Guidance on Human Gene Transfer Clinical Trials Under IBC Oversight

March 25, 2020

We are providing this guidance for principal investigators and clinical staff involved with human gene transfer clinical trials. Your Institutional Biosafety Committee (IBC) plays a critical role in helping assure your human gene transfer clinical trials are conducted in compliance with the NIH Guidelines for Research Involving Recombinant or Synthetic Nucleic Acid Molecules (the NIH Guidelines), and that the recombinant study agents are handled in a manner that assures the safety of the pharmacy staff, clinical staff, other patients, and visitors to your clinic or hospital.

We realize that clinics and hospitals around the country are needing to evolve their practices, facilities, and priorities in response to the expanding COVID-19 pandemic. In many cases this includes coping with a shortage of personal protective equipment, a shortage of trained staff, and the need to reallocate physical facilities.

With this in mind, we are providing you with the following guidance:

  1. Your IBC approved the use of specific facilities, practices, and personal protective equipment to be used when handling the study agent associated with each human gene transfer clinical trial. The NIH Guidelines require that you comply with these determinations.
  2. If you are not able to comply with these determinations, we encourage you to consider postponing enrollment in your human gene transfer clinical trial until such time that the necessary resources are again available.
  3. If you wish to change the facilities, practices, or personal protection equipment associated with your human gene transfer clinical trial, please contact Clinical Biosafety Services. We can help with an initial assessment and schedule a convened meeting of your IBC to review the proposed change(s). Please keep in mind that, in most cases, your IBC will need to review and approve such changes before they may be implemented.
  4. We recognize that clinical trial integrity, and the safety of individual trial participants, are important. If you feel that it is necessary to deviate from the determinations of your IBC in order to continuing dosing of trial participants that have already enrolled in your human gene transfer clinical trial, please notify CBS immediately so that we can provide advice regarding your current circumstances, provide advice regarding reporting requirements, and schedule a convened meeting of your IBC in order to review the situation and consider alternative facilities, practices, and personal protective equipment.
  5. Please note that deviations from facilities, practices, and personal protective equipment without prior review and approval by your IBC may need to be reported to the National Institutes of Health as unapproved research. CBS staff have experience with such reporting, and we are here to help assist you if reporting is required.

We appreciate your attention to this guidance, and are ready to help with your biosafety and regulatory needs during these difficult times. If you need assistance with any of the issues described above, or would like to discuss your current circumstances with a biosafety professional, we can be reached at:

Clinical Biosafety Services
Phone: (888) 442-2472

Mission Statement

Our mission and expertise at Clinical Biosafety Services is to provide accelerated, compliant review of gene transfer/gene therapy clinical trials to a convened review of a site-specific Institutional Biosafety Committee (IBC). We do this by understanding the challenges in the two client relationship of serving an institution and a sponsor.

Our focus is to accelerate the trial, and remove regulatory burden and uncertainty by having our highly trained experts provide friendly, knowledgeable support during the review process. We understand these therapeutics will change medicine, and our service is to bring the trial to enrollment fruition, quickly and compliantly.