Long-Term Follow-Up of Recipient of Gene Transfer Research

Sponsor

M.D. Anderson Cancer Center (Other)

Overall Status

Recruiting

CT.gov ID

NCT01492036

Collaborator

National Center for Research Resources (NCRR) (NIH), National Cancer Institute (NCI) (NIH), Alaunos Therapeutics (Industry)

130

Enrollment

Location

192

Anticipated Duration (Months)

0.7

Patients Per Site Per Month

Study Details

Study Description

Brief Summary

The goal of this clinical research study is to collect information about the long-term safety of gene transfer therapy. This study is also designed to create a database of medical information about patients who have received gene transfer therapy, in order for researchers to track and review the long-term safety and any effects (good or bad) of gene transfer therapy.

Condition or Disease	Intervention/Treatment	Phase
Retroviridae Infections Cancer	Other: Blood Tests Behavioral: Questionnaires

Detailed Description

Study Procedures for Years 1-5:

If you agree to take part in this study, you will come to the clinic for study visits once a year during Years 1-5. It is your choice whether to visit your regular local doctor or your study doctor at MD Anderson. (If you visit your regular local doctor, you will need to have your test results and blood samples mailed to the study staff.) You will receive a reminder phone call once a year, when it is time to schedule these visits.

At these visits, the following procedures will be performed:

Blood (about 2-4 tablespoons each time) will be drawn for routine tests.
You will have a physical exam, including measurement of your vital signs (blood pressure, heart rate, temperature, and breathing rate).
You will fill out a questionnaire that asks about your medical history (your general health, the status of any medical conditions, and any illnesses or hospitalizations that may have occurred). It should take about 15-30 minutes to complete.
You will be asked if there have been any changes in your contact information.
If necessary to check the status of the disease, you will have scans (x-rays and/or computed tomography [CT] scans) and/or additional routine blood tests (about 1-2 tablespoons).

Study Procedures for Years 6-15:

Once a year during Years 6-15, the research staff will contact you. Either over the phone, by email, or by mail (but usually by mail, with an enclosed questionnaire), you will be asked about your medical history (including if you have had any children since the infusion) and whether there have been any changes in your contact information. The phone calls and questionnaires should take about 15-30 minutes each time.

Results of Study Testing:

If any of the tests that are performed in this study suggest that you may have a medical condition and/or side effect that is likely to be related to the gene transfer therapy, the study doctor will contact you. You will receive a referral in case you would like to seek medical care for the condition and/or side effect.

You and/or your insurance provider will be responsible for the costs of any CT scans and x-rays that may be needed for this study.

Study Database:

Your medical information that is collected for this study will be stored in a research database at MD Anderson for use in future research related to the safety and effects of gene transfer therapy.

Your data will be given a code number. No identifying information will be directly linked to your data. Only the researcher or authorized personnel in charge of the database will have access to the code numbers and be able to link the data to you. This is to allow medical information related to your data to be updated as needed. Other researchers using your data will not be able to link this data to you.

Changes in Parent/Guardian Contact:

If you are younger than 18 years old when joining this study but turn 18 during this study, starting then, the research staff will contact you directly for this study instead of your parent/guardian. However, if you would prefer for your parent/guardian to still be the main contact person, please tell the study staff.

Length of Study Participation:

After 15 years, your participation in this study will be over unless the FDA decides that follow-up must continue.

This is an investigational study.

Up to 130 patients will take part in this study. All will be enrolled at MD Anderson.

Study Design

Study Type:

Observational

Anticipated Enrollment :

130 participants

Observational Model:

Cohort

Time Perspective:

Prospective

Official Title:

Long-Term Follow-Up Study of Recipients of Gene Transfer Research Protocols

Actual Study Start Date :

Dec 1, 2011

Anticipated Primary Completion Date :

Dec 1, 2026

Anticipated Study Completion Date :

Dec 1, 2027

Arms and Interventions

Arm	Intervention/Treatment
Gene Transfer Therapy Study participants receiving gene therapy product at MD Anderson Cancer Center	Other: Blood Tests If participant received retroviral gene therapy, blood test performed to check for a type of infection called the replication-competent retrovirus (RCR). Blood (up to 4 tablespoons each time) drawn every 3 months for first year after gene transfer therapy and once a year after that. Other Names: Blood draw Behavioral: Questionnaires Once a year during Years 1-5, questionnaire completions that asks about medical history (general health, the status of any medical conditions, and any illnesses or hospitalizations that may have occurred). It should take about 15-30 minutes to complete. Once a year during Years 6-15, completion of mailed questionnaire. Participant will be asked about medical history (including if participant has had any children since the infusion) and whether there have been any changes in contact information. The questionnaires should take about 15-30 minutes each time. Other Names: Surveys

Arm

Intervention/Treatment

Gene Transfer Therapy

Study participants receiving gene therapy product at MD Anderson Cancer Center

Other: Blood Tests

If participant received retroviral gene therapy, blood test performed to check for a type of infection called the replication-competent retrovirus (RCR). Blood (up to 4 tablespoons each time) drawn every 3 months for first year after gene transfer therapy and once a year after that.

Other Names:

Blood draw

Behavioral: Questionnaires

Once a year during Years 1-5, questionnaire completions that asks about medical history (general health, the status of any medical conditions, and any illnesses or hospitalizations that may have occurred). It should take about 15-30 minutes to complete. Once a year during Years 6-15, completion of mailed questionnaire. Participant will be asked about medical history (including if participant has had any children since the infusion) and whether there have been any changes in contact information. The questionnaires should take about 15-30 minutes each time.

Other Names:

Surveys

Outcome Measures

Primary Outcome Measures

Document Long-Term Safety of Gene-Transfer Research [15 years]
First 5 years following infusion of vector or vector-treated cells: Participants evaluated on annual basis. Years 6 to 15 following infusion of vector or vector-treated cells: Participants mailed questionnaires. Participants in retroviral studies: Up to 60 mL (max 1 mL/kg) blood will be obtained to check for replication-competent retrovirus (RCR) every 3 months during the first year, and then blood archived annually thereafter. Replication competent vector testing performed when there is a clinical indication or when requested by the FDA.

Eligibility Criteria

Criteria

Ages Eligible for Study:

N/A and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

Yes

Inclusion Criteria:

Patient or patient's legal representative(s) provided written informed consent to participate in a gene transfer study at the University of Texas MD Anderson Cancer Center (MD Anderson).
Patient will receive vector or vector-treated cells at MD Anderson.
Patient or patient's legal representative(s) is/are able to provide written informed consent to participate in this long-term follow-up protocol for gene transfer participants for up to 15 years following administration of gene therapy product.
Agree to allow clinical samples to be collected and stored at MD Anderson and/or at a NIH designated facility such as the NGVB.