Epidermolysis Bullosa (EB) is a rare, inherited skin condition that makes the skin extremely fragile, causing painful blisters and wounds from even minor friction or injury. There is currently no cure, and because EB is uncommon, doctors still have limited high-quality data to guide the best treatment and long-term care. This study is part of a large North American effort to collect and organize health information from people with EB into a secure database. By tracking how the disease progresses over time, along with symptoms, complications, and treatments, researchers hope to better understand EB and improve care for future patients. Participation involves consenting to share medical record information and optionally completing brief questionnaires during routine clinic visits or by email. No experimental treatments or extra medical procedures are involved. While there is no direct benefit to participants, the knowledge gained may help improve care and support the development of new treatments in the future.
The purpose of this clinical research study is to learn more about the use of an investigational medicine, called brepocitinib, for the treatment of Lichen Planopilaris (LPP). The study will also look at how safe and effective brepocitinib is and will monitor the long-term safety of brepocitinib when taken for a period up to 52 weeks.
This research study will evaluate the long-term safety and effectiveness of APG777 in patients with moderate-to-severe atopic dermatitis (AD) who have already completed treatment in a previous APG777 study. The study is multicenter and double-blind, and participants will continue with the same dose and injection schedule as in their prior study. The study includes three periods: a screening visit, an extended treatment period of about 92 weeks, and a post-treatment follow-up of up to 52 weeks. Patients who met certain improvement criteria in the previous study will continue their maintenance regimen, while others will enter an open-label Escape Arm with a different dosing schedule. The study aims to determine whether long-term use of APG777 is safe and effective for patients who may benefit from continued treatment.
This study is for participants that have been diagnosed with Intermediate or High-risk Primary or Secondary Myelofibrosis. This study is testing an investigational drug called Nuvisertib. "Investigational" means it has not been approved by the United States Food and Drug Administration (FDA). The main purpose of this study is to evaluate the efficacy and safety of Nuvisertib. Nuvisertib is an oral PIM1 selective inhibitor. A PIM1 selective inhibitor is a drug that specifically targets and blocks the activity of the PIM-1 kinase, an enzyme implicated in cancer cell growth. This drug is given to participants by mouth. Participants in this study can expect to be in the treatment phase of this study for 19 months and the long term follow up phase for 3 years.
This study will continue using the study drug levosiminden to determine if it can help people with the heart failure with preserved ejection fraction with pulmonary hypertension (HFpEF-PH). Levosimenden is an investigational drug. Investigational means that this drug is not yet approved for use in any settings outside of clinical research studies like this one. This drug is thought to assist in helping with decreasing your heart failure symptoms and increase your quality of life.
Participation in this study will last up to 60 month and will consist of about 13 clinic visits. Some tests required include physical exams, and dispensing of study medication, bloodwork.
This study is for patients that have been diagnosed with diagnosed with primary advanced or recurrent endometrial cancers that express high levels of the human epidermal growth factor receptor 2 (HER2) protein and that have a genetic characteristic known as mismatch repair proficiency (pMMR). The study is testing investigational drugs called Trastuzumab Deruxtecan (T-DXd) and Rilvegostomig. "Investigational" means it has not been approved by the United States Food and Drug Administration (FDA). The purposed of this study is to learn more about whether T-DXd with Rilvegostomig or Pembrolizumab works better and is safe for the treatment of primary advanced or recurrent endometrial cancers that express the HER2 protein in high levels and that have a genetic characteristic known as mismatch repair proficiency (pMMR), when compared to chemotherapy. The study drugs are given by infusion. Participants in this study can expect to be in this study for as long as you are receiving benefit from the treatment.
This observational, multi-country cohort study examines the long-term safety of Filsuvez in real-world clinical practice. Researchers will collect both primary data and use existing patient registry data to gather information on Filsuvez exposure, skin malignancies, medical history, and other clinical characteristics. The study will include patients with confirmed diagnoses of DEB and JEB, regardless of whether they use Filsuvez, as long as they meet the approved indication. Patients previously exposed to Filsuvez through clinical trials, early access programs, or compassionate use programs may also participate. The study does not require any protocol-mandated visits or procedures, and the frequency of patient visits will follow local standard practice and individual patient needs.
This is a Phase 3, open-label, follow-up study to evaluate the long-term safety,
tolerability, and efficacy of sotatercept when added to background PAH therapy for the treatment of PAH. Participants enrolled in this study are from the following parent studies: PULSAR, SPECTRA, STELLAR, HYPERION, and ZENITH. Participants eligible to enroll in this study will have participated in and completed the requirements of the MK-7962-004 study (Amendment 07 or later). In this study, participants will receive open-label sotatercept . The duration of participation for each participant will vary based on their enrollment date, but it is anticipated to be up to 3 years.
The main reason for this research study is for researchers to evaluate the relationship between congenital heart disease and development. Currently, there is not enough long-term information available to researchers to predict a child's development if they have been diagnosed with Ductal Dependent Pulmonary Blood Flow (DDPBF), a type of congenital heart disease.
This is an open-label, long-term study assessing the safety and efficacy of abrocitinib in participants aged 2 years and older with moderate-to-severe AD. It includes an extension cohort and a de novo cohort to meet regulatory requirements for a minimum of 300 participants exposed to 52 weeks of abrocitinib. Extension cohort participants must have completed 16 weeks of treatment in parent studies B7451023 or B7451030 and remain eligible. De novo cohort participants must be aged 6 to under 12 years at enrollment and not have participated in previous abrocitinib studies. Enrollment for the de novo cohort will begin after enrollment in Study B7451023 is complete. The study will have two periods lasting up to 2 years or until commercial availability, whichever comes first. All participants will receive abrocitinib oral suspension.