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

Study Description

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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
Severe Combined Immunodeficiency; Malignancy, Hematologic; Neuroblastoma; Neoplasm; Mucopolysaccharidosis I	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

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Study Type :	Observational
Estimated 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 :	January 4, 2007
Estimated Primary Completion Date :	December 2036
Estimated Study Completion Date :	December 2036

Groups and Cohorts

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Group/Cohort	Intervention/treatment
Participants; Venipuncture	Procedure: Venipuncture; Blood draw; Other Names: Follow-Up Studies; Prospective Studies; Cancer vaccines; Biological Specimen Banks

Outcome Measures

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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). [ 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).

Biospecimen Retention:   Samples With DNA

Blood samples

Eligibility Criteria

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Ages Eligible for Study:	Child, Adult, Senior
Sexes Eligible for Study:	All
Accepts Healthy Volunteers:	No
Sampling Method:	Non-Probability Sample

Study Population

Gene therapy 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

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Study Description Study Design Groups and Cohorts Outcome Measures Eligibility Criteria Contacts and Locations More Information

Contacts

Contact: Stephen Gottschalk, MD	866-278-5833	referralinfo@stjude.org

Locations

United States, Tennessee
St. Jude Children's Research Hospital	Recruiting
Memphis, Tennessee, United States, 38105
Contact: Stephen Gottschalk, MD 866-278-5833 referralinfo@stjude.org
Principal Investigator: Stephen Gottschalk, MD

Sponsors and Collaborators

St. Jude Children's Research Hospital

Investigators

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

More Information

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Study Description Study Design Groups and Cohorts Outcome Measures Eligibility Criteria Contacts and Locations More Information

Additional Information:

St. Jude Children's Research Hospital 

Clinical Trials Open at St. Jude 

Responsible Party:	St. Jude Children's Research Hospital
ClinicalTrials.gov Identifier:	NCT00695279 History of Changes
Other Study ID Numbers:	GENEFU
First Posted:	June 11, 2008
Last Update Posted:	November 20, 2017
Last Verified:	November 2017

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:

Gene therapy

Additional relevant MeSH terms:

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