NCT00001476

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

Completed Phase 1 Last updated 2 July 2017

What this trial tests

Phase 1 trial testing Gene Therapy Method for CGD in Chronic Granulomatous Disease in 14 participants. Completed in 13 December 2010.

Timeline

1 June 1995

Primary endpoint
13 December 2010

13 December 2010

Quick facts

Lead sponsor	National Institute of Allergy and Infectious Diseases (NIAID)
Phase	Phase 1
Status	Completed
Study type	INTERVENTIONAL
Primary purpose	treatment
Enrollment	14
Start date	1 June 1995
Primary completion	13 December 2010
Estimated completion	13 December 2010
Sites	1 location across United States

Drugs / interventions tested

• Gene Therapy Method for CGD — full drug profile →
• Isolex 300i Magnetic Cell Selector

Conditions studied

• Chronic Granulomatous Disease — all drugs for Chronic Granulomatous Disease →
• Communicable Disease — all drugs for Communicable Disease →

Sponsor

National Institute of Allergy and Infectious Diseases (NIAID)

Who can join

5 and older, any sex, with Chronic Granulomatous Disease or Communicable Disease. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

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.

Publications & conference data

1 peer-reviewed publication reference this trial (live from Europe PMC):

1. Cellular unfolded protein response against viruses used in gene therapy.
   Sen D, Balakrishnan B, Jayandharan GR. · · 2014 · cited 14× · PMID 24904562 · DOI 10.3389/fmicb.2014.00250