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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT00695279
Recruitment Status : Recruiting
First Posted : June 11, 2008
Last Update Posted : December 10, 2019
Sponsor:
Information provided by (Responsible Party):
St. Jude Children's Research Hospital

Tracking Information
First Submitted Date June 9, 2008
First Posted Date June 11, 2008
Last Update Posted Date December 10, 2019
Actual Study Start Date January 4, 2007
Estimated Primary Completion Date December 2036   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures
 (submitted: July 19, 2016)
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). [ Time Frame: 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).
Original Primary Outcome Measures
 (submitted: June 9, 2008)
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 SJCRH. [ Time Frame: 30 years ]
Change History
Current Secondary Outcome Measures Not Provided
Original Secondary Outcome Measures Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
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
Study Design Observational Model: Cohort
Time Perspective: Prospective
Target Follow-Up Duration Not Provided
Biospecimen Retention:   Samples With DNA
Description:
Blood samples
Sampling Method Non-Probability Sample
Study Population Gene therapy recipient patients
Condition
  • Severe Combined Immunodeficiency
  • Malignancy, Hematologic
  • Neuroblastoma
  • Neoplasm
  • Mucopolysaccharidosis I
Intervention Procedure: Venipuncture
Blood draw
Other Names:
  • Follow-Up Studies
  • Prospective Studies
  • Cancer vaccines
  • Biological Specimen Banks
Study Groups/Cohorts Participants
Venipuncture
Intervention: Procedure: Venipuncture
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status Recruiting
Estimated Enrollment
 (submitted: June 9, 2008)
100
Original Estimated Enrollment Same as current
Estimated Study Completion Date December 2036
Estimated Primary Completion Date December 2036   (Final data collection date for primary outcome measure)
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.
Sex/Gender
Sexes Eligible for Study: All
Ages Child, Adult, Older Adult
Accepts Healthy Volunteers No
Contacts
Contact: Stephen Gottschalk, MD 866-278-5833 referralinfo@stjude.org
Listed Location Countries United States
Removed Location Countries  
 
Administrative Information
NCT Number NCT00695279
Other Study ID Numbers GENEFU
Has Data Monitoring Committee No
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement Not Provided
Responsible Party St. Jude Children's Research Hospital
Study Sponsor St. Jude Children's Research Hospital
Collaborators Not Provided
Investigators
Principal Investigator: Stephen Gottschalk, MD St. Jude Children's Research Hospital
PRS Account St. Jude Children's Research Hospital
Verification Date December 2019