NCT00695279

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

Completed Last updated 30 October 2024

What this trial tests

trial testing Venipuncture in Severe Combined Immunodeficiency in 41 participants. Completed in 17 October 2024.

Timeline

4 January 2007

Primary endpoint
17 October 2024

17 October 2024

Quick facts

Lead sponsor	St. Jude Children's Research Hospital
Status	Completed
Study type	OBSERVATIONAL
Enrollment	41
Start date	4 January 2007
Primary completion	17 October 2024
Estimated completion	17 October 2024
Sites	1 location across United States

Drugs / interventions tested

• Venipuncture — full drug profile →

Conditions studied

• Severe Combined Immunodeficiency — all drugs for Severe Combined Immunodeficiency →
• Malignancy, Hematologic — all drugs for Malignancy, Hematologic →
• Neuroblastoma — all drugs for Neuroblastoma →
• Neoplasm — all drugs for Neoplasm →

Sponsor

St. Jude Children's Research Hospital

Who can join

Eligibility, any sex, with Severe Combined Immunodeficiency or Malignancy, Hematologic. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

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.

Publications & conference data

2 peer-reviewed publications reference this trial (live from Europe PMC):

1. Current and Future Prospects for Gene Therapy for Rare Genetic Diseases Affecting the Brain and Spinal Cord.
   Jensen TL, Gøtzsche CR, Woldbye DPD. · · 2021 · cited 53× · PMID 34690692 · DOI 10.3389/fnmol.2021.695937
2. Stem cell manipulation, gene therapy and the risk of cancer stem cell emergence.
   Clément F, Grockowiak E, Zylbersztejn F, Fossard G, et al · · 2017 · cited 22× · PMID 28815178 · DOI 10.21037/sci.2017.07.03