Safety and Efficacy Clinical Study of Severe Hemophilia B Treatment With KL001

• Status: Not yet recruiting
• Trial end date: 2025-07-10
• Target enrollment: 0
• Participant gender: Male

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 levels.

• Phase: Early Phase 1
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Zhejiang University

Criteria

Inclusion Criteria:

1. Male ≥12 years of age.

2. Moderate/severe or severe hemophilia B (baseline FIX activity ≤2% of normal or documented history of FIX activity ≤2%).

3. At least 3 bleeding episodes per year that require on-demand treatment with FIX OR are treated with a prophylactic regimen of FIX.

4. At least 100 days exposure history to recombinant or plasma-derived FIX protein products.

5. No neutralizing antibodies to exogenous FIX protein products.

6. Willing and able to comply with study procedures and requirements.

Exclusion Criteria:

1. Suffering from chronic inflammatory muscle disease.

2. Positive in Hepatitis B or Hepatitis C.

3. Infection with HIV-1 or HIV-2 and CD4+T cell count ≤ 200/ μ L.

4. History of thrombosis or susceptibility to thrombosis.

5. Current or previous participation in another gene therapy study.

6. Has any other significant medical condition that the investigator feels would be a risk to the subject or would impede the study.