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Clinical Trial Details — Status: Not yet recruiting

Administrative data

NCT number NCT06119659
Other study ID # CP-KL001-001/01
Secondary ID
Status Not yet recruiting
Phase Early Phase 1
First received
Last updated
Start date November 20, 2023
Est. completion date July 10, 2025

Study information

Verified date November 2023
Source Zhejiang University
Contact He Huang, PhD&MD
Phone +86 13605714822
Email huanghe@zju.edu.cn
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial 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.


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 a total 5 years.


Recruitment information / eligibility

Status Not yet recruiting
Enrollment 9
Est. completion date July 10, 2025
Est. primary completion date June 30, 2024
Accepts healthy volunteers No
Gender Male
Age group 18 Years and older
Eligibility 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.

Study Design


Related Conditions & MeSH terms


Intervention

Drug:
Low dose KL001
Subjects will be dosed with single dose of KL001 at 2.5x10^12 vg/kg.
Middle dose KL001
Subjects will be dosed with single dose of KL001 at 5.0x10^12 vg/kg.
High dose KL001
Subjects will be dosed with single dose of KL001 at 1.0x10^13 vg/kg.

Locations

Country Name City State
n/a

Sponsors (1)

Lead Sponsor Collaborator
Zhejiang University

Outcome

Type Measure Description Time frame Safety issue
Primary 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
Primary 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
Primary 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 vector-derived FIX: C activity levels Peak and steady-state activity levels of vector-derived FIX: C From dosing day to week 52
Secondary The annualized bleeding rate Before and After KL001 Infusion The annualized bleeding rate (ABR) will be calculated for all subjects through Week 52 From dosing day to week 52
Secondary 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. From dosing day to week 52
See also
  Status Clinical Trial Phase
Completed NCT04072237 - Study of Coagulation Faction VIIa Variant Marzeptacog Alfa (Activated) in Adult Subjects With Hemophilia Phase 1
Not yet recruiting NCT06125756 - Safety and Efficacy Clinical Study of Severe Hemophilia B Treatment With KL001 Early Phase 1