Status:
UNKNOWN
Safety and Efficacy Clinical Study of Severe Hemophilia B Treatment With KL001
Lead Sponsor:
Affiliated Hospital of Guangdong Medical University
Conditions:
Hemophilia B Without Inhibitor
Eligibility:
MALE
12+ years
Phase:
EARLY_PHASE1
Brief Summary
This is an open- label, non- randomized, uncontrolled, dose-escalation pilot study to evaluate the safety and efficacy of KL001 injection solution in hemophilia B subjects with ≤2 IU/dl residual FIX l...
Detailed Description
This is an open- label, non- randomized, uncontrolled, dose-escalation pilot study to evaluate the safety and efficacy of KL001 injection solution in hemophilia B subjects with ≤2 IU/dl residual FIX l...
Eligibility Criteria
Inclusion
- Male ≥12 years of age.
- Moderate/severe or severe hemophilia B (baseline FIX activity ≤2% of normal or documented history of FIX activity ≤2%).
- At least 3 bleeding episodes per year that require on-demand treatment with FIX or are treated with a prophylactic regimen of FIX.
- At least 100 days exposure history to recombinant or plasma-derived FIX protein products.
- No neutralizing antibodies to exogenous FIX protein products.
- Willing and able to comply with study procedures and requirements.
Exclusion
- Suffering from chronic inflammatory muscle disease.
- Positive in Hepatitis B or Hepatitis C.
- Infection with HIV-1 or HIV-2 and CD4+T cell count ≤ 200/ μ L.
- History of thrombosis or susceptibility to thrombosis.
- Current or previous participation in another gene therapy study.
- Has any other significant medical condition that the investigator feels would be a risk to the subject or would impede the study.
Key Trial Info
Start Date :
December 20 2023
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
October 30 2025
Estimated Enrollment :
9 Patients enrolled
Trial Details
Trial ID
NCT06125756
Start Date
December 20 2023
End Date
October 30 2025
Last Update
November 9 2023
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