Sickle Cell DiseasePhase 1/2NCT04774536
Transplantation of Clustered Regularly Interspaced Short Palindromic Repeats Modified Hematopoietic Progenitor Stem Cells (CRISPR_SCD001) in Patients With Severe Sickle Cell Disease
A Phase 1/2 study testing CRISPR_SCD001 in people with Sickle Cell Disease, run by Mark Walters, MD at 3 sites in United States.
Trial summary
This is an open label, non-randomized, 2-center, phase 1/2 trial of a single infusion of sickle allele modified cluster of differentiation (CD34+) hematopoietic stem progenitor cells (HSPCs) in subjects with in subjects ≥12 years old to 35 years old severe Sickle Cell Disease (SCD). Read more about this study
- Who is it for?
- Ages 12 to 35 years
- Conditions the study is for
- Sickle Cell Disease
- What is being tested?
- CRISPR_SCD001
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 9 participants
- Where
- 3 sitesUnited States
- Study dates
- 22 Aug 2025to 1 Mar 2030
- Phase
- Phase 1/2
- 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 12 to 35 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Male or female 12.00 - 34.99 years of age (at time of consent) who have one or more of the following:
- 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); ACS is defined as a new radiodensity on chest imaging accompanied by fever and/or respiratory symptoms, treated in the hospital with supplemental oxygen at a minimum.
- History of at least 4 severe vaso-occlusive pain events in the 2-year period preceding enrollment despite the institution of supportive care measures (i.e. a pain management plan and/or treatment with hydroxyurea); painful episodes related to or any sickle-related acute event are acceptable; a severe painful vaso-occlusive event is defined as receiving analgesic treatment (opioid or other analgesic via parenteral (PN) route) for longer than 24 -hours in a hospital or emergency room (ER) observation unit visit or at least 2 visits in a day unit or ER over 72 hours with both visits requiring parenteral analgesics.
- 2 or more episodes of splenic sequestration, defined as an acute decrease in hemoglobin by at least 2 g/dL accompanied by splenomegaly in the 2 years before enrollment.
- Recurrent priapism (episodes lasting at least 4 hours at least twice in the last 12 months or 3 times in the last 24 months before enrollment) recalcitrant to medical treatment
- Any episode of hepatic sequestration defined as right upper quadrant pain, hepatomegaly, and rapidly decreasing hemoglobin level with hepatic dysfunction within the 2 years before enrollment.
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- Leg ulcer recalcitrant to treatment within 2 years prior to enrollment.
- Participants must have adequate physical function as measured by all of the following:
- Karnofsky performance score ≥60.
- Cardiac function: Left ventricular ejection fraction (LVEF) >40%; or LV shortening fraction > 26% by cardiac echocardiogram or by (multiple-gated acquisition) MUGA scan.
- Pulmonary function: Pulse oximetry with a baseline O2 saturation of ≥85% and diffusion capacity of lung for carbon monoxide(DLCO) > 40% (corrected for hemoglobin).
- Renal function: Serum creatinine ≤ 1.5 x upper limit of normal for age and estimated or measured creatinine clearance ≥ 70 mL/min/1.73 m2.
- Hepatic function:
- Serum conjugated (direct) bilirubin < 2x upper limit of normal for age as per local laboratory. Participants with hyperbilirubinemia as the result of hyperhemolysis, or a severe drop in hemoglobin post blood transfusion, are not excluded.
- Alanine aminotransferase (ALT) and aspartate aminotransferase (AS < 5 times upper limit of normal as per local laboratory.
- Participants who have liver iron concentration (LIC) on liver MRI of ≥12 mg Fe/g liver dry weight should also have MR elastography (MRE) or Ultrasound elastography obtained. If severe fibrosis is present, no liver biopsy will be performed and participant is excluded. Children with LIC >12 and negative elastography imaging are eligible if a clinically indicated liver biopsy shows no significant fibrosis.
- Baseline prothrombin time or partial thromboplastin time <1.5 ULN except if receiving a prophylactic anticoagulant which causes an elevated prothrombin or partial thromboplastin time.
- Written informed consent or assent obtained from subject or subject's legal representative and ability for subject to comply with the requirements of the study.
Who cannot join
- Participants with uncontrolled bacterial, viral or fungal infection in the 6 weeks before enrollment (currently taking medication with evidence of progression of clinical symptoms or radiologic findings).
- Participants with evidence of HIV infection or seropositivity for HIV or active hepatitis B or C.
- Participants who have received a Hematopoietic Cell Transplant (HCT)
- Participants who have received a solid organ transplant.
- Participants who have participated in another clinical trial in which the participant received an investigational or off-label use of a drug or device within 3 months prior to enrollment.
- Females who are pregnant or breast feeding.
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- Females of child bearing potential (to include all female participants > 10 years of age, unless postmenopausal for a minimum of 1 year before the time of consent or surgically sterilized) who do not agree to practice two (2) effective methods of contraception at the same time, or who do not agree to practice true abstinence when this is in line with the preferred and usual lifestyle of the subject, from the time of signing of informed consent through 12 months post-stem cell infusion.
- Males (even if surgically sterilized) who do not agree to practice effective barrier contraception, or who do not agree to practice true abstinence from the time of signing informed consent through 12 months post-stem cell infusion.
- Participants who have had a stroke OR who are receiving red blood cell (RBC) transfusions to prevent primary stroke or silent cerebral infarction.
- Patients who have a suitable human leukocyte antigen identical (HLA-ID) sibling donor willing and able to donate bone marrow.
- Presence of a condition or abnormality that in the opinion of the Investigator would compromise the safety of the patient or the quality of the data.
- Any non-homozygous sickle hemoglobin (HbSS) genotype of SCD
- Either or both of the following findings on screening bone marrow aspirate/biopsy:
- diagnosis of myelodysplastic syndrome (MDS) based on morphology and/or cytogenetics (based on WHO definitions) OR
- any evidence of a pathogenic clonal variant in any candidate gene detected by a standard, licensed next-generation sequencing clinical assay for gene mutations associated with hematological malignancies.
- The participant has an identified pathogenic mutation associated with myeloid malignancy as identified by RHP or a variant of unknown significance (VUS) judged to be pathogenic for myeloid malignancy as determined by one or more members of the adjudication panel.
Trial locations and contacts
3 sites in 1 country. Contact the study team or a site near you to ask about taking part.
United States3 sites, 3 recruiting
- Recruiting
University of California, Davis
Davis, California, 95817
Crystal Gariano, 916-501-9393, cagariano@health.ucdavis.edu
Mehrdad Abedi, MD
- Recruiting
University of California, Los Angeles
Los Angeles, California, 90095
Augustine Fernandes, PhD, afernandes@mednet.ucla.edu
Vladimir Kustanovich, PhD, VKustanovich@mednet.ucla.edu
- Recruiting
UCSF Benioff Children's Hospital
Oakland, California, 94609
Marci Moriarty, RN BSN, (510) 428-3885 ext. 5396, marci.moriarty@ucsf.edu
Cyrus Bascon, (510) 428-3885 ext. 6953, cyrus.bascon@ucsf.edu
About this study
Official title: Transplantation of CRISPRCas9 Corrected Hematopoietic Stem Cells (CRISPR_SCD001) in Patients With Severe Sickle Cell Disease
This is an open label, non-randomized, 2-center, phase 1/2 trial of a single infusion of sickle allele modified cluster of differentiation (CD34+) hematopoietic stem progenitor cells (HSPCs) in subjects with in subjects ≥12 years old to 35 years old severe Sickle Cell Disease (SCD). The study will evaluate the hematopoietic stem cell transplantation (HSCT) using CRISPR/Cas9 edited red blood cells (known as CRISPR_SCD001 Drug Product).
Read the detailed description
This is an open label, non-randomized, 2-center, phase 1/2 trial of a single infusion of sickle allele modified CD34+ HSPCs in subjects with severe SCD. The primary endpoint of the trial will determine the safety of CRISPR_SCD001 through a 3+3 design with staggered enrollment and a pause in enrollment for safety review after each of the first 3 patients has had drug product infused. After safety is assessed in the 3rd patient, enrollment of the next 3 patients will not be staggered. The first six subjects will be adults. If CRISPR_SCD001 is determined to be safe in the first six subjects, the trial will continue to enroll 3 adolescents 12 - 18 years of age to evaluate the safety in younger patients. The younger age cohort also will follow staggered enrollment.
- Conditions
- Sickle Cell Disease
- Being tested
- CRISPR_SCD001
- 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.
CRISPR_SCD001Drug
CRISPR_SCD001 is administered by IV infusion following myeloablative conditioning with busulfan.
| Study group | Role | What they receive |
|---|---|---|
| CRISPR_SCD001 Drug Product | Experimental | 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. Receives: Drug: CRISPR_SCD001 |
What is being measured
The primary outcome is the main question the study is designed to answer.
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)
The adverse event rate will be summarized using descriptive statistics, together with 95% confidence intervals where appropriate. No formal statistical hypothesis testing will be performed. Adverse events defined: failure of engraftment, malignant clonal expansion related to genomic editing or death.
Measured: 24 months post-transplant
Secondary outcomes (12)
Change in the annualized vaso-occlusive pain event (VOE) rates.
Change in the annualized vast-occlusive pain event (VOE) rates comparing the 24 months before with 24 months after the infusion of drug product.
Measured: 24 months pre-transplant to 24 months post-transplant
Graft rejection defined as having an absolute neutrophil count (ANC) < 500 at 42 days post-infusion
A delay in platelet and/or neutrophil engraftment will be captured.
Measured: 42 days post-transplant
Time to neutrophil recovery defined as the first of 3 measurements on different days when the patient has an absolute neutrophil count (ANC) of ≥500/µL after transplant.
Neutrophil recovery as part of the overall hematological recovery.
Measured: 24 months post-transplant
Time to platelet recovery is defined as the first day of a minimum of 3 measurements on different days that the patient has achieved a platelet count > 50,000/µL AND did not receive a platelet transfusion in the previous 7 days.
Platelet recovery as part of the overall hematological recovery.
Measured: baseline, through 24 months post-transplant
Rate of improvement in Hemoglobin S (HbS) fraction as measured by hemoglobin electrophoresis, as percent of total.
Hemolysis markers.
Measured: baseline, through 24 months post-transplant
Rate of normalization in hemoglobin (hgb) level as measured by clinical hematology (laboratory) test.
Hemolysis markers.
Measured: baseline, through 24 months post-transplant
Rate of normalization of lactate dehydrogenase (LDH), reticulocyte count, and haptoglobin, as measured by clinical hematology and serum chemistry (laboratory) tests.
Hemolysis markers.
Measured: baseline, through 24 months post-transplant
Frequency of hepatic veno-occlusive disease (VOD) as measured by Seattle or Baltimore Criteria for VOD Diagnosis.
Hematologic and non-hematologic toxicities of autologous transplantation with CRISPR-Cas9 sickle-corrected HSPCs.
Measured: 24 months post-transplant
Frequency of hepatic idiopathic pneumonia syndrome (IPS) as measured by the Idiopathic pneumonia syndrome (IPS) criteria.
Hematologic and non-hematologic toxicities of autologous transplantation with CRISPR-Cas9 sickle-corrected HSPCs.
Measured: 24 months post-transplant
Frequency of central nervous system (CNS) toxicity (reversible posterior leukoencephalopathy syndrome [RPLS] or posterior reversible encephalopathy syndrome [PRES], hemorrhage, and seizures).
Hematologic and non-hematologic toxicities of autologous transplantation with CRISPR-Cas9 sickle-corrected HSPCs.
Measured: 24 months post-transplant
Frequency of cytomegalovirus (CMV) infection, invasive fungal infection, and any other serious viral or bacterial infection
Hematologic and non-hematologic toxicities of autologous transplantation with CRISPR-Cas9 sickle-corrected HSPCs.
Measured: 24 months post-transplant
Frequency of sickle gene-editing (correction and insertion/deletion) and off-target site-1 editing in marrow and peripheral blood mononuclear cells.
Stability of gene-editing in hematopoietic cells by genotyping studies.
Measured: 3 months, 1 and 2 years post-transplant
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Mar 2021
- Study start22 Aug 2025
- Primary completion1 Mar 2030 (estimated)
- Study completion1 Mar 2030 (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.
- 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 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 9 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Mark Walters, MD
- Sponsor type
- University, hospital or other
- Collaborators
- University of California, Los Angeles, University of California, Berkeley
- Responsible party
- Sponsor-investigator, Mark Walters, MD, University of California, San Francisco
- Investigators
- Mark Walters, MD, Principal investigator, UCSF Benioff Children's Hospital Oakland
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04774536
- Sponsor's protocol ID
- 21-33287
- Status last verified
- Apr 2026
- First submitted
- 17 Feb 2021
- Last updated
- 20 Oct 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 20 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Transplantation of CRISPRCas9 Corrected Hematopoietic Stem Cells (CRISPR_SCD001) in Patients With Severe Sickle Cell Disease
- ClinicalTrials.gov ID
- NCT04774536
- Acronym / sponsor ID
- 21-33287
- Conditions
- Sickle Cell Disease
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 9 participants (estimated)
- Lead sponsor
- Mark Walters, MDUniversity, hospital or other
- Collaborators
- University of California, Los Angeles, University of California, Berkeley
- Study start
- 22 Aug 2025 (actual)
- Primary completion
- 1 Mar 2030 (estimated)
- Study completion
- 1 Mar 2030 (estimated)
- First posted
- 1 Mar 2021 (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 is an open label, non-randomized, 2-center, phase 1/2 trial of a single infusion of sickle allele modified cluster of differentiation (CD34+) hematopoietic stem progenitor cells (HSPCs) in subjects with in subjects ≥12 years old to 35 years old severe Sickle Cell Disease (SCD). The study will evaluate the hematopoietic stem cell transplantation (HSCT) using CRISPR/Cas9 edited red blood cells (known as CRISPR_SCD001 Drug Product).
Detailed description
This is an open label, non-randomized, 2-center, phase 1/2 trial of a single infusion of sickle allele modified CD34+ HSPCs in subjects with severe SCD. The primary endpoint of the trial will determine the safety of CRISPR_SCD001 through a 3+3 design with staggered enrollment and a pause in enrollment for safety review after each of the first 3 patients has had drug product infused. After safety is assessed in the 3rd patient, enrollment of the next 3 patients will not be staggered. The first six subjects will be adults. If CRISPR_SCD001 is determined to be safe in the first six subjects, the trial will continue to enroll 3 adolescents 12 - 18 years of age to evaluate the safety in younger patients. The younger age cohort also will follow staggered enrollment.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| CRISPR_SCD001 Drug Product | Experimental | Drug: CRISPR_SCD001CRISPR_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. |
Who may qualify
- Ages
- 12 to 35 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (18 listed)
- Male or female 12.00 - 34.99 years of age (at time of consent) who have one or more of the following:
- Participants must have adequate physical function as measured by all of the following:
- Participants who have liver iron concentration (LIC) on liver MRI of ≥12 mg Fe/g liver dry weight should also have MR elastography (MRE) or Ultrasound elastography obtained. If severe fibrosis is present, no liver biopsy will be performed and participant is excluded. Children with LIC >12 and negative elastography imaging are eligible if a clinically indicated liver biopsy shows no significant fibrosis.
- Baseline prothrombin time or partial thromboplastin time <1.5 ULN except if receiving a prophylactic anticoagulant which causes an elevated prothrombin or partial thromboplastin time.
- Written informed consent or assent obtained from subject or subject's legal representative and ability for subject to comply with the requirements of the study.
Key exclusion criteria (16 listed)
- Participants with uncontrolled bacterial, viral or fungal infection in the 6 weeks before enrollment (currently taking medication with evidence of progression of clinical symptoms or radiologic findings).
- Participants with evidence of HIV infection or seropositivity for HIV or active hepatitis B or C.
- Participants who have received a Hematopoietic Cell Transplant (HCT)
- Participants who have received a solid organ transplant.
- Participants who have participated in another clinical trial in which the participant received an investigational or off-label use of a drug or device within 3 months prior to 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 |
|---|---|---|
| CRISPR_SCD001CRISPR_SCD001 is administered by IV infusion following myeloablative conditioning with busulfan. | Drug | CRISPR_SCD001 Drug Product |
Primary outcome
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)
Time frame: 24 months post-transplant
The adverse event rate will be summarized using descriptive statistics, together with 95% confidence intervals where appropriate. No formal statistical hypothesis testing will be performed. Adverse events defined: failure of engraftment, malignant clonal expansion related to genomic editing or death.
12 secondary outcomes
Change in the annualized vaso-occlusive pain event (VOE) rates.
Time frame: 24 months pre-transplant to 24 months post-transplant
Graft rejection defined as having an absolute neutrophil count (ANC) < 500 at 42 days post-infusion
Time frame: 42 days post-transplant
Time to neutrophil recovery defined as the first of 3 measurements on different days when the patient has an absolute neutrophil count (ANC) of ≥500/µL after transplant.
Time frame: 24 months post-transplant
Time to platelet recovery is defined as the first day of a minimum of 3 measurements on different days that the patient has achieved a platelet count > 50,000/µL AND did not receive a platelet transfusion in the previous 7 days.
Time frame: baseline, through 24 months post-transplant
Rate of improvement in Hemoglobin S (HbS) fraction as measured by hemoglobin electrophoresis, as percent of total.
Time frame: baseline, through 24 months post-transplant
Rate of normalization in hemoglobin (hgb) level as measured by clinical hematology (laboratory) test.
Time frame: baseline, through 24 months post-transplant
Rate of normalization of lactate dehydrogenase (LDH), reticulocyte count, and haptoglobin, as measured by clinical hematology and serum chemistry (laboratory) tests.
Time frame: baseline, through 24 months post-transplant
Frequency of hepatic veno-occlusive disease (VOD) as measured by Seattle or Baltimore Criteria for VOD Diagnosis.
Time frame: 24 months post-transplant
Frequency of hepatic idiopathic pneumonia syndrome (IPS) as measured by the Idiopathic pneumonia syndrome (IPS) criteria.
Time frame: 24 months post-transplant
Frequency of central nervous system (CNS) toxicity (reversible posterior leukoencephalopathy syndrome [RPLS] or posterior reversible encephalopathy syndrome [PRES], hemorrhage, and seizures).
Time frame: 24 months post-transplant
Frequency of cytomegalovirus (CMV) infection, invasive fungal infection, and any other serious viral or bacterial infection
Time frame: 24 months post-transplant
Frequency of sickle gene-editing (correction and insertion/deletion) and off-target site-1 editing in marrow and peripheral blood mononuclear cells.
Time frame: 3 months, 1 and 2 years post-transplant
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 20 Oct 2026.
- Individual participant dataSharing planned: No
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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.
Mark Walters, MD principal investigator
UCSF Benioff Children's Hospital Oakland
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing CRISPR_SCD001 in people with Sickle Cell Disease, run by Mark Walters, MD at 3 sites 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 18 inclusion and 16 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 20 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 3 sites 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 combined 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.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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 Mark Walters, MD ((510) 428-3374, Mark.Walters@ucsf.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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) (time frame: 24 months post-transplant). 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 20 Oct 2026.