Gene Therapy for Chronic Granulomatous Diseases - Long-term Follow-up
Status:
Completed
Trial end date:
2010-12-13
Target enrollment:
Participant gender:
Summary
This protocol will follow patients who participated in NIAID's study Gene Therapy Approach
for Chronic Granulomatous Diseases (95-I-0134). No further gene therapy treatments will be
given under this protocol. However, because gene therapy is a new technology and involves a
permanent change in the genetic code of some cells, patients who have had this treatment
require long-term health monitoring.
Participants will be asked to provide updated address and telephone information and the names
of two contact persons, such as siblings or friends. Patients will be seen about once a year
at the NIH Clinical Center to provide an update on their health status and donate a small
blood sample (about 2 teaspoons), which will be frozen and stored. If a patient acquires a
serious illness, such as cancer, his or her stored blood will be tested; another of blood or
tissue sample may also be requested for further study. If a patient develops a medical
problem that is thought possibly to be related to gene therapy, the illness will be
investigated. The annual follow-up visits will continue indefinitely or until the patient
declines to continue participation.
Participants may also agree to store some of their blood future research on chronic
granulomatous diseases and other medical conditions. Stored samples may be labeled with a
code, such as a number, that only the study team can link with the patient. Any identifying
information about the patient will be kept confidential as is permitted by law.
Phase:
Phase 1
Details
Lead Sponsor:
National Institute of Allergy and Infectious Diseases (NIAID)