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

NCT06119659 | Unknown Early Phase 1

• 1 other identifier: CP-KL001-001/01
• Study Type: interventional
• Target: 9
• Locations: 0 countries
• Sites: N/A

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.

Conditions

Hemophilia B Without Inhibitor

Outcome Measures

Primary Outcomes (3)

• Incidence of treatment- related adverse events

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

  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

  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

  Infusion to the completion of study, about 52 weeks

Secondary Outcomes (3)

• vector-derived FIX: C activity levels

  From dosing day to week 52

• The annualized bleeding rate Before and After KL001 Infusion

  From dosing day to week 52

• The annualized use of FIX

  From dosing day to week 52

Study Arms (1)

KL001 injection solution

EXPERIMENTAL

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

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

Interventions

Low dose KL001 DRUG

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

Also known as: rAAV vector

KL001 injection solution

Middle dose KL001 DRUG

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

Also known as: rAAV vector

KL001 injection solution

High dose KL001 DRUG

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

Also known as: rAAV vector

KL001 injection solution

Eligibility Criteria

• Age: 18 Years+
• Sex: male
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Male ≥12 years of age.
• Moderate/severe or severe hemophilia B (baseline FIX activity ≤2% of normal or documented history of FIX activity ≤2%).
• At least 3 bleeding episodes per year that require on-demand treatment with FIX OR are treated with a prophylactic regimen of FIX.
• At least 100 days exposure history to recombinant or plasma-derived FIX protein products.
• No neutralizing antibodies to exogenous FIX protein products.
• Willing and able to comply with study procedures and requirements.

You may not qualify if:

• Suffering from chronic inflammatory muscle disease.
• Positive in Hepatitis B or Hepatitis C.
• Infection with HIV-1 or HIV-2 and CD4+T cell count ≤ 200/ μ L.
• History of thrombosis or susceptibility to thrombosis.
• Current or previous participation in another gene therapy study.
• Has any other significant medical condition that the investigator feels would be a risk to the subject or would impede the study.

Sponsors & Collaborators

• Zhejiang University: lead

MeSH Terms

Interventions

KL001

Study Officials

• He Huang, PhD&MD

  First Affiliated Hospital of Zhejiang University

  PRINCIPAL INVESTIGATOR

Central Study Contacts

He Huang, PhD&MD

CONTACT

+86 13605714822; huanghe@zju.edu.cn

Yongxian Hu, PhD&MD

CONTACT

+8615957162012; huyongxian2000@aliyun.com

Study Design

• Study Type: interventional
• Phase: early phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Chief physician

Study Record Dates

• First Submitted: October 31, 2023
• First Posted: November 7, 2023
• Study Start: November 20, 2023
• Primary Completion: June 30, 2024
• Study Completion: July 10, 2025
• Last Updated: November 7, 2023

Record last verified: 2023-11

Data Sharing

• IPD Sharing: Will not share