Search Results

The goal of this study is to provide comprehensive longitudinal assessments of a cohort of PD patients before, during, and after DBS surgery, including neurological, neurophysiological, and neuropsychological data.

This is a clinical research study exploring the safety and tolerability of a single dose of VX-522 for people with cystic fibrosis (CF) who are not expected to benefit from CFTR modulators.

AML is a type of blood cancer where infection fighting cells called white blood cells (WBCs) don’t grow up or “mature” like they are supposed to. Instead, they stay stuck as infant or immature “blast” cells. This study will investigate the potential risks and benefits of adding a targeted therapy called ziftomenib to intensive therapy (7+3) OR non-intensive (Venetoclax + Azacitidine) for patients whose cancers are found to have KMT2A rearrangements or mutations in the NPM1 gene. We will also see if the use of ziftomenib as maintenance therapy following consolidation is beneficial.

To determine if the addition of an ultrasound guided left sided stellate ganglion block with bupivacaine in patients undergoing esophagectomy, pneumonectomy, or lobectomy will result in lower rates of postoperative atrial fibrillation as compared to standard of care.

The purpose of this study to examine the effects of cigarette smoking and drinking alcohol on the formation of DNA damage in the mouth. The overall goal is to identify DNA damage that may be important to the development of head and neck cancers.

This study is being done to find out if vagus nerve stimulation (VNS) affects how different parts of the brain interact with each other and process information. Participants must be in the REVEAL study and have a new VNS device implanted for treatment of depression. The study will last for about 19 weeks after the VNS is implanted.

The purpose of this study is to evaluate the long-term safety and ability of a transplant with gene modified stem cells (autologous stem cell transplant) to treat sickle cell disease. Participants must have received investigational gene therapy with bb1111 in a clinical study sponsored by bluebird bio. There is no additional treatment associated with this study as this is a long-term follow-up study.

We are comparing treatment of early stage, hormone positive breast cancer with and without radiation therapy. One group will receive radiation therapy with endocrine therapy and the other group will receive endocrine therapy alone. We want to find out if there is any difference in how often breast cancer recurs in the same breast.

The purpose of this research is to evaluate the safety, efficacy, and pharmacokinetics of a new investigational drug called CRN04894, also called atumelnant for treating pediatric participants with Congenital Adrenal Hyperplasia (CAH). We want to see how safe atumelnant is at different doses and how well the body accepts (tolerates) it and how it moves through the body (how it gets in, spreads around, gets used, and then leaves), also known as pharmacokinetics (PK). We also want to see if if atumelnant produces the expected effect in the body, if it helps control the CAH, and if we can reduce the steroid (GC) dose.

To determine the prevalence, type, and severity of chronic health conditions (CHC) in survivors of Down syndrome-associated acute leukemia (DS-AL), and to compare CHC with frequency-matched DS individuals that have no cancer history.

The treatment (chemotherapy and transplant procedures) is considered standard clinical care that are usually given to the patients with this disease. The research aspect of this study is to collect data on the patients who are being treated on this plan. Patients will be followed throughout the course of their clinical care and for three years after their transplant.

To determine the prognostic factors for visual outcome for newly diagnosed NF1-OPGs. Hypothesis: Patients (<18 years of age) with tumors involving the optic tracts and/or radiations will demonstrate worse visual outcomes compared to those without optic tract involvement.

The purpose of the study is to assess if the study treatment, pelacarsen, taken by people with mild or moderate CAVS and elevated lipoprotein(a) can safely help slow the progression of calcific aortic valve stenosis (CAVS). Pelacarsen is a treatment being tested that acts on a particle called Lp(a), which if elevated, may play a role in CAVS.

The research aspect of this trial is the use of a new machine to remove specific lymphocytes from the donor’s peripheral blood stem cells (PBSCs). This is called T cell receptor alpha/beta T cell depletion. This machine does such a good job at removing the unwanted donor T cells, and as a result we think patients will need fewer drugs to suppress their immune system.

The purpose of this study is to determine if replacing the mitral valve without open-heart surgery is as safe and effective as standard mitral valve surgery in patients with similar medical conditions. This system allows a bioprosthetic mitral valve (investigational valve) to be implanted through a long, thin, flexible tube that is inserted through an incision in the side of the chest or through an incision made in the groin area and through a vein in the leg. Participation in the study is expected to last approximately 5 years from the day the valve is implanted.

The purpose of this research is to test how well the investigational study drug, quizartinib, works in patients with AML without a FLT3-ITD mutation who have not received any prior treatment for the disease. The study will test how it works when taken with standard chemotherapy and then taken alone to prevent the disease from coming back.

We want to find out whether lifileucel is safe and works in treating untreated, unresectable or metastatic melanoma. Lifileucel is a type of medicine, known as immunotherapy, that uses your body’s immune system to fight cancer. Lifileucel is also called “tumor infiltrating lymphocytes” (TIL) and is made up of specialized white blood cells known as lymphocytes or “T cells” obtained from a piece of your tumor. T cells are a part of your immune system that help your body fight against infections and diseases including fight cancer.

This research study is testing a potential new treatment for pulmonary hypertension (high lung blood pressure due to heart failure) called TX000045 (study drug). Participants will be assigned to a study group and receive active study drug or a placebo (drug with no active ingredients). The study will last approximately 9 months

The Cordella™ Pulmonary Artery Sensor System is a possible treatment for New York Heart Association (NYHA) Class II and III heart failure. The main purpose of this study is to investigate the safety and effectiveness of the study device in helping to reduce Heart Failure hospitalizations.

In this study, we are going to compare two ways of treating mild respiratory illnesses in children with Cystic Fibrosis (CF). One is immediate antibiotics (increase airway clearance and start oral antibiotics right away) and the other is tailored therapy (increase airway clearance right away but only start oral antibiotics if symptoms worsen or do not improve). We are looking at how safe each option is and how well each one works. To do this, we will randomize participants (like flipping a coin) to one of these two approaches if a mild respiratory illness happens over the next 12 months.

The purpose of this study is to learn if removing the donor T cells from the donor product using this new method will be a better way to reduce the risk of GVHD. The benefit of removing these cells with this new method is that they will prevent GVHD without requiring drugs to suppress the immune system. Potentially, the immune system will recover from the transplant faster, which in turn will also lessen the risk of severe infections. As well, the patient will not have the other common undesired side effects of these immunosuppressive drugs.

This research study is about early development in children diagnosed with congenital cytomegalovirus infection (cCMV), identified by newborn screening that has been confirmed with a follow-up test. Study visits, when the child is 12, 24 and 36 months of age, will include questionnaires, direct assessment of child and interviews with parent(s). A brain MRI will also be completed at about 12 months. Parents will receive a report summarizing a child’s developmental, language, and behavioral assessments, reported as within normal ranges or if clinical follow-up is recommended.

The purpose of this research is to measure the safety and effectiveness of ivonescimab compared to pembrolizumab. Ivonescimab is an antibody designed to block proteins that help cancer cells grow and spread, and by blocking these proteins may potentially slow cancer progression. Participants will have a 50% chance of being assigned to either the ivonescimab treatment group or the pembrolizumab treatment group.

We are doing this study because we want to find out if observation is as good as the usual care for breast cancer. The usual approach for patients with early-stage triple-negative breast cancer (TNBC) who receive preoperative chemotherapy plus pembrolizumab is to continue to receive FDA-approved pembrolizumab for up to 27 weeks after surgery. Participants will either get pembrolizumab for up to 27 weeks, or will not receive any treatment and will be observed for up to 27 weeks. We will continue to follow participants every 6 months for 5 years and watch for side effects or cancer coming back. After that, participants will be checked every year for a total of 10 years after the study.

A clinical research study for primary focal segmental glomerulosclerosis (FSGS), or genetic focal segmental glomerulosclerosis (FSGS), or focal segmental glomerulosclerosis (FSGS) of undetermined cause in pediatric (12-17 years) and adult patients. Eligible participants will be assigned to receive either DMX-200 (repagermanium) or placebo (50/50 chance) over a treatment period, with total participation up to 28 month, with potential for participation in an Open Label Extension study period. The main purpose of this study is to see if DMX-200 reduces proteinuria and slows the loss of kidney function in those with FSGS.

To evaluate the safety of the EN-374 treatment regimen (HSC mobilization, immune prophylaxis, EN-374 dose and administration, and enrichment of HSCs with O6BG/TMZ).

The rate of lung cancer screening is not optimal among Chinese American high-risk smokers, although there is a high lung cancer rate among this population. We want to validate the adapted Chinese Lung Cancer Screening Health Belief Scale, enable its usage in Chinese Americans, and further explore the relationship between lung cancer screening health beliefs and health behavior among Chinese Americans. We will also aim to identify the differences in health beliefs regarding lung cancer screening between high-risk populations in China and Chinese Americans, and to examine factors influencing these differences.

We are doing this study to compare the effectiveness of Single Fraction Stereotactic Radiosurgery (SRS) to radiation treatment called fractionated SRS (or FSRS). SRS delivers a high dose of radiation in a single treatment to the small areas of cancer in the brain. FSRS that delivers a high dose of radiation given over 3 treatments. Both SRS and FSRS only deliver radiation to the small areas of cancer in the brain and minimizes dose to the surrounding normal brain tissue.

Ritlecitinib (LITFULO™, PF-06651600) 50 mg daily is currently approved for people 12 years of age and older to treat severe alopecia areata (hair loss). Ritlecitinib 100 mg will be compared to Ritlecitinib 50 mg to find out if a larger daily dose is safe and can improve regrowth of hair. The study will last about 57 weeks and will have up to 9 visits. Everyone will get one of the two doses of the study drug.

The goal of this study is to find out whether combining different study drugs makes tumors shrink in people with advanced head and neck cancer. This type of cancer is in the mouth, nose, throat, and sinuses. The study drugs are antibodies that act in different ways against cancer. BI 770371 and pembrolizumab may help the immune system fight cancer. Cetuximab blocks growth signals and may prevent the tumor from growing. Participants are put into 1 out of 3 groups, by chance (like drawing names from a hat), and which of the study treatments participants will receive.