Gene Therapy for Chronic Granulomatous Diseases - Long-term Follow-up

Completed · Phase 1

Conditions studied: Chronic Granulomatous Disease, Communicable Disease

In brief

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.

Key facts

Study ID

NCT00001476

Run by

National Institute of Allergy and Infectious Diseases (NIAID)

People needed

14

Starts

1995-06-01

Expected to finish

2010-12-13

Last updated by the study team

2017-07-02

Who can join

Age: 5 and older. Sex: any. Healthy volunteers: not accepted.

You may not qualify if…

• Female patients who are pregnant or lactating as determined by history and/or positive pregnancy test.
• While patients may have an active infection under treatment and still be included in the study, patients are excluded who are in shock, manifested by severe hypotension (less than 100 systolic or less than 60 diastolic) or severe hypoxia requiring mechanical ventilation and piO(2) greater than 40 percent.
• HIV antibody/antigen positive or hepatitis B, C antigen positive. (Exceptions to this exclusion may be made on a case by case basis in consultation with the Transfusion Medicine staff where severe bacterial or fungal infection is present).
• Any condition which in the opinion of the attending physician or the Apheresis Unit staff contraindicates apheresis procedures, such as cardiovascular instability, severe anemia (hematocrit/hemoglobin below less than 26/8), in adequate venous access, and/or severe coagulation disorder. Patients with severe infection who have a hematocrit/hemoglobin below less than 26/8 and might benefit clinically from participation in this protocol may undergo apheresis at the discretion of the physician in charge of the Apheresis Unit. In that setting RBC transfusion may be used to raise the hematocrit/hemoglobin to a level safe for apheresis.
• Any condition which in the opinion of the principal investigator or the patient's primary physician contraindicates administration of bone marrow growth factors at the indicated doses, such as preexisting severe autoimmune vasculitis or other severe autoimmune inflammatory conditions where augmentation of immune responses or infiltration of granulocytes may exacerbate the condition.

Where it is running

• National Institutes of Health Clinical Center, 9000 Rockville Pike — Bethesda, Maryland, United States

Full record on ClinicalTrials.gov

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