A 52-week Double-blind Placebo-controlled Randomized Phase 3 study Intended to Determine the Effects of seladelpar on normalization of Alkaline phosphatase Levels in Subjects with Primary Biliary Cholangitis (PBC) and an Incomplete Response or Intolerance to Ursodeoxycholic Acid (UDCA)
This is a multicenter, randomized, double-blind, placebo-controlled, parallel-group study of seladelpar in patients with PBC and ALP values greater than ULN but less than 1.67×ULN with either concomitant UDCA treatment or an intolerance to UDCA. Approximately 75 subjects will be randomized in a 2:1 ratio (seladelpar:placebo) across approximately 26 sites.Screening PeriodThe Screening Period will be up to 4 weeks, during which time subject eligibility will be confirmed. Subjects must use the study-issued electronic device or app for at least 14 days. On Day 1, subjects will be randomized and enter the Treatment Period.Double-blind Treatment PeriodOn Day 1 (Visit 2), subjects will be randomized into 1 of 2 treatment groups (seladelpar 10 mg or placebo) in a 2:1 ratio. Subjects will be stratified at randomization according to ALP levels (1×ULN < ALP < 1.3×ULN versus 1.3×ULN = ALP < 1.67×ULN) to ensure balance across the treatment groups. Subjects will receive double-blinded treatment for 52 weeks.Clinic visits will occur at Weeks 4, Week 12, Week 26, Week 39, and Week 52 (Visits 3 to 7). Week 52 is the End of Treatment (EOT) visit.Safety Follow-up PeriodSubjects will have a follow-up visit performed 2 weeks after the last dose of study drug, including subjects who discontinue treatment early.
A clinical study to assess whether the gut microbiome affects the response of children with short stature (SS) to growth hormone (GH) therapy
Title of ProtocolA clinical study to assess whether the gut microbiome affects the response of children with short stature (SS) to GH therapy Research questions: • Does the composition of the gut microbiome of SS children differ from their “normal” siblings or age/sex-matched controls (with normal height 10-90%) ? • Does the composition of the gut microbiome change in response to GH therapy? • Can the initial composition of the gut microbiome predict response to therapy? Background and significance:Short stature (SS) is defined as a standing height below 2 SDS for sex/age matched controls of a well-nourished subject. Short stature can be due to various causes such as growth hormone deficiency (GHD), syndromes or idiopathic short stature (ISS). The Utah Growth Study (1), which is the largest population-based survey of growth in nearly 115,000 American children, found that approximately 44,000 children in the US have SS (2).SS children can be treated with rhGH till attainment of final adult height prediction. The response to GH therapy usually assessed every 3-4 months during therapy (3). GH usually improves growth velocity and final height in SS children, however the response varies greatly (4). In the past few years there is an increasing rate of poor or unsatisfactory response to GH treatment among SS subjects (i.e., not leading to significant catch-up growth) (5). The reasons for the variable response are not clear. GH dosing is weight based and monitoring and dose adjustments are based on blood tests and growth. Using data from the Genentech National Cooperative Growth Study (NCGS), collected over a 25-year period, to examine the responsiveness to rhGH in1,186 SS patients it was found that during the first year, lower BMI SDS predicted worse response to GH therapy (6).This data are in line with a previous report showing that children with SS have lower BMI than children in the normal population (7).
A computational approach to optimal deactivation of cochlear implant electrodes
The goal of the present study is to use computationally driven models of speech understanding in CI users to guide the search for which combination of active electrodes can yield the best speech understanding for a specific patient. It is hypothesized that model-recommended settings will result in significantly better speech understanding than standard-of-care settings.
A Digital Health Framework To Assess Glycemia and Physical Activity in Kidney Transplant Recipients
This pilot is a prospective exploratory study in 20 patients 50 years of age or older with no cognitive impairments who are scheduled to receive a kidney transplant at NYULH. The goal of this pilot is to examine the feasibility of using wearable digital devices, specifically a continuous glucose monitor (CGM) and accelerometer, in this population.
A DOUBLE-BLIND RANDOMIZED PLACEBO-CONTROLLED MULTICENTER OUTPATIENT PARALLEL-GROUP STUDY TO ASSESS THE EFFICACY AND SAFETY OF STACCATO ALPRAZOLAM IN PARTICIPANTS 12 YEARS OF AGE AND OLDER WITH EPILEPSY WITH A PREDICTABLE SEIZURE PATTERN.
Staccato alprazolam is being developed for the indication of rapid cessation of a prolonged focal or generalized seizure that has not progressed to status epilepticus in patients with epilepsy 12 years of age and older. After completing the study, eligible study participants will be allowed to enroll in an open-label extension (OLE) study.
A First-in-Human Phase 1a/1b Trial to Assess the Safety Tolerability and Preliminary Efficacy of LY4170156 an Antibody-Drug Conjugate Targeting Folate Receptor a Expressing Tumor Cells in Participants with Selected Advanced Solid Tumors
This study will test a new drug called LY4170156, an antibody drug conjugate (ADC) to see how it works in the body, and if it helps patients with different types of cancer, like ovarian, endometrial, cervical, lung, breast, pancreatic, and colorectal cancers. The study has two phases. In Phase 1a, doctors will find the best dose of LY4170156 by giving it to patients and checking for side effects and how well it works. If needed, they will try different doses to see which one is the safest and most effective. In Phase 1b, the best dose from Phase 1a will be given to more patients to see how well it works as a single treatment. Patients will be divided into groups based on the type of cancer and a specific antigen in their tumors to which the ADC binds, which will be tested in a lab. The study will make sure that the drug is given at the right dose and checks how patients feel during the treatment.
A First in Human Study of theMenin-KMT2A (MLL1)Inhibitor JNJ-75276617 in Participants with Acute Leukemia
A First in Human Study of theMenin-KMT2A (MLL1)Inhibitor JNJ-75276617 in Participants with Acute Leukemia
A Follow-Up Open-Label Research Evaluation of Sustained Treatment with Aficamten (CK-3773274) in Hypertrophic Cardiomyopathy (HCM)
This is an open-label study of CK-3773274 in patients with HCM. The treatment duration is anticipated to be multiple years, concluding when marketing authorization is achieved in the patient’s country or Cytokinetics terminates the study. Approximately 600 patients will be enrolled in this study. After (up to) 56-days of screening, eligible patients will be administered a daily dose of CK-3773274. The highest maximum tolerated dose of CK-3773274 in CY 6022 will be informed by the ongoing conduct of other studies of CK-3773274. Each patient will start at the lowest prespecified dose and undergo echocardiography-guided dose titration to their maximum tolerated dose (not to exceed the highest prespecified dose). Dose adjustment may be made no more frequently than every 2 weeks.Approximately 95 investigative sites worldwide.
A green activity prescription program for Chinese Americans living with dementia
This study will iteratively develop and test the feasibility and acceptability of a 12-week green prescription program among Chinese American people living with dementia and their care partners. Development of the intervention will be guided by focus groups consisting of experts in green activities, Alzheimer's disease and related dementias, occupational therapy, BE, implementation science, and Chinese American people living with dementia and their care partners. Stakeholders will also identify preferred recruitment strategies and meaningful outcomes to prepare the green intervention for future pilot testing.
A LONGITUDINAL SINGLE-CENTER STUDY: Rapid Quantitative Assessment of Knee Joint with Compressed Sensing
60 participants will be recruited for this study (30 patients and 30 healthy controls). All participants will attend one study visit for a baseline MRI, and only the patients will have a follow up visit 2 years after their baseline MRI. A covariance analysis will be performed as well as Pearson and Spearman rank correlation coefficients.