 2022

open study