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Gene Therapy for Chinese Hemophilia A

NCT04728841 · Status: RECRUITING · Phase: NA · Type: INTERVENTIONAL · Enrollment: 12

Last updated 2025-08-01

No results posted yet for this study

Summary

IHBDH-GTHA-2020 is an open- label, non- randomized study to evaluate the safety, tolerability and kinetics of a single intravenous infusion of GS001 in hemophilia A subjects with \<1 IU/dl residual FVIII levels.

Conditions

Interventions

GENETIC

Injection of GS001

Patients will be enrolled sequentially every 3 weeks or more between cohorts. Dose escalation may occur after a single patient has been safely dosed if the resulting FVIII activity at Week 3 is \< 5 IU/dL.The dose levels are as follows: 1. 2×10\^12 vg/kg 2. 6×10\^12vg/kg or other recommended doses 3. 2×10\^13 vg/kg or other recommended doses

Sponsors & Collaborators

  • Institute of Hematology & Blood Diseases Hospital, China

    lead OTHER

Principal Investigators

  • Lei Zhang, MD · Chinese Academy of Medical Science and Blood Disease Hospital

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
18 Years
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2021-03-04
Primary Completion
2028-07-31
Completion
2028-07-31

Countries

  • China

Study Locations

More Related Trials

Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04728841 on ClinicalTrials.gov