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Bone Marrow Transplant (BMT) for Sickle Cell Disease (TRANSFORM Trial)
TRANSFORM Trial Summary
This trial is testing if bone marrow transplants from half-matched related donors can be used to treat people with severe sickle cell disease. Before transplant, patients receive chemotherapy and radiation.
- Sickle Cell Disease
TRANSFORM Trial Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowTRANSFORM Trial Timeline
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Side effects data
From 2021 Phase 2 trial • 10 Patients • NCT02757885TRANSFORM Trial Design
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Who is running the clinical trial?
Frequently Asked Questions
How many people are signing up for this experiment?
"Yes, an update on September 13th indicates that this study is actively recruiting the 15 participants it needs from a single site."
What is the minimum age requirement for participants in this test?
"The age limit for inclusion in this study is 40 years old, with a minimum age of 15."
What are some of the risks associated with BMT?
"Since there is only some data supporting safety, and none yet to suggest it is effective, BMT received a score of 2."
Can you give me some more information on other BMT clinical trials?
"Out of the 960 ongoing trials studying Bone Marrow Transplants (BMT), 170 are currently in Phase 3. Most of these studies taking place in Philadelphia, however there are 29043 locations running clinical trials for this treatment globally."
To whom does this study appeal?
"This study is looking for 15 participants between the ages of 15 and 40 who have anemia or sickle cell. Participants must also meet the following criteria: -Clinically significant neurologic event (stroke) or any neurological deficit lasting > 24 hours;-Karnofsky/Lansky performance score ≥ 60; -Disease severity: Participants with SCD who have 1 or more of the following (i-v); -Age: Patients must be 15 - 40 years of age inclusive OR if younger than 15 years must be pubertal; -History of three or"
Are there any patients currently needed for this research project?
"According to the clinicaltrials.gov website, this study is still open for enrollment and actively recruiting patients. The trial was originally posted on April 1st, 2016 with a most recent update on September 13th, 2020."
What types of cancer respond best to treatment with BMT?
"BMT is a potential treatment for malignant melanoma of the skin as well as other conditions such as leukemia, neoplasm metastasis, and sickle cell anemia."
Why was this research project undertaken?
"The primary objective of this study is to evaluate the rate of late graft rejection. Secondary objectives include assessing the frequency of idiopathic pneumonia syndrome (IPS) and stroke, as well as the veno-occlusive disease (VOD) rate."
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