A Study to Evaluate the Safety and Efficacy of VGB-R04 in Adult Hemophilia B Patients

NCT ID: NCT05441553

Last Updated: 2022-07-01

Basic Information

• Recruitment Status: UNKNOWN
• Clinical Phase: PHASE1/PHASE2
• Total Enrollment: 26 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2022-07-31
• Study Completion Date: 2025-01-31

Brief Summary

A multicenter, open, non-randomized, phase I/II, two-phase clinical study. The dose exploration phase was phase I, and the dose extension phase was phase II.

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 study is intended to evaluate the safety, tolerability and efficacy of a single IV infusion of VGB-R04. All subjects in this study will provide informed consent and then undergo screening assessments up to 6 weeks before administration of VGB-R04. All subjects will undergo 52 weeks of safety observation and will be 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

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

VGB-R04

Single intravenous (i.v.) infusion of VGB-R04 Intervention: Gene Therapy / Gene Transfer

Group Type: EXPERIMENTAL

VGB-R04

Intervention Type: GENETIC

A novel, bioengineered adeno-associated viral (AAV) vector carrying human factor IX variant

Interventions

VGB-R04

A novel, bioengineered adeno-associated viral (AAV) vector carrying human factor IX variant

Intervention Type: GENETIC

Eligibility Criteria

Inclusion Criteria

1. Male ≥18 years and ≤65years of age;

2. Confirmed diagnosis of hemophilia B (baseline FIX activity ≤ 2% of normal);

3. At least 100 days exposure history to FIX;

4. Currently receiving FIX Prophylaxis therapy or on-demand treatment to prevent bleeding;

5. Have acceptable laboratory values:

1. Hemoglobin ≥110 g/L;

2. Platelets ≥100×109 /L;

3. AST, ALT, alkaline phosphatase ≤2×upper limit of normal (ULN) at the testing laboratory;

4. Bilirubin ≤3× ULN ;

5. Creatinine ≤1.5× ULN.

6. No measurable factor IX inhibitor as assessed by the central laboratory and have no prior history of inhibitors to factor IX protein;

7. Agree to use reliable contraception until 2 consecutive samples are negative for vector sequences;

Exclusion Criteria

1. Have significant underlying liver disease within the past 6 months prior to or at Screening, including but not limited to:

1. Preexisting diagnosis of portal hypertension;

2. Splenomegaly;

3. Encephalopathy;

4. Reduction of serum albumin;

5. Evidence of significant liver fibrosis;

2. Have anti-VGB-R04 neutralizing antibody titers ≥1:5;

3. Evidence of severe infection disease, i.e., human immunodeficiency virus (HIV) infection, syphilis, tuberculosis, etc.;

4. Novel coronavirus infection occurred in the 6 weeks prior to entry into the group

5. Evidence of active hepatitis B virus infection (HBsAg positive) or hepatitis C virus infection (HCV-RNA positive);

6. Evidence of malignant tumours or those with a previous history of malignant tumours;

7. Have a history of chronic infection or other chronic diseases that the Investigator considers to constitute an unacceptable risk;

8. Any immunodeficiency;

9. planned surgery may be required within one year;

10. Past thromboembolic events (arterial or venous thromboembolic events);

11. Hypertensive patients with poor blood pressure control (systolic blood pressure ≥150 mmHg or diastolic blood pressure ≥90mmHg after antihypertensive drug treatment);

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 65 Years
• Eligible Sex: MALE
• Accepts Healthy Volunteers: No

Sponsors

Shanghai Vitalgen BioPharma Co., Ltd.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Lei Zhang, PhD

Role: PRINCIPAL_INVESTIGATOR

Institute of Hematology & Blood Diseases Hospital, China

Locations

Shanghai Vitalgen Biopharma Co.,Ltd.

Shanghai, , China

Countries

China

Central Contacts

Min Li

Role: CONTACT

m.li@vitalgen.com

18822167237

Facility Contacts

Min Li, Bachelor's

Role: primary

m.li@vitalgen.com

18822167237

Other Identifiers

VGB-R04-101

Identifier Type: -

Identifier Source: org_study_id