Clinical Translational Core
The overall goal of the Clinical Translational Core (CTC) is to efficiently and effectively move basic and applied intellectual and developmental disability research (IDDR) findings into human clinical trials and ultimately clinical practice. The CTC provides assistance and services to IDDRC investigators at each stage of the clinical and translational research. This process is facilitated by a support team and navigators.
The direct functions of the CTC will include:
- Data mining of large real-world evidence data sources (informatics)
- Patient recruitment and study performance across multiple venues
- Consultative support in biostatistics/informatics for planning, designing and analyzing IDDR studies
- Connection and collaboration with other IDDRC cores for deep phenotyping/genotyping and biobanking
- Training and dissemination through a web-based investigator toolkit
To build a phenomics framework with a digital platform that integrates clinical and 'omics' data and a companion biorepository.
The CTC will serve as the coordinating and integrating center for the services provided by the other DC-IDDRC cores as related to deep phenotyping/‘omics’. This will include using the REDCap and other digital tools and our CTC informatics capacity to assist IDDR by integrating information from the Genomics and Bioinformatics Core, Human and Animal Imaging Core, Cell and Tissue Microscopy Core, and Neurobehavioral Evaluation Core. The CTC will also house a biorepository which is important to assist in:
- Biomarker development for following clinical disease progression or response to disease modifiers
- Tests of proof of concept
- Exploration of underlying tissue response or characteristics of a disease or therapy
- Developing biologic assays for diagnosis
- Studying functional relevance of genetic variations
To employ a multi-dimensional strategy to enhance participant recruitment and study implementation/performance.
The Washington, D.C., metro area has one of the most ethnically and racially diverse populations in the country. We will enhance recruitment of individuals with IDD into research studies through a combination of community “open science” programs, collaboration with HSCSN (the Children’s National managed care organization for 5,000 children with special health care needs), the DCPS Department of Special Education, the Children’s National CTSA, the NCATS Rare Diseases Clinical Research Network, the national IDDRC network and other networks and patient advocacy groups. Research navigators will serve to connect IDDR investigators with these organizations.
We will also use the real-world evidence data lake (HealtheIntent Enterprise Data Warehouse) created by a collaboration with our EMR partner, Cerner and its Power Trials application for identification of potential subjects for IDDR clinical trials. In addition, Children’s National Hospital has made progress towards becoming one of the few autism- and IDD-friendly hospitals in the country which will assist in promoting recruitment and retention of individuals with IDD into clinical research.
To provide IDDR-relevant study design, biostatistical and informatics support.
Biostatistics, epidemiology and informatics faculty of the CTC, in collaboration with their CTSA colleagues, will work with IDD investigators in applying best practice guidelines in study design and reporting for both interventional and observational studies. These collaborations will lead to innovations in IDDR as well as in the development of quantitative methods of particular interest to IDD researchers. They will include:
- Methods leveraging large real-world evidence data sources such as registries and the CNH data lake
- Reducing dimensionality of large complex data in genomics, neuroimaging and neuropsychology using text mining and machine learning tools in collaboration with other IDD cores
- Innovations in design and analyses of small sample studies
To advance bench to bedside translation through technology transfer and commercialization.
The core will assist investigators in the areas of technology transfer and commercialization through its collaboration with the CNH/GWU Offices of Technology Transfer and with the JLABS startup incubator on the new Children’s National Research & Innovation Campus. The core will also promote dissemination through its Science Cafés, website, social media presence and publications.
To develop an IDDR investigator toolkit.
A priority of the CTC will be to augment the CTSA training resources by supporting an online investigator toolkit in areas of knowledge and practice specific to IDDR and its translation. These include:
- Acquiring knowledge and skills in performance of studies in vulnerable populations
- Accommodating testing for individuals with sensory, motor or cognitive deficits and other aspects of research in IDD
- Working with other stakeholders such as patient advocacy groups. The CTC has multiple mechanisms to accomplish this aim, especially for investigators newly entering the IDDR field, including seminars, online e-learning and a mentoring program
Provided Services and Technology
- Deep phenotyping of research participants
- Recruitment across a diverse population of children with IDD as well as age-matched controls
- Statistical support for study feasibility, design and analysis
- Specimen Biobank
- Assistance with implementation, commercialization and dissemination
- Training of junior investigators and dissemination of IDDR toolkit