This is a prospective, longitudinal, multicenter, nonrandomized observational study to obtain research quality data across key outcome measures in people with Cystic Fibrosis who are ineligible and/or not taking a approved CFTR modulator and who are not receiving an investigational therapy.
Participants will be seen at study sites for research visits to include spirometry, patient reported outcomes (PROs), and blood collections on Day 0, Day 90 (3 months), Day 180 (6 months), and Day 360 (12 months). Participants will complete home spirometry as well during this period.
This study is being conducted at approximately 150 research centers worldwide and is expected to enroll approximately 675 pediatric subjects in total with moderately to severely AD. This study will have 2 cohorts, a Randomized Cohort, and a Dupilumab-Inadequate Responder / Dupilumab Medically Inadvisable Cohort. The study comprises a 35-day Screening Period; a 16-week, open-label, efficacy assessor blinded study treatment period for the subjects in the randomized cohort; an open-label period up to Week 160 for subjects in the upadacitinib study treatment arms across both cohorts (Randomized Cohort and Dupi-IR/Dupilumab Medically Inadvisable Cohort); an open label period up to Week 52 for subjects in the dupilumab arm; and a 30-day Follow up Visit/call after the last dose is administered for upadacitinib or dupilumab.
Tobacco-free oral nicotine pouches (such as Zyn brand) are a less harmful alternative to combustible tobacco (cigarette/cigarillo/little cigar) smoking. Pouches, however, contain nicotine, and addictive substance that is not risk-free. The present study is evaluating how well nicotine pouches, at different nicotine levels, help people switch away from smoking combustible tobacco. People who smoke will be asked to answer questions about their tobacco product use and provide breath samples and cheek swab samples at an in-person visit to MUSC Charleston. Participants will then be provided with a 28-day supply of nicotine pouches, and will be asked to switch from smoking to pouches over the course of 4 weeks. Finally, participants will complete a final visit at MUSC, and will answer more questions about their tobacco use 1-month later.
The purpose of this research is to gather information about the effectiveness and safety of Left Atrial Appendage Occlusion (LAAO) device procedures in patients using a Watchman device performed on days where doctors perform a large number of procedures.
The study primary outcome is to evaluate complications during the procedure up to 30 days after the procedure.
The final outcome is to determine successful placement within 31 - 90 days after implant.
This study is for subjects that have been diagnosed with mantle cell lymphoma that has spread and has not responded to treatment. This study is testing an "investigational" (not yet FDA approved) study drug called glofitamab. The purpose of this study is to compare the effects, good or bad, of glofitamab (experimental arm) versus bendamustine plus rituximab (BR) or rituximab plus lenalidomide (R-Len;the control arm) on subjects with relapsed/refractory mantle cell lymphoma. Your total time in the study and the number of assessments in the follow up visits, will depend on how your MCL responds to study treatment. This could range from 1 day to more than 24 months. The screening period may last up to 28 days (4 weeks) and may involve more than one visit to the clinic.
In this pilot study we will assemble a portable TMS(transcranial magnetic stimulation) unit, in a van, and then test out delivering TMS in Medical University of South Carolina (MUSC)-affiliated facilities in South Carolina, all within 90 minutes of driving from Charleston, SC. This work will hopefully open up access to TMS for millions of patients with treatment-resistant depression who cannot be treated in the current model.
We will recruit up to 30 treatment-resistant depression patients who live near one of the MUSC-affiliated clinics. We will treat these patients, open-label, with FDA-approved accelerated TMS (6 sessions each day, over 2 hours, for 5 days, spread over one or two weeks). We will measure TMS effectiveness using standard depression rating scales.
In this pilot study we will assemble a portable TMS(transcranial magnetic stimulation) unit, in a van, and then test out delivering TMS in Medical University of South Carolina (MUSC)-affiliated facilities in South Carolina, all within 90 minutes of driving from Charleston, SC. This work will hopefully open up access to TMS for millions of patients with treatment-resistant depression who cannot be treated in the current model.
We will recruit up to 30 treatment-resistant depression patients who live near one of the MUSC-affiliated clinics. We will treat these patients, open-label, with FDA-approved accelerated TMS (6 sessions each day, over 2 hours, for 5 days, spread over one or two weeks). We will measure TMS effectiveness using standard depression rating scales.
In this pilot study we will assemble a portable TMS(transcranial magnetic stimulation) unit, in a van, and then test out delivering TMS in Medical University of South Carolina (MUSC)-affiliated facilities in South Carolina, all within 90 minutes of driving from Charleston, SC. This work will hopefully open up access to TMS for millions of patients with treatment-resistant depression who cannot be treated in the current model.
We will recruit up to 30 treatment-resistant depression patients who live near one of the MUSC-affiliated clinics. We will treat these patients, open-label, with FDA-approved accelerated TMS (6 sessions each day, over 2 hours, for 5 days, spread over one or two weeks). We will measure TMS effectiveness using standard depression rating scales.
Multiple Myeloma (MM) is a cancer of the blood's plasma cells ( blood cell). The cancer is typically found in the bones and bone marrow (the spongy tissue inside of the bones) and can cause bone pain, fractures, infections, weaker bones, and kidney failure. Treatments are available, but MM can come back (relapsed) or may not get better (refractory) with treatment. This is a study to determine adverse events and change in disease symptoms of ABBV-383 in adult participants with relapsed/refractory (R/R) MM. ABBV-383 is an investigational drug being developed for the treatment of R/R Multiple Myeloma (MM). This study is broken into 2 Arms; Arm A (Parts 1 and 2) and Arm B. Arm A includes 2 parts: step-up dose optimization (Part 1) and dose expansion (Part 2). In Part 1, different level of step-up doses are tested followed by the target dose of ABBV-383. In Part 2, the step-up dose identified in Part 1 (Dose A) will be used followed by the target dose A of ABBV-383. In Arm B a flat dose of ABBV-383 will be tested. Around 120 adult participants with relapsed/refractory multiple myeloma will be enrolled at approximately 30 sites across the world. Participants will receive ABBV-383 as an infusion into the vein in 28 day cycles for approximately 3 years. There may be higher treatment burden for participants in this trial compared to their standard of care. Participants will attend regular visits during the study at a hospital or clinic. The effect of the treatment will be checked by medical assessments, blood tests, checking for side effects and questionnaires.
Alpha-1 antitrypsin (AAT) deficiency is a condition in which the body does not make enough of AAT, a protein that protects the lungs and liver from damage. This condition is inherited, meaning you get the faulty gene from one or both of your parents.
The purpose of this study is to assess the safety, tolerability, pharmacokinetics, and pharmacodynamics of oral administration of BMN 349 in adult participants with the PiZZ genotype (also defined as having a severe deficiency or AAT ≤ 60mg/dL) or PiMZ genotype with metabolic dysfunction-associated steatohepatitis (MASH). Participants will receive an oral single dose of BMN 349 (250 mg), a medication designed to assist Z-alpha-1 antitrypsin to get out of the liver cell. The study drug BMN 349 has not been approved by the Federal Drug Administration (FDA).
This is a phase 1 study in which participants will get a single pill dose of drug or placebo to measure the amount of alpha-1 that gets out of the liver cells and into the bloodstream. Study details include:
• Study duration: up to 78 days
• Treatment duration: 1 day (single dose).
• Observations: The study will collect data on medical history, physical examination, vital signs, electrocardiogram readings, clinical laboratory parameters, pulmonary function tests, and drug distribution.
• Visit frequency: The Screening Visit, dosing, and post dosing evaluations will be conducted at MUSC on 3 consecutive days. Visits at days 8 and 36 days will occur at study site. Other procedures/assessments may be performed at the study site or at home by a healthcare professional and/or by telemedicine.