Lead-in Study of VGB-R04 Gene Therapy for Hemophilia B-- An Observational Survey Analysis Study

NCT05442528 | Unknown

• 1 other identifier: VGB-R04-LI
• Study Type: observational
• Target: 20
• Locations: 0 countries
• Sites: N/A

Brief 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.

Conditions

Hemophilia B

Outcome Measures

Primary Outcomes (1)

• Annualized bleeding rate changes from baseline

  The number of bleeding episodes per participant will be recorded, and the annualized number of bleeding episodes.

  Baseline up to Week 26

Secondary Outcomes (5)

• Annualized FIX consumption changes from baseline

  Baseline up to Week 26

• Total number of annualized bleeding

  Baseline up to Week 26

• FIX:C activity level.

  Baseline up to Week 26

• Number of target joints

  Baseline up to Week 26

• The incidence of adverse Events of special interest

  Baseline up to Week 26

Study Arms (1)

Experimental: FIX replacement therapy.

Subject's previous treatment plan will be followed

Eligibility Criteria

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

Study Population

Hemophilia B (baseline FIX activity ≤ 2% of normal)

You may qualify if:

• 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;
• 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;

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

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

  Institute of Hematology & Blood Diseases Hospital, China

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Gui Cao

CONTACT

15156012172; g.cao@vitalgen.com

Study Design

• Study Type: observational
• Observational Model: CASE ONLY
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 28, 2022
• First Posted: July 5, 2022
• Study Start: August 1, 2022
• Primary Completion: December 1, 2024
• Study Completion: December 1, 2024
• Last Updated: July 5, 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.