Hemophilia BNCT05442528
Lead-in Study of VGB-R04 Gene Therapy for Hemophilia B-- An Observational Survey Analysis Study
An observational study of Hemophilia B, run by Shanghai Vitalgen BioPharma Co., Ltd..
Trial summary
This is a prospective, open, multicenter, observational lead-in study，to collect prospective efficacy and safety data of current replacement therapy in adult hemophilia B patients. Read more about this study
- Who is it for?
- Ages 18 to 65 years, men only
- Conditions the study is for
- Hemophilia B
- How many people
- 20 participants
- Where
- No sites listed yet
- Study dates
- Aug 2022to Dec 2024
- Phase
- Observational
- 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 to 65 years
- Men only
- No healthy volunteers
- Adult, Older adult
Study population: Hemophilia B (baseline FIX activity ≤ 2% of normal)
Who may be able to join
- Male ≥18 years and ≤65years of age;
- Confirmed diagnosis of hemophilia B (baseline FIX activity ≤ 2% of normal);
- At least 50 days exposure history to FIX;
- Currently receiving FIX Prophylaxis therapy or on-demand treatment to prevent bleeding;
- Have acceptable laboratory values:
- Hemoglobin ≥110 g/L;
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- Platelets ≥100×109 /L;
- AST, ALT, alkaline phosphatase ≤2×upper limit of normal (ULN) at the testing laboratory;
- Bilirubin ≤3× ULN ;
- Creatinine ≤1.5× ULN.
- No measurable factor IX inhibitor as assessed by the central laboratory and have no prior history of inhibitors to factor IX protein;
Who cannot join
- Have significant underlying liver disease within the past 6 months prior to or at Screening, including but not limited to:
- Preexisting diagnosis of portal hypertension;
- Splenomegaly;
- Encephalopathy;
- Reduction of serum albumin;
- Evidence of significant liver fibrosis;
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- Have anti-VGB-R04 neutralizing antibody titers ≥1:5;
- Evidence of severe infection disease, i.e., human immunodeficiency virus (HIV) infection, syphilis, tuberculosis, etc.;
- Novel coronavirus infection occurred in the 6 weeks prior to entry into the group
- Evidence of active hepatitis B virus infection (HBsAg positive) or hepatitis C virus infection (HCV-RNA positive);
- Evidence of malignant tumours or those with a previous history of malignant tumours;
- Have a history of chronic infection or other chronic diseases that the Investigator considers to constitute an unacceptable risk;
- Any immunodeficiency;
- planned surgery may be required within one year;
- Past thromboembolic events (arterial or venous thromboembolic events);
- Hypertensive patients with poor blood pressure control (systolic blood pressure ≥150 mmHg or diastolic blood pressure ≥90mmHg after antihypertensive drug treatment);
Trial locations and contacts
No sites are listed yet.
About this study
Official title: Six-month lead-in Study to Collect Prospective Efficacy and Safety Data of Current Replacement Therapy in Adult Hemophilia B Patients -- An Observational Survey Analysis Study
This is a prospective, open, multicenter, observational lead-in study，to collect prospective efficacy and safety data of current replacement therapy in adult hemophilia B patients.
Read the detailed description
Hemophilia B is a genetic bleeding disorder caused by pathogenic variants (eg, mutations, deletion) in the FIX gene. HB patients have frequent and potentially life-threatening bleeding and often develop progressive physical disability and pain from chronic haemarthropathy. Current replacement therapy needs regular treatment in the life-long time, bringing heavy economic and social burdens.VGB-R04 is a novel AAV vector carrying a high specific activity factor IX variant.
This is a prospective, open, multicenter, observational lead-in study. To evaluate the efficacy and safety of current treatment regiments in patients with moderate to severe HB (FIX:C activity ≤2%). The efficacy and safety data collected in this study may be used as baseline data for subsequent related clinical trials of VGB-R04. All subjects in this study will provide informed consent and then undergo screening assessments up to 4 weeks. All subjects will undergo 26 weeks of efficacy and safety observation .Eligible subjects will be invited to a key clinical study（phase 2 study）of VGB-R04,and then encouraged to enroll in an Long-term follow-up study to evaluate the long-term safety of VGB-R04 for a total of five years.
- Conditions
- Hemophilia B
- Medical subject headings
- Hemophilia B
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| Experimental: FIX replacement therapy. | Subject's previous treatment plan will be followed |
What is being measured
The primary outcome is the main question the study is designed to answer.
Annualized bleeding rate changes from baseline
The number of bleeding episodes per participant will be recorded, and the annualized number of bleeding episodes.
Measured: Baseline up to Week 26
Secondary outcomes (5)
Annualized FIX consumption changes from baseline
The use of on-demand FIX replacement therapy will be recorded by dose (IU/kg) administered, and the annualized use of FIX replacement therapy will be calculated.
Measured: Baseline up to Week 26
Total number of annualized bleeding
Total number of annualized bleeding including spontaneous or traumatic bleeding.
Measured: Baseline up to Week 26
FIX:C activity level.
FIX:C activity level and its change from baseline .
Measured: Baseline up to Week 26
Number of target joints
The criterion of the target joint is a minimum of three bleeds into a single joint within a consecutive three-month period.
Measured: Baseline up to Week 26
The incidence of adverse Events of special interest
Adverse Events of special interest (serious or non-serious) are a category of events of scientific and medical concern that are particularly relevant to the sponsor's drug or research program. Adverse Events of special interest include liver dysfunction, inhibitors, thromboembolic events, and allergic reactions.
Measured: Baseline up to Week 26
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Jul 2022
- Study startAug 2022 (estimated)
- Primary completionDec 2024 (estimated)
- Study completionDec 2024 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hemophilia BStudies of the same condition, open and under way.
- 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
- Observational
- Observational model
- Case only
- Time perspective
- Prospective
- Enrolment
- 20 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Shanghai Vitalgen BioPharma Co., Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Lei Zhang, Principal investigator, Institute of Hematology & Blood Diseases Hospital, China
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05442528
- Sponsor's protocol ID
- VGB-R04-LI
- Status last verified
- Jun 2022
- First submitted
- 28 Jun 2022
- Last updated
- 5 Jul 2022
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: IPD will be shared with other researchers when VGB-R04 is fully approved.
Study at a glance
From the registry record, last updated by the sponsor on 5 Jul 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Six-month lead-in Study to Collect Prospective Efficacy and Safety Data of Current Replacement Therapy in Adult Hemophilia B Patients -- An Observational Survey Analysis Study
- ClinicalTrials.gov ID
- NCT05442528
- Acronym / sponsor ID
- VGB-R04-LI
- Conditions
- Hemophilia B
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 20 participants (estimated)
- Lead sponsor
- Shanghai Vitalgen BioPharma Co., Ltd.Industry (pharma, biotech, devices)
- Study start
- Aug 2022 (estimated)
- Primary completion
- Dec 2024 (estimated)
- Study completion
- Dec 2024 (estimated)
- First posted
- 5 Jul 2022 (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 a prospective, open, multicenter, observational lead-in study，to collect prospective efficacy and safety data of current replacement therapy in adult hemophilia B patients.
Detailed description
Hemophilia B is a genetic bleeding disorder caused by pathogenic variants (eg, mutations, deletion) in the FIX gene. HB patients have frequent and potentially life-threatening bleeding and often develop progressive physical disability and pain from chronic haemarthropathy. Current replacement therapy needs regular treatment in the life-long time, bringing heavy economic and social burdens.VGB-R04 is a novel AAV vector carrying a high specific activity factor IX variant.
This is a prospective, open, multicenter, observational lead-in study. To evaluate the efficacy and safety of current treatment regiments in patients with moderate to severe HB (FIX:C activity ≤2%). The efficacy and safety data collected in this study may be used as baseline data for subsequent related clinical trials of VGB-R04. All subjects in this study will provide informed consent and then undergo screening assessments up to 4 weeks. All subjects will undergo 26 weeks of efficacy and safety observation .Eligible subjects will be invited to a key clinical study（phase 2 study）of VGB-R04,and then encouraged to enroll in an Long-term follow-up study to evaluate the long-term safety of VGB-R04 for a total of five years.
Study design
- Observational model
- Case only
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Experimental: FIX replacement therapy. | – | –Subject's previous treatment plan will be followed |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- Hemophilia B (baseline FIX activity ≤ 2% of normal)
Key inclusion criteria (11 listed)
- Male ≥18 years and ≤65years of age;
- Confirmed diagnosis of hemophilia B (baseline FIX activity ≤ 2% of normal);
- At least 50 days exposure history to FIX;
- Currently receiving FIX Prophylaxis therapy or on-demand treatment to prevent bleeding;
- Have acceptable laboratory values:
Key exclusion criteria (16 listed)
- Have significant underlying liver disease within the past 6 months prior to or at Screening, including but not limited to:
- Have anti-VGB-R04 neutralizing antibody titers ≥1:5;
- Evidence of severe infection disease, i.e., human immunodeficiency virus (HIV) infection, syphilis, tuberculosis, etc.;
- Novel coronavirus infection occurred in the 6 weeks prior to entry into the group
- Evidence of active hepatitis B virus infection (HBsAg positive) or hepatitis C virus infection (HCV-RNA positive);
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Annualized bleeding rate changes from baseline
Time frame: Baseline up to Week 26
The number of bleeding episodes per participant will be recorded, and the annualized number of bleeding episodes.
5 secondary outcomes
Annualized FIX consumption changes from baseline
Time frame: Baseline up to Week 26
Total number of annualized bleeding
Time frame: Baseline up to Week 26
FIX:C activity level.
Time frame: Baseline up to Week 26
Number of target joints
Time frame: Baseline up to Week 26
The incidence of adverse Events of special interest
Time frame: Baseline up to Week 26
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Jul 2022.
- Individual participant dataSharing planned: Yes. IPD will be shared with other researchers when VGB-R04 is fully approved.
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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.
Lei Zhang principal investigator
Institute of Hematology & Blood Diseases Hospital, China
Questions about this trial
What is this clinical trial studying?
An observational study of Hemophilia B, run by Shanghai Vitalgen BioPharma Co., Ltd..
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 11 inclusion and 16 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 5 Jul 2022. 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.
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 Gui Cao (15156012172, g.cao@vitalgen.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Annualized bleeding rate changes from baseline (time frame: Baseline up to Week 26). 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 5 Jul 2022.