- Gene therapy involves changing the genes inside the body s cells to stop disease. It is
very closely regulated. People who have had this therapy may have problems months or even
years later. Researchers do not know the long-term side effects, so they want to study people
who have had the therapy. They want the study to continue over the next 15 years.
- To study over time the negative side effects from genetically engineered cellular therapy.
This will be studied in people who have been in Pediatric Oncology Branch (POB) gene therapy
- People who are currently or were previously in a research study with gene therapy in the
National Cancer Institute POB.
- Participants blood will be tested right before they get the genetically changed cells.
They will get the cells as part of another study.
- For the next year, they will come back to the clinic or see their doctor at home at
least every 3 months. They will answer questions about their health and blood will be
- For the next 5 years, they will go to the clinic or see their own doctor once a year.
They will have physical exam and blood will be drawn.
- For 10 years after that, they will be asked every year for health information.
- Participants will keep their contact information up to date with researchers. They may
be phoned for more health information.
- If the participant was under 18 years old when given the gene therapy and turns 18
during this follow-up, they will be asked to sign a new consent form when they turn 18.
- Study subjects exposed to gene therapy interventions may be at risk for delayed or long
term adverse consequences. The U.S. Food and Drug Administration (FDA) has issued
Guidance for Industry: Gene Therapy Clinical Trials Observing Participants for Delayed
Adverse Events, which outlines a framework to assess the risk of gene therapy-related
delayed adverse events and the essential elements appropriate for the conduct of
long-term follow up observations1.
- Accordingly some vector designs (i.e. use of an adenovirus), based on duration of
persistence accompanied by cumulative preclinical and clinical evidence, may require
shorter periods of observation than other vector designs (i.e. use of a lentivirus).
- To conduct long term safety evaluations after administration of genetically engineered
cellular therapy to subjects who have participated in POB clinical trials.
- Subjects who have received at least one dose of a genetically engineered cellular
therapy on a POB gene therapy clinical trial are eligible to participate.
- Examples of POB clinical trials and their genetically engineered cellular therapy
products include, but are not limited to: 12-C-0112/anti-CD19 CAR transduced T cells;
11-C-0113/anti-NY-ESO-1 TCR transduced T cells; 14-C-0059/anti-GD2 CAR transduced T
- Subjects will be evaluated for long term safety and occurrence of adverse events
according to the requirements established by the POB gene therapy parent protocol, the
FDA guidance and the NIH Guidelines for Research Involving Recombinant or Synthetic
Nucleic Acid Molecules (NIH Guidelines), to include:
- Long term follow up after genetically engineered cellular therapy:
- Post-cell infusion evaluations for T-Cell persistence (if applicable)
- Monitoring for replication-competent retrovirus (RCR) or replication-competent
- For 5 years: Physical examination with medical history
- For 10 additional years: Complete Questionnaire
- INCLUSION CRITERIA:
- Subjects must be participating in or have participated in a POB gene therapy clinical
trial and have received/or be scheduled to receive a genetically engineered cellular