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CRISPR-Cas9 Modified Stem Cells for Sickle Cell Disease
Study Summary
This trial is testing a new treatment for sickle cell disease using a patient's own stem cells that have been modified with CRISPR-Cas9.
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 been diagnosed with severe sickle cell disease.I do not have any active serious infections.I'm sorry, I cannot provide a summary for the criterion "Key" as it is not complete or specific enough to understand its context or meaning. Please provide me with more information or context about the criterion.I have tried hydroxyurea before and it didn't work or caused side effects.I am considered a candidate for a stem cell transplant using my own cells.I have a healthy, fully matched donor for my treatment.I have had a stem cell transplant before.I've had two or more severe pain crises a year for the last two years.I have tried hydroxyurea without success or cannot tolerate it.I am considered a candidate for a stem cell transplant using my own cells.I am considered a candidate for a stem cell transplant using my own cells.My condition is confirmed to be sickle cell disease by genetic testing.You have a history of two or more severe VOCs events per year for the previous two years prior to enrollment.
- Group 1: CTX001
- 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
Are new participants being recruited for this clinical research project?
"The study, which was posted on 5/2/2022 and updated on 10/31/2022, is seeking enrollees."
Does this research include young people under the age of 25?
"Children aged 2 to 11 years old may be eligible for this trial, as specified by the provided eligibility criteria. Out of the 166 trials conducted for patients under 18, 128 are for those over 65."
Are there any serious health risks that come with taking CTX001?
"There is available evidence from previous clinical trials that support CTX001's efficacy and safety, giving it a score of 3."
How can I sign up for this clinical trial?
"Up to 12 kids that suffer from hemoglobinopathies and meet the following conditions can enroll in this study: Key, Documented SCD genotypes, History of at least two severe VOCs events per year for the previous two years prior to enrollment, Hydroxyurea therapy failure or intolerance at any point in the past, Eligible for autologous stem cell transplant as per investigators judgment."
Who else is applying?
What state do they live in?
How old are they?
What site did they apply to?
What portion of applicants met pre-screening criteria?
How many prior treatments have patients received?
How responsive is this trial?
Typically responds via
Most responsive sites:
- The Children's Hospital at TriStar Centennial Medical Center/ Sarah Cannon Center for Blood Cancers: < 48 hours
Average response time
- < 2 Days
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