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Gene Modified T Cells for Bone Marrow Failure Syndrome
Study Summary
This trial is testing a new way to give gene-modified T cells to people who have had a stem cell transplant from a donor with a different blood type. Researchers want to find out if it is safe and helps prevent a complication called GVHD.
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 have a non-cancerous condition that can be treated with a stem cell transplant.My donor and I match at least one allele in all required HLA types.I currently have an active infection.I am either male or female.I have a serious organ problem.I have Fanconi anemia and either AML or MDS.I am eligible for a transplant from a donor.I am between 4 months and 55 years old.I don't have a perfect match donor or my disease is worsening too quickly.
- Group 1: BPX-501 and Rimiducid
- 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 the application process still open for prospective participants in this trial?
"Unfortunately, recruitment for this clinical trial has concluded. It was first posted on February 1st 2015 and last edited on July 10th 2022. For those interested in alternative trials, 1147 studies are currently seeking participants with hemoglobinopathies and 16 studies have opened up for BPX-501 or Rimiducid research."
Does this research involve adults aged 65 and over?
"According to the criteria listed, this trial is open to individuals between 4 months and 55 years of age. There are 356 trials for those younger than 18 and 757 for seniors over 65."
What overarching goals are investigators pursuing with this research?
"Bellicum Pharmaceuticals, the clinical trial sponsor for this study, states that Adverse Events will be monitored over a 28-day period as the primary outcome. Additionally, Graft rejection incidence and severity of acute/chronic GvHD will also be evaluated in order to gauge Immune Reconstitution."
Have there been any other investigations involving BPX-501 and Rimiducid?
"Currently, 16 clinical trials are underway for BPX-501 and Rimiducid with none in their terminal phase. These trails are located primarily in Nashville Tennessee but span a total of 94 sites globally."
What is the cap on enrollment for this research project?
"Unfortunately, the recruitment period for this trial has ended; the first post was on February 1st 2015 and it was last edited on July 10th 2022. But those with hemoglobinopathies have 1147 viable trials to choose from if they are looking for a clinical study, while 16 studies related to BPX-501 and Rimiducid are actively recruiting participants."
Have the drugs BPX-501 and Rimiducid been authorized for use by the U.S. Food and Drug Administration?
"The safety of BPX-501 and Rimiducid is assessed at a score of 1, as this clinical trial is in Phase 1. This implies that there is restricted data available to support the efficacy and security of these two medications."
Who qualifies to take part in this clinical research?
"This research project is recruiting one person aged between 4 months old and 55 years that has a hemoglobinopathy. As well as the aforementioned condition, they must meet these criteria: age over four months and under fifty-five, diagnosis of nonmalignant disorder treatable by haematopoietic cell transplantation (HCT), eligible for allogeneic HCT with either an exact 5/10 match or no suitable donor at hand; Fanconi anaemia sufferers requiring red blood cells or platelet transfusions due to bone marrow failure also qualify."
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