Human Gene Transfer (HGT) - IBC Registration
Human Gene Transfer” or “HGT” is used to describe research involving the transfer of recombinant or synthetic nucleic acid molecules, or DNA or RNA derived from recombinant or synthetic nucleic acid molecules into human subjects. It is also sometimes referred to as “Gene Therapy.” Human gene transfer is the process of transferring genetic material (DNA or RNA) into a person. Nucleic acids (DNA or RNA) may be transferred as "naked" nucleic acid, encapsulated nucleic acid,or nucleic acid within another organism, such as a virus.
At present, human gene transfer is experimental and is being studied to determine whether or not it can treat certain health problems by compensating for defective genes, producing a potentially therapeutic substance, or triggering the immune system to fight disease.
Special provisions are necessary for conducting HGT research at University of Cincinnati (UC). Principal Investigators (PIs) must complete a process of multiple reviews and approvals at both federal and local/institutional levels. First, HGT proposals must be submitted to the NIH Office of Biotechnology Activities (OBA)/ Recombinant DNA Advisory Committee (RAC). Once NIH OBA/RAC review is complete, the project must be submitted to both UC Institutional Biosafety Committee (IBC) and Institutional Review Board (IRB) for review. IBC approval must be obtained from each institution at which recombinant or synthetic nucleic acid material will be administered to human subjects.
Protocols may be submitted to IBC and IRB at the same time, but IRB approval will only be granted after IBC approval.
No research participant may be enrolled in an HGT study until the OBA/RAC review process is completed and IBC and IRB approvals and applicable regulatory authorizations are obtained.
Initial Documentation to Be Submitted to the IBC
- Complete the UC HGT form. The Biosafety office can be contacted for assistance preparing this form.
- OBA/RAC letter certifying protocol review completion - and any additional RAC-Sponsor or -investigator correspondence
- Complete Appendix M of NIH Guidelines
- Clinical Protocol and Investigator Brochure
- Proposed Consent Form
Reporting Serious Adverse Events for HGT Studies
A Serious Adverse Event (SAE) is defined as:
“An unexpected and related to the intervention, occurring at any dose that results in any of the following outcomes; death, a life-threatening event, in-patient hospitalization or prolongation of existing hospitalization, a persistent or significant disability/incapacity, or a congenital anomaly/birth defect. Important medical events that may not result in death, be life-threatening or require hospitalization also may be considered a serious adverse event when, based upon appropriate medical judgment, they may jeopardize the human gene transfer research subject and may require medical or surgical intervention to prevent one of the outcomes listed in this definition."
Investigators should not await definitive proof of association before reporting such events.
PIs at all sites must report qualifying SAEs to the OBA according to the guidance provided in Appendix M-I-C-4 of the NIH Guidelines. When SAE is fatal or life-threatening, report should not exceed 7 calendar days and for those that are not fatal or life-threatening, report should be submitted not later than 15 calendar days.
Principal Investigators may delegate to another party, such as a corporate sponsor, the reporting functions set forth in Appendix M, with written notification to the NIH OBA of the delegation and of the name(s), address, telephone and fax numbers of the contact(s). The Principal Investigator is responsible for ensuring that the reporting requirements are fulfilled and will be held accountable for any reporting lapses.
In either case, any OBA reports concerning UC enrolled participants should also be submitted to the IRB and IBC. To report an SAE, complete and follow instructions in the UC SAE HGT Form.
UC HGT - IBC form
UC SAE HGT form
NIH – SAE online report
NIH Guidelines – Appendix M
NIH FAQ for HGT Studies
NIH – RAC information
51 Goodman Drive, suite 300 Cincinnati, Ohio 45221
513-558-6182 & 513-558-0332
Marcia Espinola, DVM, MS, CBSP
Director, Biological Safety
Biological Safety Officer
Gary E. Dean, PhD