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Gene Therapy Trial Report

Summary

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


NCTID NCT05441553 (View at clinicaltrials.gov)
Description
Development Status Active
Indication Hemophilia B
Disease Ontology Term DOID:12259
Compound Name VGB-R04
Sponsor Shanghai Vitalgen BioPharma Co., Ltd.
Funder Type Industry
Recruitment Status
Unknown
Enrollment Count 26 (ESTIMATED)
Results Posted Not Available

Therapy Information


Target Gene/Variant F9
Therapy Type Gene transfer
Therapy Route In-vivo
Mechanism of Action Functional gene replacement
Route of Administration Intravenous
Drug Product Type Viral vector
Delivery System Viral transduction
Vector Type AAV8
Editor Type none
Dose 1 Undisclosed dose (ranging from 4E11 - 2E12 vg/kg)

Study Record Dates


Current Stage Phase1, Phase2
Submit Date 2022-06-15
Completion Date 2025-01
Last Update 2022-07-01

Participation Criteria


Eligible Age 18 Years - 65 Years
Standard Ages Adult, Older adult
Sexes Eligible for Study MALE
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);
View Inclusion and Exclusion Criteria at ClinicalTrials.gov

Locations


No.of Trial Sites 1
Locations China

Regulatory Information


Has US IND False
FDA Designations Orphan Drug Designation
Recent Updates Granted ODD in December 2021

Resources/Links