Long Term Follow Up Of Patients Who Have Received Gene Therapy Or Gene Marked Products
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT00695279|
Recruitment Status : Recruiting
First Posted : June 11, 2008
Last Update Posted : November 27, 2018
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|
|Study Type :||Observational|
|Estimated Enrollment :||100 participants|
|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|
- 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
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00695279
|Contact: Stephen Gottschalk, MDfirstname.lastname@example.org|
|United States, Tennessee|
|St. Jude Children's Research Hospital||Recruiting|
|Memphis, Tennessee, United States, 38105|
|Contact: Stephen Gottschalk, MD 866-278-5833 email@example.com|
|Principal Investigator: Stephen Gottschalk, MD|
|Principal Investigator:||Stephen Gottschalk, MD||St. Jude Children's Research Hospital|