Gene Therapy Follow-up Protocol for People Previously Enrolled in CAR-T Cell Studies

NCT02473757 | Enrolling By Invitation

• 2 other identifiers: 15014115-C-0141
• Study Type: observational
• Target: 1,000
• Locations: 1 country
• Sites: 1

Brief Summary

Background: \- Gene therapy is a way to treat or prevent disease using genes. It is monitored very closely by regulators because there can be long-term, unexpected side effects. NIH is required to try to contact people who have been treated with gene therapy at least annually for up to 15 years. This is to see if they have had any bad side effects. This trial does not include any therapy and is only for patients previously treated on gene-therapy trials at the NCI Surgery Branch who are no longer enrolled on their original gene therapy clinical trial. Objective: \- To collect of long-term follow-up data on people who have been in gene transfer studies. This follow-up is required by regulators. Eligibility: \- People age 18 and older who have been in a previous NCI Surgery Branch gene therapy research study. Design:

• After they get the genetically modified cells, participants will:
• Have blood drawn 3, 6, and 12 months later.
• Have an annual clinic visit for the next 4 years. They will have a physical exam. They will answer questions about any signs of neurological, autoimmune, or blood disorders, or any new cancers. Blood may be drawn.
• Be called or emailed annually for the next 10 years. They will answer health questions. Blood samples may need to be taken.
• Participants will be asked for their current address and phone number. They will also be asked for the address and phone number of 1 or 2 people who will know their whereabouts. One of these should be a family member if possible,
• At the time of the participant s death, researchers will request permission from their family for an autopsy.

Conditions

Lyphoma, B-Cell; Leukemia, B-cell; Multiple Myeloma; Hematologic Malignancies

Keywords

Recombinant DNA Product; Laboratory Evaluation; NIH Office of Biotechnology Activities; Gamma-Retroviral Vectors; Natural History

Outcome Measures

Primary Outcomes (1)

• To provide long term follow up of patients previously enrolled on treatment protocols in the NCI ETIB Branch.

  List of long time adverse event frequency after Gene therapy drug

  15 years

Study Arms (1)

1/Cohort 1

Subjects who have received treatment on an NCI Surgery Branch CAR T-cell gene therapy protocols.

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Probability Sample

Study Population

To facilitate collection of long term follow up information on subjects who have participated in gene transfer studies as required by the U.S. Food and Drug Administration and other regulatory groups

You may qualify if:

• Subjects who have received treatment on a CAR T-cell gene therapy protocol. Age \>= 18 years as children are generally excluded from CAR T-cell gene therapy studies.

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

Leukemia, B-Cell; Multiple Myeloma; Hematologic Neoplasms

Condition Hierarchy (Ancestors)

Leukemia, Lymphoid; Leukemia; Neoplasms by Histologic Type; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases; Lymphoproliferative Disorders; Lymphatic Diseases; Immunoproliferative Disorders; Immune System Diseases; Neoplasms, Plasma Cell; Hemostatic Disorders; Vascular Diseases; Cardiovascular Diseases; Paraproteinemias; Blood Protein Disorders; Hemorrhagic Disorders; Neoplasms by Site

Study Officials

• James N Kochenderfer, 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: June 13, 2015
• First Posted: June 17, 2015
• Study Start: September 24, 2015
• Primary Completion (Estimated): July 1, 2034
• Study Completion (Estimated): August 1, 2050
• Last Updated: December 1, 2025

Record last verified: 2025-11-26

Locations

US (1)