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Human Subjects Research

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

Deliberate transfer of recombinant or 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
  • Change the genetic makeup of the cell either temporarily or permanently 

Human gene transfer protocols are subject to dual review by the IBC and the Institutional Review Board (IRB). The primary focus of the IBC is to assess risk posed by the proposed gene transfer to other humans or the environment, and to define appropriate containment conditions based on those risks. No research participant shall be enrolled until the IBC, IRB approvals have been obtained and the NIH protocol registration process has been completed.

Types of Human Gene Transfer (HGT):

  • Gene Therapy
  • Cellular Therapy
    • CAR-T Cell therapy
    • Genetically-modified stem cells
    • Induced pluripotent stem cells
  • Gene Ending
    • CRISPR
    • TALEN
    • Zinc Finger
  • Recombinant Oncolytic Viruses
  • Molecular Immunotherapy
  • Genes delivered by:
    • In vivo or ex vivo viral vectors such as adenovirus or lentivirus
    • Plasmid DNA

How to Obtain HGT Approval

The IBC and IRB submission process vary depending on whether the study is a multi-site or single-site study. The process will also vary depending on whether it is determined that review by the NIH RAC is needed.

Instructions on how to obtain IBC approval, submit amendments, annual reports, adverse events and protocol deviations can be found in the Guide, Conducting Human Gene Transfer Research at WCM. For additional assistance, please contact us at ibc@med.cornell.edu  or (646) 962-7233.

Biological agents as defined by IBC are: non-FDA approved 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.

Research involving work with biological agents in human subjects at WCM is subject to dual review by the IBC and the Institutional Review Board (IRB).

Examples of biological agents/therapies used in the research with human subjects at the WCM that are subject to IBC review and approval include:

  1. Vaccine trials
  2. Human microbiome studies (studies using human microbiota)

How to Obtain Biological Agent Approval

To obtain IBC approval for use of Biological Agents in Human Subject Research, take the following steps:

Step 1: Submit your protocol to the IRB according to IRB submission procedures. See the IRB FAQ and IRB website for instructions.

Step 2: Resolve all protocol and informed consent issues that the IRB has identified, before you submit to the IBC. The only remaining IRB issues should be the missing IBC approval letter and administrative issues that would not impact the IBC's review.

Step 3: Submit all of the following documents to the IBC via submit2ibc@med.cornell.edu for review:

  • Cover letter stating the contents of your submission
  • IBC Application for the Use of Biological Agents in Human Subjects Research, outlining the safe handling, receipt, packaging, storage, aliquot preparation, transport, administration, and disposal of the biological agents to be administered
  • IRB Protocol
  • Informed Consent Form(s)
  • Investigator’s Brochure

The submission will undergo review by the convened committee, the IBC determines the appropriate biological safety level and issues approval.

Changes that Could Impact the Biosafety Risk Assessment

IBC applicable amendments, any changes that could potentially impact the initial biosafety risk assessment must be submitted to the IBC for review prior to initiating these changes, following the same process, including any altered documentation as clean and highlighted copies.

For more information refer to our Guide, Use of Biological Agents in Human Subjects Research at WCM. For further assistance contact us at ibc@med.cornell.edu or (646) 962-7233.

Adverse Events and Protocol Deviations

The principal investigator must submit all adverse events and protocol deviations in accordance with WCM Immediate Reporting Policy. Immediate Reports should be sent to the IBC at submit2ibc@med.cornell.edu.

Annual Review

The PI must submit an annual review to the IBC, which is due on or before the IRB Continuing Review expiration date. The protocol's IBC Annual Report should be submitted to submit2ibc@med.cornell.edu and must include a letter providing 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.
  • Copy of the updated IRB protocol
  • Copy of the updated clinical protocol
  • Copy of the updated Investigator’s Brochure

Clinical Training

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 Hospital Annual Hospital Training meets this training requirement
  • Complete on-line Clinical and General Safety Refresher Training

EHS Website/ Lab Training

NIH Office of Science Policy (OSP)

NIH Guidelines

Recombinant DNA Advisory Committee (RAC) 

The NIH Review Process for Human Gene Transfer Trials

The NIH Guidelines Vaccine Exemption

Clinical Resources

Additional NIH HGT Resources

Gene Therapy Explained

The American Society of Gene and Cell Therapy

Clinical FAQ

Gene and Cell Therapy FAQs

NIH Review Process for Human Gene Transfer Trials

Contact IBC