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Clinical Trial Summary

The GO-8 study focuses on assessing safety and efficacy of gene therapy for patients with severe haemophilia A


Clinical Trial Description

Haemophilia A is an x-linked, life threatening bleeding disorder arising from defects in the coagulation factor VIII (FVIII) gene. Current treatment for haemophilia A, the commonest inherited bleeding disorder (prevalence of 1 in 5000 individuals) consists of life-long, 2-3X/week, intravenous injection of clotting factor concentrates, which is demanding, exceedingly expensive not widely available nor curative. In contrast, gene therapy offers the potential of a cure for haemophilia A as illustrated by our first unequivocal success in a related condition, haemophilia B. In that study the investigators showed that a single intravenous administration of a serotype 8 based adeno-associated virus, (AAV8) vector encoding the factor IX (FIX) gene resulted in stable (>6 years) therapeutic expression of FIX without long-lasting toxicity. The investigators plan to use the same AAV8 platform to evaluate a novel FVIII expression cassette, AAV2/8-HLP-FVIII-V3, in patient with haemophilia A. Extensive preclinical studies demonstrate that AAV2/8-HLP-FVIII-V3 leads to long-term, endogenous expression of FVIII in mouse and non-human primate models without toxicity even when fifty-fold higher doses than the proposed starting clinical trial dose were used. Therefore, an open label, Phase I/II dose escalation study entailing a single systemic administration of AAV2/8-HLP-FVIII-V3 in adults (>18 years of age) with severe haemophilia A who have baseline factor FVIII levels of <1% of normal has been designed to establish safety and efficacy of our approach. Dosing will begin at 6x10^11 vector genome (vg)/kg progressing sequentially to 2x10^12vg/kg and ultimately 6x10^12vg/kg in the absence of toxicity. A minimum of 2 patients will be recruited at each dose with a possibility of expanding the dose cohort to a maximum of 6 patients based on safety and efficacy. The study duration for each patient will be 15 years after vector infusion. ;


Study Design


Related Conditions & MeSH terms


NCT number NCT03001830
Study type Interventional
Source University College, London
Contact Mark Phillips
Phone +442078302068
Email [email protected]
Status Recruiting
Phase Phase 1
Start date June 14, 2017
Completion date June 2025

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