The purpose of this study is to investigate the safety (what side effects the study treatment may have) and effectiveness (how well the study treatment works to relieve symptoms) of BOTOX when injected into the bladder of adult females for the treatment of Interstitial Cystitis/ Bladder Pain Syndrome. Interstitial Cystitis/Bladder Pain Syndrome (IC/BPS) is a chronic and debilitating urological condition characterized by symptoms of bladder pain or discomfort and accompanied by lower urinary tract symptoms such as frequent toilet visits, often also during bedtime. BOTOX might be able to temporarily reduce bladder pain/discomfort and improve your accompanying urinary symptoms. There will be a total of 9 study visits over a period of approximately 28 weeks which includes up to 28 days (4 weeks of screening), a treatment visit, and 24 weeks of follow-up if you receive only one treatment. If you choose to receive a second treatment (which will be BOTOX), you could have a total of up to 13 visits and be in the study for an additional 12 weeks for a total of up to 44 weeks (depending on when you request retreatment) which includes the 28 days (4 weeks of screening). If you are already
receiving treatment for IC-BPS, after you have signed the informed consent, you may have to washout of your medication prior to screening and throughout your study participation. If you have a urinary tract infection [UTI] that requires treatment, an additional 14-day extension is allowed to receive Treatment 2 for total study
participation of up to 46 weeks. You may need to attend additional unscheduled visits for safety or other reasons.
The primary objective of this study is to evaluate the efficacy, safety, and tolerability of upadacitinib for the treatment of adults and adolescents with Non-Segmental Vitiligo who are eligible for systemic therapy. This study is comprised of a 35-day Screening Period; a 48-week placebo-controlled, double-blinded treatment period (Period A); a 112-week open-label extension period (Period B); and a 30-day follow-up period. In Period A, adult and adolescent subjects who meet eligibility criteria will be randomized 2:1 to receive daily oral doses of upadacitinib 15 mg or matching placebo. Subjects who complete Period A will continue into Period B and will receive open-label upadacitinib 15 mg.
This is a double arm, open label, 20-week Phase III study with three and six-month follow up periods, in patients with a documented history of generalised vitiligo.
Up to 200 eligible patients across study sites will be enrolled and randomised in equal numbers to one of the following treatment groups:
• Group A will receive NB-UVB twice weekly from Day 0 (40 treatments in total), and SCENESSE® (one implant administered on Days 0, 21 (±4), 42 (±4), 63 (±4), 84 (±4), 105 (±4) and 126 (±4) (seven implants in total));
• Group B will receive NB-UVB light only (administered twice weekly for 20 weeks, 40 treatments in total).
To determine eligibility for study participation, patients will undergo a screening evaluation within a 28-day period before receiving the first study treatment.
This phase I trial will determine the maximum tolerated dose of lenalidomide when given in combination with high-dose systemic methotrexate and rituximab, with or without nivolumab, as induction treatment of primary central nervous system lymphoma. In addition, whether the combination of drugs can extend the control of CNS lymphoma by being used as maintenance (prolonged treatment) after control is achieved with the initial chemotherapy regimen (induction) will be judged. If decided to take part in the study, participants will complete pre-study testing, and if allowed to participate in study different people will get different doses of the study drug lenalidomide during induction chemotherapy. If the drug does not cause serious side effects, the next group of people in the study will get a higher dose, and the doses will continue to increase for every new group until people have serious side effects that require the dose to be lower. Lenalidomide will be taken by mouth on days 5 to 14 of each induction cycle. Once the dose of lenalidomide is found, the next group of people in the study will receive nivolumab in combination with the other drugs (methotrexate, rituximab, and lenalidomide). The first drug administered in each cycle is rituximab, which is given as an intravenous infusion typically in the infusion center. The day after rituximab, participants will be admitted to the hospital for the infusion of methotrexate. Enrolled participants that present benefit after induction will receive lenalidomide and nivolumab as prolonged therapy (maintenance) for an additional 12 months (12 cycles and each cycle is 28 days) or until the disease gets worse or the side effects become too severe. After treatment is completed the study doctor will continue to follow up on participants condition for 2 years to observe side effects. After 2 years the doctor will continue to follow up either in clinic or by phone for up to 5 years after registration. The most common side effects known are kidney damage, infusion reaction, blood clots, birth defects, immune toxicity, fever and infections, and there may be some risks that the study doctor is not aware of yet. Once the combination is proven safe, this study will allow for future studies to determine whether the combination of these four drugs can improve the response to treatment and help increase the understanding of their use in primary CNS lymphoma treatment. It is unclear whether these drugs will help participants live longer than the usual approach alone.
This study is for patients with hypertrophic cardiomyopathy (HCM). HCM is a condition where the heart muscle becomes abnormally thickened, which can sometimes block the blood flow out of the heart and results in the heart muscle working harder to pump blood to the body. Participants who have completed participation in a previous HCM study investigating the study drug, called aficamten (CK-3773274), will be eligible to participate in this study.
The study is done to collect long-term safety and tolerability data, including assessments of cardiac structure and function during chronic dosing with aficamten. Aficamten is a tablet taken by mouth. This is an open label study (the participants and study team will know the dose of aficamten taken at any given time). If your screening results show you are eligible to continue in the study, you will visit the research site for the "first dosing day" (Day 1), followed by visits at Weeks 2, 4, 6, 12, then every 12 weeks thereafter. Study related procedures include blood work, echocardiograms (ultrasound test of the heart), electrocardiogram (recording of heart's electrical activity), physical exams, and questionnaires. Risks associated with this study include shortness of breath, nausea, diarrhea, headaches and dizziness.
This is a global, Phase 3b/4, randomized, open-label, efficacy assessor-blinded, multi-center study that will evaluate upadacitinib compared to dupilumab in adult subjects with moderate to severe AD and inadequate response to dupilumab after at least 6 months of current use. The study consists of a 35-day Screening Period; an 8-week randomized, open-label, efficacy assessor blinded treatment period for all participants (Period 1); a 24-week open-label, efficacy assessor-blinded extension period for all participants who finish Period 1 (Period 2) (total duration of Period 1 and Period 2 is 32 weeks); and a 30-day Follow-up visit.
This is an inpatient clinical trial of a new form of transcranial magnetic stimulation with 10 treatments/day for 5 days, called SAINT. It is jointly funded by the NIH and a TMS company, MAGNUS. It is being done at 5 enrolling sites in the US, with total sampe of 100. Depressed and suicidal inpatients will have an MRI scan, then 5 days of treatment, and then a followup MRI scan. Outcome measures are depression and suicide ratings, as well as MRI changes. It is randomized and double blind, so some patients will not be getting active TMS. Importantly this is an adjunctive treatment study on top of all routine clinical treatments.
The purpose of this medical research study is to evaluate the safety and effectiveness of a new medication called imatinib mesylate in the treatment of Lymphangioleiomyomatosis (LAM). LAM is a rare disease in which abnormal cells (called LAM cells) grow out of control. Over time, LAM cells destroy healthy lung tissue and cause respiratory disease or failure.
Many patients with LAM are currently treated with a medication called sirolimus (rapamycin). Sirolimus slows the growth of LAM cells.
Imatinib mesylate (hereafter called imatinib) is approved by the Food and Drug Administration (FDA) for the treatment of some cancers that share common pathways with LAM cells. Laboratory studies suggest that imatinib could completely block the growth of LAM cells through initiation of targeted cell death.
An important purpose of this research is to determine the safety of imatinib in people with LAM. This study will also evaluate the short-term effectiveness of imatinib. Participants will be randomized to receiving imatinib (study medication) or placebo (no treatment) for the 180 day duration of participation. The study is being conducted at the Medical University of South Carolina and at Columbia University in New York (CUMC). Each site will enroll 10 participants.
This study is for subjects that have been diagnosed with mantle cell lymphoma. The purpose of this study is to compare continuous treatment with zanubrutinib to treatment with zanubrutinib that is stopped after the initial six 28-day cycles of chemotherapy and restarted at the first time your disease gets worse following the initial six 28-day cycles of chemotherapy. The subject may remain in the study for up to 10 years.
This is a pivotal Phase 3, global, randomized, double-blinded, placebo-controlled, multicenter study to evaluate efficacy and safety of lutikizumab in subjects 16 years of age and older with moderate to severe HS. The study comprises a 35-day Screening Period, a 16-week placebo-controlled double-blinded period (Period 1), a 36-week double-blinded extension (Period 2), and a 10-week (70 days) Follow-Up Period after last study drug dosing or an option to enter a separate long term extension study.