Sickle Cell DiseasePhase 1NCT06506461
Gene Editing For Sickle Cell Disease
A Phase 1 study testing Plerixafor, Busulfan and Gene-modified CD34+ cells in people with Sickle Cell Disease, run by St. Jude Children's Research Hospital at 1 site in United States.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 18 to 24 years
- Conditions the study is for
- Sickle Cell Disease
- What is being tested?
- Plerixafor, Busulfan and Gene-modified CD34+ cells
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 25 participants
- Where
- 1 siteUnited States
- Study dates
- 21 Mar 2025to Dec 2032
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 24 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- Age ≥18 years and ≤24.9 years.
- Patients with SCD (Hb SS, Hb SB0 and Hb SB+ genotype) who have experienced EITHER (a) 2 or more SCD-related vaso-occlusive events (acute pain events, acute chest syndrome, priapism and splenic sequestration) per year in the 2-year period before screening, OR (b) administration of regular red blood cell (RBC) transfusions (≥8 transfusions in the 12 months preceding enrollment) EXCEPT if the RBC transfusions are being administered for primary or secondary stroke prevention and, in the opinion of the treating hematologist, cannot be safely discontinued after infusion of the gene modified drug product.
- Failure, intolerance, or refusal of hydroxyurea therapy.
- Patients must be eligible for autologous stem cell transplant as per investigator's judgment.
- Females of childbearing potential (i.e., those who are post-menarchal with an intact uterus and at least 1 ovary, and those who are less than 1 year postmenopausal) must agree to use acceptable method(s) of contraception from start of mobilization through at least 6 months post-infusion.
- Males must agree to use effective contraception from start of mobilization through at least 6 months post-infusion.
- Patients should be willing to participate in an additional long-term follow-up study after completion of this trial.
Who cannot join
- Availability of an human leukocyte antigen (HLA)-matched sibling who is willing and able to donate an appropriate graft for hematopoietic cell transplantation (HCT).
- Karnofsky or Lansky performance score < 80.
- Pregnant, as confirmed by positive serum or urine pregnancy test within 14 days before enrollment (if female).
- Breastfeeding.
- Uncontrolled (undergoing appropriate treatment and with progression of clinical symptoms) or clinically significant bacterial, viral, or fungal infections within 1 month before enrollment.
- Patients with confirmed Hepatitis B or Hepatitis C infections.
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- Patients with confirmed seropositivity or positive nucleic acid amplification test (NAAT) for human immunodeficiency virus (HIV) or human T-cell lymphotropic virus (HTLV).
- Patients with a history of stroke.
- Serum conjugated (direct) bilirubin > 2× the upper limit of normal for age, or serum alanine transaminase (ALT) > 3× the upper limit of normal for age as per the local laboratory. Participants with hyperbilirubinemia or elevated aspartate aminotransferase (AST) as the result of hyperhemolysis, or with a severe drop in hemoglobin post blood transfusion, are not excluded as long as these values downtrend and return to acceptable limits subsequently.
- Left ventricular shortening fraction < 25% or ejection fraction < 45% by echocardiogram.
- Estimated creatinine clearance less than 60 mL/min/1.73m^2.
- Diffusion capacity of carbon monoxide (DLCO) < 50% (adjusted for hemoglobin) OR baseline oxygen saturation < 85% in patients unable to perform pulmonary function tests.
- Prior HCT or gene therapy.
- Known hepatic cirrhosis, bridging hepatic fibrosis, or active hepatitis. Appropriate ultrasound or magnetic resonance (MR) imaging may be used to define the presence and degree of cirrhosis. Liver biopsy may be performed at the discretion of the attending physician or principal investigator if there are concerns regarding the presence of severe hepatic fibrosis or cirrhosis such that participation in this trial will not be in the patient's best interest.
- Active known malignancy, myelodysplasia, abnormal cytogenetics, or immunodeficiency.
- Patients with history of a significant bleeding disorder.
- Cerebrovascular procedure within 6 months, including pial synangiosis for moyamoya.
- Patients with history of untreated moyamoya disease or presence of moyamoya disease at screening that in the opinion of the investigator puts the subjects at the risk of bleeding.
- Evidence of a pathogenic clonal variant in any candidate gene detected by a standard, licensed next-generation sequencing clinical assay for gene mutations associated hematological malignancies.
- Patients with history of intolerance, contraindication, or known sensitivity to plerixafor or motixafortide or busulfan. Prior anaphylactic reaction with excipients of the proposed product.
- Patients with participation in another clinical study with an investigational drug/product within 30 days of screening or fewer than 5 half-lives of the investigational agent whichever is longer from screening.
- Patients with history of alloimmunization to RBC antigens and for whom the investigator anticipates that there will be insufficient RBC units available for the duration of the study.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
St. Jude Children's Research Hospital
Memphis, Tennessee, 38105
Akshay Sharma, MBBS, MSc, 888-226-4343, referralinfo@stjude.org
About this study
Official title: St. Jude Autologous Genome Edited Stem Cells For Sickle Cell Disease-1
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.
Read the detailed description
Participants will receive a daily subcutaneous (under the skin) dose of motixafortide for up to 3 consecutive days to mobilize their hematopoietic stem and progenitor cells (HSPCs) into peripheral blood. Participants who cannot tolerate motixafortide may receive a daily subcutaneous dose of plerixafor as an alternative for 3-5 consecutive days. The collection of HSPCs will be done via apheresis. The collected HSPCs will be sent to a lab to genetically modify them using CRISPR/Cas9.
In the lab, the researchers will take the stem cells and purify them. The stem cells will then be mixed with the CRISPR-Cas9 gRNA ribonucleoprotein (RNP) complex to change (edit) the genes in the cells and produce the new gene edited cellular product. This gene edited drug product will be frozen until ready for infusion.
Once the cellular product is ready, participants will be given Busulfan (a chemotherapy medicine) intravenously (IV) for 4 days. The thawed gene product will be given IV about 48 hours after the completion of the last dose of busulfan.
Participants will be followed for 3 years on this study. After the three years, participants will be followed for 12 more years on a long-term follow-up study.
- Conditions
- Sickle Cell Disease
- Being tested
- Plerixafor, Busulfan, Gene-modified CD34+ cells, Motixafortide
- Keywords
- Plerixafor, Autologous, CD34+ cells, Apheresis, Conditioning, Busulfan, Gene Editing
- Medical subject headings
- Anemia, Sickle Cell
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PlerixaforDrug
Also called Mozobil®
Given Subcutaneous (under the skin)
BusulfanDrug
Also called Myleran®, Busulfex
Given Intravenous (IV)
Gene-modified CD34+ cellsBiological
Given Intravenous (IV)
MotixafortideDrug
Also called Aphexda
Given Subcutaneous (under the skin)
| Study group | Role | What they receive |
|---|---|---|
| Autologous, genetically modified CD34+ HSPCs Treatment | Experimental | All eligible participants receive intervention as described in the Detailed Description with the following: motixafortide, plerixafor, busulfan, and autologous, gene-modified CD34+ cells Receives: Drug: Plerixafor; Drug: Busulfan; Biological: Gene-modified CD34+ cells; Drug: Motixafortide |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of neutrophil engraftment by day +42 after infusion of the CRISPR/Cas9-edited CD34+ HSPCs.
Upon completion of the trial, summary statistics will be computed for the time to neutrophil engraftment.
Measured: Within 42 days of the cellular product infusion
Incidence of platelet engraftment by day +60 after infusion of the CRISPR/Cas9-edited CD34+ HSPCs.
Upon completion of the trial, summary statistics will be computed for the time to platelet engraftment.
Measured: Within 60 days of the cellular product infusion
Sustenance of multi-lineage engraftment and polyclonal hematopoiesis as measured by counts of different clones of myeloid cells, T cells, B cells, and NK cells at 1 year after infusion of the CRISPR/Cas9-edited CD34+ HSPCs.
Sustenance of multi-lineage engraftment will be described using descriptive statistics.
Measured: Within 1 year of the cellular product infusion
Frequency of off-target editing after infusion of the CRISPR/Cas9-edited CD34+ HSPCs.
Frequency of off-target editing will be described using descriptive statistics.
Measured: Within 3 years of the cellular product infusion
Occurrence of secondary graft failure, clonal hematopoiesis, MDS, or AML
Occurrence will be described using descriptive statistics.
Measured: Within 3 years of the cellular product infusion
Secondary outcomes (4)
Estimate the change in the annualized rate of SCD-related vaso-occlusive events (such as pain crises and acute chest syndrome events), starting at 3 months after the infusion of autologous CRISPR/Cas9-edited CD34+ HSPCs.
We will evaluate the change in the annualized rate of SCD-related vaso-occlusive events starting at 3 months after the infusion of autologous gene-edited CD34+ HSPCs relative to the annualized rate calculated during the 2-year period before study enrollment. The changes will be summarized using descriptive statistics and displayed graphically.
Measured: From 3 months post the cellular product infusion to 3 years post infusion
Compare the change from baseline in the total blood hemoglobin concentration.
The change from baseline (at the time of screening) in the total blood hemoglobin concentration will be calculated at various timepoints as described in the protocol. The changes will be summarized using descriptive statistics and displayed graphically.
Measured: From time of screening to 3 years post infusion
Compare the change from baseline in the fraction of red blood cells (RBCs) containing HbF.
The change from baseline (at the time of screening) in the fraction of RBCs containing HbF by immunostaining will be calculated at various timepoints as described in the protocol. The changes will be summarized using descriptive statistics and displayed graphically.
Measured: From time of screening to 3 years post infusion
Change in incidence of packed RBC transfusions.
The change from baseline (at the time of screening) in the incidence of packed RBC transfusions will be calculated at various timepoints as described in the protocol. The changes will be summarized using descriptive statistics and displayed graphically.
Measured: Within 1 year prior to infusion and within 3 months to 1 year post infusion
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Jul 2024
- Study start21 Mar 2025
- Primary completionDec 2029 (estimated)
- Study completionDec 2032 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Sickle Cell DiseaseStudies of the same condition, open and under way.
- More trials run by St. Jude Children's Research HospitalThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- Patients with severe sickle cell disease (SCD)
- Masking
- None (open label)
- Enrolment
- 25 participants (estimated)
Sponsor and investigators
- Lead sponsor
- St. Jude Children's Research Hospital
- Sponsor type
- University, hospital or other
- Collaborators
- National Heart, Lung, and Blood Institute (NHLBI)
- Responsible party
- Sponsor
- Investigators
- Akshay Sharma, MBBS, MSc, Principal investigator, St. Jude Children's Research Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06506461
- Sponsor's protocol ID
- SAGES1
- Other IDs
- U01HL163983
- Status last verified
- Oct 2026
- First submitted
- 14 Jun 2024
- Last updated
- 15 Oct 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: Individual participant de-identified datasets containing the variables analyzed in the published article will be made available (related to the study primary or secondary objectives contained in the publication). Supporting documents such as the protocol, statistical analyses plan, and informed consent are available through the CTG website for the specific study. Data used to generate the published article will be made available at the time of article publication. Investigators who seek access to individual level de-identified data will contact the computing team in the Department of Biostatistics (ClinTrialDataRequest@stjude.org) who will respond to the data request.
Study at a glance
From the registry record, last updated by the sponsor on 15 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- St. Jude Autologous Genome Edited Stem Cells For Sickle Cell Disease-1
- ClinicalTrials.gov ID
- NCT06506461
- Acronym / sponsor ID
- SAGES1
- Conditions
- Sickle Cell Disease
- Keywords
- Plerixafor, Autologous, CD34+ cells, Apheresis, Conditioning, Busulfan, Gene Editing
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 25 participants (estimated)
- Lead sponsor
- St. Jude Children's Research HospitalUniversity, hospital or other
- Collaborators
- National Heart, Lung, and Blood Institute (NHLBI)
- Study start
- 21 Mar 2025 (actual)
- Primary completion
- Dec 2029 (estimated)
- Study completion
- Dec 2032 (estimated)
- First posted
- 17 Jul 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
Participants will receive a daily subcutaneous (under the skin) dose of motixafortide for up to 3 consecutive days to mobilize their hematopoietic stem and progenitor cells (HSPCs) into peripheral blood. Participants who cannot tolerate motixafortide may receive a daily subcutaneous dose of plerixafor as an alternative for 3-5 consecutive days. The collection of HSPCs will be done via apheresis. The collected HSPCs will be sent to a lab to genetically modify them using CRISPR/Cas9.
In the lab, the researchers will take the stem cells and purify them. The stem cells will then be mixed with the CRISPR-Cas9 gRNA ribonucleoprotein (RNP) complex to change (edit) the genes in the cells and produce the new gene edited cellular product. This gene edited drug product will be frozen until ready for infusion.
Once the cellular product is ready, participants will be given Busulfan (a chemotherapy medicine) intravenously (IV) for 4 days. The thawed gene product will be given IV about 48 hours after the completion of the last dose of busulfan.
Participants will be followed for 3 years on this study. After the three years, participants will be followed for 12 more years on a long-term follow-up study.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Patients with severe sickle cell disease (SCD)
| Arm | Type | Receives |
|---|---|---|
| Autologous, genetically modified CD34+ HSPCs Treatment | Experimental | Drug: Plerixafor, Drug: Busulfan, Biological: Gene-modified CD34+ cells, Drug: MotixafortideAll eligible participants receive intervention as described in the Detailed Description with the following: motixafortide, plerixafor, busulfan, and autologous, gene-modified CD34+ cells |
Who may qualify
- Ages
- 18 to 24 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (7 listed)
- Age ≥18 years and ≤24.9 years.
- Patients with SCD (Hb SS, Hb SB0 and Hb SB+ genotype) who have experienced EITHER (a) 2 or more SCD-related vaso-occlusive events (acute pain events, acute chest syndrome, priapism and splenic sequestration) per year in the 2-year period before screening, OR (b) administration of regular red blood cell (RBC) transfusions (≥8 transfusions in the 12 months preceding enrollment) EXCEPT if the RBC transfusions are being administered for primary or secondary stroke prevention and, in the opinion of the treating hematologist, cannot be safely discontinued after infusion of the gene modified drug product.
- Failure, intolerance, or refusal of hydroxyurea therapy.
- Patients must be eligible for autologous stem cell transplant as per investigator's judgment.
- Females of childbearing potential (i.e., those who are post-menarchal with an intact uterus and at least 1 ovary, and those who are less than 1 year postmenopausal) must agree to use acceptable method(s) of contraception from start of mobilization through at least 6 months post-infusion.
Key exclusion criteria (22 listed)
- Availability of an human leukocyte antigen (HLA)-matched sibling who is willing and able to donate an appropriate graft for hematopoietic cell transplantation (HCT).
- Karnofsky or Lansky performance score < 80.
- Pregnant, as confirmed by positive serum or urine pregnancy test within 14 days before enrollment (if female).
- Breastfeeding.
- Uncontrolled (undergoing appropriate treatment and with progression of clinical symptoms) or clinically significant bacterial, viral, or fungal infections within 1 month before enrollment.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| PlerixaforAlso: Mozobil®Given Subcutaneous (under the skin) | Drug | Autologous, genetically modified CD34+ HSPCs Treatment |
| BusulfanAlso: Myleran®, BusulfexGiven Intravenous (IV) | Drug | Autologous, genetically modified CD34+ HSPCs Treatment |
| Gene-modified CD34+ cellsGiven Intravenous (IV) | Biological | Autologous, genetically modified CD34+ HSPCs Treatment |
| MotixafortideAlso: AphexdaGiven Subcutaneous (under the skin) | Drug | Autologous, genetically modified CD34+ HSPCs Treatment |
Primary outcome
Incidence of neutrophil engraftment by day +42 after infusion of the CRISPR/Cas9-edited CD34+ HSPCs.
Time frame: Within 42 days of the cellular product infusion
Upon completion of the trial, summary statistics will be computed for the time to neutrophil engraftment.
Incidence of platelet engraftment by day +60 after infusion of the CRISPR/Cas9-edited CD34+ HSPCs.
Time frame: Within 60 days of the cellular product infusion
Upon completion of the trial, summary statistics will be computed for the time to platelet engraftment.
Sustenance of multi-lineage engraftment and polyclonal hematopoiesis as measured by counts of different clones of myeloid cells, T cells, B cells, and NK cells at 1 year after infusion of the CRISPR/Cas9-edited CD34+ HSPCs.
Time frame: Within 1 year of the cellular product infusion
Sustenance of multi-lineage engraftment will be described using descriptive statistics.
Frequency of off-target editing after infusion of the CRISPR/Cas9-edited CD34+ HSPCs.
Time frame: Within 3 years of the cellular product infusion
Frequency of off-target editing will be described using descriptive statistics.
Occurrence of secondary graft failure, clonal hematopoiesis, MDS, or AML
Time frame: Within 3 years of the cellular product infusion
Occurrence will be described using descriptive statistics.
4 secondary outcomes
Estimate the change in the annualized rate of SCD-related vaso-occlusive events (such as pain crises and acute chest syndrome events), starting at 3 months after the infusion of autologous CRISPR/Cas9-edited CD34+ HSPCs.
Time frame: From 3 months post the cellular product infusion to 3 years post infusion
Compare the change from baseline in the total blood hemoglobin concentration.
Time frame: From time of screening to 3 years post infusion
Compare the change from baseline in the fraction of red blood cells (RBCs) containing HbF.
Time frame: From time of screening to 3 years post infusion
Change in incidence of packed RBC transfusions.
Time frame: Within 1 year prior to infusion and within 3 months to 1 year post infusion
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 Oct 2026.
- St. Jude Children's Research HospitalLink provided by the sponsor.
- Clinical Trials Open at St. JudeLink provided by the sponsor.
- Individual participant dataSharing planned: Yes. Individual participant de-identified datasets containing the variables analyzed in the published article will be made available (related to the study primary or secondary objectives contained in the publication). Supporting documents such as the protocol, statistical analyses plan, and informed consent are available through the CTG website for the specific study. Data used to generate the published article will be made available at the time of article publication. Investigators who seek access to individual level de-identified data will contact the computing team in the Department of Biostatistics (ClinTrialDataRequest@stjude.org) who will respond to the data request.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Akshay Sharma, MBBS, MSc principal investigator
St. Jude Children's Research Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Plerixafor, Busulfan and Gene-modified CD34+ cells in people with Sickle Cell Disease, run by St. Jude Children's Research Hospital at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 22 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 15 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Akshay Sharma, MBBS, MSc (888-226-4343, referralinfo@stjude.org), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of neutrophil engraftment by day +42 after infusion of the CRISPR/Cas9-edited CD34+ HSPCs. (time frame: Within 42 days of the cellular product infusion). The study lists 5 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 15 Oct 2026.