Long-Term Follow-Up Study of Recipients of Gene Transfer Research Protocols
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 M. D. 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
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.
Blood Tests for RCR:
The study doctor will tell you if the type of gene transfer therapy you received was called
"retroviral" therapy. If so, you will have an additional blood test performed to check to
make sure you do not have a type of infection called the replication-competent retrovirus
(RCR). For this test, blood (up to 4 tablespoons each time) will be drawn every 3 months
for the first year after your gene transfer therapy and once a year after that. (If you
join this study more than a year after your gene transfer therapy, however, this RCR test
will be performed once a year for all 15 years of the study, rather than your having the
blood draws every 3 months in Year 1.)
For these RCR blood tests, it is your choice whether to visit your regular local doctor,
your study doctor at M. D. Anderson. If you visit your regular local doctor, you will need
to have your test results and blood samples mailed to either the National Gene Vector
Biorepository and Coordinating Center (NGVB) or M. D. Anderson. The study staff will tell
you where to have the results and samples mailed, and you will receive a kit with supplies
for mailing the blood samples.
If the RCR test results during Year 1 of the study show that you do not have the RCR
infection, the rest of your leftover blood samples (left over from RCR testing in Years
2-15) will be stored at M. D. Anderson for safety reasons. This is so researchers can study
any changes in your blood (related to RCR) that may arise in Years 2-15.
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. (This includes, for example, the RCR infection described
above). You will receive a referral in case you would like to seek medical care for the
condition and/or side effect.
Your medical information that is collected for this study will be stored in a research
database at M. D. 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 100 patients will take part in this study. All will be enrolled at M. D. Anderson.
Observational Model: Cohort, Time Perspective: Prospective
Document Long-Term Safety of Gene-Transfer Research
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.
Laurence JN Cooper, MD, PHD
UT MD Anderson Cancer Center
United States: Institutional Review Board
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