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

Early Phase 1UnknownNCT06125756

Affiliated Hospital of Guangdong Medical University9 enrolled

Overview

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.

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Hemophilia B Without Inhibitor

Interventions

Low dose KL001DRUG

Subjects will be dosed with single dose of KL001 at 2.5x10\^12 vg/kg.

Middle dose KL001DRUG

Subjects will be dosed with single dose of KL001 at 5.0x10\^12 vg/kg.

High dose KL001DRUG

Subjects will be dosed with single dose of KL001 at 1.0x10\^13 vg/kg.

Eligibility

Sex: MALEMin age: 12 Years

Medical Language ↔ Plain English

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.

Outcomes

Primary Outcomes

Incidence of treatment- related adverse events

Number and severity of adverse events and serious adverse events and relationship to KL001

Time frame: Infusion to the completion of study, about 52 weeks

Antibody against KL001 AAV vector capsid protein

Immune response against AAV capsid will be evaluated by measurement of the total antibody and neutralizing antibody against AAV capsid protein in plasma samples collected at multiple timepoints after dosing up to 52 weeks

Time frame: Infusion to the completion of study, about 52 weeks

Factor IX inhibitor

Development of inhibitor against vector-derived Factor IX protein will be measured using bethesda method.

Time frame: Infusion to the completion of study, about 52 weeks

Secondary Outcomes

vector-derived FIX: C activity levels

Peak and steady-state activity levels of vector-derived FIX: C

Time frame: From dosing day to week 52

The annualized bleeding rate Before and After KL001 Infusion

The annualized bleeding rate (ABR) will be calculated for all subjects through Week 52

Time frame: From dosing day to week 52

The annualized use of FIX

The use of on-demand FIX replacement therapy was recorded by dose (IU/kg) administered, and the annualized use of FIX replacement therapy was calculated for all subjects through Week 52.

Time frame: From dosing day to week 52

Central Contacts

Liang Liang, MD

CONTACT

+86 13560539300 13560539300@139.com

Data from ClinicalTrials.gov

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