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

NCT07436767 · Status: NOT_YET_RECRUITING · Phase: NA · Type: INTERVENTIONAL · Enrollment: 3

Last updated 2026-02-27

No results posted yet for this study

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

Interventions

GENETIC

KL003 Cell Injection

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.

Sponsors & Collaborators

  • Institute of Hematology & Blood Diseases Hospital, China

    lead OTHER

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
12 Years
Max Age
50 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2026-03-01
Primary Completion
2028-09-30
Completion
2028-12-31

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Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT07436767 on ClinicalTrials.gov