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 Community-Based Intervention To Address Health-Related Social Needs For Patients On LAI-PrEP
The purpose of this study is to evaluate the effect of addressing health-related social needs for people taking long-acting injectable HIVprevention medication (LAI-PrEP). We are particularly interested inrecruiting people that are most vulnerable to HIV infection and least likelyto be prescribed PrEP, including cisgender women of color and genderdiverse persons (transgender or non-binary individuals). If you are eligible and agree to participate, you would start LAI-PrEP aspart of your standard of care and would have 5 research study visits. Atthe study visits, you would be asked to complete various surveys and 1interview. If you decide to participate in this study, you would be asked to come to 5in-person study visits over a period of approximately 1 year. You would be compensated for each in-person study visit.
A Comparison of NeuroSpan Bridge NeuraGen Nerve Guide and Nerve Autograft for Peripheral Nerve Repair (NeuroSpan-1)
The purpose of this research study is to compare three treatments for nerve injuries in the arms or legs: the NeuroSpan Bridge, the NeuraGen Nerve Guide, and using a person’s own nerve (nerve autograft). These treatments help repair damaged nerves and restore movement or feeling.
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 Double-Blind Randomized Multicenter Trial Evaluating the Efficacy and Safety of PRAX-628 in Adults with Focal Seizures (POWER 1)
The main purpose of this research is to find out how well PRAX-628 works and how safe it is when used to treat focal seizures compared to placebo (inactive drug). The study also looks at the side effects of taking PRAX-628 compared with placebo and how the body processes it. The active study drug or placebo will be taken in addition to your regular anti-seizure medications.
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 Feasibility Pilot Study of Home-Based Intermittent 60Hz Light Therapy for the Treatment of Depression
The purpose of this study is to see how easy it is to use an intermittent light therapy device in adults with a major depressive episode (MDE), and to see how mood is impacted by the light therapy. Participants will complete 15 non-invasive light stimulation sessions, online surveys, and assessments.
A First-in-human (FIH) Study of Inhibitory Interneurons (NRTX- 1001) in Drug-Resistant Unilateral Mesial Temporal Lobe Epilepsy (MTLE)
The purpose of this two part study is to evaluate the safety (Stage 1 and Stage 2) and efficacy (Stage 2) of NRTX-1001 in subjects with drug resistant mesial temporal lobe epilepsy (MTLE).
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