Human Research

Clinical trials involving the use of biological products (biologics), including human gene products-therapies in human subjects, requiring the IBC review and approval before initiation, include but are not limited to the following:

Gene Therapy

Cellular Therapy

• CAR-T Cell Therapy
• Genetically-Modified Stem Cells
• Induced Pluripotent Stem Cells

Gene Editing

• CRISPR
• TALEN
• Zinc Finger

Recombinant Oncolytic Viruses

Molecular Immunotherapy

Genes delivered by:

• In vivo or Ex vivo viral vectors such as Adenovirus, Lentivirus, or Plasmid DNA

Vaccine Trials

Human Microbiome Studies (Studies Using Human Microbiota)

The IBC submission and review process occurs within the Research Safety module of 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 the 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 ibc@med.cornell.edu 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

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).

WRG Instructions on how to submit the IBC Application

Instructions on how to obtain access to WRG-RS

Any changes that could potentially impact the initial biosafety risk assessment require an amendment to the previously approved IBC Application. These changes must be submitted to the IBC for review before being initiated, following the same submission process within the WRG-RS by creating an amendment to the 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 the biological product.
• Infection control or containment requirements.
• Risk decryption in the informed consent form, how ICF communicates the risks associated with the use of the biological product.

For more information, refer to the IBC Clinical Research Guide. For further assistance, contact us at ibc@med.cornell.edu or (646) 962-7233.

The Principal Investigator (PI) is required to submit an Immediate Report when all of the following conditions are met:

1. The adverse event is unexpected.

2. The event is related to, or possibly related to, the use of a biological product, human gene product, or therapy.

3. The event suggests that the research exposes WCM subjects to a greater risk of harm than was previously known or recognized.

If all three criteria are satisfied, the PI must submit the report in accordance with the WCM Immediate Reporting Policy.

Once a Serious Adverse Event (SAE) associated with an IBC-approved human subjects protocol has been submitted, the Weill Research Gateway (WRG) will automatically notify the Institutional Biosafety Committee (IBC). A separate submission to the IBC is not required.

The IBC will reach out to the research team if additional clarification or information is needed.

The Principal Investigator must submit an annual report to the IBC, which is due on or before the expiration date of the IBC Application. 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 the 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 bloodborne pathogens or hazardous chemicals, but do not work in a laboratory setting. Participation is required on an annual basis under OSHA regulations for all staff and students who do not work in a laboratory but come into direct contact with patients, handle clinical specimens, work routinely in a clinical environment, or handle biological waste (red bag/sharps). This includes graduate students, medical students, physicians, post-doctoral fellows, and 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.

For additional details on completing the training, please visit this page.

• Gene and Cell Therapy FAQs

Training Resources

• What is Gene Therapy?
• Gene Therapy Basics
• Cell Therapy Basics
• Gene Editing Explained
• Vectors 101

• NIH Office of Science Policy (OSP)
• NIH Guidelines
• Novel and Exceptional Technology and Research Advisory Committee (NExTRAC)
• Human Gene Therapy for Neurodegenerative Diseases
• Gene Therapy Explained
• The American Society of Gene and Cell Therapy
• Expanded Access Protocols Information