Actively Recruiting
Gene Editing For Sickle Cell Disease
Led by St. Jude Children's Research Hospital · Updated on 2026-05-05
25
Participants Needed
1
Research Sites
401 weeks
Total Duration
On this page
Sponsors
S
St. Jude Children's Research Hospital
Lead Sponsor
N
National Heart, Lung, and Blood Institute (NHLBI)
Collaborating Sponsor
AI-Summary
What this Trial Is About
This study is being done to test the safety of a new treatment called gene editing in Sickle Cell Disease (SCD) patients and to see if a single dose of this genetically modified cellular product will increase the amount of a certain hemoglobin called fetal hemoglobin (HbF) and help reduce the symptoms of SCD. Primary Objective * To assess the safety of autologous infusion of clustered regularly interspaced palindromic repeats (CRISPR)/ CRISPR associated protein (Cas9)-edited CD34+ hematopoietic stem and progenitor cells (HSPCs) in patients with severe SCD. Secondary Objective * To assess the efficacy autologous infusion of CRISPR/Cas9 genome-edited CD34+ HSPCs into patients with severe SCD.
CONDITIONS
Official Title
Gene Editing For Sickle Cell Disease
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Age 18 to 24.9 years.
- Diagnosis of Sickle Cell Disease with Hb SS, Hb SB0, or Hb SB+ genotype.
- History of 2 or more SCD-related vaso-occlusive events per year in the 2 years before screening, OR regular red blood cell transfusions (8 or more in 12 months) except when for stroke prevention.
- History of failure, intolerance, or refusal of hydroxyurea therapy.
- Eligibility for autologous stem cell transplant by investigator judgment.
- Females of childbearing potential must agree to use contraception from mobilization start through 6 months post-infusion.
- Males must agree to use contraception from mobilization start through 6 months post-infusion.
- Willingness to participate in additional long-term follow-up study after trial completion.
You will not qualify if you...
- Availability of an HLA-matched sibling donor for hematopoietic cell transplantation.
- Karnofsky or Lansky performance score below 80.
- Pregnancy confirmed by positive test within 14 days before enrollment.
- Breastfeeding.
- Uncontrolled or clinically significant infections within 1 month before enrollment.
- Confirmed Hepatitis B or C infection.
- Confirmed HIV or HTLV infection.
- History of stroke.
- Serum conjugated bilirubin above 2 times normal or ALT above 3 times normal, unless values improve.
- Left ventricular shortening fraction under 25% or ejection fraction under 45%.
- Estimated creatinine clearance below 60 mL/min/1.73m².
- Diffusion capacity of carbon monoxide under 50% or baseline oxygen saturation under 85% if unable to perform lung tests.
- Prior hematopoietic cell transplant or gene therapy.
- Known liver cirrhosis, bridging fibrosis, or active hepatitis.
- Active malignancy, myelodysplasia, abnormal cytogenetics, or immunodeficiency.
- History of significant bleeding disorder.
- Cerebrovascular procedure within 6 months.
- Untreated or risky moyamoya disease.
- Presence of pathogenic clonal mutations linked to blood cancers.
- Intolerance or allergy to plerixafor, motixafortide, or busulfan.
- Participation in another investigational drug study within 30 days or less than 5 half-lives of the drug.
- History of alloimmunization to red blood cell antigens with anticipated insufficient blood units for study duration.
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 1 location
1
St. Jude Children's Research Hospital
Memphis, Tennessee, United States, 38105
Actively Recruiting
Research Team
A
Akshay Sharma, MBBS, MSc
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
Not the Right Trial for You?
Explore thousands of other clinical trials that might be a better match.
Sign up to get personalized trial recommendations delivered to your inbox.
Already have an account? Log in here