Sickle Cell DiseaseNCT07436767
Safety and Efficacy of KL003 Cell Injection in Severe Sickle Cell Disease
A study testing KL003 Cell Injection in people with Sickle Cell Disease, run by Institute of Hematology & Blood Diseases Hospital, China at 1 site in China.
Trial summary
It is a single-arm, single-center, open-label, single-dose study. A total of three subjects with severe sickle cell disease (SCD), aged 12-50 years (inclusive), are planned to receive cell infusion. Read more about this study
- Who is it for?
- Ages 12 to 50 years
- Conditions the study is for
- Sickle Cell Disease
- What is being tested?
- KL003 Cell Injection
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 3 participants
- Where
- 1 siteChina
- Study dates
- 1 Mar 2028to 31 Dec 2030
- Phase
- Interventional
- Are there results?
- None posted in the registry
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 50 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 12-50 years of age (inclusive) at the time of screening
- Diagnosed with sickle cell disease (SCD) with a βS/βS, βS/β0 or βS/β+ genotype.
- Experienced at least 4 severe vaso-occlusive events (VOEs) in the two years before informed consent, despite supportive care measures (e.g., a pain management plan).
- Have a Karnofsky Performance Status (KPS) score (for subjects ≥16 years) or Lansky Performance Status (LPS) score (for subjects <16 years) of ≥60.
- Have experienced either hydroxyurea (HU) failure at any time in the past or demonstrated intolerance to HU.
- Subject must have been treated and followed for at least two years before informed consent at the medical center that maintained detailed records of their sickle cell disease history.
- Be willing and able to comply with all study procedures and visit schedules.
- The subject and/or their legally authorized representative must voluntarily agree to participate, sign the informed consent form, and be capable of completing all follow-up assessments required by the protocol.
Who cannot join
- Have any severe active infection (fungal, bacterial, viral, tuberculosis, or other), including active Hepatitis B (defined as serum HBV-DNA ≥2000 IU/mL), active Hepatitis C virus (HCV) infection, positive human immunodeficiency virus (HIV) antibody, or active syphilis.
- Inadequate bone marrow function, as defined by an absolute neutrophil count of <1.0 x 10^9/L (<0.5 x 10^9/L for subjects on hydroxyurea treatment) or a platelet count <100 x 10^9/L.
- Have severe cerebrovascular disease, including a history of significant ischemic or hemorrhagic stroke, abnormal Transcranial Doppler (TCD) flow velocities requiring chronic transfusion therapy (>200 cm/s), occlusion or stenosis of the circle of Willis, or any history of moyamoya disease.
- Baseline oxygen saturation < 90% without supplemental oxygen (excluding periods of SCD crisis, severe anemia or infection).
- Baseline carbon monoxide diffusing capacity (DLCO) < 50% (corrected for Hb) in the absence of infection. If DLCO cannot be assessed due to age or cognitive limitations, there must be a normal respiratory exam, a chest radiograph without pulmonary infiltrates, and oxygen saturation by pulse oximetry ≥ 90% on room air.
- Baseline left ventricular ejection fraction (LVEF) < 45%
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- Clinically significant pulmonary hypertension at baseline, as defined by the requirement for ongoing pharmacologic treatment or the consistent or intermittent use of supplemental oxygen.
- Baseline estimated glomerular filtration rate (eGFR) <70 mL/min/1.73 m^2
- Advanced liver disease
- Have a definite contraindication to stem cell collection.
- Have any prior or current malignancy, myeloproliferative disorder, or immunodeficiency disease.
- Have white blood count <3.0 x 10^9/L and/or platelet count <100.0 x 10^9/L not due to hypersplenism.
- Have a diagnosis of compound alpha-thalassemia (excluding silent carrier).
- Have significant iron overload at screening, defined as severe iron overload on liver MRI, or serum ferritin levels >2000 ng/mL, or cardiac T2* <10 ms.
- Have positive irregular red cell antibodies or platelet antibodies.
- Be eligible for allogeneic hematopoietic stem cell transplantation and have an identified willing, fully HLA-matched donor.
- Prior receipt of gene therapy or allogeneic hematopoietic stem cell transplantation.
- Immediate family member with a known or suspected Familial Cancer Syndrome (including but not limited to hereditary breast and ovarian cancer syndrome, hereditary non-polyposis colorectal cancer syndrome, and familial adenomatous polyposis).
- Have a diagnosed major psychiatric disorder or predisposition that, in the Investigator's opinion, would severely compromise the ability to participate in the clinical study.
- Have an uncorrectable coagulation disorder or a history of severe hemorrhagic disease.
- Have any other condition that, in the opinion of the treating physician, renders the subject unsuitable for hematopoietic stem cell transplantation.
- Have a known allergy to the investigational drug(s) (e.g., plerixafor, busulfan) or their components.
- Have participated in or are currently participating in another interventional clinical study within 3 months before screening.
- Received a live vaccine within 6 weeks before screening.
- Be pregnant or breastfeeding.
- The subject or their partner is unwilling to use medically acceptable effective contraception during the 32-month study period.
- Unable to receive red blood cell transfusion.
- The subject or their parent/guardian is unable to adhere to the study protocol.
- Have any other condition deemed by the Investigator to make the subject unsuitable for participation in this study.
Trial locations and contacts
1 site in 1 country.
About this study
It is a single-arm, single-center, open-label, single-dose study. A total of three subjects with severe sickle cell disease (SCD), aged 12-50 years (inclusive), are planned to receive cell infusion. After successful hematopoietic stem cell engraftment is achieved in the first subject, cell infusion will be initiated for subsequent subjects.
- Conditions
- Sickle Cell Disease
- Being tested
- KL003 Cell Injection
- 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.
KL003 Cell InjectionGenetic
KL003 is an autologous CD34⁺ hematopoietic stem cell gene therapy product in which the βA-T87Q-globin gene is transduced via a lentiviral vector. Through genetic modification, the patient's autologous CD34⁺ hematopoietic stem cells are engineered to differentiate into red blood cells expressing functional β-globin, thereby increasing overall hemoglobin levels, improving anemia, and ultimately eliminating transfusion dependence.
| Study group | Role | What they receive |
|---|---|---|
| Experimental | Experimental | A single intravenous infusion of ≥ 3.0 × 10^6 CD34+ cells/kg was administered. Receives: Genetic: KL003 Cell Injection |
What is being measured
The primary outcome is the main question the study is designed to answer.
The proportion of subjects who achieve successful engraftment of CD34⁺ cells modified with the βA-T87Q-globin lentiviral vector
Achieving neutrophil engraftment, defined as an absolute neutrophil count ≥ 0.5 × 10^9/L for three consecutive days.
Measured: From Day 0 to Day 42 after cell infusion
Incidence and severity of adverse events
Use Common Terminology Criteria for Adverse Events (CTCAE) Version 5 to assess the adverse event
Measured: Adverse events will be monitored from baseline through study completion, up to 24 months.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Feb 2026
- Study start1 Mar 2028 (estimated)
- Primary completion30 Sep 2030 (estimated)
- Study completion31 Dec 2030 (estimated)
Why it stopped: Difficulty identifying eligible patients.
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 Institute of Hematology & Blood Diseases Hospital, ChinaThe 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 N/A
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 3 participants (estimated)
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Jun Shi, Institute of Hematology & Blood Diseases Hospital, China
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07436767
- Sponsor's protocol ID
- CP-KL003-006/02
- Status last verified
- Nov 2026
- First submitted
- 14 Feb 2026
- Last updated
- 5 Nov 2026
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 5 Nov 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Safety and Efficacy of KL003 Cell Injection in Severe Sickle Cell Disease
- ClinicalTrials.gov ID
- NCT07436767
- Acronym / sponsor ID
- CP-KL003-006/02
- Conditions
- Sickle Cell Disease
- Study type
- Interventional
- Recruitment status
- SuspendedReason stopped: Difficulty identifying eligible patients.
- Enrolment
- 3 participants (estimated)
- Lead sponsor
- Institute of Hematology & Blood Diseases Hospital, ChinaUniversity, hospital or other
- Study start
- 1 Mar 2028 (estimated)
- Primary completion
- 30 Sep 2030 (estimated)
- Study completion
- 31 Dec 2030 (estimated)
- First posted
- 27 Feb 2026 (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.
It is a single-arm, single-center, open-label, single-dose study. A total of three subjects with severe sickle cell disease (SCD), aged 12-50 years (inclusive), are planned to receive cell infusion. After successful hematopoietic stem cell engraftment is achieved in the first subject, cell infusion will be initiated for subsequent subjects.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Experimental | Experimental | Genetic: KL003 Cell InjectionA single intravenous infusion of ≥ 3.0 × 10^6 CD34+ cells/kg was administered. |
Who may qualify
- Ages
- 12 to 50 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (8 listed)
- 12-50 years of age (inclusive) at the time of screening
- Diagnosed with sickle cell disease (SCD) with a βS/βS, βS/β0 or βS/β+ genotype.
- Experienced at least 4 severe vaso-occlusive events (VOEs) in the two years before informed consent, despite supportive care measures (e.g., a pain management plan).
- Have a Karnofsky Performance Status (KPS) score (for subjects ≥16 years) or Lansky Performance Status (LPS) score (for subjects <16 years) of ≥60.
- Have experienced either hydroxyurea (HU) failure at any time in the past or demonstrated intolerance to HU.
Key exclusion criteria (29 listed)
- Have any severe active infection (fungal, bacterial, viral, tuberculosis, or other), including active Hepatitis B (defined as serum HBV-DNA ≥2000 IU/mL), active Hepatitis C virus (HCV) infection, positive human immunodeficiency virus (HIV) antibody, or active syphilis.
- Inadequate bone marrow function, as defined by an absolute neutrophil count of <1.0 x 10^9/L (<0.5 x 10^9/L for subjects on hydroxyurea treatment) or a platelet count <100 x 10^9/L.
- Have severe cerebrovascular disease, including a history of significant ischemic or hemorrhagic stroke, abnormal Transcranial Doppler (TCD) flow velocities requiring chronic transfusion therapy (>200 cm/s), occlusion or stenosis of the circle of Willis, or any history of moyamoya disease.
- Baseline oxygen saturation < 90% without supplemental oxygen (excluding periods of SCD crisis, severe anemia or infection).
- Baseline carbon monoxide diffusing capacity (DLCO) < 50% (corrected for Hb) in the absence of infection. If DLCO cannot be assessed due to age or cognitive limitations, there must be a normal respiratory exam, a chest radiograph without pulmonary infiltrates, and oxygen saturation by pulse oximetry ≥ 90% on room air.
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 |
|---|---|---|
| KL003 Cell InjectionKL003 is an autologous CD34⁺ hematopoietic stem cell gene therapy product in which the βA-T87Q-globin gene is transduced via a lentiviral vector. Through genetic modification, the patient's autologous CD34⁺ hematopoietic stem cells are engineered to differentiate into red blood cells expressing functional β-globin, thereby increasing overall hemoglobin levels, improving anemia, and ultimately eliminating transfusion dependence. | Genetic | Experimental |
Primary outcome
The proportion of subjects who achieve successful engraftment of CD34⁺ cells modified with the βA-T87Q-globin lentiviral vector
Time frame: From Day 0 to Day 42 after cell infusion
Achieving neutrophil engraftment, defined as an absolute neutrophil count ≥ 0.5 × 10^9/L for three consecutive days.
Incidence and severity of adverse events
Time frame: Adverse events will be monitored from baseline through study completion, up to 24 months.
Use Common Terminology Criteria for Adverse Events (CTCAE) Version 5 to assess the adverse event
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Nov 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. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing KL003 Cell Injection in people with Sickle Cell Disease, run by Institute of Hematology & Blood Diseases Hospital, China at 1 site in China.
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 8 inclusion and 29 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: paused, as of the sponsor's last update on 5 Nov 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 China. 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.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
The proportion of subjects who achieve successful engraftment of CD34⁺ cells modified with the βA-T87Q-globin lentiviral vector (time frame: From Day 0 to Day 42 after cell infusion). The study lists 2 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 5 Nov 2026.