NCT06125756

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

Safety and Dose Finding Study of KL001 in Moderately Severe to Severe Hemophilia B Subjects

• Status: Unknown
• Phase: EARLY_Phase 1
• Study type: Interventional
• Enrollment: 9 (estimated)
• Sponsor: Affiliated Hospital of Guangdong Medical University · Academic / Other
• Sex: Male
• Age: 12 Years
• Healthy volunteers: Not accepted

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.

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 levels. Nine subjects will be enrolled and administered with three different doses of KL001. Subjects will provide informed consent and then undergo screening assessments up to 1 month prior administration of KL001. All subjects will undergo 52 weeks safety observation and will be encouraged to enroll in an extension study to evaluate long- term safety of KL001 for total 5 years.

Conditions

• Hemophilia B Without Inhibitor

Interventions

Type	Name	Description
DRUG	Low dose KL001	Subjects will be dosed with single dose of KL001 at 2.5x10\^12 vg/kg.
DRUG	Middle dose KL001	Subjects will be dosed with single dose of KL001 at 5.0x10\^12 vg/kg.
DRUG	High dose KL001	Subjects will be dosed with single dose of KL001 at 1.0x10\^13 vg/kg.

Timeline

• Start date: 2023-12-20
• Primary completion: 2024-09-20
• Completion: 2025-10-30
• First posted: 2023-11-09
• Last updated: 2023-11-09