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Study Summary
This trial is a study to test a new treatment for SCID, in which patients receive low doses of targeted busulfan followed by infusion of their own stem cells that have been modified with a virus to correct the SCID gene. The trial will enroll subjects over 3 years and follow them for 2 years afterwards to see how well the treatment works.
Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowTimeline
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Trial Design
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Who is running the clinical trial?
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- I am on a mechanical ventilator or use CPAP.I have an infection that hasn't improved after 2 weeks of treatment.My liver tests are much higher than normal.Your heart's pumping function is not strong enough, as shown in an echocardiogram.I have a condition that affects how my body repairs DNA.I have a cancer diagnosis that is not related to EBV.I am HIV-1 positive.I do not have severe birth defects that threaten my life.I have seizures that are not controlled by medication.I am 5 years old or younger.I had an allogeneic transplant and it's confirmed that the donor T cells did not engraft.I do not have a family member who is a perfect match for a bone marrow transplant.My kidney function is very low or I am on dialysis.I have been diagnosed with encephalopathy.I have SCID-X1 confirmed by a specific gene mutation and poor T cell function.I am willing to follow up for 15 years after treatment as required.I am at least 8 weeks old at the time of receiving busulfan.
- Group 1: Treatment arm
- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
Frequently Asked Questions
What is the current level of participant enrollment in this experiment?
"Affirmative. The information hosted on clinicaltrials.gov indicates that this scientific experiment is presently recruiting participants; it was initially posted on January 19th 2018 and has recently been updated on July 19th 2022. Recruitment for 10 patients between 4 sites is necessary to run the trial successfully."
Are there any Canadian healthcare centers currently conducting this clinical trial?
"This clinical trial is being conducted at four locations, including Mattel Children's Hospital - UCLA in Los Angeles, Emory University/Childrens Healthcare of Atlanta in Atlanta, and Cincinnati Children's Hospital Medical Center."
Are there any openings available in this clinical trial yet?
"Affirmative. Evidence hosted on clinicaltrials.gov confirms that this research endeavour, which was initially made public on January 19th 2018, is actively seeking out volunteers. At present, 10 participants must be recruited from 4 distinct medical centres."
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