Hemophilia A Clinical Trial
Official title:
GO-8: Gene Therapy for Haemophilia A Using a Novel Serotype 8 Capsid Pseudotyped Adeno-associated Viral Vector Encoding Factor VIII-V3
The GO-8 study focuses on assessing safety and efficacy of gene therapy for patients with severe haemophilia A
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, 2-3 times/week, intravenous injection of clotting factor concentrates, which is demanding and expensive. In contrast, gene therapy offers the potential of a cure for haemophilia A. In a previous gene therapy study in haemophilia B 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 twenty-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 5 years after vector infusion. ;
Status | Clinical Trial | Phase | |
---|---|---|---|
Completed |
NCT03834727 -
Characterizing the Impact and Treatment of Reproductive Tract Bleeding on Women and Post-menarchal Girls With Bleeding Disorders
|
||
Completed |
NCT03191799 -
A Study to Evaluate the Safety and Tolerability of Prophylactic Emicizumab in Hemophilia A Patients With Inhibitors
|
Phase 3 | |
Completed |
NCT01599819 -
BAX 855 Dose-Escalation Safety Study
|
Phase 1 | |
Terminated |
NCT04541628 -
Safety & Efficacy of Encapsulated Allogeneic FVIII Cell Therapy in Haemophilia A
|
Phase 1/Phase 2 | |
Completed |
NCT02847637 -
A Clinical Trial to Evaluate Prophylactic Emicizumab Versus no Prophylaxis in Hemophilia A Participants Without Inhibitors
|
Phase 3 | |
Completed |
NCT04072237 -
Study of Coagulation Faction VIIa Variant Marzeptacog Alfa (Activated) in Adult Subjects With Hemophilia
|
Phase 1 | |
Completed |
NCT04085458 -
Study to Gain More Information on How Safe and Effective Jivi Works in Patients With Severe Hemophilia A (Post-marketing Investigation)
|
Phase 4 | |
Completed |
NCT04565236 -
A Post Approval Commitment Study to Gain More Information on How Safe and Effective KOVALTRY is in Chinese Children, Adolescents /Adults With Severe Hemophilia A
|
Phase 4 | |
Recruiting |
NCT05987449 -
A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of NXT007 in Persons With Severe or Moderate Hemophilia A
|
Phase 1/Phase 2 | |
Active, not recruiting |
NCT04621916 -
Preventing Inhibitor Recurrence Indefinitely
|
Phase 4 | |
Not yet recruiting |
NCT02888223 -
Pharmacokinetic Study of SCT800 in Previously Treated Patients With Hemophilia A
|
Phase 1 | |
Completed |
NCT02528968 -
National Study of a Pharmacokinetic-Focused Educational Package for Patients With Severe Haemophilia A
|
N/A | |
Completed |
NCT02225483 -
Phenotypic Heterogeneity in Hemophilia A: An Investigation of the Role of Platelet Function
|
N/A | |
Completed |
NCT02199717 -
An Institutional Pilot Study to Investigate Physical Activity Patterns in Boys With Hemophilia
|
N/A | |
Completed |
NCT01217255 -
Comparing the Burden of Illness of Hemophilia in the Developing and the Developed World
|
||
Completed |
NCT00969319 -
Effekt-2 - Efficacy and Safety of Long-term Treatment With KOGENATE® FS in Latin America
|
N/A | |
Terminated |
NCT00995046 -
Individually Tailored Prophylaxis in Patients With Severe Hemophilia A
|
N/A | |
Completed |
NCT00868530 -
Study Evaluating On-Demand Treatment Of Xyntha In Chinese Subjects
|
Phase 3 | |
Completed |
NCT00839202 -
Activity and Content of Factor VIII (FVIII) in Human Plasma: The Assessment of a Novel Immunoassay
|
N/A | |
Completed |
NCT00629837 -
Pharmacokinetics and Safety of a Single Intravenous Infusion of BAY 79-4980
|
Phase 1 |