HGT Clinical Trials

Human Gene Transfer (HGT) is the deliberate transfer into human research participants of either:

1. Recombinant nucleic acid molecules, or DNA or RNA derived from recombinant nucleic acid molecules, OR
2. Synthetic nucleic acid molecules, or DNA or RNA derived from synthetic nucleic acid molecules, that meet any one of the following criteria:
   • Contain more than 100 nucleotides, OR
   • Possess biological properties that enable integration into the genome (e.g., cis elements involved in integration), OR
   • Have the potential to replicate in a cell, OR
   • Can be translated or transcribed.

HGT research is usually designated as a clinical trial and is sometimes referred to as “Gene Therapy”. NOTE: HGT research cannot be initiated until Institutional Biosafety Committee (IBC) and all other applicable institutional and regulatory authorization(s) and approvals have been obtained.

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IBC Protocol Review and Approval

The outline below summarizes the review and approval process for clinical trial protocols submitted to the IBC. PI, Clinical Investigations Support Office (CISO), and EH&S have defined roles in preparing the protocol for the IBC.

1. PI informs CISO of new HGT clinical trial from a sponsor. NOTE: Sponsor may utilize the USC IBC only or an external IBC. For HGT clinical trials that utilize external IBC assistance, the USC IBC still has to approve the protocols for clinical trials occurring at USC before patient enrollment.
2. PI completes a new BUA in iStar to be reviewed by USC IBC. NOTE: CISO will be responsible for assisting the PI with the BUA submission and making necessary changes based on enrollment, serious adverse events (SAEs), and/or changes related to the phase of the study.
3. EH&S Biosafety assists both the USC IBC and the external IBC (if external IBC services were utilized by the sponsor) with any requests regarding the details of the clinical trials. NOTE: Download the Required Documents Checklist (see button below) for common items reviewed by IBCs.
4. USC IBC and the external IBC (if external IBC services were utilized by the sponsor) concurrently review the HGT clinical trial for proper biosafety practices and provide recommendations/stipulations for approval as necessary.
5. Full IBC approval is then granted for the HGT clinical trial.

For a detailed, step-wise outline of the IBC protocol review and approval process for clinical trials, click on the button below to download the SOP.

Additionally, use the External IBC Review Required Documents Checklist to collect the critical documents for review.