At a glance
ClinicalIndex Comparison Record- ✓Male subjects ≥18 years of age
- ✓Hemophilia A with ≤1% endogenous FVIII activity at screening
- ✓No measurable FVIII inhibitor on two assessments ≥1 week apart, or documented no history after 150 EDs with no clinical signs of decreased response
- ✓Able to understand study purpose and risks and provide informed consent
- ✕Hepatitis B or C with clinical significance (exceptions: natural clearers and those cleared on antiviral therapy)
- ✕Currently receiving antiviral therapy for hepatitis B and C
- ✕History of chronic infections or other chronic diseases posing risk to study participation
- ✕Prior gene therapy trial within last 52 weeks or investigational drug within past 30 days
Standardized by ClinicalIndex from the ClinicalTrials.gov record · verify against the source.
Clinical Exploration of Clinical Exploration Adeno-associated Virus Vector Expression of Human Coagulation Factor VIII Gene Therapy for Hemophilia A
In Brief
A clinical study evaluating Injection of GS001 for Hemophilia A and Gene Therapy. Currently recruiting, targeting 12 participants across 1 site.
Detailed 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.
Study Details
Timeline
Interventions
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