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Stem Cell Transplant for Immune Deficiency Syndrome
Study Summary
This trial is for people with GATA2 deficiency to see if stem cell transplants are successful in treating the condition.
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?
Media Library
- I will have an adult caregiver with me after my transplant.My cancer has spread to my brain or spinal cord.I am between 8 and 70 years old.I have ongoing active hepatitis B.I have a GATA2 gene mutation or related health issues.I have GATA2 deficiency and had a serious infection.I have an active cancer in an organ other than my blood.I don't have a closely matched donor for a transplant.I have a donor who is a close match to my tissue type.I have an infection that hasn't improved with treatment.I am HIV-positive.My blood disorder shows a certain level of immature cells.
- 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.
- Group 1: Arm A
- Group 2: Arm B
- Group 3: Arm C (combined with Arm B per Amendment N)
- Group 4: Arm D (Deleted this arm per amendment I)
- Group 5: Arm E (Deleted this arm per amendment O)
Frequently Asked Questions
Will this research be enrolling any participants who are over 50 years old?
"The age requirements for this trial state that the youngest a participant can be is 8 years old, while the oldest a patient can be and still participate is 70."
Are there any unfilled vacancies for participants in this research project?
"Yes, this trial is still open to recruitment and information on clinicaltrials.gov supports this claim. The listing was originally posted on July 24th, 2013 with the most recent update being made on October 31st, 2020."
How can I become a subject in this clinical trial?
"This clinical trial is looking for 144 participants, within the ages of 8 and 70 who have immunodeficiencies. It is required that patients also meet the following criteria:-Creatinine: Adult patients: less than or equal to 2.0 mg/dl and creatinine clearance greater than or equal to 30 ml/min; Pediatric patients (<18 years old): creatinine <1.5 mg/dL and a creatinine clearance , using the Schwartz Formula, > 30 mL/min/1.73m(2).-INCLUSION CRITERIA- Recipient,"
Are the risks of Arm B outweighed by its benefits?
"Arm B's safety is rated a 2 because there is only evidence of its safety and no efficacy data."
What is the standard care that Arm B is used to compare against in this study?
"While Arm B is the most popular treatment for leukemia, it can also be used to effectively manage osteoporosis caused by glucocorticoid, lung cancers, and dermatitis, atopic."
How many people are included in the data set for this research?
"This is accurate. The website provides evidence that this study, which was originally posted on July 24th 2013 and last updated on October 31st 2020, is currently looking for 144 participants from 2 locations."
Is Arm B a new experimental treatment?
"At the moment, there are 1077 ongoing studies that focus on Arm B. Out of those 189 trials are currently in Phase 3. The majority of clinical trials for Arm B are located in Philadelphia, however, there 29790 different locations running studies for Arm B."
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