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

This study is currently recruiting participants. (see Contacts and Locations)
Verified May 2014 by St. Jude Children's Research Hospital
Sponsor:
Information provided by (Responsible Party):
St. Jude Children's Research Hospital
ClinicalTrials.gov Identifier:
NCT00695279
First received: June 9, 2008
Last updated: May 21, 2014
Last verified: May 2014
  Purpose

This protocol 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 Intervention
Severe Combined Immunodeficiency
Malignancy, Hematologic
Neuroblastoma
Neoplasm
Mucopolysaccharidosis I
Procedure: Venipuncture

Study Type: Observational
Study Design: 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

Resource links provided by NLM:


Further study details as provided by St. Jude Children's Research Hospital:

Primary Outcome Measures:
  • Obtain histories for detection of significant delayed medical events 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 ] [ Designated as safety issue: No ]
    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).


Biospecimen Retention:   Samples With DNA

Blood samples


Estimated Enrollment: 100
Study Start Date: December 2006
Estimated Study Completion Date: December 2036
Estimated Primary Completion Date: December 2036 (Final data collection date for primary outcome measure)
Groups/Cohorts Assigned Interventions
1 Procedure: Venipuncture
Blood draw
Other Names:
  • Follow-Up Studies
  • Prospective Studies
  • Cancer vaccines
  • Biological Specimen Banks

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.

  Eligibility

Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population

Transplant recipient patients

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.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00695279

Contacts
Contact: Brian Sorrentino, MD 1-866-278-5833 info@stjude.org

Locations
United States, Tennessee
St. Jude Children's Research Hospital Recruiting
Memphis, Tennessee, United States, 38105
Contact: Brian Sorrentino, MD    866-278-5833    info@stjude.org   
Principal Investigator: Brian Sorrentino, MD         
Sponsors and Collaborators
St. Jude Children's Research Hospital
Investigators
Principal Investigator: Brian Sorrentino, MD St. Jude Children's Research Hospital
  More Information

Additional Information:
No publications provided

Responsible Party: St. Jude Children's Research Hospital
ClinicalTrials.gov Identifier: NCT00695279     History of Changes
Other Study ID Numbers: GENEFU
Study First Received: June 9, 2008
Last Updated: May 21, 2014
Health Authority: United States: Institutional Review Board

Keywords provided by St. Jude Children's Research Hospital:
Gene therapy

Additional relevant MeSH terms:
Immunologic Deficiency Syndromes
Mucopolysaccharidoses
Mucopolysaccharidosis I
Neuroblastoma
Severe Combined Immunodeficiency
Carbohydrate Metabolism, Inborn Errors
Connective Tissue Diseases
DNA Repair-Deficiency Disorders
Genetic Diseases, Inborn
Immune System Diseases
Infant, Newborn, Diseases
Lysosomal Storage Diseases
Metabolic Diseases
Metabolism, Inborn Errors
Mucinoses
Neoplasms
Neoplasms by Histologic Type
Neoplasms, Germ Cell and Embryonal
Neoplasms, Glandular and Epithelial
Neoplasms, Nerve Tissue
Neoplasms, Neuroepithelial
Neuroectodermal Tumors
Neuroectodermal Tumors, Primitive
Neuroectodermal Tumors, Primitive, Peripheral

ClinicalTrials.gov processed this record on November 23, 2014