Hemophilia B Without InhibitorEarly Phase 1NCT06119659
Safety and Efficacy Clinical Study of Severe Hemophilia B Treatment With KL001
A Early Phase 1 study testing Low dose KL001, Middle dose KL001 and High dose KL001 in people with Hemophilia B Without Inhibitor, run by Zhejiang University.
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. Read more about this study
- Who is it for?
- Ages 18 years and older, men only
- Conditions the study is for
- Hemophilia B Without Inhibitor
- What is being tested?
- Low dose KL001, Middle dose KL001 and High dose KL001
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 9 participants
- Where
- No sites listed yet
- Study dates
- 20 Nov 2023to 10 Jul 2025
- Phase
- Early Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- Men only
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
No sites are listed yet.
About this study
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.
Read the 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 a total 5 years.
- Conditions
- Hemophilia B Without Inhibitor
- Being tested
- Low dose KL001, Middle dose KL001, High dose KL001
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Low dose KL001Drug
Also called rAAV vector
Subjects will be dosed with single dose of KL001 at 2.5x10^12 vg/kg.
Middle dose KL001Drug
Also called rAAV vector
Subjects will be dosed with single dose of KL001 at 5.0x10^12 vg/kg.
High dose KL001Drug
Also called rAAV vector
Subjects will be dosed with single dose of KL001 at 1.0x10^13 vg/kg.
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: Drug: Low dose KL001; Drug: Middle dose KL001; Drug: High dose KL001 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of treatment- related adverse events
Number and severity of adverse events and serious adverse events and relationship to KL001
Measured: 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
Measured: 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
Measured: Infusion to the completion of study, about 52 weeks
Secondary outcomes (3)
vector-derived FIX: C activity levels
Peak and steady-state activity levels of vector-derived FIX: C
Measured: From dosing day to week 52
The annualized bleeding rate Before and After KL001 Infusion
The annualized bleeding rate (ABR) will be calculated for all subjects through Week 52
Measured: From dosing day to week 52
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.
Measured: From dosing day to week 52
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Nov 2023
- Study start20 Nov 2023 (estimated)
- Primary completion30 Jun 2024 (estimated)
- Study completion10 Jul 2025 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hemophilia B Without InhibitorStudies of the same condition, open and under way.
- More trials run by Zhejiang UniversityThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Early Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 9 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Zhejiang University
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, He Huang, Zhejiang University
- Investigators
- He Huang, PhD&MD, Principal investigator, First Affiliated Hospital of Zhejiang University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06119659
- Sponsor's protocol ID
- CP-KL001-001/01
- Status last verified
- Nov 2023
- First submitted
- 31 Oct 2023
- Last updated
- 7 Nov 2023
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 7 Nov 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Safety and Efficacy Clinical Study of Severe Hemophilia B Treatment With KL001
- ClinicalTrials.gov ID
- NCT06119659
- Acronym / sponsor ID
- CP-KL001-001/01
- Conditions
- Hemophilia B Without Inhibitor
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 9 participants (estimated)
- Lead sponsor
- Zhejiang UniversityUniversity, hospital or other
- Study start
- 20 Nov 2023 (estimated)
- Primary completion
- 30 Jun 2024 (estimated)
- Study completion
- 10 Jul 2025 (estimated)
- First posted
- 7 Nov 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 a total 5 years.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| KL001 injection solution | Experimental | Drug: Low dose KL001, Drug: Middle dose KL001, Drug: High dose KL001Subjects will be dosed with three different dose of KL001 injection solution at 2.5x10^12 vg/kg to 1.0x10^13 vg/kg. |
Who may qualify
- Ages
- 18 years and older
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- 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.
Key exclusion criteria (6 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Low dose KL001Also: rAAV vectorSubjects will be dosed with single dose of KL001 at 2.5x10^12 vg/kg. | Drug | KL001 injection solution |
| Middle dose KL001Also: rAAV vectorSubjects will be dosed with single dose of KL001 at 5.0x10^12 vg/kg. | Drug | KL001 injection solution |
| High dose KL001Also: rAAV vectorSubjects will be dosed with single dose of KL001 at 1.0x10^13 vg/kg. | Drug | KL001 injection solution |
Primary outcome
Incidence of treatment- related adverse events
Time frame: Infusion to the completion of study, about 52 weeks
Number and severity of adverse events and serious adverse events and relationship to KL001
Antibody against KL001 AAV vector capsid protein
Time frame: Infusion to the completion of study, about 52 weeks
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
Factor IX inhibitor
Time frame: Infusion to the completion of study, about 52 weeks
Development of inhibitor against vector-derived Factor IX protein will be measured using bethesda method
3 secondary outcomes
vector-derived FIX: C activity levels
Time frame: From dosing day to week 52
The annualized bleeding rate Before and After KL001 Infusion
Time frame: From dosing day to week 52
The annualized use of FIX
Time frame: From dosing day to week 52
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 7 Nov 2023.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
He Huang, PhD&MD principal investigator
First Affiliated Hospital of Zhejiang University
Questions about this trial
What is this clinical trial studying?
A Early Phase 1 study testing Low dose KL001, Middle dose KL001 and High dose KL001 in people with Hemophilia B Without Inhibitor, run by Zhejiang University.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 7 Nov 2023. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact He Huang, PhD&MD (+86 13605714822, huanghe@zju.edu.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of treatment- related adverse events (time frame: Infusion to the completion of study, about 52 weeks). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 7 Nov 2023.