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Bone Marrow Transplant for Sickle Cell Disease
Study Summary
This trial is testing a bone marrow transplant as a possible treatment for sickle cell disease in children. Eligible participants will undergo a pre-transplant evaluation, conditioning, and then the transplant itself, followed by routine care and checkups for at least 5 years.
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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- I have a brain condition without symptoms, confirmed by specific brain scans.My parent or guardian does not understand the risks of the HSCT process.I cannot undergo a bone marrow harvest or general anesthesia due to health or personal reasons.I have had a stroke with symptoms lasting over 24 hours and confirmed by brain scans.I've had at least 3 painful episodes a year that needed strong painkillers or hospital visits.I have symptoms of sickle cell disease.I have had 2 or more episodes of acute chest syndrome in my life.I have severe lung disease and may need oxygen during the day.I've had 3 or more episodes of chest pain and blocked blood flow yearly for 3 years.I have had at least 3 acute chest syndrome events requiring blood transfusions.I am between 2 and 12 years old with a sickle cell disease.My sibling under 13 can donate to me and doesn't have sickle cell disease.My condition is considered severe.I've had 2 or more painful episodes needing strong painkillers at a hospital or clinic.My heart's pumping ability is reduced.I have severe liver scarring.I do not have severe neurological impairments, except possibly hemiplegia.I do not have symptoms of HbSS or HbSβ°thalassemia.My kidney function is below 50% of the expected level for my age.I have a long-term health issue that could make a stem cell transplant too risky for me.I do not have any active infections.My daily activities are significantly limited.My condition is not considered severe.I have had at least 3 episodes of acute chest syndrome or pain crises in my life.
- Group 1: Reduced Intensity Conditioning with FAM
- All Individual Drugs Already Approved - Therapies where all constituent drugs have already been approved are likely to have better-understood side effect profiles.
- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
- Approved for 5 Other Conditions - This treatment demonstrated efficacy for 5 other conditions.
- 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 for this investigation still a possibility?
"Per information hosted on clinicaltrials.gov, this medical trial is actively recruiting participants as of August 3rd 2022; the study was initially posted in March 22nd 2019."
How many venues are currently hosting this experiment?
"Currently, there are 11 study sites that are taking part in this trial. These locations span from Kent to Charleston and Jackson as well as other nearby towns. To reduce travel needs, it may be beneficial to seek out the closest centre for enrollment purposes."
What potential harms could Fludarabine pose to patients?
"Fludarabine's safety was assessed and ranked as a 2, since the Phase 2 clinical trial has only produced some evidence of its protective properties but no data on efficacy."
Does the eligibility criterion for this research project include adults aged 18 or older?
"The intended patient population for this trial are minors between the ages of two and thirteen."
How many individuals are currently engaged in this medical research?
"Affirmative. According to information found on clinicaltrials.gov, this medical research project is in its recruitment phase. It was initially posted on March 22nd 2019 and updated most recently on August 3rd 2022. The study needs 58 volunteers across 11 healthcare sites."
Who meets the requirements to participate in this experiment?
"Aspiring participants of this research should meet the criteria of anemia, sickle cell, and between 2 to 13 years old. The trial is seeking 58 volunteers in total."
What conditions has Fludarabine been administered to treat?
"Fludarabine is a viable treatment for multiple sclerosis, as well as organ transplant rejection, kidney issues, B-lymphocyte overproduction, and amyloidosis."
Has there been prior research into the efficacy of Fludarabine?
"Fludarabine was first clinically investigated in 1997 at the City of Hope Comprehensive Cancer Center. Since then, 654 trials have been completed and 463 are presently recruiting participants. Notably, a significant portion of these studies originate from Kent, Ohio."
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