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

NCT ID: NCT06125756

Last Updated: 2023-11-09

Basic Information

• Recruitment Status: NOT_YET_RECRUITING
• Clinical Phase: EARLY_PHASE1
• Total Enrollment: 9 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2023-12-20
• Study Completion Date: 2025-10-30

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 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

Study Design

• Allocation Method: NA
• Intervention Model: SEQUENTIAL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

KL001 injection solution

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

Group Type: EXPERIMENTAL

Low dose KL001

Intervention Type: DRUG

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

Middle dose KL001

Intervention Type: DRUG

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

High dose KL001

Intervention Type: DRUG

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

Interventions

Low dose KL001

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

Intervention Type: DRUG

Middle dose KL001

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

Intervention Type: DRUG

High dose KL001

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

Intervention Type: DRUG

Other Intervention Names

rAAV vector; rAAV vector; rAAV vector

Eligibility 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.

• Minimum Eligible Age: 12 Years
• Eligible Sex: MALE
• Accepts Healthy Volunteers: No

Sponsors

Affiliated Hospital of Guangdong Medical University

OTHER

Sponsor Role: lead

Responsible Party

Liang Liang

associate chief physician

Responsibility Role: PRINCIPAL_INVESTIGATOR

Principal Investigators

Liang Liang, MD

Role: PRINCIPAL_INVESTIGATOR

Affiliated Hospital of Guangdong Medical University

Central Contacts

Liang Liang, MD

Role: CONTACT

13560539300@139.com

+86 13560539300

Other Identifiers

CP-KL001-001/01

Identifier Type: -

Identifier Source: org_study_id