Gene Therapy Follow up for Subjects Previously Enrolled in NCI Experimental Transplantation and Immunology Branch Studies

Brief Title

Gene Therapy Follow up for Subjects Previously Enrolled in NCI Genitourinary Malignancies Branch or Experimental Transplantation and Immunology Branch Studies

Official Title

Gene Therapy Follow up Protocol for Subjects Previously Enrolled in NCI Genitourinary Malignancies Branch or Experimental Transplantation and Immunology Branch Studies

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
      Experimental Transplantation and Immunology Branch 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 GMB/ETIB 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 GMB or ETIB 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

Primary Outcome

To provide long term follow up of patients previously enrolled on treatment protocols in the NCI ETIB Branch.

Condition

Cervical Intraepithelial Neoplasia

Study Arms / Comparison Groups

 Cohort 1

Description:  Subjects who have received treatment on an NCI GMB/ETIB or GMB gene therapy protocol.

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Estimated Enrollment

1000

Start Date

May 8, 2020

Completion Date

October 1, 2050

Primary Completion Date

October 1, 2035

Eligibility Criteria

        -  INCLUSION CRITERIA:

          -  Subjects who have received gene therapy on an applicable NCI GMB/ETIB protocol. Age
             greater than or equal to 18 years. Note: Children are generally excluded from GMB/ETIB
             gene therapy studies and inclusion on this follow-up study will be limited to 18 years
             and older.

        EXCLUSION CRITERIA:

        -Children
      

Gender

All

Ages

18 Years - N/A

Accepts Healthy Volunteers

No

Contacts

Scott M Norberg, D.O., (240) 760-6163, [email protected]

Location Countries

United States

Location Countries

United States

NCT ID

NCT04266093

Organization ID

200051

Secondary IDs

20-C-0051

Responsible Party

Sponsor

Study Sponsor

National Cancer Institute (NCI)

Study Sponsor

Scott M Norberg, D.O., Principal Investigator, National Cancer Institute (NCI)

Verification Date

December 15, 2020