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

NCT02315599 | Enrolling By Invitation FDA Drug

• 2 other identifiers: 15002815-C-0028
• Study Type: observational
• Target: 500
• Locations: 1 country
• Sites: 1

Brief Summary

Background: \- 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. Objective: \- 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. Eligibility: \- People who are currently or were previously in a research study with gene therapy in the National Cancer Institute POB. Design:

• 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.

Conditions

Hematologic Malignancies; Pediatric Cancers; Solid Tumors

Keywords

Replication Competent Retrovirus (RCR); Replication Competent Lentivirus (RCL); T Cell Persistence; Natural History

Outcome Measures

Primary Outcomes (1)

• Conduct long term safety evaluations after gene therapy

  PCR results (for RCR/RCL, gene transduced cells)

  Every 3 months X 1 year then annually X 15 years

Study Arms (1)

1

Patients screening for, 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.

Drug: anti-CD19 CAR

Interventions

anti-CD19 CAR DRUG

1

Eligibility Criteria

• Age: 1 Year - 99 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Participants screening for, 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.

You may qualify if:

• Participants must be identified eligible for, participating in, or have participated in a POB genetically engineered cellular therapy clinical trial and have received/or be scheduled to receive a genetically engineered cellular therapy.
• All monitoring and testing described in this protocol will pertain ONLY to genetically engineered cellular therapy received at the National Institutes of Health (NIH). RCR/RCL monitoring and persistence will NOT be followed for cells administered at another institution.

Sponsors & Collaborators

• National Cancer Institute (NCI): lead

Study Sites (1)

National Institutes of Health Clinical Center

Bethesda, Maryland, 20892, United States

Location

Related Links

• NIH Clinical Center Detailed Web Page

MeSH Terms

Conditions

Hematologic Neoplasms

Condition Hierarchy (Ancestors)

Neoplasms by Site; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases

Study Officials

• John W Glod, M.D.

  National Cancer Institute (NCI)

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: NIH
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 11, 2014
• First Posted: December 12, 2014
• Study Start: December 23, 2014
• Primary Completion (Estimated): April 1, 2035
• Study Completion (Estimated): August 1, 2050
• Last Updated: May 6, 2026

Record last verified: 2026-05-01

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, ICF
• Time Frame: Clinical data available during the study and indefinitely.
• Access Criteria: Clinical data will be made available via subscription to BTRIS and with the permission of the study PI.

Locations

US (1)