Status:
TERMINATED
Gene Therapy for Chronic Granulomatous Disease
Lead Sponsor:
National Institute of Allergy and Infectious Diseases (NIAID)
Conditions:
Chronic Granulomatous Disease
Eligibility:
MALE
3-55 years
Phase:
EARLY_PHASE1
Brief Summary
X-linked Chronic Granulomatous Disease (CGD) is an inherited disorder caused by an abnormal gene that fails to make the protein known as gp91 phox. This protein is part of a group of proteins that wor...
Detailed Description
X-linked Chronic Granulomatous Disease (CGD) is an inherited disorder caused by an abnormal gene that fails to make the protein known as gp91 phox. This protein is part of a group of proteins that wor...
Eligibility Criteria
Inclusion
- INCLUSION CRITERIA:
- Have a diagnosis of X-linked CGD (i.e., a gp91-phox gene mutation/defect).
- Have a minimum of 5.0 x10(6) CD34 plus cells per kg body weight (collected and cryopreserved, prior to enrollment) available for transduction.
- Weight greater or equal to 20 kg.
- Unresponsive or incurable infection as defined by either/or:
- Continued (stable or progressive) infection despite standard antimicrobial therapy
- Stable and/or does not completely resolve despite a minimum of 2 months of treatment
- OR
- Progressive as shown by increase in size or new sites of infection despite therapy for a minimum of two weeks.
- Multidrug resistant organism as determined by tissue analysis
- Not have a suitable sibling who is HLA-matched for stem cell or bone marrow donation.
- Males aged 3-55 years.
- Must use two approved methods of contraception, such as barrier method (condom) with a spermicidal if sexually active
- Willingness to remain hospitalized for several weeks
- Have a primary care physician at home
- Consent to permit storage of blood and/or other tissues samples
- Patients with a Grade 3 toxicity due to active infection may enter this trial.
- EXCLUSION CRITERIA:
- Weigh less than 20 kg.
- Be hemodynamically unstable or requiring pressor support.
- Require ventilatory assistance with high levels of oxygen.
- Have an HLA-matched suitable sibling for stem cell or bone marrow donation.
- Intolerance to busulfan.
- Failure to use two approved methods of contraception, such as barrier method (such as a condom with a spermicidal).
- Participation in another Gene Therapy clinical trial
- If pre-conditioning and pre-infusion evaluations are found to match a criterion for Grade 4 toxicity as defined in Toxicity Table for grading severity of AEs
- Presence of an anti-GP91 antibody.
- When the condition or parameter has returned to the criterion for Grade 3 or less for that condition or parameter twice over an interval of at least 4 weeks prior to the infusion, eligibility may be resumed.\<TAB\>\<TAB\>
Exclusion
Key Trial Info
Start Date :
October 30 2006
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
April 8 2014
Estimated Enrollment :
3 Patients enrolled
Trial Details
Trial ID
NCT00394316
Start Date
October 30 2006
End Date
April 8 2014
Last Update
July 5 2018
Active Locations (1)
Enter a location and click search to find clinical trials sorted by distance.
1
National Institutes of Health Clinical Center, 9000 Rockville Pike
Bethesda, Maryland, United States, 20892