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

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.

Study Overview

• Status: Enrolling by invitation
• Conditions: Cervical Intraepithelial Neoplasia; Oropharyngeal Neoplasms; Squamous Intraepithelial Lesions of the Cervix; Cervical Neoplasms; Hematologic Malignacies
• Intervention / Treatment: Drug: GeneTherapy

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 Type: Observational
• Enrollment (Estimated): 1000

Contacts and Locations

Study Locations

• United States
  • Maryland
    • Bethesda, Maryland, United States, 20892
      • National Institutes of Health Clinical Center

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

• 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

Description

• INCLUSION CRITERIA:
• Subjects who have received gene therapy on an applicable NCI CIO 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

Study Plan

How is the study designed?

Design Details

• Observational Models: Cohort
• Time Perspectives: Prospective

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Cohort 1; Subjects who have received treatment on an NCI CIO or CIO gene therapy protocol.	Drug: GeneTherapy; Observation/ Gene Therapy Long-term Follow-up. Subjects who have received treatment on an applicable NCI CIO gene therapy protocol.

What is the study measuring?

Primary Outcome Measures

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

Collaborators and Investigators

• Sponsor: National Cancer Institute (NCI)
• Investigators: Principal Investigator: Scott M Norberg, D.O., National Cancer Institute (NCI)

Publications and helpful links

Helpful Links

• NIH Clinical Center Detailed Web Page

Study record dates

Study Major Dates

• Study Start (Actual): May 8, 2020
• Primary Completion (Estimated): October 1, 2035
• Study Completion (Estimated): October 1, 2050

Study Registration Dates

• First Submitted: February 11, 2020
• First Submitted That Met QC Criteria: February 11, 2020
• First Posted (Actual): February 12, 2020

Study Record Updates

• Last Update Posted (Actual): April 17, 2024
• Last Update Submitted That Met QC Criteria: April 16, 2024
• Last Verified: November 14, 2023

More Information

Terms related to this study

• Keywords: Natural History; Gene Transfer; Recombinant DNA Product; Gamma-Retroviral Vectors; Laboratory Evaluation
• Additional Relevant MeSH Terms: Urogenital Neoplasms; Neoplasms by Site; Uterine Neoplasms; Genital Neoplasms, Female; Uterine Cervical Diseases; Uterine Diseases; Pharyngeal Neoplasms; Otorhinolaryngologic Neoplasms; Head and Neck Neoplasms; Pharyngeal Diseases; Stomatognathic Diseases; Otorhinolaryngologic Diseases; Precancerous Conditions; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Genital Diseases; Genital Diseases, Female; Neoplasms; Uterine Cervical Neoplasms; Uterine Cervical Dysplasia; Oropharyngeal Neoplasms; Squamous Intraepithelial Lesions of the Cervix
• Other Study ID Numbers: 200051; 20-C-0051

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No