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

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Brief Title

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

Official Title

Long Term Follow Up Of Patients Who Have Received Gene Therapy Or Gene Marked Products at St. Jude Children's Research Hospital

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.
    

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 Type

Observational


Primary Outcome

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).


Condition

Severe Combined Immunodeficiency

Intervention

Venipuncture

Study Arms / Comparison Groups

 Participants
Description:  Venipuncture

Publications

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

Recruitment Information


Recruitment Status

Procedure

Estimated Enrollment

100

Start Date

January 4, 2007

Completion Date

December 2036

Primary Completion Date

December 2036

Eligibility Criteria

        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.
      

Gender

All

Ages

N/A - N/A

Accepts Healthy Volunteers

No

Contacts

Stephen Gottschalk, MD, 866-278-5833, [email protected]

Location Countries

United States

Location Countries

United States

Administrative Informations


NCT ID

NCT00695279

Organization ID

GENEFU


Responsible Party

Sponsor

Study Sponsor

St. Jude Children's Research Hospital


Study Sponsor

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


Verification Date

December 2019