Clinical Trial: Gene Therapy for Chronic Granulomatous Diseases - Long-term Follow-up
Study Status: Completed
Recruit Status: Completed
Study Type: Interventional
Official Title: Gene Therapy Approach for Chronic Granulomatous Disease
Brief 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.
Detailed Summary: This is a Phase I/II clinical trial to determine the efficacy and safety of a method of ex vivo gene therapy to treat both X-linked gp91phox deficient Chronic Granulomatous Disease (CGD) and autosomal recessive p47phox deficient CGD. CGD is an inherited immune deficiency in which blood neutrophils and monocytes fail to produce superoxide and other antimicrobial oxidants, and patients get recurrent life-threatening infections. 30 CGD patients of either sex at least 5 years of age may be enrolled in addition to the 5 patients enrolled in the first phase of this trial. Patients less than 16 years of age must have an active infection or a recent relapse of infection at the time of enrollment. Patients may receive up to 6 cycles of stem cell mobilization for gene therapy at intervals of 4 weeks or longer. For up to first 3 cycles, a cycle of stem cell mobilization for gene therapy will begin with 8 daily subcutaneous injections of the combination of flt3-ligand (flt3L) at 50 micro g/kg/day plus granulocyte-macrophage colony stimulating factor (GM-CSF) at 5 micro g/kg/day for mobilization of CD34+ cells. For 3 or more of the cycles of mobilization of CD34+ cells for gene therapy (up to the maximum of 6 cycles total), the mobilization will begin with 6 daily injections of granulocyte colony stimulating factor (G-CSF) at 10 micro g/kg/day. On the last two or three days of marrow growth factor administration for mobilization of CD34+ cells, the patients will have an apheresis procedure to harvest blood mononuclear cells, thus completing a cycle of mobilization and stem cell harvest. CD34+ progenitors will be selected from the apheresis collection using the Isolex(Registered Trademark) 300i anti-CD34 monoclonal antibody/magnetic bead selection system. The purified CD34+ cells may either be placed directly into culture or may be cryopreserved as per standard blood bank procedure (freezing in autologous serum with 10% dimethylsufoxide {DMSO}) and stored frozen at liquid nitroge
Sponsor: National Institute of Allergy and Infectious Diseases (NIAID)
Current Primary Outcome:
Original Primary Outcome:
Current Secondary Outcome:
Original Secondary Outcome:
Information By: National Institutes of Health Clinical Center (CC)
Dates:
Date Received: November 3, 1999
Date Started: June 1, 1995
Date Completion:
Last Updated: January 24, 2017
Last Verified: December 13, 2010
