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Study Summary
This trial is studying a gene therapy for sickle cell disease. The FDA has put a partial clinical hold on the trial for patients less than 18 years of age, which means that no new enrollment, cell collection, conditioning, or drug product infusion can be initiated in pediatric patients. The study is still open to patients 18 years of age and older.
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 am currently pregnant, breastfeeding, or planning to become pregnant.I cannot receive red blood cell transfusions.My bone marrow is not working properly.I do not have any serious ongoing infections.I am mostly able to care for myself and carry out daily activities.I am mostly able to care for myself and carry out daily activities.I have a genetic mutation linked to a higher risk of MDS or AML.A close family member has or might have a hereditary cancer syndrome.I have genetic mutations that affect my α-globin genes.I have sickle cell disease with a specific genetic makeup.I have been treated for sickle cell disease at a center that keeps detailed records for at least 2 years.I cannot take hydroxyurea due to past failure or intolerance as judged by my doctor.My liver disease is in an advanced stage.I have severe blood vessel problems in my brain, including a stroke or significant narrowing/blockage.I have had an allogeneic transplant or gene therapy before.I am between 2 and 50 years old.I am a candidate for a stem cell transplant from a donor and have a matching donor available.I have been treated for sickle cell disease and followed at a medical center with detailed records for the past 2 years.I need blood-thinning medication from the start of my treatment until my platelet count recovers.I have had 4 or more severe pain episodes related to my condition in the last 2 years.I agree to use effective birth control during and for 6 months after treatment.I have had a bad reaction to or did not respond to hydroxyurea.I have sickle cell disease with a specific genetic makeup.I am between 2 and 50 years old.I have no current cancer or immune disorders, except for cured, non-threatening tumors.I am not allergic or unable to take plerixafor, busulfan, or similar medications.
- Group 1: bb1111
- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
- Pivotal Trial - The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.
- 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
Does this research only involve elderly patients?
"This particular study is only enrolling patients that are aged 2-50. In contrast, there are 133 clinical trials for those under 18 and 85 for senior citizens."
How many individuals are enrolled in this study currently?
"The sponsor, bluebird bio, needs to recruit 35 patients that meet the clinical trial's inclusion criteria in order to operate the study. The trial will be conducted out of various locations including Virginia Commonwealth University (VCU) in Richmond, Virginia and University of Minnesota in Minneapolis, Minnesota."
Who makes for an ideal candidate to test this new medication?
"This trial is currently seeking 35 young patients, aged 2-50, that suffer from anemia and sickle cell. Eligible applicants must have experienced a minimum of 4 protocol-defined VOEs in the 24 months prior to informed consent, have either experienced HU failure at any point in the past or be intolerant to HU, agree to use 1 method of highly effective contraception from Screening to at least 6 months after drug product infusion, have a diagnosis of SCD with βS/βS, βS/β0, or βS/β+ genotype,, weigh a minimum of 6 kg., have a Karn"
Is this research being conducted in multiple states across America?
"In addition to Virginia Commonwealth University (VCU) in Richmond, Virginia and the University of Minnesota in Minneapolis, Minnesota; this study is also enrolling patients at Fred Hutchinson Cancer Research Center in Seattle, Washington as well as 8 other locations."
What are the short-term and long-term effects of bb1111 on human health?
"There is some efficacy data and multiple rounds of safety data, so the Power team gave bb1111 a score of 3."
Are there any more patients needed for this clinical trial?
"The clinicaltrials.gov website says that this study is still looking for patients. This trial was first announced on February 14th, 2020 and received its most recent update on July 15th, 2022. In total, the study needs 35 enrollees from 8 different hospitals or medical facilities."
Who else is applying?
What site did they apply to?
What portion of applicants met pre-screening criteria?
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