Safety and Tolerability of VGB-R04 in Patients With Haemophilia B

Launched by INSTITUTE OF HEMATOLOGY & BLOOD DISEASES HOSPITAL, CHINA · Nov 29, 2021

Keywords

ClinConnect Summary

This clinical trial is investigating a new treatment called VGB-R04 for men with Hemophilia B, a condition that affects blood clotting. The study aims to determine how safe the treatment is and how well patients tolerate it. It is an early-phase trial, meaning it's one of the first steps in testing this new treatment. The trial is currently recruiting male participants aged 18 to 75 who have a confirmed diagnosis of Hemophilia B and are already receiving treatment to help prevent bleeding.

To be eligible for the study, participants need to meet several criteria, including having low levels of a specific clotting factor (Factor IX) and being in generally good health with acceptable lab results. Participants can expect to receive a single dose of VGB-R04 and will be monitored for safety and any side effects. It’s important for potential participants to know that they will need to agree to certain health checks and follow-up visits throughout the study. This trial is a chance to contribute to research that could improve treatment options for Hemophilia B.

Gender

MALE

Eligibility criteria

• Inclusion Criteria:
• • 1. Male ≥18 years and ≤75years of age;
• • 2. Confirmed diagnosis of hemophilia B (baseline FIX activity ≤ 2% of normal or documented history of FIX activity ≤2%);
• • 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 ≥100×10'9 cells/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 barrier contraception until 3 consecutive samples are negative for vector sequences;
• • 8. Able to provide informed consent and comply with the requirements of the study.
• 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. Evidence of active hepatitis B virus infection (HBV-DNA \>103 IU/ml) or hepatitis C virus infection (HCV antigen and HCV-RNA positive);
• • 5. Evidence of malignant tumours or those with a previous history of malignant tumours;
• • 6. Have a history of chronic infection or other chronic diseases that the Investigator considers to constitute an unacceptable risk;
• • 7. Any immunodeficiency;
• • 8. Participated in a gene transfer trial within the last 52 weeks or in a clinical trial with an investigational drug within the last 4 weeks;
• • 9. Have used glucocorticoids, immunosuppressive drugs, or antipsychotics within the last 3 months;
• • 10. Previous history of hypersensitivity or allergic reaction to any FIX products or any immunoglobulin;
• • 11. Unable or unwilling to comply with the schedule of visits and study assessments described in the clinical protocol;
• • 12. Any concurrent clinically significant major disease or any other condition that, in the opinion of the Investigator, makes the subject unsuitable for participation in the study.