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

NCT05441553 | Unknown Phase 1

• 1 other identifier: VGB-R04-101
• Study Type: interventional
• Target: 26
• Locations: 1 country
• Sites: 1

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.

Conditions

Hemophilia B

Outcome Measures

Primary Outcomes (3)

• Incidence of adverse events

  An adverse event (AE) is any untoward medical occurrence in a clinical investigation participant administered a product; the event will not need to have a causal relationship with the treatment.

  Baseline up to Week 52

• Incidence of serious adverse events

  A serious adverse event (SAE) is any untoward medical occurrence at any dose that resulted in death; life threatening;require inpatient hospitalization or prolongation of existing hospitalization; result in persistent or significant disability/incapacity; result in congenital anomaly/birth defect

  Baseline up to Week 52

• FIX:C Antigen Level at Steady State

  FIX:C activity antigen levels were characterized by post-treatment population mean.

  Baseline up to Week 52

Secondary Outcomes (4)

• FIX:C activity level

  Baseline up to Week 52

• Vector- derived FIX antigen levels

  Baseline up to Week 52

• Annualized bleeding rate changes from baseline

  Baseline up to Week 52

• Annualized FIX consumption changes from baseline

  Baseline up to Week 52

Study Arms (1)

VGB-R04

EXPERIMENTAL

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

Genetic: VGB-R04

Interventions

VGB-R04 GENETIC

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

VGB-R04

Eligibility Criteria

• Age: 18 Years - 65 Years
• Sex: male
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Male ≥18 years and ≤65years of age;
• Confirmed diagnosis of hemophilia B (baseline FIX activity ≤ 2% of normal);
• At least 100 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;
• 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;
• Agree to use reliable contraception until 2 consecutive samples are negative for vector sequences;

You may not qualify if:

• 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;
• 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);

Sponsors & Collaborators

• Shanghai Vitalgen BioPharma Co., Ltd.: lead

Study Sites (1)

Shanghai Vitalgen Biopharma Co.,Ltd.

Shanghai, China

Location

MeSH Terms

Conditions

Hemophilia B

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Genetic Diseases, X-Linked

Study Officials

• Lei Zhang, PhD

  Institute of Hematology & Blood Diseases Hospital, China

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Min Li

CONTACT

18822167237; m.li@vitalgen.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 15, 2022
• First Posted: July 1, 2022
• Study Start: July 1, 2022
• Primary Completion: January 1, 2025
• Study Completion: January 1, 2025
• Last Updated: July 1, 2022

Record last verified: 2022-06

Data Sharing

• IPD Sharing: Will share
• Time Frame: IPD will be shared with other researchers when VGB-R04 is fully approved.
• Access Criteria: IPD will be shared with other researchers when VGB-R04 is fully approved.

Locations

CN (1)