Ignite Creation Date:
2026-03-26 @ 3:18 PM
Ignite Modification Date:
2026-03-31 @ 1:52 AM
Study NCT ID:
NCT07436767
Status:
NOT_YET_RECRUITING
Last Update Posted:
2026-02-27
First Post:
2026-02-14
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Safety and Efficacy of KL003 Cell Injection in Severe Sickle Cell Disease
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2026-03-25'}, 'conditionBrowseModule': {'meshes': [{'id': 'D000755', 'term': 'Anemia, Sickle Cell'}], 'ancestors': [{'id': 'D000745', 'term': 'Anemia, Hemolytic, Congenital'}, {'id': 'D000743', 'term': 'Anemia, Hemolytic'}, {'id': 'D000740', 'term': 'Anemia'}, {'id': 'D006402', 'term': 'Hematologic Diseases'}, {'id': 'D006425', 'term': 'Hemic and Lymphatic Diseases'}, {'id': 'D006453', 'term': 'Hemoglobinopathies'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}]}}, 'protocolSection': {'designModule': {'phases': ['NA'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 3}}, 'statusModule': {'overallStatus': 'NOT_YET_RECRUITING', 'startDateStruct': {'date': '2026-03-01', 'type': 'ESTIMATED'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2026-02', 'completionDateStruct': {'date': '2028-12-31', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2026-02-23', 'studyFirstSubmitDate': '2026-02-14', 'studyFirstSubmitQcDate': '2026-02-23', 'lastUpdatePostDateStruct': {'date': '2026-02-27', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2026-02-27', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2028-09-30', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'The proportion of subjects who achieve successful engraftment of CD34⁺ cells modified with the βA-T87Q-globin lentiviral vector', 'timeFrame': 'From Day 0 to Day 42 after cell infusion', 'description': 'Achieving neutrophil engraftment, defined as an absolute neutrophil count ≥ 0.5 × 10\\^9/L for three consecutive days.'}, {'measure': 'Incidence and severity of adverse events', 'timeFrame': 'Adverse events will be monitored from baseline through study completion, up to 24 months.', 'description': 'Use Common Terminology Criteria for Adverse Events (CTCAE) Version 5 to assess the adverse event'}]}, 'oversightModule': {'isFdaRegulatedDrug': False, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'conditions': ['Sickle Cell Disease']}, 'descriptionModule': {'briefSummary': '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.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT'], 'maximumAge': '50 Years', 'minimumAge': '12 Years', 'healthyVolunteers': False, 'eligibilityCriteria': "Inclusion Criteria:\n\n* 12-50 years of age (inclusive) at the time of screening\n* Diagnosed with sickle cell disease (SCD) with a βS/βS, βS/β0 or βS/β+ genotype.\n* 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).\n* Have a Karnofsky Performance Status (KPS) score (for subjects ≥16 years) or Lansky Performance Status (LPS) score (for subjects \\<16 years) of ≥60.\n* Have experienced either hydroxyurea (HU) failure at any time in the past or demonstrated intolerance to HU.\n* 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.\n* Be willing and able to comply with all study procedures and visit schedules.\n* 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.\n\nExclusion Criteria:\n\n* 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.\n* 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.\n* 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.\n* Baseline oxygen saturation \\< 90% without supplemental oxygen (excluding periods of SCD crisis, severe anemia or infection).\n* 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.\n* Baseline left ventricular ejection fraction (LVEF) \\< 45%\n* Clinically significant pulmonary hypertension at baseline, as defined by the requirement for ongoing pharmacologic treatment or the consistent or intermittent use of supplemental oxygen.\n* Baseline estimated glomerular filtration rate (eGFR) \\<70 mL/min/1.73 m\\^2\n* Advanced liver disease\n* Have a definite contraindication to stem cell collection.\n* Have any prior or current malignancy, myeloproliferative disorder, or immunodeficiency disease.\n* Have white blood count \\<3.0 x 10\\^9/L and/or platelet count \\<100.0 x 10\\^9/L not due to hypersplenism.\n* Have a diagnosis of compound alpha-thalassemia (excluding silent carrier).\n* 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.\n* Have positive irregular red cell antibodies or platelet antibodies.\n* Be eligible for allogeneic hematopoietic stem cell transplantation and have an identified willing, fully HLA-matched donor.\n* Prior receipt of gene therapy or allogeneic hematopoietic stem cell transplantation.\n* 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).\n* 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.\n* Have an uncorrectable coagulation disorder or a history of severe hemorrhagic disease.\n* Have any other condition that, in the opinion of the treating physician, renders the subject unsuitable for hematopoietic stem cell transplantation.\n* Have a known allergy to the investigational drug(s) (e.g., plerixafor, busulfan) or their components.\n* Have participated in or are currently participating in another interventional clinical study within 3 months before screening.\n* Received a live vaccine within 6 weeks before screening.\n* Be pregnant or breastfeeding.\n* The subject or their partner is unwilling to use medically acceptable effective contraception during the 32-month study period.\n* Unable to receive red blood cell transfusion.\n* The subject or their parent/guardian is unable to adhere to the study protocol.\n* Have any other condition deemed by the Investigator to make the subject unsuitable for participation in this study."}, 'identificationModule': {'nctId': 'NCT07436767', 'briefTitle': 'Safety and Efficacy of KL003 Cell Injection in Severe Sickle Cell Disease', 'organization': {'class': 'OTHER', 'fullName': 'Institute of Hematology & Blood Diseases Hospital, China'}, 'officialTitle': 'Safety and Efficacy of KL003 Cell Injection in Severe Sickle Cell Disease', 'orgStudyIdInfo': {'id': 'CP-KL003-006/02'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Experimental', 'description': 'A single intravenous infusion of ≥ 3.0 × 10\\^6 CD34+ cells/kg was administered.', 'interventionNames': ['Genetic: KL003 Cell Injection']}], 'interventions': [{'name': 'KL003 Cell Injection', 'type': 'GENETIC', 'description': "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.", 'armGroupLabels': ['Experimental']}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Institute of Hematology & Blood Diseases Hospital, China', 'class': 'OTHER'}, 'responsibleParty': {'type': 'PRINCIPAL_INVESTIGATOR', 'investigatorTitle': 'Director of the Red Blood Cell Diseases Center & Director of the Regenerative Medicine Clinic', 'investigatorFullName': 'Jun Shi', 'investigatorAffiliation': 'Institute of Hematology & Blood Diseases Hospital, China'}}}}