The primary purposes of this study are to:
•Provide access to cord blood units for recipients whose best choice for a cord blood unit(s) do not meet all FDA standards, but do meet standards set by the NMDP on this study.
•Assess how well and how quickly blood counts return to normal after transplant in recipients on this study.
This study if for patients that have a blood disease and it's been determined that the best option for treating that blood disease is a cord blood transplant. Cord blood (CB) is blood that is taken from the umbilical cord and placenta of healthy newborn babies after childbirth. The cord blood collected from a newborn baby is called a cord blood unit. The United States Food and Drug Administration (FDA) considers cord blood to be a biological drug. These are considered "investigational" products. This study will evaluate the safety of administration of the investigational cord blood units by carefully documenting all infusion-related problems.
We will ask 80 patients with Parkinson's disease, representing the full spectrum of motor and cognitive symptoms, to participate. Participation will include measurement of eye movements using two methods: the new computer-based saccade battery and the best available video-based eye-tracking equipment. The evaluation will be repeated about 30 days later. Data will be analyzed to determine whether the computer-based tasks are reliable and able to provide the same quality of information as the gold standard in eye-tracking. A comparison sample of 80 healthy older adults will also complete the behavioral saccade tests in order to establish normative data that will enable application in clinical settings.
The Hernia in Premies (HIP) Trial is a multi-site randomized clinical trial that is comparing two accepted treatment choices, surgery before or after neonatal intensive care unit (NICU) discharge, for inguinal hernia (IH) repair in premature infants. The reason we are doing this study is that surgeons and neonatologists currently do not know the best time to perform the hernia repair. Some providers recommend having the hernia fixed before discharge from the neonatal intensive care unit (NICU) and some believe waiting until after NICU discharge is better. Both treatments are standard options for IH repair in premature infants. Also, all babies that have IH repair receive anesthesia. We also do not know if the age of the child receiving anesthesia affects neurodevelopment. We are comparing the timing of anesthesia exposure between the two treatments (IH repair before or after NICU discharge) to help answer these questions.
By volunteering to take part in the HIP Trial, your child will be randomly assigned to have the operation before or after NICU discharge. This means that the timing of the repair operation will be determined by chance, like flipping a coin, and he/she has an equal chance of being treated before or after NICU discharge. We will be collecting information about your baby and the treatment that he or she receives, and how your baby recovers before and after the surgery. We will also collect information about your baby from clinic notes and results from your baby's routine 2 year follow-up neurodevelopmental testing, where your child will be asked questions that measure cognitive, language, social and motor development. We will stop collecting information about your baby and your participation will end when your child is 2 years old. There are no extra medical tests or blood work being done for research purposes in the HIP trial.
This study proposes to design and build a neural imager/stimulator which will let us obtain information about brain circuits and pathways by acquiring electrical and fMRI signals from the brain at the same time. By adding magnetic stimulation to the instrument we will be able to perturb these circuits at precise times and locations in order to both improve how TMS is used as an anti-depression treatment as well as to better understand how our brains function. This research has the potential to revolutionize our understanding of how best to use TMS as a treatment as well as learning how our brains function. This study simply proposes to develop the combined instrument. The use of the new instrument as a anti-depressive treatment will be presented independently after we have developed a working instrument.
Over the past 30 years we have discovered that both the efficacy and the side effects of ECT come not only from the induced seizure, but by the currents of electricity and where they go in the brain. In all patients we now determine, at the first treatment session, the minimum dose of electricity needed to produce a seizure. This is called the seizure threshold. Subsequent treatments are then given at 6 or 9 times this number. The method of titrating has not been fully explored. We propose to titrate with two different currents, one of which is much lower than standard clinical practice. We need to do this twice in each patient, on the first and second treatment sessions, and compare the difference. If we find that that lower currents are paradoxically better, then this will change ECT practice around the world. Patients will receive less overall electricity, with likely fewer side effects.
This study is for patients that have been diagnosed with acute myelogenous leukemia (AML) . The investigational drug in this study is AG-120. The purpose of this study is to identify and test the highest dose of AG-120 that can be given safely. Participants can expect to be in the study for as long as the study doctor feels it is in the participants best interest.
The purpose of this study is to determine which treatment is best for preventing stroke or systemic embolism in patients who have experienced at least one episode of SCAF (sub-clinical atrial fibrillation) detected by their pacemaker or ICD (intracardiac defibrillator) or ICM (insertable cardiac monitor) and also have other risk factors for stroke. Subjects will be randomly assigned to one of the two treatment strategies: apixaban or aspirin. Neither the subject nor the researcher will know which treatment the subject is assigned. Follow-up visits will occur every six months until the end of the study.
We recently completed an NIMH R34 in which we piloted a patient- and provider-informed tablet-based toolkit designed to facilitate delivery of Trauma-Focused Cognitive Behavioral Therapy (TF-CBT) – a treatment that was selected because it addresses a wide range of symptoms using techniques shared by other treatments for emotional and behavioral disorders. The tablet-based toolkit consists of numerous components (e.g., videos, interactive games, drawing applications) that are designed to facilitate provider-patient interactions in a way that enhances children's engagement and supports adherence to the treatment model. The tablet-based toolkit was very well received by children, caregivers, and providers in our pilot work. Moreover, all benchmarks for feasibility outlined in our NIMH R34 application were met or exceeded. We now propose to conduct a hybrid effectiveness-implementation trial to examine the extent to which the tablet intervention may improve fidelity, engagement, and children's mental health outcomes. We will conduct a randomized controlled trial with 250 mental health providers and 360 families in partnership with dozens of clinics in the Carolinas and Florida. Providers will be assigned randomly to tablet-facilitated vs. standard TF-CBT. Youth aged 8-16 years with clinically elevated symptoms of PTSD will be recruited. Baseline and 3-, 6-, 9-, and 12-month post-baseline assessments will be conducted by independent, blind evaluators. Sessions will be videorecorded for observational coding of engagement and fidelity by independent raters blind to study hypotheses. We will also examine costs and conduct semi-structured interviews with families, providers, supervisors, and agency leaders to inform future dissemination and implementation initiatives. Technology-based resources that are scalable, easy to use, and designed for efficient integration into everyday practice may have sustained national impact. The return on investment of these initiatives will ultimately rest on their potential to improve the spread of best-practice treatments and the quality with which they are delivered to the children who need them.
Behavioral health problems among Veterans have raised awareness of the critical need for more reliable, effective, and accessible ways to recognize those in need, direct them to help, and ensure that they receive the best evidence-based care available. AboutFace is a novel peer education program that features the personal stories of Veterans and is designed to improve Veterans' likelihood of engaging in PTSD specialty care. Using a randomized controlled study design we propose to compare the efficacy of AboutFace relative to standard care for improving treatment engagement and outcomes. Additional data from VA providers will provide valuable information on wide scale implementation and dissemination of AboutFace. If AboutFace increases access of services, data will have broad implications for overcoming barriers to care for Veterans with PTSD and other stigmatized conditions.