Clinical trial NCT02473757: Gene Therapy Follow-up Protocol for People Previously Enrolled in CAR-T Cell Studies
Official title: Gene Therapy Follow up Protocol for Subjects Previously Enrolled in CAR T-Cell Studies
Study type: OBSERVATIONAL
Overall status: ENROLLING_BY_INVITATION
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
Lead sponsor: National Institutes of Health Clinical Center (CC)
Sponsor class: NIH
First posted: 2015-06-17
Enrollment: 1000
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
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

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.

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.

Eligibility criteria: * 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.

Primary outcomes:
measure: To provide long term follow up of patients previously enrolled on treatment protocols in the NCI ETIB Branch.
timeFrame: 15 years
Secondary outcomes:
Other outcomes:
Locations:
city: Bethesda
country: United States
facility: National Institutes of Health Clinical Center
state: Maryland
Study officials:
affiliation: National Cancer Institute (NCI)
name: James N Kochenderfer, M.D.
role: PRINCIPAL_INVESTIGATOR
References: