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 range

18 Years

Sex

MALE

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.

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

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 range

18 Years

Sex

MALE

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.

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