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.

The BRIDGES Clinical Trial (GTM-105) is a phase 3 clinical trial for people who have been newly diagnosed with a glioblastoma (GBM) brain tumor and need surgery. The purpose of this study is to find out if adding GammaTile® during surgical removal of a brain tumor improves survival outcomes compared to standard of care treatment. GammaTile is an FDA-cleared, implantable radiation therapy device placed directly in the surgical cavity at the time of tumor removal surgery. Each GammaTile is made of a 2 cm × 2 cm collagen square that contains four radioactive sources (about the size of a grain of rice). Once implanted, GammaTile begins delivering radiation immediately, right where the tumor was removed. Participants will be randomized into one of two groups (like flipping a coin), with 2 participants being randomized to receive GammaTile for every 1 participant receiving standard care. Participants randomized into the standard care arm will undergo tumor removal surgery followed by 6 weeks of external beam radiation therapy (EBRT) and oral chemotherapy (temozolomide, TMZ), followed by additional cycles of TMZ. Participants in the GammaTile arm will have the GammaTile placed into their surgical site, and also receive all the standard care treatments. However, the radiation course will be four weeks instead of six weeks, because GammaTile will deliver part of the radiation dose. Participants can expect to be enrolled in this trial for up to three years. The most common side effects from the procedures in this trial include radiation brain injury, surgical complications, infections and fatigue.

Both external radiation and GT are FDA cleared for the treatment of malignant (cancerous) brain tumors and available to patients. The experimental part of the study is comparing standard of care to standard of care plus GT for this type of brain tumor.

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.

The purpose of this study is to determine if a drug called adalimumab can reduce or prevent emergence or progression of synuclein-related neurodegeneration, or nerve cell death, in people diagnosed with REM Sleep Behavior Disorder (RBD.) About 108 individuals, aged 50-80, will be enrolled in this study, half of whom will receive the active study drug and half of whom will receive placebo. Participants would receive 40 mg adalimumab, administered subcutaneously every 2 weeks or matching placebo for up to 2 years (96 weeks).

Epidermolysis Bullosa is a genetic disorder manifesting typically at birth comprising several genetic subtypes that manifest as blistering or erosion of the skin in response to little or no apparent trauma. This clinical study is being conducted for research purposes. The cream being studied has allantoin as the active ingredient and the aim of this study is to assess the long-term safety of this cream when applied to the skin of participants with Epidermolysis Bullosa. This study is an open label extension to study SD-007. The study will be conducted by the study doctor, in other words, the physician responsible for the clinical study at your child's study site. Your child's participation in the clinical study is expected to last for approximately 12 months and approximately eighty (80) children will participate in this study. There will be a total of five (5) study visits over the course of one year. Study visits 1, 3 and 5 will be in person and visits 2 and 4 will be telephone visits.

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 study will look at whether a topical medication called minocycline foam (AMZEEQ) can help treat confluent and reticulated papillomatosis (CARP), a skin condition that causes dark, scaly patches. We will also evaluate how safe the treatment is and how satisfied patients are with it. The study is open to participants ages 9 years and older.

Participants will apply minocycline foam to one side of the affected skin and a moisturizing cream (emollient) to the other side for 5 weeks. This allows us to compare the two treatments in the same person. After this period, participants may choose to continue using the minocycline foam on all affected areas.

During the study, participants will attend follow-up visits where photos of the skin will be taken and the condition of the skin will be evaluated. Participants will also complete brief surveys about how their skin condition affects their quality of life and how satisfied they are with the treatment. The research team will monitor treatment use and any side effects.

The goal of this study is to learn whether topical minocycline could be a safe and effective treatment option for CARP, potentially providing an alternative to oral antibiotics.

This study aims to improve our understanding of Cystic Fibrosis Transmembrane Conductance Regulator(CFTR)-related metabolic syndrome(CRMS), a term used for some infants whose newborn screening results suggest they may be at risk for cystic fibrosis but who do not currently meet the criteria for a cystic fibrosis diagnosis. CFTR stands for cystic fibrosis transmembrane conductance regulator and refers to a gene that provides instructions for making a protein that helps control the movement of salt and water in and out of cells. Changes in this gene can increase a person's risk of developing cystic fibrosis or related health concerns.

The study has two goals. First, we will review existing medical records to better understand the health conditions, symptoms, and long-term health of children with CRMS and adults with cystic fibrosis diagnosed later in life who receive care at the Medical University of South Carolina pulmonary clinic. Second, we will survey parents of children with CRMS to better understand how receiving a CRMS designation affects families emotionally and in their daily lives.