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Long Term Follow Up Of Patients Who Have Received Gene Therapy Or Gene Marked Products
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Purpose
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 | 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 |
- 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
| Estimated Enrollment: | 100 |
| Actual Study Start Date: | January 4, 2007 |
| Estimated Study Completion Date: | December 31, 2036 |
| Estimated Primary Completion Date: | December 31, 2036 (Final data collection date for primary outcome measure) |
| Groups/Cohorts | Assigned Interventions |
|---|---|
|
Participants
Venipuncture
|
Procedure: Venipuncture
Blood draw
Other Names:
|
Detailed Description:
Eligibility| Ages Eligible for Study: | Child, Adult, Senior |
| Sexes Eligible for Study: | All |
| Accepts Healthy Volunteers: | No |
| Sampling Method: | Non-Probability Sample |
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 LocationsPlease refer to this study by its ClinicalTrials.gov identifier: NCT00695279
| Contact: Brian Sorrentino, MD | 1-866-278-5833 | referralinfo@stjude.org |
| United States, Tennessee | |
| St. Jude Children's Research Hospital | Recruiting |
| Memphis, Tennessee, United States, 38105 | |
| Contact: Brian Sorrentino, MD 866-278-5833 referralinfo@stjude.org | |
| Principal Investigator: Brian Sorrentino, MD | |
| Principal Investigator: | Brian Sorrentino, MD | St. Jude Children's Research Hospital |
More Information
Additional Information:
| 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: | March 31, 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 |
ClinicalTrials.gov processed this record on September 21, 2017