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

Who is this study for? Adults with hemophilia B
What treatments are being studied? VGB-R04
Status: Recruiting
Location: See location...
Intervention Type: Genetic
Study Type: Interventional
Study Phase: Early Phase 1
SUMMARY

An Open-Label, Non-Randomized, uncontrolled, single-dose pilot study of VGB-R04 in subjects with Hemophilia B.

Eligibility
Participation Requirements
Sex: Male
Minimum Age: 18
Maximum Age: 75
Healthy Volunteers: f
View:

• Male ≥18 years and ≤75years of age;

• Confirmed diagnosis of hemophilia B (baseline FIX activity ≤ 2% of normal or documented history of FIX activity ≤2%);

• 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 ≥100×10'9 cells/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 barrier contraception until 3 consecutive samples are negative for vector sequences;

• Able to provide informed consent and comply with the requirements of the study.

Locations
Other Locations
China
Blood diseases hospital
RECRUITING
Tianjin
Contact Information
Primary
Lei Zhang, Doctor
zhanglei1@ihcams.as.cn
+86-13502118379
Time Frame
Start Date: 2021-12-28
Estimated Completion Date: 2025-12
Participants
Target number of participants: 3
Treatments
Experimental: VGB-R04
Single intravenous (i.v.) infusion of VGB-R04 Intervention: Gene Therapy / Gene Transfer
Related Therapeutic Areas
Sponsors
Leads: Institute of Hematology & Blood Diseases Hospital, China

This content was sourced from clinicaltrials.gov

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