A Phase 1/2 Study to Evaluate the Safety and Efficacy of VGB-R04 in Adult Hemophilia B Patients

Status: Not yet recruiting
Location: See location...
Intervention Type: Genetic
Study Type: Interventional
Study Phase: Phase 1/Phase 2
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.

Eligibility
Participation Requirements
Sex: Male
Minimum Age: 18
Maximum Age: 65
Healthy Volunteers: No
View:

• 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;

Locations
Other Locations
China
Shanghai Vitalgen Biopharma Co.,Ltd.
Shanghai
Contact Information
Primary
Min Li
m.li@vitalgen.com
18822167237
Time Frame
Start Date: July 2022
Estimated Completion Date: January 2025
Participants
Target number of participants: 26
Treatments
Experimental: VGB-R04
Single intravenous (i.v.) infusion of VGB-R04 Intervention: Gene Therapy / Gene Transfer
Authors
Related Therapeutic Areas
Sponsors
Leads: Shanghai Vitalgen BioPharma Co., Ltd.

This content was sourced from clinicaltrials.gov

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