Long Term Follow Up Of Patients Who Have Received Gene Therapy Or Gene Marked Products

Sponsor
St. Jude Children's Research Hospital (Other)
Overall Status
Recruiting
CT.gov ID
NCT00695279
Collaborator
(none)
100
1
358.9
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Study Details

Study Description

Brief Summary

This protocol (GENEFU) provides a mechanism for the 15-year followup period that the FDA requires for all participants in gene transfer protocols and assures that adequate followup can be maintained for a wide variety of participants on different individual gene therapy protocols at St. Jude Children's Research Hospital.

GENEFU serves as an umbrella protocol for long-term follow-up (LTFU) for recipients of gene therapy/gene marked (GT/GM) products at St. Jude Children's Research Hospital. The FDA has recommended methods to assess the risk of delayed adverse events after GT/GM and has provided specific requirements regarding the duration and design of LTFU observations. This protocol is intended to provide LTFU in accordance with the FDA guidelines for those who received a GT/GM product as part of a St. Jude-sponsored clinical trial or compassionate use treatment plan. The protocol calls for a physical examination or general health evaluation and collection of required blood samples annually for up to 15 years after the last receipt of a GT/GM product.

Goals will be to obtain clinical histories in order to detect late clinical outcomes suggestive of retroviral or lentiviral disease, including but not limited to cancer/second malignancies, neurologic disorders, autoimmune disorders, and hematologic disorders. Blood samples will be archived and tested when clinically or scientifically indicated, as in the event of development of a second malignancy. This prospective cohort study will utilize descriptive statistics in the analysis of long-term late effects outcomes. It offers a uniform approach to long-term safety monitoring in research participants who have received a gene-transduced product as part of St. Jude-sponsored GT or GM protocols and compassionate use treatment plans.

Condition or Disease Intervention/Treatment Phase
  • Procedure: Venipuncture

Detailed Description

Secondary objectives include the following: to obtain and archive blood samples to 1) detect frequency of vector marking in peripheral blood mononuclear cells when clinically or scientifically indicated, such as in the event of participant development of second malignancy, autoimmune disease, hematologic abnormality, or neurologic event, and; 2) To obtain and archive blood samples to detect and characterize clonal proliferation events occurring in gene transfer trials when clinically or scientifically indicated.

Study Design

Study Type:
Observational
Anticipated Enrollment :
100 participants
Observational Model:
Cohort
Time Perspective:
Prospective
Official Title:
Long Term Follow Up Of Patients Who Have Received Gene Therapy Or Gene Marked Products at St. Jude Children's Research Hospital
Actual Study Start Date :
Jan 4, 2007
Anticipated Primary Completion Date :
Dec 1, 2036
Anticipated Study Completion Date :
Dec 1, 2036

Arms and Interventions

Arm Intervention/Treatment
Participants

Venipuncture

Procedure: Venipuncture
Blood draw
Other Names:
  • Follow-Up Studies
  • Prospective Studies
  • Cancer vaccines
  • Biological Specimen Banks
  • Outcome Measures

    Primary Outcome Measures

    1. Obtain histories for detection of significant delayed medical events in research participants who have received an integrating vector-based gene therapy/gene marked product at St. Jude Children's Research Hospital (SJCRH). [30 years]

      Obtain histories for detection of significant delayed medical events including hematologic, malignant, autoimmune, and neurologic events in research participants who have received an integrating vector-based gene therapy/gene marked product at St. Jude Children's Research Hospital (SJCRH).

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    N/A and Older
    Sexes Eligible for Study:
    All
    Accepts Healthy Volunteers:
    No
    Inclusion Criteria:
    • Has received an integrating retroviral or lentiviral vector based GT/GM product at St. Jude Children's Research Hospital within the past 15 year time period. Patient is no later than 15 years post receipt of GT/GM product.

    Contacts and Locations

    Locations

    Site City State Country Postal Code
    1 St. Jude Children's Research Hospital Memphis Tennessee United States 38105

    Sponsors and Collaborators

    • St. Jude Children's Research Hospital

    Investigators

    • Principal Investigator: Stephen Gottschalk, MD, St. Jude Children's Research Hospital

    Study Documents (Full-Text)

    None provided.

    More Information

    Additional Information:

    Publications

    None provided.
    Responsible Party:
    St. Jude Children's Research Hospital
    ClinicalTrials.gov Identifier:
    NCT00695279
    Other Study ID Numbers:
    • GENEFU
    First Posted:
    Jun 11, 2008
    Last Update Posted:
    Dec 14, 2021
    Last Verified:
    Dec 1, 2021
    Studies a U.S. FDA-regulated Drug Product:
    No
    Studies a U.S. FDA-regulated Device Product:
    No
    Keywords provided by St. Jude Children's Research Hospital
    Additional relevant MeSH terms:

    Study Results

    No Results Posted as of Dec 14, 2021