Follow-Up Evaluation for Gene-Therapy-Related Delayed Adverse Events After Participation in Pediatric Oncology Branch Clinical Trials

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT02315599
Recruitment Status : Active, not recruiting
First Posted : December 12, 2014
Last Update Posted : July 2, 2018
Information provided by (Responsible Party):
National Institutes of Health Clinical Center (CC) ( National Cancer Institute (NCI) )

Brief Summary:


- 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 trials.


- 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 drawn.
  • 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.

Condition or disease
Pediatric Cancers Hematologic Malignancies Solid Tumors

Detailed Description:


  • 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 cells.


  • 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 lentivirus (RCL)
  • For 5 years: Physical examination with medical history
  • For 10 additional years: Complete Questionnaire

Study Type : Observational
Actual Enrollment : 63 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: Follow-Up Evaluation for Gene-Therapy Related Delayed Adverse Events After Participation in Pediatric Oncology Branch Clinical Trials
Study Start Date : December 11, 2014
Estimated Primary Completion Date : August 1, 2034
Estimated Study Completion Date : August 1, 2050

Resource links provided by the National Library of Medicine

Primary Outcome Measures :
  1. Conduct long term safety evaluations after gene therapy [ Time Frame: Every 3 months X 1 year then annually X 15 years ]

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

Ages Eligible for Study:   1 Year to 99 Years   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
  • 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 therapy.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT02315599

United States, Maryland
National Institutes of Health Clinical Center, 9000 Rockville Pike
Bethesda, Maryland, United States, 20892
Sponsors and Collaborators
National Cancer Institute (NCI)
Principal Investigator: Terry J Fry, M.D. National Cancer Institute (NCI)

Additional Information:
Responsible Party: National Cancer Institute (NCI) Identifier: NCT02315599     History of Changes
Other Study ID Numbers: 150028
First Posted: December 12, 2014    Key Record Dates
Last Update Posted: July 2, 2018
Last Verified: June 28, 2018

Keywords provided by National Institutes of Health Clinical Center (CC) ( National Cancer Institute (NCI) ):
Replication Competent Retrovirus (RCR)
Replication Competent Lentivirus (RCL)
T Cell Persistence