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Gene Editing
Gene Editing (CRISPR) for Sickle Cell Disease
Phase 1 & 2
Waitlist Available
Research Sponsored by Mark Walters, MD
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Karnofsky performance score ≥60.
History of two or more episodes of acute chest syndrome (ACS) in the 2-year period preceding enrollment despite the institution of supportive care measures (i.e. asthma therapy and/or hydroxyurea);
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 1 and 2 years post-transplant
Awards & highlights
Study Summary
This trial will test a new treatment for sickle cell disease that uses CRISPR to edit red blood cells.
Who is the study for?
This trial is for individuals aged 12 to 35 with severe Sickle Cell Disease who've had multiple pain events or acute chest syndrome despite treatment, and have good kidney, liver, heart, and lung function. It's not for those with certain infections, pregnant or breastfeeding women, men unwilling to use contraception, or anyone who has received a transplant.Check my eligibility
What is being tested?
The study tests a one-time infusion of CRISPR_SCD001-modified stem cells in patients with severe Sickle Cell Disease. This gene-editing approach aims to correct the sickle cell allele in hematopoietic stem cells to alleviate disease symptoms.See study design
What are the potential side effects?
Potential side effects are not explicitly listed but may include typical risks associated with stem cell transplantation such as immune reactions, infection risk increase due to immunosuppression during the procedure and potential off-target genetic effects.
Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowSelect...
I can care for myself but may need occasional help.
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I've had two or more acute chest syndrome episodes in the last 2 years despite treatment.
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I've had 4 or more severe pain crises due to sickle cell in the last 2 years despite treatment.
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My lung function tests show my oxygen levels and breathing capacity are within safe limits.
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My heart's pumping ability is within a healthy range.
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I am between 12 and 34 years old.
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My kidney function tests are within normal limits.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ 1 and 2 years post-transplant
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~1 and 2 years post-transplant
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
Incidence of adverse events and grade 3 or higher serious adverse events, using the National Cancer Institute's Common Terminology Criteria for Adverse Events (CTCAE)
Secondary outcome measures
Change in the annualized vaso-occlusive pain event (VOE) rates.
Frequency of central nervous system (CNS) toxicity (reversible posterior leukoencephalopathy syndrome [RPLS] or posterior reversible encephalopathy syndrome [PRES], hemorrhage, and seizures).
Frequency of cytomegalovirus (CMV) infection, invasive fungal infection, and any other serious viral or bacterial infection
+9 moreOther outcome measures
Change from baseline in cardiac-pulmonary function via echocardiogram (tricuspid regurgitant jet velocity [TRJV], LVEF).
Change from baseline in cardiac-pulmonary function via pulmonary function tests
Change from baseline in meters walked during 6-minute walk test (6MWD)
+3 moreTrial Design
1Treatment groups
Experimental Treatment
Group I: CRISPR_SCD001 Drug ProductExperimental Treatment1 Intervention
CRISPR_SCD001 Drug Product (autologous CD34+ cell-enriched population that contains cells modified by the CRISPR-Cas9 ribonucleoprotein) dose will be ≥3.0×106 CD34+ cells/kg recipient weight for each subject and the upper limit cell dose is 20 ×106 CD34+ cells/kg.
Find a Location
Who is running the clinical trial?
Mark Walters, MDLead Sponsor
University of California, BerkeleyOTHER
180 Previous Clinical Trials
721,177 Total Patients Enrolled
University of California, Los AngelesOTHER
1,528 Previous Clinical Trials
10,276,842 Total Patients Enrolled
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- I am not pregnant or breastfeeding.I can care for myself but may need occasional help.I do not have HIV or active hepatitis B or C.I am physically fit based on several health checks.My liver is working well.I've had two or more acute chest syndrome episodes in the last 2 years despite treatment.I agree to use two forms of birth control or practice abstinence for 12 months after my treatment.My sickle cell disease is not the HbSS type.I've had 4 or more severe pain crises due to sickle cell in the last 2 years despite treatment.I have had a stroke or I get blood transfusions to prevent strokes.My lung function tests show my oxygen levels and breathing capacity are within safe limits.I have a sibling donor who is a perfect HLA match.I am a male willing to use effective contraception or practice abstinence for 12 months post-treatment.My heart's pumping ability is within a healthy range.My direct bilirubin levels are within twice the normal limit, not due to blood transfusion issues.I am between 12 and 34 years old.I need a liver MRI because I've had many blood transfusions.I haven't had an uncontrolled infection in the last 6 weeks.My kidney function tests are within normal limits.You have had an organ transplant.I have received a stem cell transplant.
Research Study Groups:
This trial has the following groups:- Group 1: CRISPR_SCD001 Drug Product
Awards:
This trial has 1 awards, including:- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
Timeline:
This trial has the following timeline:- 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
These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
Is there an opportunity for me to participate in this experiment?
"This medical trial is seeking 9 candidates who suffer from anemia and sickle cell, aged between 12 and 35."
Answered by AI
Does this clinical investigation involve individuals aged 85 or older?
"According to the eligibility criteria of this research, applicants aged 12-35 are suitable for participation. Additionally, there are 131 studies available for minors and 85 trials designed specifically for seniors above 65 years old."
Answered by AI
Is there availability for enrolment in this clinical study?
"According to the clinicaltrials.gov listing, this particular trial is no longer recruiting patients for its study. Initially posted on December 1st 2022 and last updated April 25th 2022, there are still 197 other studies actively looking for participants at present."
Answered by AI
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