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Main
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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
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Register:
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ClinicalTrials.gov |
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Last refreshed on:
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12 December 2020 |
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Main ID: |
NCT00394316 |
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Date of registration:
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31/10/2006 |
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Prospective Registration:
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No |
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Primary sponsor: |
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Public title:
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Gene Therapy for Chronic Granulomatous Disease
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Scientific title:
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Autologous Transplantation of Genetically Modified Cells for the Treatment of X-Linked Chronic Granulomatous Disease |
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Date of first enrolment:
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October 30, 2006 |
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Target sample size:
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3 |
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Recruitment status: |
Terminated |
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URL:
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https://clinicaltrials.gov/show/NCT00394316 |
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Study type:
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Interventional |
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Study design:
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Allocation: Non-Randomized. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
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Phase:
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Early Phase 1
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Countries of recruitment
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United States
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Contacts
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Name:
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Elizabeth M Kang, M.D. |
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Address:
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Telephone:
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Email:
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Affiliation:
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National Institute of Allergy and Infectious Diseases (NIAID) |
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Key inclusion & exclusion criteria
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- 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.
Age minimum:
3 Years
Age maximum:
55 Years
Gender:
Male
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Health Condition(s) or Problem(s) studied
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Chronic Granulomatous Disease
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Intervention(s)
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Drug: Phagocyte Oxidase Subunit Transduced CD34 Hematopoietic Stem Cells
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Primary Outcome(s)
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Can conditioning with busulfan improve gene therapy outcomes
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Secondary Outcome(s)
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1) To evaluate further the safety of gene therapy2) To monitor long-term results of gene therapy with conditioning
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Secondary ID(s)
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07-I-0017
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070017
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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Results
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Results available:
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Date Posted:
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Date Completed:
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URL:
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