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

Affiliated Hospital of Guangdong Medical University

Status and phase: Not yet enrolling; Early Phase 1

Conditions: Hemophilia B Without Inhibitor

Treatments: Drug: Middle dose KL001; Drug: High dose KL001; Drug: Low dose KL001

Study type: Interventional

Funder types: Other

Identifiers

NCT06125756

CP-KL001-001/01

Details and patient eligibility

About

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.

Full 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.

Enrollment: 9 estimated patients

Sex: Male

Ages: 12+ years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Sequential Assignment

Masking: None (Open label)

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9 participants in 1 patient group

KL001 injection solution

Experimental group

Description:

Subjects will be dosed with three different dose of KL001 injection solution at 2.5x10\^12 vg/kg to 1.0x10\^13 vg/kg.

Treatment:

Drug: Middle dose KL001

Drug: High dose KL001

Drug: Low dose KL001