A Non-randomized, Open-label, Dose-escalation, Phase I/II Study to Evaluate the Safety, Tolerability, Kinetics and Efficacy of a Single Intravenous Infusion of ZS801 in Hemophilia B Subjects With Endogenous FIX ≤2%.

Status: Recruiting
Location: See location...
Intervention Type: Genetic
Study Type: Interventional
Study Phase: Phase 1/Phase 2
SUMMARY

A non-randomized, open-label, dose-escalation, phase I/II study to evaluate the safety, tolerability, kinetics and efficacy of a single intravenous infusion of ZS801 in hemophilia B subjects with endogenous FIX ≤2%.

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

• Male ≥18 years of age;

• Confirmed diagnosis of hemophilia B, and endogenous FIX ≤2%;

• Have had ≥100 prior exposure days (EDs) to any recombinant and/or plasma-derived FIX protein products;

• The subject had at least 3 or more bleeding events and/or chronic hemophilia arthritis in one or more joints in the previous 1 year requiring treatment with FIX;

• Agree to use reliable barrier contraception and prohibition of sperm donation until 52 weeks after the administration of ZS801.

• 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
Zhang Lei, MD
zhanglei1@ihcams.ac.cn
+86 022-23909240
Time Frame
Start Date: 2022-02-16
Estimated Completion Date: 2028-12
Participants
Target number of participants: 21
Treatments
Experimental: ZS801
Single intravenous (i.v.) infusion of ZS801 Intervention: Gene Therapy / Gene Transfer
Related Therapeutic Areas
Sponsors
Leads: Institute of Hematology & Blood Diseases Hospital, China

This content was sourced from clinicaltrials.gov