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

S

Shanghai Vitalgen BioPharma

Status

Not yet enrolling

Conditions

Hemophilia B

Study type

Observational

Funder types

Industry

Identifiers

NCT05442528
VGB-R04-LI

Details and patient eligibility

About

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.

Full 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.

Enrollment

20 estimated patients

Sex

Male

Ages

18 to 65 years old

Volunteers

No Healthy Volunteers

Inclusion criteria

  • 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;

Exclusion criteria

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

Trial design

20 participants in 1 patient group

Experimental: FIX replacement therapy.
Description:
Subject's previous treatment plan will be followed

Trial contacts and locations

0

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Central trial contact

Gui Cao

Data sourced from clinicaltrials.gov

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