UnknownEarly Phase 1

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

9 participantsStarted 2023-11-20

Plain-language 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.

Who can participate

Age range18 Years

SexMALE

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

✓. 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.

Exclusion criteria

✕. 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.

What they're measuring

Incidence of treatment- related adverse events

Timeframe: Infusion to the completion of study, about 52 weeks

Antibody against KL001 AAV vector capsid protein

Timeframe: Infusion to the completion of study, about 52 weeks

Factor IX inhibitor

Timeframe: Infusion to the completion of study, about 52 weeks

Trial details

NCT IDNCT06119659

SponsorZhejiang University

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2024-06-30

Contact for this trial

He Huang, PhD&MD

+86 13605714822 huanghe@zju.edu.cn

View on ClinicalTrials.gov More Hemophilia B Without Inhibitor trials

Who can participate

Age range18 Years

SexMALE

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

✓. 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.

Exclusion criteria

✕. 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.

What they're measuring

Incidence of treatment- related adverse events

Timeframe: Infusion to the completion of study, about 52 weeks

Antibody against KL001 AAV vector capsid protein

Timeframe: Infusion to the completion of study, about 52 weeks

Factor IX inhibitor

Timeframe: Infusion to the completion of study, about 52 weeks