Vanderbilt Clinical Trials

A first-in-human study of KK8123 in adults with X-linked hypophosphatemia.

Type: Interventional

Start Date: Oct 2024

open study

PROSPER trial is a trial to assess the efficacy of FNP-223 in slowing disease progression in participants with PSP as measured by the PSP Rating Scale (PSPRS) over 52 weeks and to assess the safety and tolerability of FNP-223 for 52 weeks in participants with PSP.

Type: Interventional

Start Date: Jul 2024

open study

The goal of this study is to learn if GS-1427 is effective in treating participants with moderate to severe ulcerative colitis. The study will compare participants in different treatment groups treated with GS-1427 with participants treated with placebo. The primary objective of this study is to assess the efficacy of GS-1427, compared with placebo control, in achieving clinical response at Week 12.

Type: Interventional

Start Date: Mar 2024

open study

The objective of the study is to assess the safety and technical feasibility of the Laplace Transcatheter Tricuspid Valve Replacement (TTVR) System

Type: Interventional

Start Date: Feb 2024

open study

This clinical trial will study the effects of aficamten (versus placebo) on the quality of life, exercise capacity, and clinical outcomes of patients with non-obstructive hypertrophic cardiomyopathy.

Type: Interventional

Start Date: Aug 2023

open study

The purpose of this protocol is to evaluate the efficacy of tulisokibart in participants with moderately to severely active ulcerative colitis. Study 1's primary hypotheses are that at least 1 tulisokibart dose level is superior to Placebo in the proportion of participants achieving clinical remission according to the Modified Mayo Score at Week 12, and that at least 1 tulisokibart dose level is superior to Placebo in the proportion of participants achieving clinical remission according to the Modified Mayo Score at week 52. Study 2's primary hypothesis is that at least 1 tulisokibart dose level is superior to Placebo in the proportion of participants achieving clinical remission according to the Modified Mayo Score at Week 12.

Type: Interventional

Start Date: Oct 2023

open study

This is a Phase III open-label study to assess if camizestrant improves outcomes compared to standard adjuvant endocrine therapy for patients with ER+/HER2- early breast cancer with intermediate-high or high risk for disease recurrence who completed definitive locoregional therapy (with or without chemotherapy). The planned duration of treatment in either arm within the study will be 7 years.

Type: Interventional

Start Date: Oct 2023

open study

This study will evaluate the clinical efficacy and safety of udenafil, an orally administered, potent and selective inhibitor of PDE5, versus placebo for the treatment of adolescent who have had the Fontan procedure.

Type: Interventional

Start Date: Oct 2023

open study

The COPE-AKI study is a randomized, pragmatic, parallel-arm trial comparing a multimodal intervention to usual care on hospital-free days through 90 days of study follow up. The primary study hypothesis is that patients randomized to the intervention will have increased odds of more hospital-free days through 90 days (primary clinical) compared to those randomized to usual care. Key secondary hypotheses will investigate the impact of the intervention on rates of major adverse kidney events, rates of recurrent AKI, and changes in patient-reported outcomes. Participants (N=2145) will be allocated 1:1 to the intervention or usual care using a web-based system to maintain allocation concealment using stratified randomization with randomly permuted blocks. Randomization will be stratified by clinical site.

Type: Interventional

Start Date: Sep 2023

open study

The goal of this clinical trial is to evaluate the safety of TOS-358 in adults with select solid tumors who meet study enrollment criteria. The main questions it aims to answer are: 1. what is the maximum tolerated dose and recommended dose for phase 2? 2. how safe and tolerable is TOS-358 at different dose levels when taken orally once or twice per day?

Type: Interventional

Start Date: Feb 2023

open study

This phase III trial compares total ablative therapy and usual systemic therapy to usual systemic therapy alone in treating patients with colorectal cancer that has spread to up to 4 body sites (limited metastatic). The usual approach for patients who are not participating in a study is treatment with intravenous (IV) (through a vein) and/or oral medications (systemic therapy) to help stop the cancer sites from getting larger and the spread of the cancer to additional body sites. Ablative means that the intention of the local treatment is to eliminate the cancer at that metastatic site. The ablative local therapy will consist of very focused, intensive radiotherapy called stereotactic ablative radiotherapy (SABR) with or without surgical resection and/or microwave ablation, which is a procedure where a needle is temporarily inserted in the tumor and heat is used to destroy the cancer cells. SABR, surgical resection, and microwave ablation have been tested for safety, but it is not scientifically proven that the addition of these treatments are beneficial for your stage of cancer. The addition of ablative local therapy to all known metastatic sites to the usual approach of systemic therapy could shrink or remove the tumor(s) or prevent the tumor(s) from returning.

Type: Interventional

Start Date: Jan 2023

open study

This is a Phase 1, open-label, first-in-human, dose-escalation and expansion study of SRF114, a monoclonal antibody that targets CCR8, as a monotherapy in patients with solid tumors.

Type: Interventional

Start Date: Dec 2022

open study

This is a Phase III, randomized, open-label, 3-arm, multicenter, international study assessing the efficacy and safety of Dato-DXd with or without durvalumab compared with ICT in participants with stage I to III TNBC with residual invasive disease in the breast and/or axillary lymph nodes at surgical resection following neoadjuvant systemic therapy.

Type: Interventional

Start Date: Nov 2022

open study

In this study, researchers will learn more about a study drug called litifilimab (BIIB059) in participants with cutaneous lupus erythematosus (CLE). The study will focus on participants who have either active subacute CLE or chronic CLE, or both. They may also have systemic lupus erythematosus (SLE). The participants did not respond to antimalarial therapy or had problems with the treatment that made it hard to continue. The main objective of the study is to learn about the effect litifilimab has on lowering the activity of the skin disease. Researchers will measure symptoms of CLE over time using a variety of scoring tools. These include the Cutaneous Lupus Erythematosus Disease Area and Severity Index (CLASI), the Cutaneous Lupus Activity of Investigator's Global Assessment-Revised (CLA-IGA-R), and the SELENA-SLEDAI Flare Index (SFI). The main questions researchers want to answer are: - How many participants have a score of 0 or 1 on the CLA-IGA-R looking at skin redness after treatment? - How many participants have their skin disease activity go down by at least 70%? Researchers will also learn more about the safety of litifilimab. They will study how participants' immune systems respond to litifilimab. Additionally, they will measure the effect litifilimab and CLE have on the quality of life of participants using a group of questionnaires. The study will be split into 2 parts - Part A and Part B. Both parts will be done as follows: - After screening, participants will be randomized to receive either litifilimab or placebo for the 1st treatment period. A placebo looks like the study drug but contains no real medicine. - Participants will receive either litifilimab or placebo as injections under the skin once every 4 weeks. - The 1st treatment period will be double blinded which means neither the researchers nor the participants will know if the participants are receiving litifilimab or placebo. - This double blinded treatment period will last 24 weeks, after which the 2nd treatment period will begin. - During the 2nd treatment period, all participants will receive litifilimab for 28 weeks. - After completing treatment in this study, participants that qualify will be given the choice to join the Long-Term Extension study, 230LE305. If they do not, they will move into a follow-up safety period that will last up to 24 weeks. - The total study duration for participants will be up to 80 weeks

Type: Interventional

Start Date: Sep 2022

open study

B-cell Lymphoma is an aggressive and rare cancer of a type of immune cells (a white blood cell responsible for fighting infections). Classic Follicular Lymphoma is a slow-growing type of non-Hodgkin lymphoma. The purpose of this study is to assess the safety of epcoritamab in adult participants in relapsed or refractory (R/R) diffuse large b-cell lymphoma (DLBCL) who have received at least 1 prior line of systemic antilymphoma therapy including at least 1 anti-CD20 monoclonal antibody-containing therapy or R/R classic follicular lymphoma (cFL). Adverse events will be assessed. Epcoritamab is an investigational drug being developed for the treatment of R/R DLBCL and R/R cFL. Study doctors will assess participants in a monotherapy treatment arm of epcoritamab. Participants will receive escalating doses of epcoritamab, until full dose is achieved. Approximately 184 adult participants with R/R DLBCL and R/R cFL will be enrolled in the study in approximately 80 sites in the United States of America. Participants will receive escalating doses of subcutaneous epcoritamab, until full dose is achieved, in 28-day cycles. There may be higher treatment burden for participants in this trial compared to their standard of care. Participants will attend regular visits during the study at an approved institution (hospital or clinic). The effect of the treatment will be frequently checked by medical assessments, blood tests, questionnaires and side effects.

Type: Interventional

Start Date: Aug 2022

open study

Genes contain genetic code which tell the body which proteins to make. Many types of cancer are caused by changes, or mutations, in a gene called KRAS. Researchers are looking for ways to stop the actions of abnormal proteins made from the mutated KRAS gene. The so-called G12D mutation in the KRAS gene is common in people with some solid tumors. ASP3082 is a potential new treatment for solid tumors in people who have the G12D mutation in their KRAS gene. Before ASP3082 is available as a treatment, the researchers need to understand how it is processed by and acts upon the body. This information will help find a suitable dose and to check for potential medical problems from the treatment. People in this study will be adults with locally advanced, unresectable or metastatic solid tumors with the G12D mutation in their KRAS gene. 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. They may have been previously treated with standard therapies. The main aims of the study are: to check the safety of ASP3082 by itself and together with cetuximab or chemotherapy, and how well it is tolerated, and to find a suitable dose of ASP3082 by itself and together with cetuximab or chemotherapy. This is an open-label study. This means that people in this study and clinic staff will know that they will receive ASP3082. This study will be in 2 parts. In Part 1, different small groups of people will receive lower to higher doses of ASP3082, by itself, or together with cetuximab. Any medical problems will be recorded at each dose. This is done to find suitable doses of ASP3082, by itself or together with cetuximab to use in Part 2 of the study. The first group will receive the lowest dose of ASP3082. A medical expert panel will check the results from this group and decide if the next group can receive a higher dose of ASP3082. The panel will do this for each group until all groups have received ASP3082 (by itself or together with cetuximab) or until suitable doses have been selected for Part 2. In Part 2, other different small groups of people will receive ASP3082 by itself or together with cetuximab or chemotherapy, with the most suitable doses worked out from Part 1. This will help find a more accurate dose of ASP3082 to use in future studies. ASP3082 (cetuximab or chemotherapy if used), will be given through a vein. This is called an infusion. Each treatment cycle is 21 or 28 days long. People will continue treatment until: they have medical problems from the treatment they can't tolerate; their cancer gets worse; they start other cancer treatment; or they ask to stop treatment. At some visits, other checks will include a medical examination, echocardiogram (ECHO) or multigated acquisition (MUGA) scan, blood and urine tests and vital signs. Vital signs include temperature, pulse, breathing rate, and blood pressure. (Blood oxygen levels will also be checked for people treated with ASP3082 together with cetuximab or chemotherapy.) Tumor samples will be taken during certain visits during treatment and when treatment has finished. People will visit the clinic on certain days during their treatment, with extra visits during the first 2 cycles of treatment. The study doctors will check for any medical problems from ASP3082 by itself or together with cetuximab or chemotherapy. At some visits, other checks will include a medical examination, echocardiogram (ECHO) or multigated acquisition (MUGA) scan, blood and urine tests and vital signs. Vital signs include temperature, pulse, breathing rate, and blood pressure. (Blood oxygen levels will also be checked for people treated with ASP3082 together with cetuximab or chemotherapy.) Tumor samples will be taken during certain visits during treatment and when treatment has finished. People will visit the clinic within 7 days after stopping treatment. The study doctors will check for any medical problems from ASP3082 by itself or together with cetuximab or chemotherapy. Other checks will include a medical examination, echocardiogram (ECHO) or multigated acquisition (MUGA) scan, urine and blood tests and vital signs. After this, people will continue to visit the clinic every 9 weeks to check the condition of their cancer. They will do this until 45 weeks after treatment stopped, or if their cancer is worse, they start other cancer treatment, or they ask to stop treatment. Also, people may visit the clinic at 30 days and 90 days after stopping treatment. At the 30-day visit, the study doctors will check for any medical problems from ASP3082 by itself or together with cetuximab or chemotherapy. People will have their vital signs checked and have some blood tests. At the 90-day visit, the study doctors will check for any medical problems from ASP3082 by itself or together with cetuximab or chemotherapy and people will have their vital signs checked.

Type: Interventional

Start Date: Jun 2022

open study

The purpose of this study is to assess the anti-tumor activity of amivantamab as a monotherapy (Cohorts A, B, and C), to characterize the safety of amivantamab when added to standard-of care (SoC) chemotherapy in participants with metastatic colorectal cancer (mCRC) (Ph2 cohorts), and to assess the recommended phase 2 combination dose (RP2CD) of amivantamab when added to SoC chemotherapy (Ph1b cohorts).

Type: Interventional

Start Date: Jul 2022

open study

This pilot trial compares drug exposure levels using a new method for dosing vincristine in infants and young children compared to the standard dosing method based on body surface area (BSA) in older children. Vincristine is an anticancer drug used to a variety of childhood cancers. The doses anticancer drugs in children must be adjusted based on the size of the child because children vary significantly in size (height, weight, and BSA) and ability to metabolize drugs from infancy to adolescence. The dose of most anticancer drugs is adjusted to BSA, which is calculated from a patient's weight and height. However, infants and young children have more severe side effects if the BSA is used to calculate their dose, so new dosing models have to be made to safely give anticancer drugs to the youngest patients. This new method uses a BSA-banded approach to determine the dose. Collecting blood samples before and after a dose of the drug will help researchers determine whether this new vincristine dosing method results in equivalent drug levels in the blood over time in infants and young children compared to older children.

Type: Observational

Start Date: Nov 2022

open study

Clinical study to investigate the efficacy and safety of savolitinib in combination with osimertinib versus platinum-based doublet chemotherapy in participants with EGFR mutated, MET-overexpressed and/or amplified, locally advanced or metastatic NSCLC who have progressed on treatment with Osimertinib.

Type: Interventional

Start Date: Aug 2022

open study

This phase III trial compares the effect of open thoracic surgery (thoracotomy) to thoracoscopic surgery (video-assisted thoracoscopic surgery or VATS) in treating patients with osteosarcoma that has spread to the lung (pulmonary metastases). Open thoracic surgery is a type of surgery done through a single larger incision (like a large cut) that goes between the ribs, opens up the chest, and removes the cancer. Thoracoscopy is a type of chest surgery where the doctor makes several small incisions and uses a small camera to help with removing the cancer. This trial is being done evaluate the two different surgery methods for patients with osteosarcoma that has spread to the lung to find out which is better.

Type: Interventional

Start Date: Apr 2022

open study

Progressive age-related cognitive deficits occurring in both AD and DS have been connected to the degeneration of several neuronal populations, but mechanisms are not fully elucidated. The most consistent neuronal losses throughout the progression of AD are seen in cholinergic neurons where these losses negatively affect cognition, particularly in attention, learning, and memory formation. Evidence of reduced cholinergic integrity in DS is largely limited to animal models and post-mortem human data. The investigators propose to use molecular, functional, and structural biomarkers to assess the cholinergic integrity in adults with DS. The investigators anticipate using the data gathered in this pilot study to inform future study designs to determine AD risk stratification in DS by identifying individuals who show an accelerated decline in cholinergic integrity that correlates with cognitive and neurobehavioral changes. Also, our cholinergic biomarkers may identify whether individuals with DS are likely to respond to pro-cholinergic interventions, including the novel cholinergic modulators that are being developed to enhance cholinergic-sensitive cognitive functioning. The investigators anticipate using the data gathered here to inform future treatment studies in TRC-DS and beyond where novel cholinergic treatments may offer opportunities for early intervention in DS and be complementary to disease-modifying approaches such as anti-amyloid treatments.

Type: Interventional

Start Date: Aug 2021

open study

To demonstrate the safety and effectiveness of the Reducer system for treatment of patients with refractory angina pectoris treated with maximally tolerated guideline-directed medical therapy who demonstrate objective evidence of reversible myocardial ischemia in the distribution of the left coronary artery and who are deemed unsuitable for revascularization. A non-randomized single-arm will further assess the safety and effectiveness of the Neovasc Reducer System in selected subjects with reversible myocardial ischemia in the distribution of the right coronary artery and who are deemed unsuitable for revascularization, subjects with reversible myocardial ischemia without documented obstructive coronary disease and subjects who cannot complete an exercise tolerance test due to an above-the-ankle amputation.

Type: Interventional

Start Date: Jan 2022

open study

The study is intended to assess the safety and efficacy of perioperative treatment with Durvalumab in combination with Oleclumab, Monalizumab, or AZD0171 and platinum doublet chemotherapy (CTX); or Volrustomig or Rilvegostomig in combination with CTX; or Datopotamab deruxtecan (Dato-DXd) in combination with Durvalumab or Rilvegostomig and single agent platinum chemotherapy in participants with resectable, early-stage non-small cell lung cancer.

Type: Interventional

Start Date: Apr 2022

open study

This study will evaluate the efficacy, safety, and pharmacokinetics of tirabrutinib monotherapy in patients with relapsed or refractory PCNSL (Part A), and tirabrutinib in combination with one of two different high dose methotrexate based regimens (methotrexate/ temozolomide/rituximab or rituximab/methotrexate/procarbazine/ vincristine) as first line therapy in patients with newly diagnosed, treatment naïve PCNSL (Part B)

Type: Interventional

Start Date: Dec 2021

open study

This Phase III Trial evaluates whether breast conservation surgery and endocrine therapy results in a non-inferior rate of invasive or non-invasive ipsilateral breast tumor recurrence (IBTR) compared to breast conservation with breast radiation and endocrine therapy.

Type: Interventional

Start Date: Jun 2021

open study