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

Sponsor
Institute of Hematology & Blood Diseases Hospital, China
Study ID
NCT07436767
Status
Not Yet Recruiting

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Conditions

Eligibility Criteria

Sex
ALL
Age
12 Years - 50 Years
Healthy Volunteers
Not accepted

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.

Study Details

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.

Key Dates

First listed
Feb 27, 2026
Start date
Mar 1, 2026
Status verified
Feb 2026
Primary completion
Sep 30, 2028
Completion
Dec 31, 2028

Study Design

Enrollment
3 participants (estimated)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

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

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 ]

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