A Non-randomized, Open-label, Dose-escalation Study to Evaluate the Safety, Tolerability, Kinetics and Efficacy of a Single Intravenous Infusion of ZS802 in Hemophilia A Subjects With Endogenous FVIII ≤2%.

Status: Recruiting
Location: See location...
Intervention Type: Genetic
Study Type: Interventional
Study Phase: Not Applicable
SUMMARY

A non-randomized, open-label, dose-escalation study to evaluate the safety, tolerability, kinetics and efficacy of a single intravenous infusion of ZS802 in hemophilia A subjects with endogenous FVIII ≤2%.

Eligibility
Participation Requirements
Sex: Male
Minimum Age: 18
Maximum Age: 65
Healthy Volunteers: f
View:

• Male ≥18 years and ≤65years of age;

• Confirmed diagnosis of hemophilia A, and endogenous FVIII ≤2%:

‣ \<1% (\<1 IU/dL) endogenous FVIII activity levels as historically documented by a certified laboratory or screening data results; OR

⁃ 1%-2% (1-2 IU/dL) endogenous FVIII activity levels and \>10 bleeding events per year (in the last 52 weeks prior to screening); OR

⁃ 1%-2% (1-2 IU/dL) endogenous FVIII activity levels and on prophylaxis;

• Have had ≥150 prior exposure days (EDs) to any recombinant and/or plasma-derived FVIII protein products.

• Agree to use reliable barrier contraception and prohibition of sperm donation until 52 weeks after the administration of ZS802.

• Subjects voluntarily participate and are fully informed, fully understand the research and can comply with the requirements of the research protocol, are willing to complete the research as planned, and voluntarily cooperate with the provision of biological samples for testing.

Locations
Other Locations
China
Institute of Hematology & Blood Diseases Hospital
RECRUITING
Tianjin
Contact Information
Primary
Lei Zhang, MD
zhanglei1@ihcams.ac.cn
+86 022-23909240
Time Frame
Start Date: 2022-09-29
Estimated Completion Date: 2025-10
Participants
Target number of participants: 6
Treatments
Experimental: ZS802
Single intravenous (i.v.) infusion of ZS802 Intervention: Gene Therapy / Gene Transfer
Related Therapeutic Areas
Sponsors
Leads: Institute of Hematology & Blood Diseases Hospital, China

This content was sourced from clinicaltrials.gov