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

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.

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Key information

Conditions

Severe Combined Immunodeficiency Carbohydrate Metabolism, Inborn Errors Congenital, Hereditary, and Neonatal Diseases and Abnormalities Connective Tissue Diseases DNA Repair-Deficiency Disorders Genetic Diseases, Inborn Hematologic Diseases Hematologic Neoplasms Hemic and Lymphatic Diseases Immune System Diseases Immunologic Deficiency Syndromes Infant, Newborn, Diseases Lysosomal Storage Diseases Malignancy, Hematologic Metabolic Diseases Metabolism, Inborn Errors Mucinoses Mucopolysaccharidoses Mucopolysaccharidosis I Neoplasm Neoplasms Neoplasms by Histologic Type Neoplasms by Site Neoplasms, Germ Cell and Embryonal Neoplasms, Glandular and Epithelial Neoplasms, Nerve Tissue Neoplasms, Neuroepithelial Neuroblastoma Neuroectodermal Tumors Neuroectodermal Tumors, Primitive Neuroectodermal Tumors, Primitive, Peripheral Nutritional and Metabolic Diseases Primary Immunodeficiency Diseases Skin and Connective Tissue Diseases

Sex eligibility

All sexes

Study type

Observational

Primary location

St. Jude Children's Research Hospital

Memphis, Tennessee, 38105, United States

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

Venipuncture

Procedure

Blood draw

Other names: Follow-Up Studies, Prospective Studies, Cancer vaccines, Biological Specimen Banks

Primary outcomes

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).

Sponsors and collaborators

Lead sponsor

St. Jude Children's Research Hospital

Other

Registry information

Official study title

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

Important dates

Study start: 2007
Primary completion: 2024
Study completion: 2024
First posted: Jun 11, 2008
Registry last updated: Oct 30, 2024

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Published trials that share one or more normalized conditions with this study.

Completed