The 12 week study is designed to assess the efficacy and safety of the twice daily ALKS2680, in narcolepsy Type 2. Administered upon wakening and a second time 3-5 hours later, 3 different doses will be compared to a placebo. The effects of ALKS2680 on excessive daytime sleepiness and sleep quality will be examined during overnight and daytime sleep studies.

The 12 week study is designed to assess the efficacy and safety of the twice daily ALKS2680, in narcolepsy Type 1. Administered upon wakening and a second time 5-7 hours later, 2 different doses (6mg and 10mg) will be compared to a placebo. The effects of ALKS2680 on excessive daytime sleepiness and sleep quality will be examined during overnight and daytime sleep studies.

This study is for patients diagnosed with a solid tumor malignancy of any stage and identified as high risk (having a 6-month mortality estimate). The study is evaluating A-PACT (algorithm-Enables Patients Activated in Cancer Care through Teams). The subjects will be randomized (like flipping a count) to determine if they will receive A-PACT with usual care or usual care alone. The primary purpose of this study is to determine if the A-PACT Program reduces unplanned hospital visits and improves communication between patients and their health care team. Participants in this study can expect to be in this study for 12 months or their withdrawal of consent.

A 95-week study comparing the efficacy and safety of a specific dose of a trial product named zenagamtide versus two different doses of semaglutide, administered once weekly via injection along with a reduced-calorie diet and increased physical activity on weight management in participants with obesity.

This study aims to learn how adolescents and young adults who received a liver transplant as children think about their transplant and prepare to manage their healthcare as they get older. Participants will complete questionnaires and may take part in an interview about their experiences, feelings, and healthcare responsibilities. The study will explore how young people view their transplant, how it fits into their sense of self, and how these factors relate to their readiness for adult healthcare. Information from this study will help researchers better understand the needs of young liver transplant recipients and guide the development of future support programs.

This study is for people with advanced head and neck cancer who cannot receive cisplatin. The purpose of this study is to compare treating head and neck cancer with radiation plus cetuximab versus radiation plus chemotherapy (carboplain and paclitaxel). Carboplatin, paclitaxel, and cetuximab are approved by the FDA to treat head and neck cancer.

If you agree to take part, we expect that your participation in this study may last up to 3 years. This includes visits to the clinic after you finish all study treatments. These follow-up visits will take place about every 2 months during the 1st year and about every 4 months thereafter.

Risks may include nausea, vomiting, headache, feeling tired, loss of appetite, and hair loss. Participants may or may not receive a direct benefit from participating in this trial, however, information learned from the trial may help other people in the future.

This study is for patients diagnosed with relapsed or refractory T-cell lymphomas. These include:

Peripheral T‑cell lymphoma, not otherwise specified (PTCL-NOS)

Follicular helper T‑cell lymphoma (TFH lymphoma)

Systemic anaplastic large‑cell lymphoma (sALCL)

The study is testing a drug called Soquelitinib. This drug is investigational, which means it has not been approved by the U.S. Food and Drug Administration (FDA).
The purpose of the study is to see how well soquelitinib works compared with standard treatment chosen by a physician. Standard treatment may include:

Belinostat (BELEODAQ®)

Pralatrexate (FOLOTYN®)

The study may last up to 6 years. Study treatment may last up to 2 years (104 weeks). Study visits will happen based on the treatment schedule for the assigned study drug.About 180 people will be screened for the study. About 150 participants will be randomly assigned to a treatment group.Soquelitinib works by blocking an enzyme called ITK. ITK is part of a signaling pathway that helps certain cancer cells grow. Blocking ITK may slow or stop the growth of cancer T cells.
Soquelitinib has not been approved for use in humans and is considered experimental. It is not known if the drug will work for this cancer. The possible side effects are also not fully known. About 70 patients have received soquelitinib in another clinical trial. In those patients, the drug was generally tolerated.In this study, treatment with soquelitinib will be compared with standard treatments used for this type of cancer.Belinostat is approved in the United States for adults with relapsed or refractory PTCL.Pralatrexate is approved in several countries, including the United States, Canada, Australia, and South Korea, for patients with relapsed or refractory PTCL.
Participants will be placed into treatment groups by randomization. Randomization means assignment to a group by chance, similar to flipping a coin. Each participant has an equal chance of receiving any of the study treatments.

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.

The ATHNdataset Registry is the largest blood disorders real-world data set in the United States and is used to answer important clinical and scientific questions about the specific causes, prevention, treatment, and the social and economic impact of blood disorders.

The registry will involve collecting health information from the medical records, bleeding and treatment records of participants, and completion of questionnaires. This information will be part of the ATHNdataset registry which will be used to answer scientific, public health, and policy questions about better ways to treat blood disorders.

In our preclinical mouse studies we have identified immune dysfunction in mouse models of osteogenesis imperfecta, also known as brittle bone disease. We then successfully restored bone strength in these mice with our patented adoptive cell therapy treatment.

In this study we will collect small volume blood samples from patients with osteogenesis imperfecta. We will then analyze these blood samples through a variety of methods to determine if osteogenesis imperfecta patients have the same immune dysfunction as we have seen in our mice studies.