Procedures for Human Gene Transfer (HGT) Research at UVA
This document contains instructions for obtaining approval to conduct HGT research at UVA including submission, oversight and reporting responsibilities. Please note that the deliberate transfer of recombinant or synthetic nucleic acids into one human research participant, conducted under an FDA-regulated individual patient expanded access IND or protocol, including for emergency use, is not research subject to the NIH Guidelines and thus does not need to be submitted to an Institutional Biosafety Committee for review and approval.
I. Principal Investigator (PI) responsibilities for Industry sponsored Protocols
- Complete the IBC Inventory and Activity Registration (IAR) at http://ehs.virginia.edu/biosafety/.
- Provide the protocol, consents and all sponsor related materials to the IBC. Prior consultation with the IBC is strongly advised.
- Submit the protocol, consents and all additional required documents to the IRB of record (Note: this may be done simultaneously with IBC registration).
II. PI responsibilities for Investigator Initiated HGT Protocols
- Write the protocol using the template found on the IRB website (Full Board or NCI CTEP templates), or similar template and submit the protocol and consent form(s) to the IBC for review.
- Complete the IBC IAR at http://ehs.virginia.edu/biosafety/. Prior consultation with the IBC is strongly advised.
- After IBC approval, submit the protocol to the IRB.
- After receiving the IRB approval submit the application to the FDA. The PI must notify the IRB 30 days following the FDA submission if the Investigational New Drug Application (IND) has not been placed on clinical hold. At that time, the IRB will grant approval to enroll subjects.
III. Details of the approval process and compliance committee responsibilities:
The IBC will:
- Assess risks to the environment and the necessity to inform medical care personnel, (e.g., nurses, technicians), of risks, precautions, and recommended health surveillance procedures.
- Notify the SoM Dean about the proposed work and request, if necessary, additional expertise to review the protocol. The Dean may request an Ad Hoc Committee.
- Review and approve the IBC registration document.
- Confirm that key personnel have completed the appropriate biosafety training.
- Perform initial and regularly scheduled inspections of facilities where gene therapy material will be handled and certify appropriate work practices and physical controls.
Ad hoc scientific review committee may be appointed by the SOM Dean or the IBC to:
- Assess the scientific and medical relevance and merit of the protocol.
- Assess the risk/benefits to subjects and others.
- Review the existing knowledge of the disease state and current therapy, route of administration, dose, and vector virulence.
- Recommend information to be provided to family and other caretakers pertaining to potential risks and precautions to be stated in either a separate document or in the consent form.
- Recommend additional information the IRB may wish to have included in the consent form pertaining to potential risks to subjects.
- Recommend additional information the IBC may wish to have included in the Biosafety Manual.
- Communicate assessments to the IBC, IRB, VPR, and PI.
IRB of Record will:
- Receive approvals from all other committees or bodies and confirm that all modifications requested by other review committees are incorporated into all final documents.
- Review the protocol, consent and other applicable documents and grant approval per all applicable regulations.
- Grant approval to enroll subjects 30 days after submission of the protocol to the FDA unless the FDA has placed a clinical hold on the protocol.
- If the IRB-HSR is not the IRB of Record, the IRB-HSR will notify the Office of the Vice President for Research after the IRB approval is granted. The VPR may ask questions, comment, or place a hold on the protocol.
Additional approvals may be required from the UVA School of Medicine Clinical Trials Office or a Scientific Review Committee
Additional information regarding the review committees or bodies may be found on their Web sites:
- NIH Office of Science Policy: http://osp.od.nih.gov
- IRB-HSR: http://www.virginia.edu/vpr/irb/hsr/index.html
- Institutional Biosafety Committee: http://ehs.virginia.edu/Biosafety-IBC.html
- Cancer Center Protocol Review Committee: https://uvahealth.com/services/cancer/research/clinical-research-infrastructure/office-for-clinical-research/protocol-review-committee/protocol-review-committee
- School of Medicine Clinical Trials Office: http://www.medicine.virginia.edu/research/offices/CTO
- UVA Pharmacy Services (intranet): http://www.medicalcenter.virginia.edu/intranet/pharmacy/
IV. PI Responsibilities for all HGT protocols after Protocol is Open to Enrollment
The PI is responsible for full compliance with the NIH Guidelines.
The PI will:
- Submit unanticipated problems, serious events, protocol violations, research-related accidents and illnesses to the IRB, IBC, Biosafety Officer, NIH OSP, and other appropriate authorities within 30 days.
- Submit all modifications/amendments to the IBC and then to the IRB of Record.
- Submit the following as applicable:
- Continuation Status Forms to the IRB of Record,
- IND annual reports to the FDA,
- Any correspondence with the FDA to IRB of Record, IBC and, if applicable, the Scientific Review Committee as required.
- Update the IBC registration annually.