Gene Therapy Follow up Protocol for Subjects Previously Enrolled in NCI Center for Immuno-Oncology Studies

NCT ID: NCT04266093

Last Updated: 2025-11-06

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

ENROLLING_BY_INVITATION

Total Enrollment

1000 participants

Study Classification

OBSERVATIONAL

Study Start Date

2020-05-08

Study Completion Date

2050-10-01

Brief Summary

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Background:

Gene therapy is closely followed by the U.S. Food and Drug Administration (FDA). The FDA requires researchers to conduct long-term follow-up of people who have had the treatment. This study collects data on people who have had gene therapy and sends it to the FDA. The data does not include participants names.

Objective:

To contact current or past participants of gene therapy protocols at least once a year for up to 15 years to ensure they have not had any harmful side effects.

Eligibility:

People aged 18 and older who have had gene therapy in a National Cancer Institute study

Design:

Participants will give their address and telephone number. They will also give and the address and phone number of 1 or 2 other people who will know where they are.

For the first year after gene therapy, participants will give blood samples 3 times (at 3, 6, and 12 months). For the next 4 years, they may have a physical exam and laboratory tests with a home physician. They will get a kit to mail in blood samples. Or they can visit the NIH Clinical Center. They will be asked if they have had any signs of neurological, autoimmune, or blood disorders, or any new cancers.

For years 6 to 15, participants will be contacted yearly via phone or email and asked questions about their health. They may give blood samples.

When the participant dies, if researchers think the death was caused by gene therapy, they will ask the participant s family to allow an autopsy.

Detailed Description

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Background:

The NCI CIO conducts clinical trials utilizing gene transfer. The current 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 protocol is necessary to ensure the necessary 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 NCI CIO treatment protocol for gene therapy.

Design:

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

Conditions

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Cervical Intraepithelial Neoplasia Cervical Neoplasms Oropharyngeal Neoplasms Squamous Intraepithelial Lesions of the Cervix Hematologic Malignacies

Keywords

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Gene Transfer Recombinant DNA Product Gamma-Retroviral Vectors Laboratory Evaluation Natural History

Study Design

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Observational Model Type

COHORT

Study Time Perspective

PROSPECTIVE

Study Groups

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Cohort 1

Subjects who have enrolled on an applicable NCI CIO gene therapy treatment protocol.

GeneTherapy

Intervention Type DRUG

Observation/ Gene Therapy Long-term Follow-up. Subjects who have enrolled on an applicable NCI CIO gene therapy treatment protocol.

Interventions

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GeneTherapy

Observation/ Gene Therapy Long-term Follow-up. Subjects who have enrolled on an applicable NCI CIO gene therapy treatment protocol.

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

* Subjects who have enrolled on an applicable NCI CIO gene therapy treatment protocol. Age \>= 18 years. Note: Children are generally excluded from CIO gene therapy studies and inclusion on this follow-up study will be limited to 18 years and older.

Exclusion Criteria

-Children
Minimum Eligible Age

18 Years

Maximum Eligible Age

120 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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National Cancer Institute (NCI)

NIH

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Scott M Norberg, D.O.

Role: PRINCIPAL_INVESTIGATOR

National Cancer Institute (NCI)

Locations

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National Institutes of Health Clinical Center

Bethesda, Maryland, United States

Site Status

Countries

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United States

Related Links

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Other Identifiers

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20-C-0051

Identifier Type: -

Identifier Source: secondary_id

200051

Identifier Type: -

Identifier Source: org_study_id