for an approved therapeutic agent. The pharmaceutical company that produces the agent has agreed to provide the oral drug and placebo free of charge. The investigator contacts the MUA HRPPP to develop a research participant protection accountability plan, although the official with overall ultimate responsibility for human protection of his protocol is the President of MUA. (The president’s authority is likely to have been delegated to a subordinate official such as the dean for research).

B. Protocol Development

The protocol was designed by the PI and his collaborators, and because the drug is not approved for this indication, the PI is the holder of the investigational new drug application (IND). The pharmaceutical company is responsible for product manufacturing, packaging, labeling, and distribution; the PI, however, will be responsible for storage, usage, and disposal at his site, and he ensures that all study procedures are standardized with well-established protocols and that all co-investigators and research staff are properly credentialed and trained. The PI has found an accredited laboratory at MUA to perform the laboratory assays. The director of the laboratory has provided the PI with documentation that the laboratory is approved according to the Clinical Laboratory Improvement Act and Amendment and that it has normative values available.

C. Protocol Review Process

The study receives scientific review both from NIH and the General Clinical Research Center (GCRC) Advisory Committee (GAC). Conflict of interest review is performed by the MUA’s Conflict of Interest Committee, if necessary, which assures that the investigator does not have a significant financial interest in the pharmaceutical company and that he or she is not receiving financial remuneration. An external oversight committee established by MUA carries out a similar review examining potential institutional conflicts of interest. The ethical research review is performed by the MUA’s Research ERB. The protocol is also reviewed by MUA’s Radiation Safety Committee because patients will undergo Magnetic Resonance Imaging procedures. The PI establishes a data and safety monitoring plan.

D. Ongoing Monitoring

Because the study involves young children, the Research ERB requests consent monitoring. Protocol amendments are submitted to MUA’s Research ERB. The PI requests that NIH establish a Data and Safety Monitoring Board/Data Monitoring Committee (DSMB/DMC) for review of the

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