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

• Conditions: Cervical Intraepithelial Neoplasia; Cervical Neoplasms; Oropharyngeal Neoplasms; Squamous Intraepithelial Lesions of the Cervix; Hematologic Malignacies
• Interventions: Drug: GeneTherapy

• Registration Number: NCT04266093
• Lead Sponsor: National Cancer Institute (NCI)

Brief Summary

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

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.

Study Timeline

• Study First Submitted: 2020-02-11
• Study First Submitted QC: 2020-02-11
• Study First Posted: 2020-02-12
• Study Start: 2020-05-08
• Status Verified: 2025-03-11
• Last Update Submitted: 2025-05-17
• Last Update Posted: 2025-05-20
• Primary Completion: 2035-10-01
• Study Completion: 2050-10-01

Recruitment & Eligibility

• Status: ENROLLING_BY_INVITATION
• Sex: All
• Target Recruitment: 1000

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Cohort 1	GeneTherapy	Subjects who have enrolled on an applicable NCI CIO gene therapy treatment protocol.

Primary Outcome Measures

Name	Time	Method
To provide long term follow up of patients previously enrolled on treatment protocols in the NCI CIO.	15 years	List of long time adverse event frequency after Gene therapy drug

Trial Locations

Locations (1)

National Institutes of Health Clinical Center; 🇺🇸 Bethesda, Maryland, United States