PORTICO Clinical Trials Toolkit

A key goal of the Rare Diseases Clinical Research Network is to promote excellence in clinical research. We also aim to develop the next generation of rare disease researchers.

To further these goals, we have partnered with another NIH-funded project, the Pediatric musculOskeletal & RheumaTology Innovation COre center (PORTICO), to offer this collection of clinical training resources. The PORTICO Clinical Trials Toolkit is a curated series of publicly accessible videos and links covering essential operational details that early investigators need to know before embarking on a clinical trial with a special focus on pediatric trials.

What is the PORTICO Clinical Trials Toolkit?

This toolkit contains essential knowledge about what researchers need to know before embarking on a clinical trial. Topics address study startup, study documents, consent, oversight, management, and safety. Find more complete information on the toolkit below or at the PORTICO Clinical Trials Toolkit web page. The PORTICO Project is a National Institutes of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) Core Center of Excellence in Clinical Research focused on Pediatric Musculoskeletal Diseases (PMSKD).

Who can benefit from this toolkit?

Many early stage investigators have received little training in the nuts and bolts of conducting clinical trials. They may be surprised or unprepared for the complexities of regulatory and administrative tasks involved in a trial. The toolkit was designed for fellows and early stage investigators who have an interest in learning about the implementation of clinical trials.

How to Use this Toolkit

The toolkit is designed for self-paced and self-directed learning. There is no test at the end. Some modules have links to more information or for advanced topics. So, if you are interested in digging deeper, please do!

When you have completed the modules, please provide feedback. For questions, contact portico@cchmc.org.

Many thanks to the PORTICO team for generously sharing these resources with the RDCRN community.

The PORTICO project was supported by the National Institutes of Arthritis and Musculoskeletal Skin Diseases under Award - Number P30AR076316. The content is solely the responsibility of the authors and does not necessarily represent the official views of the NIH.

 

 
 

Study Startup

Investigator Role/Responsibilities: Provides a quick historical/legal background while highlighting the responsibilities of the investigator in the research project.

Good Clinical Practices: Good clinical practices or GCP is the international standard by which clinical trials are conducted. The ABCs of GCP provides a quick overview of GCP and the 13 Principles of ICH, while the GCP Webinar video goes into more depth.

Study Design: In order to ensure your research meets your objectives, a solid study design is required. The first video describes the process and phases of clinical research. The second video focuses on designing a high-quality study.

Statisticians/Data Management: Good statisticians are an essential part of the research team. Watch the video to learn more about working with statisticians on your research project.

Protocol and Manual of Operating Procedures: The research protocol and the Manual of Operating Procedures (MOOP) are documents that facilitate consistency in research by detailing the study’s conduct and operations. The videos describe the purpose and development of these important documents.

Records/Source Documents: Good records and source documents are the key to good research data. This video provides a framework for good documentation practices for research.

Trial Essential Documents: Essential documents are an important part of ensuring research is being done in a qualitative and compliant manner. These documents are outlined in the video and listed in the ICH essential documents link.

Clinical Trial Registration: Clinicaltrials.gov is a web-based repository of clinical study data required as the result of the Food and Drug Administration Modernization Act of 1997. This information is maintained by the National Library of Medicine and National Institutes of Health.

Informed Consent: Informed consent is an essential process in clinical research that allows the subject to make an informed decision about participating in research. This video discusses the elements of consent. For more on consent in pediatric populations, please see section Informed Consent-Assent.

Informed Consent-Assent: Assent is the consent process for persons who are too young to give informed consent but are able to understand the research study. The understanding pediatric assent video is a good review of the process.

HIPAA and Privacy: The Health Insurance Portability and Accountability Act regulates the way certain data known as protected health information (PHI) is handled. It is important that researchers know the rules surrounding the use of PHI.

Institutional Review Board: The Institutional Review Board (IRB) is a committee responsible for reviewing research involving human participants to ensure the protection and welfare of human research subjects. This seminar reviews the basics to prepare you for your submission to the IRB.

IRB Reliance: Federal policies now require certain types of federally-funded multi-site research to use one IRB for review and approval for all sites. This video describes the history and application of these policies.

Audit and Inspections: The seminar describes the U.S. Food and Drug Administration inspection process from pronouncement to closeout meeting, including common findings.

Data and Safety Monitoring: This National Institutes of Health seminar provides an introduction, examples, basics, and samples of monitoring from data and safety monitoring boards.

Multisite Trials: This video provides an overview of the challenges of managing multi-site trials from an early investigator perspective.

Pediatric Research: Pediatric participants in research are considered vulnerable populations. This video reviews the ethical underpinnings of research in the pediatric population and utilizing real-world examples.

Recruitment and Retention: Without participants, investigators will not be able to answer their research questions. This short video provides an introduction to patient engagement to help develop research with recruitment and retention in mind.

Regulations: Electronic Systems: If your study is being completed as part of an investigational new drug (IND) or investigational device exemption (IDE) or your grant specifies, you may need to ensure the data you collect is in compliance with 21 CFR Part 11, which establishes the U. S. Food and Drug Administration regulations on electronic records and electronic signatures. This video provides a short introduction to the rule.

Safety-Adverse Events: An important investigator responsibility is monitoring the safety of your research. The videos introduce the adverse event monitoring and the reporting database used in U. S. Food and Drug Administration trials.

Safety-Unanticipated Events: This video delves into the reporting of unanticipated events. An unanticipated event is an issue that is unexpected, related to the research, and places research participants at risk of greater harm.