North Carolina Paid Clinical Trials

Primary Objective: To describe the effect of routine practice with alglucosidase alfa in patients with IOPD ≤6 months of age, on invasive ventilation-free survival after 52 weeks of treatment. Secondary Objectives: * To describe the effect of routine practice with alglucosidase alfa on invasive ventilation-free survival and survival at 12 and 18 months of age, as well as on change in left ventricular mass (LVM) Z score, Alberta Infant Motor Scale (AIMS) score, body weight, body length, and head circumference Z scores, and urinary glucose tetrasaccharide (Hex4), at Week 52 of treatment. * To describe the safety, tolerability, and immunogenicity of alglucosidase alfa in the routine practice of IOPD treatment. Read Less

The investigators aim to ascertain the effects of hiatal hernia repair and fundoplication on the distensibility of the esophagogastric junction (EGJ) as measured by FLIP topography/impedance planimetry. The investigators also aim to assess for any correlation between values of EGJ distensibility and GERD related quality of life (QOL) and dysphagia scores. Read Less

This is a multicenter, open-label, Phase 1/2a dose escalation and expansion study of orally administered emavusertib (CA-4948) monotherapy in adult patients with AML or higher- risk Myelodysplastic Syndrome (hrMDS). Patients enrolling in the Phase 1 dose escalation of the study must meet one of the following criteria prior to consenting to the study: * Relapse/refractory (R/R) AML with FMS-like tyrosine kinase-3 (FLT3) mutations who have been previously treated with a FLT3 inhibitor * R/R AML with spliceosome mutations of splicing factor 3B subunit 1 (SF3B1) or U2AF1 * R/R hrMDS with spliceosome mutations of SF3B1 or U2 small nuclear RNA auxiliary factor 1 (U2AF1) * Number of pretreatments: 1 or 2 The Phase 2a Dose Expansion will be in 3 Cohorts of patients: 1. R/R AML with FLT3 mutations who have been previously treated with a FLT3 inhibitor; 2. R/R AML with spliceosome mutations of SF3B1 or U2AF1; and 3. R/R hrMDS (Revised International Prognostic Scoring System \[IPSS-R\] score \> 3.5) with spliceosome mutations of SF3B1 or U2AF1. All patients above have had ≤ 2 lines of prior systemic anticancer treatment. In previous versions of this protocol there was a Phase 1b portion of the study, in which patients with AML or hrMDS received CA-4948 in combination with venetoclax. This part of the study is no longer open for enrollment. Read Less

The research question is: What is the risk of adverse pregnancy outcomes, including preterm birth, hypertensive disorders, and other maternal and neonatal/infant outcomes, following exposure to RSV vaccine between 32 weeks, 0 days and 36 weeks, 6 days of gestation in the CorEvitas Respiratory Syncytial Virus Vaccine Pregnancy Registry (RSV-PR)? The primary study objective is to estimate the risk of (1) preterm birth and (2) hypertensive disorders following exposure to RSV vaccine between 32 weeks, 0 days and 36 weeks, 6 days of gestation. The secondary study objective is to estimate the risk of other safety outcomes of interest following exposure to RSV vaccine between 32 weeks, 0 days and 36 weeks, 6 days of gestation, including: * Pregnancy-related outcomes: stillbirth, premature labor, premature rupture of membranes, preterm premature rupture of membranes, cesarean delivery, prolonged maternal duration of hospital stay * Maternal outcomes: thrombocytopenia, Guillain-Barré syndrome, other immune-mediated demyelinating conditions, polyneuropathies, atrial fibrillation, maternal death * Neonatal/infant outcomes: small for gestational age, large for gestational age, low birth weight, admission to a neonatal intensive care unit (NICU), NICU duration of stay, mechanical ventilation in neonatal period, neonatal death, postnatal growth at 1 year of age The exploratory study objective is to describe the most frequently reported maternal adverse events following exposure to RSV vaccine between 32 weeks, 0 days and 36 weeks, 6 days of gestation. Read Less

In this Phase I trial for subjects with advanced head \& neck cancer, breast cancer, soft tissue sarcoma or melanoma all subjects will receive open label X-PACT treatment as a intra-tumoral injection. The primary objective will be to establish the safety of X-PACT when dosed with 5 intra-tumoral injections of the combination product (the phosphor device and methoxsalen sterile solution and subsequently exposing the tumor to X-ray energy) over a period of 6 weeks (on day D1, D3 and D5 of Week 1, on D1 of Week 2, and a booster on D1 of Week 6). After the week 8 tumor assessment subjects demonstrating stable disease, partial response or unconfirmed progression assessed by iRecist, will be eligible to receive two additional booster treatments 4-6 weeks apart. Treatment will be considered safe provided ≤ 2 out of 12 patients experience a dose-limiting toxicity (DLT) during the 6 weeks after the first intra-tumoral injection. Read Less

It has been shown that restoration of the normal makeup of the bowel bacterial population is the most effective way to treat recurrent colitis due to Clostridium difficile. Restoration of the normal bowel bacterial population is best done by transplanting stool from a healthy donor. The investigators wish to transplant stool from healthy donors to treat recurrent C. difficile colitis by incorporating the stool into capsules that are administered by the oral route. Read Less

This clinical trial aims to study if a drug called anifrolumab works to treat Hidradenitis Suppurativa (HS) as well as its effect in quality of life before and after treatment. Anifrolumab is a monoclonal antibody that inhibits several processes that have been shown to be involved in the development of HS. The study lasts approximately 40 weeks separated into a screening, treatment, and follow-up phase. Researchers determine if it is safe for the you to receive the drug and if you are eligible for the study during Screening. If eligible for the study, the treatment phase lasts 24 weeks (or six months) with one follow-up visit 12 weeks after the last visit in the treatment phase. During the treatment phase, participants will be asked to come to clinic every two weeks for the first month of treatment, and monthly thereafter for a total of eight treatment visits. Participants will be asked to: * Complete questionnaires asking about the effect of HS in their daily lives and their perception of HS and treatment received. * Receive related medical evaluation * Receive the study drug intravenously * Stay 20 minutes after the infusion for monitoring Read Less

The primary objective of this study is to determine the ex-vivo prognostic accuracy of the Cybrid live tumor diagnostic platform across a basket of solid tumors, using in-vivo RECIST 1.1 as the reference method. Read Less

The goal of this clinical trial is to test how well psilocybin-assisted therapy works in treating people with depression. The main questions this study aims to answer are: * Does psilocybin with assisted therapy help improve symptoms for people with depression? * How long do the effects of this treatment last? Participants will: * Take part in a couple of screening and preparation visits. * Be given psilocybin in one or two treatment sessions. * Attend a series of follow-up sessions over the following year. * Complete forms and surveys to test how their symptoms have changed and what they thought of their experience. Researchers will also compare whether one treatment or two treatments help improve symptoms more for participants. Read Less

The hypothesis for this study is that a preparative regimen that maximizes host immunosuppression without myeloablation will be well tolerated and sufficient for engraftment of donor hematopoietic cells. It is also to determine major toxicities from these conditioning regimens, within the first 100 days after transplantation. Read Less

In the United States, around 19% of reproductive-aged couples suffer from infertility. The psychological distress associated with infertility is well-established; those diagnosed commonly experience depression and anxiety symptoms, diminished quality of life, and relationship dissatisfaction. In the current study, the investigators report on a case series, in which up to 10 infertility patients will receive 12 sessions of Acceptance and Commitment Therapy (ACT). The overarching goal for the current case series is to determine whether ACT might be well-suited for infertility patients experiencing distress. The specific aims are: 1) to describe the components of the intervention that was delivered; 2) to determine the extent to which infertility patients perceive a 12-session ACT intervention beneficial and acceptable; 3) to describe change in mood, stress, and ACT processes across 12 sessions of ACT; and 4) to highlight potential processes through which ACT might promote benefits to the patient by describing how change in mood, stress, and ACT processes relate to each other over the course of 12 sessions of ACT. Read Less

Nitric Oxide (NO) is an important molecule that is produced naturally in the body and that helps maintain healthy blood flow. Low availability of NO contributes to many diseases while administration of NO is therapeutic. In addition to being made naturally in the body, NO can be obtained through the diet via the Nitrate-Nitrite-NO cycle. Nitrate, which is abundant in green leafy vegetables and beetroot juice, is partially converted to nitrite by oral bacteria. The nitrate and nitrite are taken up into the blood and nitrite is converted into NO. Remaining nitrate in the blood is taken back up into the mouth by salivary glands and the cycle continues. Emerging studies suggest that the Nitrate-Nitrite-NO cycle may contribute to cardiovascular health. In addition, there have been many studies where dietary nitrate is given to increase NO and treat various conditions. The current study rests on the premise that the quality of the oral microbiome plays a major role in the Nitrate-Nitrite-NO cycle and hence cardiovascular health and the efficacy of dietary nitrate interventions. Investigators have begun to identify oral bacterial species that are effective nitrite producers as well as though that are nitrite depleters (those that interfere with nitrite production from nitrate). In laboratory experiments, certain bacterial species have been shown to block nitrate to nitrite conversion by other oral bacteria. These nitrite depleting species are found in a commercially available oral probiotic designed to improve oral health. The purpose of this study is to examine if use of the probiotic negatively affects the Nitrate-Nitrite-NO cycle. Nitrate to Nitrite conversion will be assessed by measuring plasma levels of nitrite before and after consumption of nitrate-rich beetroot juice. Dietary nitrate to plasma nitrite conversion will assessed at baseline and after one week of consumption of the probiotic or a placebo (follow-up). The primary hypothesis of this study is that participants that consume the probiotic will have lower nitrate to nitrite conversion at follow-up compared to baseline and that there will be no significant change in nitrate to nitrite conversion between baseline and follow-up for participants who consume the placebo. While this study does not aim to treat any specific disease, it is intended to elucidate a basic physiological function that may be relevant to cardiovascular health and certain NO-based therapeutics. Read Less