7 Clinical Trials for Mental Disability
This study is an unmatched, case-control study of 150 youth (Ages 7-17) with a parent reported Intellectual Developmental Disability (IDD) who present to Rady Children's Hospital Emergency Department with a Mental Health Crisis (MHC). Rady Children's Institute for Genomic Medicine (RCIGM) will collect biological samples (such as blood) of these participants to study their genomes, medical and psychiatric profiles to better understand specific characteristics that may predispose them to MHC's. The 150 youth will be compared to historical, publicly available cohorts of youth with IDD's
Roughly 40% of those with intellectual and developmental disabilities (IDD) have mental health needs, which is twice the national average. Nevertheless, there is dearth of evidenced-based mental health treatment for youth and young adults with IDD. The disparity in access to mental health care places those with IDD at greater risk of crisis service use. While telemental health studies demonstrate potential to enhance access to care, little of this research includes those with IDD, or crisis prevention and intervention. This project will refine and evaluate telemental health services for youth and young adults with IDD delivered within START (Systemic, Therapeutic, Assessment, Resources, and Treatment), a national, evidence-based model of crisis prevention and intervention for people with IDD. The study will begin with stakeholder feedback (service recipients, families, and providers) regarding telemental health services (Aim 1). Results will be used to refine the intervention. Our team will then compare telemental health versus in-person START services in a randomized control trial (Aim 2). To our knowledge, this will be the first trial of a telemental health crisis program for the IDD population. The final goal is to understand if outcomes vary across subpopulations (Aim 3) and to identify potential disparities. If found, the investigators will work with service users, families and providers to develop a strategy to address identified disparities in outcomes. The study will be executed by an interdisciplinary team of experts engaged with stakeholder partners. Understanding the benefits of specific telemental health methods has important implications to the design of interventions, within and outside of START. This telemental health study offers promise to address disparities in access to mental health care for people with IDD.
Adults with psychiatric disabilities get sick and die 20 to 30 years younger than same-age peers, with even greater disparities occurring when the person is from a low SES or of color. Factors explaining this difference are complex and include genetic comorbidity, iatrogenic effects of medication, life choices, and life consequences. These factors are worsened by service disparities which are often fragmented in the public health system. Peer navigators are part of a program in which providers escort people with psychiatric disabilities around the fragmented system to meet their health and wellness goals, often a demanding task for the person who has needs addressed at clinics, labs, and pharmacies spread across an urban area. Navigators are peers because they have lived experience of recovery and are often from similar ethnic groups. A community-based participatory research program supported by NIMHD and PCORI developed a peer navigator program specific to the needs of people with psychiatric disabilities. Results of two small pilots funded by NIMHD and PCORI showed the Peer Navigator Program (PNP) led to significant improved service engagement which corresponded with better health, recovery, and quality of life. The studies included fidelity measurement which showed peer navigators conducting the intervention at high levels of fidelity. The current research is an efficacy study with a more fully powered test of PNP versus treatment as usual, which is integrated care (TAU-IC). The investigators aim to recruit 300 adults with psychiatric disability who wish to improve physical health/wellness through peer health navigation randomized to TAU-IC or TAU-IC plus PNP. Individuals will participate in assigned interventions as part of 8-month cohorts with data being obtained at baseline, 4, 8, and 12 months. Data will include personal descriptors (demographics, diagnosis, life consequences report), outcomes (service engagement, physical symptoms, blood pressure, recovery, and quality of life), mediators (personal empowerment, self-determination, and perceived relationship for recovery), and process measures (fidelity, feasibility, and acceptability). Investigators hypothesize that those in PNP intervention will have improved outcomes over the integrated care as usual. A cost-benefit analysis will seek to model impact based on quality-adjusted life years. Larger effect sizes will permit post hoc identification of how PNP effects vary by participant characteristics such as ethnicity and gender.
Purpose: Conduct a wait-list randomized controlled trial (RCT) of an inclusive physical activity program called PACE for adults with intellectual disability (ID) who are not yet showing signs of Alzheimer's Disease (AD)/age-related dementias (ARD). Participants: Participants include 120 adults with ID, their caregivers, and their coaches (up to 360 individual participants, grouped as triads), recruited through the University of North Carolina at Chapel Hill and the University of Arkansas. Participants also include 16 exercise professionals. Procedures (methods): Each cohort will include 20 triads who are randomly assigned to the PACE program or the waitlist control group.
FOXP1, also known as Forkhead-box Protein P1, is a transcription factor protein belonging to the FOX gene family. Disruptions in the FOXP1 gene cause a phenotype characterized by global developmental delay, speech deficits, mild dysmorphic features, and traits of autism spectrum disorder. This study seeks to characterize FOXP1-related neurodevelopmental disorders using a number of genetic, medical and neuropsychological measures.
DDX3X syndrome is a genetic cause of intellectual disability and other neurologic features including, in some cases, autism. Variants in the DDX3X gene are thought to account for 1-3% of unexplained intellectual disability in females, making it one of the more common causes of intellectual disability.This study seeks to characterize DDX3X-related neurodevelopmental disorders using a number of genetic, medical and neuropsychological measures.
The Congenital Muscle Disease Patient and Proxy Reported Outcome Study (CMDPROS) is a longitudinal 10 year study to identify and trend care parameters, adverse events in the congenital muscle diseases using the Congenital Muscle Disease International Registry (CMDIR) to acquire necessary data for adverse event calculations (intake survey and medical records curation). To support this study and become a participant, we ask that you register in the CMDIR. You can do this by visiting www.cmdir.org. There is no travel required. The registry includes affected individuals with congenital muscular dystrophy, congenital myopathy, and congenital myasthenic syndrome and registers through the late onset spectrum for these disease groups. The CMDIR was created to identify the global congenital muscle disease population for the purpose of raising awareness, standards of care, clinical trials and in the future a treatment or cure. Simply put, we will not be successful in finding a treatment or cure unless we know who the affected individuals are, what the diagnosis is and how the disease is affecting the individual. Registering in the CMDIR means that you will enter demographic information and complete an intake survey. We would then ask that you provide records regarding the diagnosis and treatment of CMD, including genetic testing, muscle biopsy, pulmonary function testing, sleep studies, clinic visit notes, and hospital discharge summaries. Study hypothesis: 1. To use patient and proxy reported survey answers and medical reports to build a longitudinal care and outcomes database across the congenital muscle diseases. 2. To generate congenital muscle disease subtype specific adverse event rates and correlate with key care parameters.