Long Term Follow Up Of Patients Who Have Received Gene Therapy Or Gene Marked Products
- Full Text View
- Tabular View
- No Study Results Posted
- Disclaimer
- How to Read a Study Record
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 |
- 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:
|
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 |
Transplant recipient patients
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 | info@stjude.org |
| 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 | |
| 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: | December 2, 2014 |
| Health Authority: | United States: Institutional Review Board |
Keywords provided by St. Jude Children's Research Hospital:
|
Gene therapy |
Additional relevant MeSH terms:
|
Mucopolysaccharidoses Mucopolysaccharidosis I Severe Combined Immunodeficiency Carbohydrate Metabolism, Inborn Errors Connective Tissue Diseases DNA Repair-Deficiency Disorders Genetic Diseases, Inborn |
Immune System Diseases Immunologic Deficiency Syndromes Infant, Newborn, Diseases Lysosomal Storage Diseases Metabolic Diseases Metabolism, Inborn Errors Mucinoses |
ClinicalTrials.gov processed this record on March 03, 2015