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Chemotherapy + Stem Cell Transplant for Fanconi Anemia (RAFA Trial)
RAFA Trial Summary
This trial is testing a new, less intense chemotherapy treatment for people with Fanconi Anemia who are getting a stem cell transplant from a related or unrelated donor.
RAFA Trial Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowRAFA Trial Timeline
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.RAFA Trial Design
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Who is running the clinical trial?
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- I have been diagnosed with Fanconi anemia.My heart, liver, kidneys, and lungs are functioning well.I am not pregnant or breastfeeding and agree to pregnancy tests and avoiding pregnancy while in the study.I have a severe blood disorder such as SAA, MDS, or AML.You have tested positive for HIV or HTLV infection.My gender or ethnic background does not limit my participation.I do not have any ongoing serious infections.I have leukemia that has spread to my brain.My donor matches me genetically but is not my sibling.I am mostly active and can care for myself.I am not pregnant or breastfeeding.
- Group 1: Arm A: Good Risk Patients
- Group 2: Arm B: Intermediate Risk Patients
- Group 3: Arm C: High Risk Patients
- 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
Is enrollment open for this experiment?
"Affirmative. Clinicaltrials.gov states that the study, which was initially announced on April 1st 2014 and recently revised on April 7th 2022, is actively seeking participants."
What precedent has been established for research on the efficacy of Peripheral blood stem cell treatments?
"Currently, there are 995 existing clinical trials researching Peripheral blood stem cell treatments. Of those trials, 180 have advanced to the Stage 3 of testing. In Philadelphia alone, Pennsylvania is hosting a multitude of these studies; however, across the United States 30321 medical sites are running their own investigations into this therapy."
What medical applications is Peripheral blood stem cell commonly utilized for?
"The usage of peripheral blood stem cell to treat bone marrow transplantation is well established, with it also being a viable treatment for conditions such as leukemia, infection and lung cancer."
How many individuals have chosen to take part in this experiment?
"Affirmative. According to the information on clinicaltrials.gov, this research has already begun its recruitment process, having gone live on April 1st 2014 and being updated most recently on July 4th 2022. 70 volunteers must be sourced from 2 different locations for completion of the project."
Does the FDA recognize Peripheral blood stem cell as a viable treatment option?
"While there is some safety data linked to the use of peripheral blood stem cells, due to it being a Phase 2 trial, no efficacy has been established yet. Thus, our team at Power have assigned this medication an overall score of two."
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