Safety and Efficacy of KL003 Cell Injection in Severe Sickle Cell Disease

NCT07436767 | Not Yet Recruiting

• 1 other identifier: CP-KL003-006/02
• Study Type: interventional
• Target: 3
• Locations: 0 countries
• Sites: N/A

Brief 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. After successful hematopoietic stem cell engraftment is achieved in the first subject, cell infusion will be initiated for subsequent subjects.

Conditions

Sickle Cell Disease

Outcome Measures

Primary Outcomes (2)

• 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.

  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

  Adverse events will be monitored from baseline through study completion, up to 24 months.

Study Arms (1)

Experimental

EXPERIMENTAL

A single intravenous infusion of ≥ 3.0 × 10\^6 CD34+ cells/kg was administered.

Genetic: KL003 Cell Injection

Interventions

KL003 Cell Injection GENETIC

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.

Experimental

Eligibility Criteria

• Age: 12 Years - 50 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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.

You may not qualify if:

• 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%
• 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.

Sponsors & Collaborators

• Institute of Hematology & Blood Diseases Hospital, China: lead

MeSH Terms

Conditions

Anemia, Sickle Cell

Condition Hierarchy (Ancestors)

Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Director of the Red Blood Cell Diseases Center & Director of the Regenerative Medicine Clinic

Study Record Dates

• First Submitted: February 14, 2026
• First Posted: February 27, 2026
• Study Start: March 1, 2026
• Primary Completion (Estimated): September 30, 2028
• Study Completion (Estimated): December 31, 2028
• Last Updated: February 27, 2026

Record last verified: 2026-02

Data Sharing

• IPD Sharing: Will not share