Human gene transfer is the deliberate transfer into human research participants of either recombinant nucleic acid molecules, or DNA or RNA derived from recombinant nucleic acid molecules, or synthetic nucleic acid molecules.
Human Subjects Research Requiring IBC Approval
All clinical research protocols involving use of investigational biological products (biologics) in human subjects conducted by Weill Cornell Medicine’s (WCM) faculty and staff must be reviewed and approved by both the Institutional Review Board (IRB) and the Institutional Biosafety Committee (IBC) prior to initiation.
Biological agents/products subject to review by the WCM IBC are Investigational New Drugs (INDs) or vaccines containing an agent(s) that resembles a disease-causing microorganism or microbial toxin-producing agent, including: bacteria, viruses, prions, rickettsia, fungi, and parasites that have potential to affect the health of humans.
Section 351 of the Public Health Service (PHS) Act defines a biological product as a “virus, therapeutic serum, toxin, antitoxin, vaccine, blood, blood component or derivative, allergenic product, or analogous product, … applicable to the prevention, treatment, or cure of a disease or condition of human beings.”
Cellular Therapy produts include cellular immunotherapies, cancer vaccines, and other types of both autologous and allogeneic cells for certain therapeutic indications, including hematopoetic stem cells and adult and embryonic stem cells.
Gene therapy and Cellular therapy products are both biological products regulated by the FDA’s Center for Biologics Evaluation and Research (CBER). Clinical studies in humans require the submission of an investigational new drug application (IND) prior to initiating clinical studies in the United States.
Clinical trials involving the use of biological products (biologics) including human gene products-therapies in human subjects requiring the IBC review and approval prior to initiation include but are not limited to the following:
- CAR-T Cell Therapy
- Genetically-Modified Stem Cells
- Induced Pluripotent Stem Cells
- Zinc Finger
Recombinant Oncolytic Viruses
Genes delivered by:
- In vivo or Ex vivo viral vectors such as Adenovirus or Lentivirus or Plasmid DNA
Human Microbiome Studies (Studies Using Human Microbiota)
IBC Submission and Review Process
The IBC submission and review process takes place in the Research Safety module within the Weill Research Getaway (also known as WRG-RS).
Step 1: Select Clinical Safety Application for IBC/RSC - Initial.
Step 2: Enter the protocol title (must be the same as Human Subjects (IRB) protocol).
Step 3: Enter the name of the PI and click Continue. A new window will appear.
Note that your name will appear by default. If you’re entering this on behalf of a PI, please update their name!
Step 4: Click the dropdown menu and choose the application applicable to your protocol:
- IBC Application for Biological Agents in Human Subjects Protocol use this application for vaccine protocols, human microbiome studies (studies using human microbiota).
- IBC Application for Human Gene Transfer Protocol use this application for protocols involving all types of human gene products/therapies/vectors/viruses.
If uncertain which application to use, please contact the IBC at email@example.com or (646) 962-7233.
Step 5: Fill out the IBC Application.
Step 6: Link the IBC Application to corresponding Human Subjects (IRB) protocol within WRG - Human Subjects module.
Step 7: Ensure that the following documents must be available in WRG-HS module:
- Investigator’s Brochure
- Clinical Protocol
- Informed Consent Form(s) Missing documents may impact the IBC review timeline.
Step 8: Click the “Submit” button.
The submission will undergo review by the convened committee, the IBC determines the appropriate biological safety level and issues approval.
NOTE: Additional WCM approvals may be required before study initiation (e.g., Radiation Safety Committee approval, Investigational Pharmacy or Pathology approvals).
Changes that Could Impact the Biosafety Risk Assessment
Any changes that could potentially impact the initial biosafety risk assessment require an amendment to previously approved IBC Application. These changes must be submitted to the IBC for review prior to initiating these changes, following the same submission process within the WRG-RS by creating the amendment to previously approved application.
Amendments that require IBC approval may include, but are not limited to changes in one of the following:
- Changes in the amount or route of biological product administration;
- Changes in the risk associated with biological product;
- Infection control or containment requirements;
- Risk decryption in the informed consent form, how ICF communicates the risks associated with the use of biological product.
The Principal Investigator must submit the adverse events that are (1)“unexpected”, (2) “related” or “possibly related” and associated with the use of the biological product/human gene product or therapy and (3) the event suggests that the research places WCM subjects at greater risk of harm than was previously known or recognized. If ALL of the three (3) conditions are met, submit the Immediate Report in accordance with WCM Immediate Reporting Policy.
Immediate Reports should be sent to the IBC at firstname.lastname@example.org.
The Principal Investigator must submit an annual report to the IBC, which is due on or before the IBC Application expiration date. The protocol's IBC Annual Report must be submitted within the WRG-RS module.
The protocol's IBC Annual Report must include the following information:
- A brief update on the status of the protocol completed during the previous year e.g., (1) total number of participants planned for inclusion in the trial; the number entered into the trial to date; (2) the number whose participation in the trial was completed; and the number who dropped out of the trial with a brief description of the reasons; (4) status of the trial, e.g., open to accrual of subjects, closed but data collection ongoing, or fully completed, and if the trial has been completed, a brief description of any study results.
- Summary of all serious adverse events submitted during the past year associated with the use of the biological agents (1) if any deaths have occurred, the number of participants who died during participation in the investigation and causes of death; and (2) a brief description of any information obtained that is pertinent to an understanding of the biological agents actions, including, for example information about dose-response and whether the laboratory and clinical results have had an impact on biological safety.
- The following documents must be available in WRG-HS module:
Updated Investigator’s Brochure
Updated Clinical Protocol
Updated Informed Consent Form(s)
Clinical and general safety training meets the needs of clinicians and other staff who may be exposed to a bloodborne pathogen or hazardous chemical, but do not work in a laboratory. Participation is required on an annual basis under OSHA regulations for all staff and students who do not work in a laboratory but do come in direct contact with patients; handle clinical specimens; routinely work in a clinical environment; or handle biological (red bag/sharps) waste. This includes graduate students, medical students, physicians, post-doctoral fellows, clinical and housekeeping staff.
Clinical and General Safety training may be completed as follows:
- Attend Clinical and General Safety training session
- Coordinate a small group training session in the clinical area/practice (contact EHS)
- Successful completion of NewYork-Presbyterian Annual Hospital Training (AHT) meets this training requirement.
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