Treating autoimmune pulmonary alveolar proteinosis (aPAP) patients with molgramostim nebulizer solution focuses on improving how the lungs function and defend against infection. In this condition, the lungs struggle to clear a natural substance called surfactant, which can make breathing harder. A protein in the body called granulocyte macophage colony-stimulating factor (GM‑CSF) helps activate immune cells that keep the lungs clean and healthy. These cells play an important role in removing buildup and protecting against germs. Increasing their activity can help the lungs work more efficiently. It also helps reduce lung damage by boosting the number of these helpful cells. In addition, this protein supports the formation of certain blood cells that are important for overall health. Molgramostim is a man-made version of this natural protein. It is delivered directly into the lungs using a nebulizer, allowing it to work where it is needed most. Research studies have shown that this treatment is generally safe, well tolerated, and can improve symptoms in people with autoimmune PAP.
This is a research study to understand patterns of nicotine pouch use over time among three separate groups of people who: 1) co-use nicotine pouches and cigarettes, 2) co-use nicotine pouches and conventional smokeless tobacco (including chewing tobacco, dip, moist snuff, or snus), or 3) exclusively use nicotine pouches. This is a naturalistic survey study; there is nothing required or requested of participants other than to complete our surveys. Participants do not need to quit or reduce their pouch/smoking/smokeless tobacco product use if they do not want to. They do not need to travel for this study; it is completely remote, and they can participate from their home.
Participation in the study will take place over a period of 12 months in the form of: 1) Monthly Surveys (13 total), 2) Daily Diaries for the first 90 days, and 3) Follow-Up Diaries in each of the 7 days leading up to each monthly follow-up survey.
This study aims to pilot test a virtual mindfulness meditation program for pulmonary hypertension (PH) in preparation for larger testing. Individuals living with PH will be invited to learn mindfulness practices via videos and the MMPH app for 8 weeks.
They will provide feedback via surveys at four time points: before and after the intervention (PAHSS, EmPHass-10, PHQ-8, PCL-5, CAMS-R), and again 1 month and 2 months after. They will be asked to wear a wrist actigraphy device before and after the intervention.
Participants will provide their feedback via an interview about their experience after the intervention is complete.
This study is evaluating a program designed to support adults recovering from a traumatic injury, such as a motor vehicle crash, fall, or other serious injury. Recovery after an injury can be challenging, particularly for people who are experiencing emotional distress as well as difficulties with needs such as housing, food, transportation, or finances. The program, called TRRP+, combines mental health support with assistance from a Community Health Worker who can help participants identify needs, connect with available resources, and navigate services during their recovery. The study will include approximately 100 adults who have experienced a traumatic injury and report emotional distress and at least one unmet social need.
Researchers will examine whether TRRP+ is practical and helpful for patients and will gather feedback from participants, Community Health Workers, and trauma center staff about their experiences with the program. The information learned from this study will help researchers understand how programs like TRRP+ can better support patients' emotional, social, and practical needs as they recover from a traumatic injury.
The purpose of this research study is to develop a multiple health behavior change intervention for weight loss and smoking cessation using medication (Naltrexone/Bupropion) and behavior therapy for adults considering bariatric surgery. All participants will receive the intervention (medication + behavior therapy) for four months. Participants will complete an interview at the end of treatment, which will be used to improve the intervention.
A research study at the Medical University of South Carolina is looking at young adults' experiences with alcohol and e-cigarette use during a 4-week study. The study is enrolling adults ages 21 to 29 who use flavored ready-to-drink alcoholic beverages and e-cigarettes. Participants will complete in-person study visits, brief daily electronic diaries, questionnaires, and provide blood and urine samples for research testing. Some participants will be asked to avoid flavored ready-to-drink alcoholic beverages for 2 weeks, while others will continue their usual use. The purpose of the study is to learn whether this type of short-term study procedure is feasible and acceptable, and to better understand patterns of alcohol and e-cigarette use among young adults.
Smell loss has been found to be associated with mortality and neurodegenerative diseases. Unfortunately, there are no current biomarkers for olfactory loss and no specific mechanism to explain exactly how this association occurs. This study aims to uncover more about smell loss and its association with neurodegenerative disease such as Parkinson's disease, Alzheimer's disease, Multiple Sclerosis, and Mild Cognitive Impairment in the early/mild stages of those conditions. Study procedures involve questionnaires, a 40-item scratch and sniff smell test, assessment of cognitive function, blood sample, and nasal mucus and cell samples collected by a physician.
The 16-week main phase of the study is designed to assess the efficacy and safety of the once daily pitolisant, administered orally upon wakening, in comparison to a placebo. This study includes overnight sleep studies. The effects of HBS-301 on excessive daytime sleepiness, fatigue, and cataplexy (NT1 population only) will be examined.
After the main phase period, there is an option to be in the second part of the study, which will span 1 year. During this period, subjects will receive open label of the active drug, HBS-301.
This study is recruiting pediatric patients who have been diagnosed with Favorable Histology Wilms Tumor (FHWT). Wilms Tumor is the most common type of kidney cancer in children, and FHWT is the most common subtype. The goal of the study to improve outcomes within cohorts of FHWT patients with current long-term EFS rates at or below 80% and maintain outcomes despite therapy reduction within cohorts of FHWT patients with current long-term EFS rates at or above 84%. Common side effects of treatment in regards to this study include nausea, vomiting, hair loss, and fatigue (tiredness). Participants will receive the medications through an IV in their arm and orally. Study participation is expected to be approximately 5 years.
This is a randomized, double-blind, placebo-controlled, multicenter, parallel-group
study of the efficacy and safety of 2 weight-based treatment groups of plecanatide
versus placebo in children and adolescent participants 6 to less than 18 years of age with FC. Randomization will be stratified by sex to ensure balance across treatment groups.
Approximately 30 participants will be randomized to each of the Placebo, Low
Dose, and High Dose groups, within age group. Low Dose and High Dose will be
assigned once daily treatments based on the weight of the participant at
randomization to achieve weight-based dosing of 0.025 to 0.05 mg/kg in the Low
Dose treatment group and 0.05 to 0.1 mg/kg in the High Dose treatment group.