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

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

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.

Study Overview

• Status: Enrolling by invitation
• Conditions: Multiple Myeloma; Hematologic Malignancies; Leukemia, B-cell; Lyphoma, B-Cell

Detailed Description

Background:

-The National Cancer Institute Surgery Branch (NCI-SB) Branch investigator conducts clinical trials utilizing gene transfer. The current U.S. Food and Drug Administration (FDA) requirements for long term follow up may be up to fifteen years for some products. As this time period is frequently longer than studies are expected to be open, a long-term follow-up protocol is necessary to ensure the follow up of these subjects

Objectives:

-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

Eligibility:

-Enrollment on a CAR T-cell treatment protocol for gene therapy.

Design:

-Patients will undergo physical exams; laboratory evaluation or phone follow up as required by the treatment protocol and/or as clinically indicated.

• Study Type: Observational
• Enrollment (Estimated): 1000

Contacts and Locations

Study Locations

• United States
  • Maryland
    • Bethesda, Maryland, United States, 20892
      • National Institutes of Health Clinical Center

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

• 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

Description

• INCLUSION CRITERIA:
• 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.

Study Plan

How is the study designed?

Design Details

• Observational Models: Cohort
• Time Perspectives: Prospective

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort
1/Cohort 1; Subjects who have received treatment on an NCI Surgery Branch CAR T-cell gene therapy protocols.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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

Collaborators and Investigators

• Sponsor: National Cancer Institute (NCI)
• Investigators: Principal Investigator: James N Kochenderfer, M.D., National Cancer Institute (NCI)

Publications and helpful links

Helpful Links

• NIH Clinical Center Detailed Web Page

Study record dates

Study Major Dates

• Study Start (Actual): September 24, 2015
• Primary Completion (Estimated): July 1, 2034
• Study Completion (Estimated): August 1, 2050

Study Registration Dates

• First Submitted: June 13, 2015
• First Submitted That Met QC Criteria: June 13, 2015
• First Posted (Estimated): June 17, 2015

Study Record Updates

• Last Update Posted (Actual): December 28, 2023
• Last Update Submitted That Met QC Criteria: December 27, 2023
• Last Verified: December 21, 2023

More Information

Terms related to this study

• Keywords: Natural History; Recombinant DNA Product; Gamma-Retroviral Vectors; Laboratory Evaluation; NIH Office of Biotechnology Activities
• Additional Relevant MeSH Terms: Cardiovascular Diseases; Vascular Diseases; Immune System Diseases; Neoplasms by Histologic Type; Neoplasms; Lymphoproliferative Disorders; Lymphatic Diseases; Immunoproliferative Disorders; Neoplasms by Site; Hematologic Diseases; Hemorrhagic Disorders; Hemostatic Disorders; Paraproteinemias; Blood Protein Disorders; Neoplasms, Plasma Cell; Leukemia, Lymphoid; Leukemia; Hematologic Neoplasms; Multiple Myeloma; Leukemia, B-Cell
• Other Study ID Numbers: 150141; 15-C-0141

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No